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1. Outcomes of allogeneic haematopoietic cell transplantation for myelofibrosis in children and adolescents: the retrospective study of the EBMT Paediatric Diseases WP

Author: Wachowiak, J, Galimard, J, Dalissier, A, Rihani, R, Alsaedi, H, Wynn, R, Dalle, J, Peffault de Latour, R, Sedlacek, P, Balduzzi, A, Schroeder, T, Bodova, I, Gonzalez Vicent, M, Gruhn, B, Hamladji, R, Krivan, G, Patrick, K, Sobkowiak-Sobierajska, A, Stepensky, P, Unal, A, Amrolia, P, Perez Martinez, A, Rialland, F, Aljurf, M, Isgro, A, Toren, A, Bierings, M, Corbacioglu, S, Kalwak, K, Wachowiak J., Galimard J. -E., Dalissier A., Rihani R., AlSaedi H., Wynn R. F., Dalle J. -H., Peffault de Latour R., Sedlacek P., Balduzzi A., Schroeder T., Bodova I., Gonzalez Vicent M., Gruhn B., Hamladji R. -M., Krivan G., Patrick K., Sobkowiak-Sobierajska A., Stepensky P., Unal A., Amrolia P., Perez Martinez A., Rialland F., Aljurf M., Isgro A., Toren A., Bierings M., Corbacioglu S., Kalwak K., Wachowiak, J, Galimard, J, Dalissier, A, Rihani, R, Alsaedi, H, Wynn, R, Dalle, J, Peffault de Latour, R, Sedlacek, P, Balduzzi, A, Schroeder, T, Bodova, I, Gonzalez Vicent, M, Gruhn, B, Hamladji, R, Krivan, G, Patrick, K, Sobkowiak-Sobierajska, A, Stepensky, P, Unal, A, Amrolia, P, Perez Martinez, A, Rialland, F, Aljurf, M, Isgro, A, Toren, A, Bierings, M, Corbacioglu, S, Kalwak, K, Wachowiak J., Galimard J. -E., Dalissier A., Rihani R., AlSaedi H., Wynn R. F., Dalle J. -H., Peffault de Latour R., Sedlacek P., Balduzzi A., Schroeder T., Bodova I., Gonzalez Vicent M., Gruhn B., Hamladji R. -M., Krivan G., Patrick K., Sobkowiak-Sobierajska A., Stepensky P., Unal A., Amrolia P., Perez Martinez A., Rialland F., Aljurf M., Isgro A., Toren A., Bierings M., Corbacioglu S., and Kalwak K.
Abstract: This retrospective study evaluated 35 children (median age 5.2 years; range 0.4–18) with myelofibrosis (MF), including 33 with primary myelofibrosis and 2 with secondary myelofibrosis transplanted from matched sibling donor (MSD) (n = 17) or non-MSD (n = 18) between 2000 and 2022. Conditioning was usually chemotherapy-based (n = 33) and myeloablative (n = 32). Fifteen patients received bone marrow (BM), 14 haematopoietic cells (HC) from peripheral blood (PB), and 6 from cord blood (CB). Day +100 acute GvHD II–IV incidence was significantly lower after MSD-haematopoietic cell transplantation (MSD-HCT) than after non-MSD-HCT [18.8% (4.3–41.1) vs 58.8% (31–78.6); p = 0.01]. Six-year non-relapse mortality (NRM) was 18% (7.1–32.8), relapse incidence was 15.9% (5.6–30.9), progression-free survival (PFS) was 66.1% (47–79.7), GvHD-free relapse-free survival was 50% (30.6–66.7), and overall survival (OS) was 71.1% (51.4–84). Six-year PFS and OS were significantly higher after BM transplantation compared to HCT from other sources [85.1% (52.3–96.1) vs 50.8% (26.3–71), p = 0.03, and 90.9% (50.8–98.7) vs 54% (28.1–74.2), p = 0.01, respectively], whereas NRM was significantly lower [0% vs 32% (12.3–53.9); p = 0.02]. This first multicentre study on outcomes of allogeneic HCT in children with myelofibrosis proves feasibility and curative effect of transplantation in these children, suggests that bone marrow transplantation is associated with better outcomes, and indicates the need for further studies.
Published: 2024

2. Supportive Care During Pediatric Hematopoietic Stem Cell Transplantation: Prevention of Infections. A Report From Workshops on Supportive Care of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author: Ifversen, M, Meisel, R, Sedlacek, P, Kalwak, K, Sisinni, L, Hutt, D, Lehrnbecher, T, Balduzzi, A, Diesch, T, Jarisch, A, Gungor, T, Stein, J, Yaniv, I, Bonig, H, Kuhlen, M, Ansari, M, Nava, T, Dalle, J, Diaz-de-Heredia, C, Trigoso, E, Falkenberg, U, Hartmann, M, Deiana, M, Canesi, M, Broggi, C, Bertaina, A, Gibson, B, Krivan, G, Vettenranta, K, Matic, T, Buechner, J, Lawitschka, A, Peters, C, Yesilipek, A, Yalcin, K, Lucchini, G, Bakhtiar, S, Turkiewicz, D, Niinimaki, R, Wachowiak, J, Cesaro, S, Dalissier, A, Corbacioglu, S, Willasch, A, Bader, P, Ifversen M., Meisel R., Sedlacek P., Kalwak K., Sisinni L., Hutt D., Lehrnbecher T., Balduzzi A., Diesch T., Jarisch A., Gungor T., Stein J., Yaniv I., Bonig H., Kuhlen M., Ansari M., Nava T., Dalle J. -H., Diaz-de-Heredia C., Trigoso E., Falkenberg U., Hartmann M., Deiana M., Canesi M., Broggi C., Bertaina A., Gibson B., Krivan G., Vettenranta K., Matic T., Buechner J., Lawitschka A., Peters C., Yesilipek A., Yalcin K., Lucchini G., Bakhtiar S., Turkiewicz D., Niinimaki R., Wachowiak J., Cesaro S., Dalissier A., Corbacioglu S., Willasch A. M., Bader P., Ifversen, M, Meisel, R, Sedlacek, P, Kalwak, K, Sisinni, L, Hutt, D, Lehrnbecher, T, Balduzzi, A, Diesch, T, Jarisch, A, Gungor, T, Stein, J, Yaniv, I, Bonig, H, Kuhlen, M, Ansari, M, Nava, T, Dalle, J, Diaz-de-Heredia, C, Trigoso, E, Falkenberg, U, Hartmann, M, Deiana, M, Canesi, M, Broggi, C, Bertaina, A, Gibson, B, Krivan, G, Vettenranta, K, Matic, T, Buechner, J, Lawitschka, A, Peters, C, Yesilipek, A, Yalcin, K, Lucchini, G, Bakhtiar, S, Turkiewicz, D, Niinimaki, R, Wachowiak, J, Cesaro, S, Dalissier, A, Corbacioglu, S, Willasch, A, Bader, P, Ifversen M., Meisel R., Sedlacek P., Kalwak K., Sisinni L., Hutt D., Lehrnbecher T., Balduzzi A., Diesch T., Jarisch A., Gungor T., Stein J., Yaniv I., Bonig H., Kuhlen M., Ansari M., Nava T., Dalle J. -H., Diaz-de-Heredia C., Trigoso E., Falkenberg U., Hartmann M., Deiana M., Canesi M., Broggi C., Bertaina A., Gibson B., Krivan G., Vettenranta K., Matic T., Buechner J., Lawitschka A., Peters C., Yesilipek A., Yalcin K., Lucchini G., Bakhtiar S., Turkiewicz D., Niinimaki R., Wachowiak J., Cesaro S., Dalissier A., Corbacioglu S., Willasch A. M., and Bader P.
Abstract: Specific protocols define eligibility, conditioning, donor selection, graft composition and prophylaxis of graft vs. host disease for children and young adults undergoing hematopoietic stem cell transplant (HSCT). However, international protocols rarely, if ever, detail supportive care, including pharmaceutical infection prophylaxis, physical protection with face masks and cohort isolation or food restrictions. Supportive care suffers from a lack of scientific evidence and implementation of practices in the transplant centers brings extensive restrictions to the child's and family's daily life after HSCT. Therefore, the Board of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) held a series of dedicated workshops since 2017 with the aim of initiating the production of a set of minimal recommendations. The present paper describes the consensus reached within the field of infection prophylaxis.
Published: 2021

3. Correction: ABO incompatibile graft management in pediatric transplantation (Bone Marrow Transplantation, (2020), 10.1038/s41409-020-0981-7)

Author: Balduzzi A., Balduzzi, A, Bonig, H, Jarisch, A, Nava, T, Ansari, M, Cattoni, A, Prunotto, G, Lucchini, G, Krivan, G, Matic, T, Kalwak, K, Yesilipek, A, Ifversen, M, Svec, P, Buechner, J, Vettenranta, K, Meisel, R, Lawitschka, A, Peters, C, Gibson, B, Dalissier, A, Corbacioglu, S, Willasch, A, Dalle, J, Bader, P, Bielorai, B, Cesaro, S, Diesch, T, Kato, K, Gomez, S, Mellgren, K, Palma, J, Rossig, C, Sauer, M, Sedlacek, P, Shaw, P, Schultz, K, Stein, J, Tayfun, G, Turkiewicz, D, Wachowiak, J, Balduzzi A., Bonig H., Jarisch A., Nava T., Ansari M., Cattoni A., Prunotto G., Lucchini G., Krivan G., Matic T., Kalwak K., Yesilipek A., Ifversen M., Svec P., Buechner J., Vettenranta K., Meisel R., Lawitschka A., Peters C., Gibson B., Dalissier A., Corbacioglu S., Willasch A., Dalle J. -H., Bader P., Bielorai B., Cesaro S., Diesch T., Kato K., Gomez S., Mellgren K., Palma J., Rossig C., Sauer M., Sedlacek P., Shaw P., Schultz K., Stein J., Tayfun G., Turkiewicz D., Wachowiak J., Balduzzi A., Balduzzi, A, Bonig, H, Jarisch, A, Nava, T, Ansari, M, Cattoni, A, Prunotto, G, Lucchini, G, Krivan, G, Matic, T, Kalwak, K, Yesilipek, A, Ifversen, M, Svec, P, Buechner, J, Vettenranta, K, Meisel, R, Lawitschka, A, Peters, C, Gibson, B, Dalissier, A, Corbacioglu, S, Willasch, A, Dalle, J, Bader, P, Bielorai, B, Cesaro, S, Diesch, T, Kato, K, Gomez, S, Mellgren, K, Palma, J, Rossig, C, Sauer, M, Sedlacek, P, Shaw, P, Schultz, K, Stein, J, Tayfun, G, Turkiewicz, D, Wachowiak, J, Balduzzi A., Bonig H., Jarisch A., Nava T., Ansari M., Cattoni A., Prunotto G., Lucchini G., Krivan G., Matic T., Kalwak K., Yesilipek A., Ifversen M., Svec P., Buechner J., Vettenranta K., Meisel R., Lawitschka A., Peters C., Gibson B., Dalissier A., Corbacioglu S., Willasch A., Dalle J. -H., Bader P., Bielorai B., Cesaro S., Diesch T., Kato K., Gomez S., Mellgren K., Palma J., Rossig C., Sauer M., Sedlacek P., Shaw P., Schultz K., Stein J., Tayfun G., Turkiewicz D., and Wachowiak J.
Abstract: An amendment to this paper has been published and can be accessed via a link at the top of the paper.
Published: 2021

4. Correction: Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study (Bone Marrow Transplantation, (2020), 55, 8, (1540-1551), 10.1038/s41409-020-0854-0)

Author: Willasch A. M., Willasch, A, Peters, C, Sedlacek, P, Dalle, J, Kitra-Roussou, V, Yesilipek, A, Wachowiak, J, Lankester, A, Prete, A, Hamidieh, A, Ifversen, M, Buechner, J, Krivan, G, Hamladji, R, Diaz-de-Heredia, C, Skorobogatova, E, Michel, G, Locatelli, F, Bertaina, A, Veys, P, Dupont, S, Or, R, Gungor, T, Aleinikova, O, Sufliarska, S, Sundin, M, Rascon, J, Kaare, A, Nemet, D, Fagioli, F, Klingebiel, T, Styczynski, J, Bierings, M, Nagy, K, Abecasis, M, Afanasyev, B, Ansari, M, Vettenranta, K, Alseraihy, A, Chybicka, A, Robinson, S, Bertrand, Y, Kupesiz, A, Ghavamzadeh, A, Campos, A, Pichler, H, Dalissier, A, Labopin, M, Corbacioglu, S, Balduzzi, A, Galimard, J, Bader, P, Willasch A. M., Peters C., Sedlacek P., Dalle J. -H., Kitra-Roussou V., Yesilipek A., Wachowiak J., Lankester A., Prete A., Hamidieh A. A., Ifversen M., Buechner J., Krivan G., Hamladji R. -M., Diaz-de-Heredia C., Skorobogatova E., Michel G., Locatelli F., Bertaina A., Veys P., Dupont S., Or R., Gungor T., Aleinikova O., Sufliarska S., Sundin M., Rascon J., Kaare A., Nemet D., Fagioli F., Klingebiel T. E., Styczynski J., Bierings M., Nagy K., Abecasis M., Afanasyev B., Ansari M., Vettenranta K., Alseraihy A., Chybicka A., Robinson S., Bertrand Y., Kupesiz A., Ghavamzadeh A., Campos A., Pichler H., Dalissier A., Labopin M., Corbacioglu S., Balduzzi A., Galimard J. -E., Bader P., Willasch A. M., Willasch, A, Peters, C, Sedlacek, P, Dalle, J, Kitra-Roussou, V, Yesilipek, A, Wachowiak, J, Lankester, A, Prete, A, Hamidieh, A, Ifversen, M, Buechner, J, Krivan, G, Hamladji, R, Diaz-de-Heredia, C, Skorobogatova, E, Michel, G, Locatelli, F, Bertaina, A, Veys, P, Dupont, S, Or, R, Gungor, T, Aleinikova, O, Sufliarska, S, Sundin, M, Rascon, J, Kaare, A, Nemet, D, Fagioli, F, Klingebiel, T, Styczynski, J, Bierings, M, Nagy, K, Abecasis, M, Afanasyev, B, Ansari, M, Vettenranta, K, Alseraihy, A, Chybicka, A, Robinson, S, Bertrand, Y, Kupesiz, A, Ghavamzadeh, A, Campos, A, Pichler, H, Dalissier, A, Labopin, M, Corbacioglu, S, Balduzzi, A, Galimard, J, Bader, P, Willasch A. M., Peters C., Sedlacek P., Dalle J. -H., Kitra-Roussou V., Yesilipek A., Wachowiak J., Lankester A., Prete A., Hamidieh A. A., Ifversen M., Buechner J., Krivan G., Hamladji R. -M., Diaz-de-Heredia C., Skorobogatova E., Michel G., Locatelli F., Bertaina A., Veys P., Dupont S., Or R., Gungor T., Aleinikova O., Sufliarska S., Sundin M., Rascon J., Kaare A., Nemet D., Fagioli F., Klingebiel T. E., Styczynski J., Bierings M., Nagy K., Abecasis M., Afanasyev B., Ansari M., Vettenranta K., Alseraihy A., Chybicka A., Robinson S., Bertrand Y., Kupesiz A., Ghavamzadeh A., Campos A., Pichler H., Dalissier A., Labopin M., Corbacioglu S., Balduzzi A., Galimard J. -E., and Bader P.
Abstract: The article “Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study,” written by Andre Manfred Willasch, Christina Peters, Petr Sedlácek, Jean-Hugues Dalle, Vassiliki Kitra-Roussou, Akif Yesilipek, Jacek Wachowiak, Arjan Lankester, Arcangelo Prete, Amir Ali Hamidieh, Marianne Ifversen, Jochen Buechner, Gergely Kriván, Rose-Marie Hamladji, Cristina Diaz-de-Heredia, Elena Skorobogatova, Gérard Michel, Franco Locatelli, Alice Bertaina, Paul Veys, Sophie Dupont, Reuven Or, Tayfun Güngör, Olga Aleinikova, Sabina Sufliarska, Mikael Sundin, Jelena Rascon, Ain Kaare, Damir Nemet, Franca Fagioli, Thomas Erich Klingebiel, Jan Styczynski, Marc Bierings, Kálmán Nagy, Manuel Abecasis, Boris Afanasyev, Marc Ansari, Kim Vettenranta, Amal Alseraihy, Alicja Chybicka, Stephen Robinson, Yves Bertrand, Alphan Kupesiz, Ardeshir Ghavamzadeh, Antonio Campos, Herbert Pichler, Arnaud Dalissier, Myriam Labopin, Selim Corbacioglu, Adriana Balduzzi, Jacques-Emmanuel Galimard, Peter Bader, on behalf of the EBMT Paediatric Diseases Working Party, was originally published online first without Open Access. After publication in volume 55, issue 8, page 1540–1551, the author decided to opt for Open Choice and to make the article an Open Access publication. Therefore, the copyright of the article has been changed to © The Author(s) 2020 and the article is forthwith distributed under the terms of the Creative Commons Attribution 4.0 InternationalS License, which permits use, sharing, adaptation, distribution, and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third-party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Comm
Published: 2021

5. Correction: Supportive care during pediatric hematopoietic stem cell transplantation: beyond infectious diseases. A report from workshops on supportive care of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) (Bone Marrow Transplantation, (2020), 55, 6, (1126-1136), 10.1038/s41409-020-0818-4)

Author: Nava T., Nava, T, Ansari, M, Dalle, J, de Heredia, C, Gungor, T, Trigoso, E, Falkenberg, U, Bertaina, A, Gibson, B, Jarisch, A, Balduzzi, A, Boenig, H, Krivan, G, Vettenranta, K, Matic, T, Buechner, J, Kalwak, K, Lawitschka, A, Yesilipek, A, Lucchini, G, Peters, C, Turkiewicz, D, Niinimaki, R, Diesch, T, Lehrnbecher, T, Sedlacek, P, Hutt, D, Dalissier, A, Wachowiak, J, Yaniv, I, Stein, J, Yalcin, K, Sisinni, L, Deiana, M, Ifversen, M, Kuhlen, M, Meisel, R, Bakhtiar, S, Cesaro, S, Willasch, A, Corbacioglu, S, Bader, P, Nava T., Ansari M., Dalle J. -H., de Heredia C. D., Gungor T., Trigoso E., Falkenberg U., Bertaina A., Gibson B., Jarisch A., Balduzzi A., Boenig H., Krivan G., Vettenranta K., Matic T., Buechner J., Kalwak K., Lawitschka A., Yesilipek A., Lucchini G., Peters C., Turkiewicz D., Niinimaki R., Diesch T., Lehrnbecher T., Sedlacek P., Hutt D., Dalissier A., Wachowiak J., Yaniv I., Stein J., Yalcin K., Sisinni L., Deiana M., Ifversen M., Kuhlen M., Meisel R., Bakhtiar S., Cesaro S., Willasch A., Corbacioglu S., Bader P., Nava T., Nava, T, Ansari, M, Dalle, J, de Heredia, C, Gungor, T, Trigoso, E, Falkenberg, U, Bertaina, A, Gibson, B, Jarisch, A, Balduzzi, A, Boenig, H, Krivan, G, Vettenranta, K, Matic, T, Buechner, J, Kalwak, K, Lawitschka, A, Yesilipek, A, Lucchini, G, Peters, C, Turkiewicz, D, Niinimaki, R, Diesch, T, Lehrnbecher, T, Sedlacek, P, Hutt, D, Dalissier, A, Wachowiak, J, Yaniv, I, Stein, J, Yalcin, K, Sisinni, L, Deiana, M, Ifversen, M, Kuhlen, M, Meisel, R, Bakhtiar, S, Cesaro, S, Willasch, A, Corbacioglu, S, Bader, P, Nava T., Ansari M., Dalle J. -H., de Heredia C. D., Gungor T., Trigoso E., Falkenberg U., Bertaina A., Gibson B., Jarisch A., Balduzzi A., Boenig H., Krivan G., Vettenranta K., Matic T., Buechner J., Kalwak K., Lawitschka A., Yesilipek A., Lucchini G., Peters C., Turkiewicz D., Niinimaki R., Diesch T., Lehrnbecher T., Sedlacek P., Hutt D., Dalissier A., Wachowiak J., Yaniv I., Stein J., Yalcin K., Sisinni L., Deiana M., Ifversen M., Kuhlen M., Meisel R., Bakhtiar S., Cesaro S., Willasch A., Corbacioglu S., and Bader P.
Abstract: An amendment to this paper has been published and can be accessed via a link at the top of the paper.
Published: 2020

6. A Multi-center, Open-Label, Single-Arm Trial to Evaluate the Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency (Jan, 10.1007/s10875-021-01181-6, 2022)

Author: Santamaria, M, Neth, O, Douglass, JA, Krivan, G, Kobbe, R, Bernatowska, E, Grigoriadou, S, Bethune, C, Chandra, A, Horneff, G, Borte, M, Sonnenschein, A, Kralickova, P, Ramon, SS, Langguth, DD, Gonzalez-Granado, LI, Alsina, L, Querolt, M, Griffin, R, Hames, C, Mondou, E, Price, J, Sanz, A, Lin, J, Santamaria, M, Neth, O, Douglass, JA, Krivan, G, Kobbe, R, Bernatowska, E, Grigoriadou, S, Bethune, C, Chandra, A, Horneff, G, Borte, M, Sonnenschein, A, Kralickova, P, Ramon, SS, Langguth, DD, Gonzalez-Granado, LI, Alsina, L, Querolt, M, Griffin, R, Hames, C, Mondou, E, Price, J, Sanz, A, and Lin, J
Published: 2022

7. A Multi-Center, Open-Label, Single-Arm Trial to Evaluate the Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency

Author: Santamaria, M, Neth, O, Douglass, JA, Krivan, G, Kobbe, R, Bernatowska, E, Grigoriadou, S, Bethune, C, Chandra, A, Horneff, G, Borte, M, Sonnenschein, A, Kralickova, P, Ramon, SS, Langguth, D, Gonzalez-Granado, LI, Alsina, L, Querolt, M, Griffin, R, Hames, C, Mondou, E, Price, J, Sanz, A, Lin, J, Santamaria, M, Neth, O, Douglass, JA, Krivan, G, Kobbe, R, Bernatowska, E, Grigoriadou, S, Bethune, C, Chandra, A, Horneff, G, Borte, M, Sonnenschein, A, Kralickova, P, Ramon, SS, Langguth, D, Gonzalez-Granado, LI, Alsina, L, Querolt, M, Griffin, R, Hames, C, Mondou, E, Price, J, Sanz, A, and Lin, J
Abstract: PURPOSE: The purpose of this phase 3 study was to evaluate the efficacy, pharmacokinetics (PK), and safety of Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) in patients with primary immunodeficiency (PI). METHODS: Immunoglobulin treatment-experienced subjects with PI received 52 weeks of IGSC 20% given weekly at the same dose as the subject's previous IgG regimen (DAF 1:1); the minimum dose was 100 mg/kg/week. The primary endpoint was serious bacterial infections (SBIs [null vs alternative hypothesis: SBI rate per person per year ≥ 1 vs < 1]). IgG subclasses and specific pathogen antibody levels were also measured. RESULTS: Sixty-one subjects (19 children [≤ 12 years], 10 adolescents [> 12-16 years], and 32 adults) were enrolled. The rate of SBIs per person per year was 0.017. The 1-sided 99% upper confidence limit was 0.036 (< 1), and the null hypothesis was rejected. The rate of hospitalization due to infection per person per year was 0.017 (2-sided 95% confidence interval: 0.008-0.033) overall. The mean trough total IgG concentrations were comparable to the previous IgG replacement regimen. The average of the individual mean trough ratios (IGSC 20%:previous regimen) was 1.078 (range: 0.83-1.54). The average steady-state mean trough IgG concentrations were 947.64 and 891.37 mg/dL, respectively. Seven subjects had serious treatment-emergent adverse events (TEAEs); none was drug-related. The rate of all TEAEs, including local infusion site reactions, during 3045 IGSC 20% infusions was 0.135. Most TEAEs were mild or moderate. CONCLUSIONS: IGSC 20% demonstrated efficacy and good safety and tolerability in subjects with PI.
Published: 2022

8. Disease Progression of WHIM Syndrome in an International Cohort of 66 Pediatric and Adult Patients

Author: Geier, CB, Ellison, M, Cruz, R, Pawar, S, Leiss-Piller, A, Zmajkovicova, K, McNulty, SM, Yilmaz, M, Evans, MO, Gordon, S, Ujhazi, B, Wiest, I, Abolhassani, H, Aghamohammadi, A, Barmettler, S, Bhar, S, Bondarenko, A, Bolyard, AA, Buchbinder, D, Cada, M, Cavieres, M, Connelly, JA, Dale, DC, Deordieva, E, Dorsey, MJ, Drysdale, SB, Ehl, S, Elfeky, R, Fioredda, F, Firkin, F, Forster-Waldl, E, Geng, B, Goda, V, Gonzalez-Granado, L, Grunebaum, E, Grzesk, E, Henrickson, SE, Hilfanova, A, Hiwatari, M, Imai, C, Ip, W, Jyonouchi, S, Kanegane, H, Kawahara, Y, Khojah, AM, Kim, VH-D, Kojic, M, Koltan, S, Krivan, G, Langguth, D, Lau, Y-L, Leung, D, Miano, M, Mersyanova, I, Mousallem, T, Muskat, M, Naoum, FA, Noronha, SA, Ouederni, M, Ozono, S, Richmond, GW, Sakovich, I, Salzer, U, Schuetz, C, Seeborg, FO, Sharapova, SO, Sockel, K, Volokha, A, von Bonin, M, Warnatz, K, Wegehaupt, O, Weinberg, GA, Wong, K-J, Worth, A, Yu, H, Zharankova, Y, Zhao, X, Devlin, L, Badarau, A, Csomos, K, Keszei, M, Pereira, J, Taveras, AG, Beaussant-Cohen, SL, Ong, M-S, Shcherbina, A, Walter, JE, Geier, CB, Ellison, M, Cruz, R, Pawar, S, Leiss-Piller, A, Zmajkovicova, K, McNulty, SM, Yilmaz, M, Evans, MO, Gordon, S, Ujhazi, B, Wiest, I, Abolhassani, H, Aghamohammadi, A, Barmettler, S, Bhar, S, Bondarenko, A, Bolyard, AA, Buchbinder, D, Cada, M, Cavieres, M, Connelly, JA, Dale, DC, Deordieva, E, Dorsey, MJ, Drysdale, SB, Ehl, S, Elfeky, R, Fioredda, F, Firkin, F, Forster-Waldl, E, Geng, B, Goda, V, Gonzalez-Granado, L, Grunebaum, E, Grzesk, E, Henrickson, SE, Hilfanova, A, Hiwatari, M, Imai, C, Ip, W, Jyonouchi, S, Kanegane, H, Kawahara, Y, Khojah, AM, Kim, VH-D, Kojic, M, Koltan, S, Krivan, G, Langguth, D, Lau, Y-L, Leung, D, Miano, M, Mersyanova, I, Mousallem, T, Muskat, M, Naoum, FA, Noronha, SA, Ouederni, M, Ozono, S, Richmond, GW, Sakovich, I, Salzer, U, Schuetz, C, Seeborg, FO, Sharapova, SO, Sockel, K, Volokha, A, von Bonin, M, Warnatz, K, Wegehaupt, O, Weinberg, GA, Wong, K-J, Worth, A, Yu, H, Zharankova, Y, Zhao, X, Devlin, L, Badarau, A, Csomos, K, Keszei, M, Pereira, J, Taveras, AG, Beaussant-Cohen, SL, Ong, M-S, Shcherbina, A, and Walter, JE
Abstract: Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome (WS) is a combined immunodeficiency caused by gain-of-function mutations in the C-X-C chemokine receptor type 4 (CXCR4) gene. We characterize a unique international cohort of 66 patients, including 57 (86%) cases previously unreported, with variable clinical phenotypes. Of 17 distinct CXCR4 genetic variants within our cohort, 11 were novel pathogenic variants affecting 15 individuals (23%). All variants affect the same CXCR4 region and impair CXCR4 internalization resulting in hyperactive signaling. The median age of diagnosis in our cohort (5.5 years) indicates WHIM syndrome can commonly present in childhood, although some patients are not diagnosed until adulthood. The prevalence and mean age of recognition and/or onset of clinical manifestations within our cohort were infections 88%/1.6 years, neutropenia 98%/3.8 years, lymphopenia 88%/5.0 years, and warts 40%/12.1 years. However, we report greater prevalence and variety of autoimmune complications of WHIM syndrome (21.2%) than reported previously. Patients with versus without family history of WHIM syndrome were diagnosed earlier (22%, average age 1.3 years versus 78%, average age 5 years, respectively). Patients with a family history of WHIM syndrome also received earlier treatment, experienced less hospitalization, and had less end-organ damage. This observation reinforces previous reports that early treatment for WHIM syndrome improves outcomes. Only one patient died; death was attributed to complications of hematopoietic stem cell transplantation. The variable expressivity of WHIM syndrome in pediatric patients delays their diagnosis and therapy. Early-onset bacterial infections with severe neutropenia and/or lymphopenia should prompt genetic testing for WHIM syndrome, even in the absence of warts.
Published: 2022

9. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

Author: Albert, M. H., Sirait, T., Eikema, D. -J., Bakunina, K., Wehr, C., Suarez, F., Fox, M. L., Mahlaoui, N., Gennery, A. R., Lankester, A. C., Beier, R., Bernardo, M. E., Bigley, V., Lindemans, C. A., Burns, S. O., Carpenter, B., Dybko, J., Gungor, T., Hauck, F., Lum, S. H., Balashov, D., Meisel, R., Moshous, D., Schulz, A., Speckmann, C., Slatter, M. A., Strahm, B., Uckan-Cetinkaya, D., Meyts, I., Vallee, T. C., Wynn, R., Neven, B., Morris, E. C., Aiuti, A., Maschan, A., Aljurf, M., Gedde-Dahl, T., Gurman, G., Bordon, V., Krivan, G., Locatelli, Franco, Porta, F., Valcarcel, D., Beguin, Y., Faraci, M., Kroger, N., Kulagin, A., Shaw, P. J., Veelken, J. H., Diaz de Heredia, C., Fagioli, F., Felber, M., Gruhn, B., Holter, W., Rossig, C., Sedlacek, P., Apperley, J., Ayas, M., Bodova, I., Choi, G., Cornelissen, J. J., Sirvent, A., Khan, A., Kupesiz, A., Lenhoff, S., Ozdogu, H., von der Weid, N., Rovira, M., Schots, R., Vinh, D. C., Locatelli F. (ORCID:0000-0002-7976-3654), Albert, M. H., Sirait, T., Eikema, D. -J., Bakunina, K., Wehr, C., Suarez, F., Fox, M. L., Mahlaoui, N., Gennery, A. R., Lankester, A. C., Beier, R., Bernardo, M. E., Bigley, V., Lindemans, C. A., Burns, S. O., Carpenter, B., Dybko, J., Gungor, T., Hauck, F., Lum, S. H., Balashov, D., Meisel, R., Moshous, D., Schulz, A., Speckmann, C., Slatter, M. A., Strahm, B., Uckan-Cetinkaya, D., Meyts, I., Vallee, T. C., Wynn, R., Neven, B., Morris, E. C., Aiuti, A., Maschan, A., Aljurf, M., Gedde-Dahl, T., Gurman, G., Bordon, V., Krivan, G., Locatelli, Franco, Porta, F., Valcarcel, D., Beguin, Y., Faraci, M., Kroger, N., Kulagin, A., Shaw, P. J., Veelken, J. H., Diaz de Heredia, C., Fagioli, F., Felber, M., Gruhn, B., Holter, W., Rossig, C., Sedlacek, P., Apperley, J., Ayas, M., Bodova, I., Choi, G., Cornelissen, J. J., Sirvent, A., Khan, A., Kupesiz, A., Lenhoff, S., Ozdogu, H., von der Weid, N., Rovira, M., Schots, R., Vinh, D. C., and Locatelli F. (ORCID:0000-0002-7976-3654)
Abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) is the gold standard curative therapy for infants and children with many inborn errors of immunity (IEI), but adolescents and adults with IEI are rarely referred for transplant. Lack of published HSCT outcome data outside small, single-center studies and perceived high risk of transplant-related mortality have delayed the adoption of HSCT for IEI patients presenting or developing significant organ damage later in life. This large retrospective, multicenter HSCT outcome study reports on 329 IEI patients (age range, 15-62.5 years at HSCT). Patients underwent first HSCT between 2000 and 2019. Primary endpoints were overall survival (OS) and event-free survival (EFS). We also evaluated the influence of IEI-subgroup and IEI-specific risk factors at HSCT, including infections, bronchiectasis, colitis, malignancy, inflammatory lung disease, splenectomy, hepatic dysfunction, and systemic immunosuppression. At a median follow-up of 44.3 months, the estimated OS at 1 and 5 years post-HSCT for all patients was 78% and 71%, and EFS was 65% and 62%, respectively, with low rates of severe acute (8%) or extensive chronic (7%) graft-versus-host disease. On univariate analysis, OS and EFS were inferior in patients with primary antibody deficiency, bronchiectasis, prior splenectomy, hepatic comorbidity, and higher hematopoietic cell transplant comorbidity index scores. On multivariable analysis, EFS was inferior in those with a higher number of IEI-associated complications. Neither age nor donor had a significant effect on OS or EFS. We have identified age-independent risk factors for adverse outcome, providing much needed evidence to identify which patients are most likely to benefit from HSCT.
Published: 2022

10. A Multi-Center, Open-Label, Single-Arm Trial to Evaluate the Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency

Author: Santamaria M, Neth O, Douglass JA, Krivan G, Kobbe R, Bernatowska E, Grigoriadou S, Bethune C, Chandra A, Horneff G, Borte M, Sonnenschein A, Kralickova P, Ramón SS, Langguth D, Gonzalez-Granado LI, Alsina L, Querolt M, Griffin R, Hames C, Mondou E, Price J, Sanz A, and Lin J
Subjects: Primary immunodeficiency, immunoglobulin replacement therapy, GTI1503, subcutaneous, 20% immunoglobulin
Abstract: Purpose The purpose of this phase 3 study was to evaluate the efficacy, pharmacokinetics (PK), and safety of Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) in patients with primary immunodeficiency (PI). Methods Immunoglobulin treatment-experienced subjects with PI received 52 weeks of IGSC 20% given weekly at the same dose as the subject's previous IgG regimen (DAF 1:1); the minimum dose was 100 mg/kg/week. The primary endpoint was serious bacterial infections (SBIs [null vs alternative hypothesis: SBI rate per person per year >= 1 vs < 1]). IgG subclasses and specific pathogen antibody levels were also measured. Results Sixty-one subjects (19 children [ 12-16 years], and 32 adults) were enrolled. The rate of SBIs per person per year was 0.017. The 1-sided 99% upper confidence limit was 0.036 (< 1), and the null hypothesis was rejected. The rate of hospitalization due to infection per person per year was 0.017 (2-sided 95% confidence interval: 0.008-0.033) overall. The mean trough total IgG concentrations were comparable to the previous IgG replacement regimen. The average of the individual mean trough ratios (IGSC 20%:previous regimen) was 1.078 (range: 0.83-1.54). The average steady-state mean trough IgG concentrations were 947.64 and 891.37 mg/dL, respectively. Seven subjects had serious treatment-emergent adverse events (TEAEs); none was drug-related. The rate of all TEAEs, including local infusion site reactions, during 3045 IGSC 20% infusions was 0.135. Most TEAEs were mild or moderate. Conclusions IGSC 20% demonstrated efficacy and good safety and tolerability in subjects with PI.
Published: 2022

11. In a high-dose melphalan setting, palifermin compared with placebo had no effect on oral mucositis or related patient’s burden

Author: Blijlevens, N, de Château, M, Krivan, G, Rabitsch, W, Szomor, A, Pytlik, R, Lissmats, A, Johnsen, H E, de Witte, T, Einsele, H, Ruutu, T, and Niederwieser, D
Published: 2013
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12. Correction: Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study (Bone Marrow Transplantation, (2020), 55, 8, (1540-1551), 10.1038/s41409-020-0854-0)

Author: Willasch, A. M., Peters, C., Sedlacek, P., Dalle, J. -H., Kitra-Roussou, V., Yesilipek, A., Wachowiak, J., Lankester, A., Prete, A., Hamidieh, A. A., Ifversen, M., Buechner, J., Krivan, G., Hamladji, R. -M., Diaz-de-Heredia, C., Skorobogatova, E., Michel, G., Locatelli, F., Bertaina, A., Veys, P., Dupont, S., Or, R., Gungor, T., Aleinikova, O., Sufliarska, S., Sundin, M., Rascon, J., Kaare, A., Nemet, D., Fagioli, F., Klingebiel, T. E., Styczynski, J., Bierings, M., Nagy, K., Abecasis, M., Afanasyev, B., Ansari, M., Vettenranta, K., Alseraihy, A., Chybicka, A., Robinson, S., Bertrand, Y., Kupesiz, A., Ghavamzadeh, A., Campos, A., Pichler, H., Dalissier, A., Labopin, M., Corbacioglu, S., Balduzzi, A., Galimard, J. -E., and Bader, P.
Subjects: Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, conditioning regimens
Published: 2021

13. ABO incompatibile graft management in pediatric transplantation

Author: Balduzzi, A, Bönig, H, Järisch, A, Nava, T, Ansari, M, Cattoni, A, Prunotto, G, Lucchini, G, Krivan, G, Matic, T, Kalwak, K, Yesilipek, A, Ifversen, M, Svec, P, Büchner, J, Vettenranta, K, Meisel, R, Lawitschka, A, Peters, C, Gibson, B, Dalissier, A, Corbacioglu, S, Willasch, A, Dalle, J, Bader, P, Balduzzi, Adriana, Bönig, Halvard, Järisch, Andrea, Nava, Tiago, Ansari, Marc, Cattoni, Alessandro, Prunotto, Giulia, Lucchini, Giovanna, Krivan, Gergely, Matic, Toni, Kalwak, Krzyzstof, Yesilipek, Akif, Ifversen, Marianne, Svec, Peter, Büchner, Jochen, Vettenranta, Kim, Meisel, Roland, Lawitschka, Anita, Peters, Christina, Gibson, Brenda, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, André, Dalle, Jean-Hugues, Bader, Peter, Balduzzi, A, Bönig, H, Järisch, A, Nava, T, Ansari, M, Cattoni, A, Prunotto, G, Lucchini, G, Krivan, G, Matic, T, Kalwak, K, Yesilipek, A, Ifversen, M, Svec, P, Büchner, J, Vettenranta, K, Meisel, R, Lawitschka, A, Peters, C, Gibson, B, Dalissier, A, Corbacioglu, S, Willasch, A, Dalle, J, Bader, P, Balduzzi, Adriana, Bönig, Halvard, Järisch, Andrea, Nava, Tiago, Ansari, Marc, Cattoni, Alessandro, Prunotto, Giulia, Lucchini, Giovanna, Krivan, Gergely, Matic, Toni, Kalwak, Krzyzstof, Yesilipek, Akif, Ifversen, Marianne, Svec, Peter, Büchner, Jochen, Vettenranta, Kim, Meisel, Roland, Lawitschka, Anita, Peters, Christina, Gibson, Brenda, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, André, Dalle, Jean-Hugues, and Bader, Peter
Abstract: Up to 40% of donor-recipient pairs in SCT have some degree of ABO incompatibility, which may cause severe complications. The aim of this study was to describe available options and survey current practices by means of a questionnaire circulated within the EBMT Pediatric Diseases Working Party investigators. Major ABO incompatibility (donor’s RBCs have antigens missing on the recipient’s cell surface, towards which the recipient has circulating isohemagglutinins) requires most frequently an intervention in case of bone marrow grafts, as immediate or delayed hemolysis, delayed erythropoiesis and pure red cell aplasia may occur. RBC depletion from the graft (82%), recipient plasma-exchange (14%) were the most common practices, according to the survey. Graft manipulation is rarely needed in mobilized peripheral blood grafts. In case of minor incompatible grafts (donor has isohemagglutinins directed against recipient RBC antigens), isohemagglutinin depletion from the graft by plasma reduction/centrifugation may be considered, but acute tolerability of minor incompatible grafts is rarely an issue. According to the survey, minor ABO incompatibility was either managed by means of plasma removal from the graft, especially when isohemagglutinin titer was above a certain threshold, or led to no intervention at all (41%). Advantages and disadvantages of each method are discussed.
Published: 2021

14. Supportive care during pediatric hematopoietic stem cell transplantation: prevention of infections:a report from workshops on supportive care of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author: Ifversen, M. (Marianne), Meisel, R. (Roland), Sedlacek, P. (Petr), Kalwak, K. (Krzysztof), Sisinni, L. (Luisa), Hutt, D. (Daphna), Lehrnbecher, T. (Thomas), Balduzzi, A. (Adriana), Diesch, T. (Tamara), Jarisch, A. (Andrea), Güngör, T. (Tayfun), Stein, J. (Jerry), Yaniv, I. (Isaac), Bonig, H. (Halvard), Kuhlen, M. (Michaela), Ansari, M. (Marc), Nava, T. (Tiago), Dalle, J.-H. (Jean-Hugues), Diaz-de-Heredia, C. (Cristina), Trigoso, E. (Eugenia), Falkenberg, U. (Ulrike), Hartmann, M. (Mihaela), Deiana, M. (Marco), Canesi, M. (Marta), Broggi, C. (Chiara), Bertaina, A. (Alice), Gibson, B. (Brenda), Krivan, G. (Gergely), Vettenranta, K. (Kim), Matic, T. (Toni), Buechner, J. (Jochen), Lawitschka, A. (Anita), Peters, C. (Christina), Yesilipek, A. (Akif), Yalçin, K. (Koray), Lucchini, G. (Giovanna), Bakhtiar, S. (Shahrzad), Turkiewicz, D. (Dominik), Niinimäki, R. (Riitta), Wachowiak, J. (Jacek), Cesaro, S. (Simone), Dalissier, A. (Arnaud), Corbacioglu, S. (Selim), Willasch, A. M. (Andre Manfred), Bader, P. (Peter), Ifversen, M. (Marianne), Meisel, R. (Roland), Sedlacek, P. (Petr), Kalwak, K. (Krzysztof), Sisinni, L. (Luisa), Hutt, D. (Daphna), Lehrnbecher, T. (Thomas), Balduzzi, A. (Adriana), Diesch, T. (Tamara), Jarisch, A. (Andrea), Güngör, T. (Tayfun), Stein, J. (Jerry), Yaniv, I. (Isaac), Bonig, H. (Halvard), Kuhlen, M. (Michaela), Ansari, M. (Marc), Nava, T. (Tiago), Dalle, J.-H. (Jean-Hugues), Diaz-de-Heredia, C. (Cristina), Trigoso, E. (Eugenia), Falkenberg, U. (Ulrike), Hartmann, M. (Mihaela), Deiana, M. (Marco), Canesi, M. (Marta), Broggi, C. (Chiara), Bertaina, A. (Alice), Gibson, B. (Brenda), Krivan, G. (Gergely), Vettenranta, K. (Kim), Matic, T. (Toni), Buechner, J. (Jochen), Lawitschka, A. (Anita), Peters, C. (Christina), Yesilipek, A. (Akif), Yalçin, K. (Koray), Lucchini, G. (Giovanna), Bakhtiar, S. (Shahrzad), Turkiewicz, D. (Dominik), Niinimäki, R. (Riitta), Wachowiak, J. (Jacek), Cesaro, S. (Simone), Dalissier, A. (Arnaud), Corbacioglu, S. (Selim), Willasch, A. M. (Andre Manfred), and Bader, P. (Peter)
Abstract: Specific protocols define eligibility, conditioning, donor selection, graft composition and prophylaxis of graft vs. host disease for children and young adults undergoing hematopoietic stem cell transplant (HSCT). However, international protocols rarely, if ever, detail supportive care, including pharmaceutical infection prophylaxis, physical protection with face masks and cohort isolation or food restrictions. Supportive care suffers from a lack of scientific evidence and implementation of practices in the transplant centers brings extensive restrictions to the child’s and family’s daily life after HSCT. Therefore, the Board of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) held a series of dedicated workshops since 2017 with the aim of initiating the production of a set of minimal recommendations. The present paper describes the consensus reached within the field of infection prophylaxis.
Published: 2021

15. Treatment and Outcome Analysis of 639 Relapsed Non-Hodgkin Lymphomas in Children and Adolescents and Resulting Treatment Recommendations

Author: Burkhardt, B, Taj, M, Garnier, N, Minard-Colin, V, Hazar, V, Mellgren, K, Osumi, T, Fedorova, A, Myakova, N, Verdu-Amoros, J, Andres, M, Kabickova, E, Attarbaschi, A, Chiang, A, Bubanska, E, Donska, S, Hjalgrim, L, Wachowiak, J, Pieczonka, A, Uyttebroeck, A, Lazic, J, Loeffen, J, Buechner, J, Niggli, F, Csoka, M, Krivan, G, Palma, J, Burke, G, Beishuizen, A, Koeppen, K, Mueller, S, Herbrueggen, H, Woessmann, W, Zimmermann, M, Balduzzi, A, Pillon, M, Burkhardt, Birgit, Taj, Mary, Garnier, Nathalie, Minard-Colin, Veronique, Hazar, Volkan, Mellgren, Karin, Osumi, Tomoo, Fedorova, Alina, Myakova, Natalia, Verdu-Amoros, Jaime, Andres, Mara, Kabickova, Edita, Attarbaschi, Andishe, Chiang, Alan Kwok Shing, Bubanska, Eva, Donska, Svetlana, Hjalgrim, Lisa Lyngsie, Wachowiak, Jacek, Pieczonka, Anna, Uyttebroeck, Anne, Lazic, Jelena, Loeffen, Jan, Buechner, Jochen, Niggli, Felix, Csoka, Monika, Krivan, Gergely, Palma, Julia, Burke, G A Amos, Beishuizen, Auke, Koeppen, Kristin, Mueller, Stephanie, Herbrueggen, Heidi, Woessmann, Wilhelm, Zimmermann, Martin, Balduzzi, Adriana, Pillon, Marta, Burkhardt, B, Taj, M, Garnier, N, Minard-Colin, V, Hazar, V, Mellgren, K, Osumi, T, Fedorova, A, Myakova, N, Verdu-Amoros, J, Andres, M, Kabickova, E, Attarbaschi, A, Chiang, A, Bubanska, E, Donska, S, Hjalgrim, L, Wachowiak, J, Pieczonka, A, Uyttebroeck, A, Lazic, J, Loeffen, J, Buechner, J, Niggli, F, Csoka, M, Krivan, G, Palma, J, Burke, G, Beishuizen, A, Koeppen, K, Mueller, S, Herbrueggen, H, Woessmann, W, Zimmermann, M, Balduzzi, A, Pillon, M, Burkhardt, Birgit, Taj, Mary, Garnier, Nathalie, Minard-Colin, Veronique, Hazar, Volkan, Mellgren, Karin, Osumi, Tomoo, Fedorova, Alina, Myakova, Natalia, Verdu-Amoros, Jaime, Andres, Mara, Kabickova, Edita, Attarbaschi, Andishe, Chiang, Alan Kwok Shing, Bubanska, Eva, Donska, Svetlana, Hjalgrim, Lisa Lyngsie, Wachowiak, Jacek, Pieczonka, Anna, Uyttebroeck, Anne, Lazic, Jelena, Loeffen, Jan, Buechner, Jochen, Niggli, Felix, Csoka, Monika, Krivan, Gergely, Palma, Julia, Burke, G A Amos, Beishuizen, Auke, Koeppen, Kristin, Mueller, Stephanie, Herbrueggen, Heidi, Woessmann, Wilhelm, Zimmermann, Martin, Balduzzi, Adriana, and Pillon, Marta
Abstract: Despite poor survival, controversies remain in the treatment for refractory or relapsed pediatric non-Hodgkin lymphoma (r/r NHL). The current project aimed to collect international experience on the re-induction treatment of r/r NHL, hematopoietic stem cell transplantation (HSCT), risk factors associated with outcome, and to suggest treatment recommendations. Inclusion criteria were (i) refractory disease, disease progression or relapse of any NHL subtype except anaplastic large cell lymphoma, (ii) age < 18 years at initial diagnosis, (iii) diagnosis in/after January 2000. Data from 639 eligible patients were evaluable. The eight-year probability of overall survival was 34 ± 2% with highly significant differences according to NHL subtypes: 28 ± 3% for 254 Burkitt lymphoma/leukemia, 50 ± 6% for 98 diffuse large B-cell lymphomas, 57 ± 8% for 41 primary mediastinal large B-cell lymphomas, 27 ± 3% for 177 T-lymphoblastic lymphomas, 52 ± 10% for 34 precursor-B-cell lymphoblastic lymphomas and 30 ± 9% for 35 patients with rare NHL subtypes. Subtype-specific factors associated with survival and treatment recommendations are suggested. There were no survivors without HSCT, except in few very small subgroups. Conclusions: There is an urgent need to further improve survival in r/r NHL. The current study provides the largest real-world series, which underlines the role of HSCT and suggests treatment recommendations.
Published: 2021

16. OUTCOMES OF ALLOGENEIC CORD BLOOD TRANSPLANTATION FOR LEUKODYSTROPHIES; A JOINT STUDY OF EUROCORD AND “INBORN ERRORS WP-EBMT”: PH-O069

Author: Hol, J., Ruggeri, A., Rocha, V., Michel, G., Ayas, M., OʼBrien, T., Krivan, G., Bordon, V., Schots, R., Rovelli, A., Elorza, I., Moraleda, J. M., Shaw, P. J., Renard, M., Sykora, K.-W., Or, R., Zuckerman, T., Abecasis, M., Bredius, R., Yesilipek, A., Diaz, M. A., Gennery, A., Gluckman, E., and Boelens, J. J.
Published: 2014

17. Plerixafor combined with standard regimens for hematopoietic stem cell mobilization in pediatric patients with solid tumors eligible for autologous transplants: two-arm phase I/II study (MOZAIC)

Author: Morland, B., Kepak, T., Dallorso, S., Sevilla, J., Murphy, D., Luksch, R., Yaniv, I., Bader, P., Rossler, J., Bisogno, G., Maecker-Kolhoff, B., Lang, P., Zwaan, C. M., Sumerauer, D., Krivan, G., Bernard, J., Liu, Q., Doyle, E., Locatelli, Franco, Locatelli F. (ORCID:0000-0002-7976-3654), Morland, B., Kepak, T., Dallorso, S., Sevilla, J., Murphy, D., Luksch, R., Yaniv, I., Bader, P., Rossler, J., Bisogno, G., Maecker-Kolhoff, B., Lang, P., Zwaan, C. M., Sumerauer, D., Krivan, G., Bernard, J., Liu, Q., Doyle, E., Locatelli, Franco, and Locatelli F. (ORCID:0000-0002-7976-3654)
Abstract: This study (NCT01288573) investigated plerixafor’s safety and efficacy in children with cancer. Stage 1 investigated the dosage, pharmacokinetics (PK), pharmacodynamics (PD), and safety of plerixafor + standard mobilization (G-CSF ± chemotherapy). The stage 2 primary endpoint was successful mobilization (doubling of peripheral blood CD34+ cell count in the 24 h prior to first apheresis) in patients treated with plerixafor + standard mobilization vs. standard mobilization alone. In stage 1, three patients per age group (2–<6, 6–<12, and 12–<18 years) were treated at each dose level (160, 240, and 320 µg/kg). Based on PK and PD data, the dose proposed for stage 2 was 240 µg/kg (patients 1–<18 years), in which 45 patients were enrolled (30 plerixafor arm, 15 standard arm). Patient demographics and characteristics were well balanced across treatment arms. More patients in the plerixafor arm (24/30, 80%) met the primary endpoint of successful mobilization than in the standard arm (4/14, 28.6%, p = 0.0019). Adverse events reported as related to study treatment were mild, and no new safety concerns were identified. Plerixafor + standard G-CSF ± chemotherapy mobilization was generally well tolerated and efficacious when used to mobilize CD34+ cells in pediatric cancer patients.
Published: 2020

18. Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman–Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT)

Author: Cesaro, S., Pillon, M., Sauer, M., Smiers, F., Faraci, M., de Heredia, C. D., Wynn, R., Greil, J., Locatelli, Franco, Veys, P., Uyttebroeck, A., Ljungman, P., Chevalier, P., Ansari, M., Badell, I., Gungor, T., Salim, R., Tischer, J., Tecchio, C., Russell, N., Chybicka, A., Styczynski, J., Krivan, G., Smith, O., Stein, J., Afanasyev, B., Pochon, C., Menconi, M. C., Bosman, P., Mauro, M., Tridello, G., de Latour, R. P., Dufour, C., Locatelli F. (ORCID:0000-0002-7976-3654), Cesaro, S., Pillon, M., Sauer, M., Smiers, F., Faraci, M., de Heredia, C. D., Wynn, R., Greil, J., Locatelli, Franco, Veys, P., Uyttebroeck, A., Ljungman, P., Chevalier, P., Ansari, M., Badell, I., Gungor, T., Salim, R., Tischer, J., Tecchio, C., Russell, N., Chybicka, A., Styczynski, J., Krivan, G., Smith, O., Stein, J., Afanasyev, B., Pochon, C., Menconi, M. C., Bosman, P., Mauro, M., Tridello, G., de Latour, R. P., Dufour, C., and Locatelli F. (ORCID:0000-0002-7976-3654)
Abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative procedure in patients with Shwachman–Diamond syndrome (SDS) with bone marrow abnormalities. The results of 74 patients with SDS (6 acute myeloid leukemia, 7 myelodysplastic syndrome, and 61 bone marrow failure) treated with HSCT between 1988 and 2016 are reported. The donor source was: 24% sibling, 8% parent, and 68% unrelated donor. The stem cell source was: 70% bone marrow, 19% peripheral blood stem cells, and 11% cord blood. The conditioning regimen was myeloablative in 54% and reduced intensity in 46%. Neutrophil engraftment was achieved in 84% of patients after a median time of 17.5 days. Graft failure occurred in 15% of HSCTs. Grades I–IV acute and chronic GVHD were observed in 55% and 20% of patients, respectively. After a median follow-up of 7.3 years (95% CI 4.8–10.2), 28 patients died for progression/relapse (7) or toxicity (21). The 5-year overall survival and nonrelapse mortality were 63.3% (95% CI 50.8–73.4) and 19.8% (95% CI 10.8–30.8), respectively. In conclusion, this is the largest series so far reported and confirms that HSCT is a suitable option for patients with SDS. Further efforts are needed to lower transplant-related toxicity and reduce graft failure.
Published: 2020

19. Correction: Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman–Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT) (Bone Marrow Transplantation, (2020), 55, 9, (1796-1809), 10.1038/s41409-020-0863-z)

Author: Cesaro, S., Pillon, M., Sauer, M., Smiers, F., Faraci, M., de Heredia, C. D., Wynn, R., Greil, J., Locatelli, Franco, Veys, P., Uyttebroeck, A., Ljungman, P., Chevalier, P., Ansari, M., Badell, I., Gungor, T., Salim, R., Tischer, J., Tecchio, C., Russell, N., Chybicka, A., Styczynski, J., Krivan, G., Smith, O., Stein, J., Afanasyev, B., Pochon, C., Menconi, M. C., Bosman, P., Mauro, M., Tridello, G., de Latour, R. P., Dufour, C., Locatelli F. (ORCID:0000-0002-7976-3654), Cesaro, S., Pillon, M., Sauer, M., Smiers, F., Faraci, M., de Heredia, C. D., Wynn, R., Greil, J., Locatelli, Franco, Veys, P., Uyttebroeck, A., Ljungman, P., Chevalier, P., Ansari, M., Badell, I., Gungor, T., Salim, R., Tischer, J., Tecchio, C., Russell, N., Chybicka, A., Styczynski, J., Krivan, G., Smith, O., Stein, J., Afanasyev, B., Pochon, C., Menconi, M. C., Bosman, P., Mauro, M., Tridello, G., de Latour, R. P., Dufour, C., and Locatelli F. (ORCID:0000-0002-7976-3654)
Abstract: An amendment to this paper has been published and can be accessed via a link at the top of the paper.
Published: 2020

20. Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter EBMT-PDWP study

Author: Willasch, A. M., Peters, C., Sedlacek, P., Dalle, J. -H., Kitra-Roussou, V., Yesilipek, A., Wachowiak, J., Lankester, A., Prete, A., Hamidieh, A. A., Ifversen, M., Buechner, J., Krivan, G., Hamladji, R. -M., Diaz-de-Heredia, C., Skorobogatova, E., Michel, G., Locatelli, Franco, Bertaina, A., Veys, P., Dupont, S., Or, R., Gungor, T., Aleinikova, O., Sufliarska, S., Sundin, M., Rascon, J., Kaare, A., Nemet, D., Fagioli, F., Klingebiel, T. E., Styczynski, J., Bierings, M., Nagy, K., Abecasis, M., Afanasyev, B., Ansari, M., Vettenranta, K., Alseraihy, A., Chybicka, A., Robinson, S., Bertrand, Y., Kupesiz, A., Ghavamzadeh, A., Campos, A., Pichler, H., Dalissier, A., Labopin, M., Corbacioglu, S., Balduzzi, A., Galimard, J. -E., Bader, P., Locatelli F. (ORCID:0000-0002-7976-3654), Willasch, A. M., Peters, C., Sedlacek, P., Dalle, J. -H., Kitra-Roussou, V., Yesilipek, A., Wachowiak, J., Lankester, A., Prete, A., Hamidieh, A. A., Ifversen, M., Buechner, J., Krivan, G., Hamladji, R. -M., Diaz-de-Heredia, C., Skorobogatova, E., Michel, G., Locatelli, Franco, Bertaina, A., Veys, P., Dupont, S., Or, R., Gungor, T., Aleinikova, O., Sufliarska, S., Sundin, M., Rascon, J., Kaare, A., Nemet, D., Fagioli, F., Klingebiel, T. E., Styczynski, J., Bierings, M., Nagy, K., Abecasis, M., Afanasyev, B., Ansari, M., Vettenranta, K., Alseraihy, A., Chybicka, A., Robinson, S., Bertrand, Y., Kupesiz, A., Ghavamzadeh, A., Campos, A., Pichler, H., Dalissier, A., Labopin, M., Corbacioglu, S., Balduzzi, A., Galimard, J. -E., Bader, P., and Locatelli F. (ORCID:0000-0002-7976-3654)
Abstract: Although most children with acute lymphoblastic leukemia (ALL) receive fractionated total body irradiation (FTBI) as myeloablative conditioning (MAC) for allogeneic hematopoietic stem cell transplantation (allo-HSCT), it is an important matter of debate if chemotherapy can effectively replace FTBI. To compare outcomes after FTBI versus chemotherapy-based conditioning (CC), we performed a retrospective EBMT registry study. Children aged 2–18 years after MAC for first allo-HSCT of bone marrow (BM) or peripheral blood stem cells (PBSC) from matched-related (MRD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to control pretreatment imbalances of the observed variables. 3.054 patients were analyzed. CR1 (1.498): median follow-up (FU) after FTBI (1.285) and CC (213) was 6.8 and 6.1 years. Survivals were not significantly different. CR2 (1.556): median FU after FTBI (1.345) and CC (211) was 6.2 years. Outcomes after FTBI were superior as compared with CC with regard to overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and nonrelapse mortality (NRM). However, we must emphasize the preliminary character of the results of this retrospective “real-world-practice” study. These findings will be prospectively assessed in the ALL SCTped 2012 FORUM trial.
Published: 2020

21. Supportive care during pediatric hematopoietic stem cell transplantation: beyond infectious diseases. A report from workshops on supportive care of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author: Nava, T, Ansari, M, Dalle, J, de Heredia, C, Güngör, T, Trigoso, E, Falkenberg, U, Bertaina, A, Gibson, B, Jarisch, A, Balduzzi, A, Boenig, H, Krivan, G, Vettenranta, K, Matic, T, Büchner, J, Kalwak, K, Lawitschka, A, Yesilipek, A, Lucchini, G, Peters, C, Turkiewicz, D, Niinimäki, R, Diesch, T, Lehrnbecher, T, Sedlacek, P, Hutt, D, Dalissier, A, Wachowiak, J, Yaniv, I, Stein, J, Yalçin, K, Sisinni, L, Deiana, M, Ifversen, M, Kuhlen, M, Miesel, R, Bakhtiar, S, Cesaro, S, Willasch, A, Corbacioglu, S, Bader, P, Nava, Tiago, Ansari, Marc, Dalle, Jean-Hugues, de Heredia, Christina Diaz, Güngör, Tayfun, Trigoso, Eugenia, Falkenberg, Ulrike, Bertaina, Alice, Gibson, Brenda, Jarisch, Andrea, Balduzzi, Adriana, Boenig, Halvard, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Büchner, Jochen, Kalwak, Krzysztof, Lawitschka, Anita, Yesilipek, Akif, Lucchini, Giovanna, Peters, Christina, Turkiewicz, Dominik, Niinimäki, Riitta, Diesch, Tamara, Lehrnbecher, Thomas, Sedlacek, Petr, Hutt, Daphna, Dalissier, Arnaud, Wachowiak, Jacek, Yaniv, Isaac, Stein, Jerry, Yalçin, Koray, Sisinni, Luisa, Deiana, Marco, Ifversen, Marianne, Kuhlen, Michaela, Miesel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, Bader, Peter, Nava, T, Ansari, M, Dalle, J, de Heredia, C, Güngör, T, Trigoso, E, Falkenberg, U, Bertaina, A, Gibson, B, Jarisch, A, Balduzzi, A, Boenig, H, Krivan, G, Vettenranta, K, Matic, T, Büchner, J, Kalwak, K, Lawitschka, A, Yesilipek, A, Lucchini, G, Peters, C, Turkiewicz, D, Niinimäki, R, Diesch, T, Lehrnbecher, T, Sedlacek, P, Hutt, D, Dalissier, A, Wachowiak, J, Yaniv, I, Stein, J, Yalçin, K, Sisinni, L, Deiana, M, Ifversen, M, Kuhlen, M, Miesel, R, Bakhtiar, S, Cesaro, S, Willasch, A, Corbacioglu, S, Bader, P, Nava, Tiago, Ansari, Marc, Dalle, Jean-Hugues, de Heredia, Christina Diaz, Güngör, Tayfun, Trigoso, Eugenia, Falkenberg, Ulrike, Bertaina, Alice, Gibson, Brenda, Jarisch, Andrea, Balduzzi, Adriana, Boenig, Halvard, Krivan, Gergely, Vettenranta, Kim, Matic, Toni, Büchner, Jochen, Kalwak, Krzysztof, Lawitschka, Anita, Yesilipek, Akif, Lucchini, Giovanna, Peters, Christina, Turkiewicz, Dominik, Niinimäki, Riitta, Diesch, Tamara, Lehrnbecher, Thomas, Sedlacek, Petr, Hutt, Daphna, Dalissier, Arnaud, Wachowiak, Jacek, Yaniv, Isaac, Stein, Jerry, Yalçin, Koray, Sisinni, Luisa, Deiana, Marco, Ifversen, Marianne, Kuhlen, Michaela, Miesel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, and Bader, Peter
Abstract: Hematopoietic stem cell transplantation (HSCT) is currently the standard of care for many malignant and nonmalignant blood diseases. As several treatment-emerging acute toxicities are expected, optimal supportive measurements critically affect HSCT outcomes. The paucity of good clinical studies in supportive practices gives rise to the establishment of heterogeneous guidelines across the different centers, which hampers direct clinical comparison in multicentric studies. Aiming to harmonize the supportive care provided during the pediatric HSCT in Europe, the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) promoted dedicated workshops during the years 2017 and 2018. The present paper describes the resulting consensus on the management of sinusoidal obstructive syndrome, mucositis, enteral and parenteral nutrition, iron overload, and emesis during HSCT.
Published: 2020

22. P636 Medical resource use of two different dosing regimens of palifermin to prevent mucositis in multiple myeloma patients receiving one-day administration of high-dose melphalan

Author: Blijlevens, N. M.A., de Chateau, M., Krivan, G., Rabitsch, W., Szomor, A., Pytlik, R., Lissmats, A., Johnsen, H. E., Ruutu, T., Einsele, H., and Niederwieser, D.
Published: 2011

23. Successful allogeneic peripheral haematopoietic stem cell transplantation for myelodysplastic syndrome associated Behçet's disease: case report

Author: Gopcsa, L., Barta, A., Torbagyi, E., Lengyel, L., Lueff, S., Rasonyi, R., Tremmel, A., Krivan, G., Csomor, J., Kadar, J., Remenyi, P., and Masszi, T.
Published: 2008

24. Hematopoietic stem cell transplantation for CD40 ligand deficiency : Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study

Author: Ferrua, F., Galimberti, S., Courteille, V., Slatter, M.A., Booth, C., Moshous, D., Neven, B., Blanche, S., Cavazzana, M., Laberko, A., Shcherbina, A., Balashov, D., Soncini, E., Porta, F., Al-Mousa, H., Al-Saud, B., Al-Dhekri, H., Arnaout, R., Formankova, R., Bertrand, Y., Lange, A., Smart, J., Wolska-Kusnierz, B., Aquino, V.M., Dvorak, C.C., Fasth, A., Fouyssac, F., Heilmann, C., Hoenig, M., Schuetz, C., Kelecic, J., Bredius, R.G.M., Lankester, A.C., Lindemans, C.A., Suarez, F., Sullivan, K.E., Albert, M.H., Kalwak, K., Barlogis, V., Bhatia, M., Bordon, V., Czogala, W., Alonso, L., Dogu, F., Gozdzik, J., Ikinciogullari, A., Krivan, G., Ljungman, P., Meyts, I., Mustillo, P., Smith, A.R., Speckmann, C., Sundin, M., Keogh, S.J., Shaw, P.J., Boelens, J.J., Schulz, A.S., Sedlacek, P., Veys, P., Mahlaoui, N., Janda, A., Davies, E.G., Fischer, A., Cowan, M.J., Gennery, A.R., SCETIDE, PIDTC, EBMT, ESID IEWP, Ferrua, F, Galimberti, S, Courteille, V, Slatter, M, Booth, C, Moshous, D, Neven, B, Blanche, S, Laberko, A, Shcherbina, A, Balashov, D, Soncini, E, Porta, F, Al-Mousa, H, Al-Saud, B, Al-Dhekri, H, Arnaout, R, Formankova, R, Bertrand, Y, Lange, A, Smart, J, Wolska-Kusnierz, B, Aquino, V, Dvorak, C, Fasth, A, Fouyssac, F, Heilmann, C, Hoenig, M, Schuetz, C, Kelečić, J, Bredius, R, Lankester, A, Lindemans, C, Suarez, F, Sullivan, K, Albert, M, Kałwak, K, Barlogis, V, Bhatia, M, Bordon, V, Czogala, W, Alonso, L, Dogu, F, Gozdzik, J, Ikinciogullari, A, Kriván, G, Ljungman, P, Meyts, I, Mustillo, P, Smith, A, Speckmann, C, Sundin, M, Keogh, S, Shaw, P, Boelens, J, Schulz, A, Sedlacek, P, Veys, P, Mahlaoui, N, Janda, A, Davies, E, Fischer, A, Cowan, M, and Gennery, A
Subjects: 0301 basic medicine, Oncology, medicine.medical_specialty, medicine.medical_treatment, Immunology, Hematopoietic stem cell transplantation, X-Linked Combined Immunodeficiency Diseases, primary immunodeficiency, Donor lymphocyte infusion, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, X-linked hyper-IgM syndrome, Humans, Medicine, Immunology and Allergy, Risk factor, Child, Prospective cohort study, business.industry, Infant, Newborn, Infant, medicine.disease, Treatment Outcome, 030104 developmental biology, medicine.anatomical_structure, Graft-versus-host disease, surgical procedures, operative, Supportive psychotherapy, Child, Preschool, hematopoietic stem cell transplantation, Primary immunodeficiency, Bone marrow, CD40 ligand, business, 030215 immunology
Abstract: BACKGROUND: CD40 ligand (CD40L) deficiency, an X-linked primary immunodeficiency, causes recurrent sinopulmonary, Pneumocystis and Cryptosporidium species infections. Long-term survival with supportive therapy is poor. Currently, the only curative treatment is hematopoietic stem cell transplantation (HSCT). OBJECTIVE: We performed an international collaborative study to improve patients' management, aiming to individualize risk factors and determine optimal HSCT characteristics. METHODS: We retrospectively collected data on 130 patients who underwent HSCT for CD40L deficiency between 1993-2015. We analyzed outcome and variables' relevance with respect to survival and cure. RESULTS: Overall survival (OS), event-free survival (EFS), and disease-free survival (DFS) were 78.2%, 58.1%, and 72.3% 5 years after HSCT. Results were better in transplantations performed in 2000 or later and in children less than 10 years old at the time of HSCT. Pre-existing organ damage negatively influenced outcome. Sclerosing cholangitis was the most important risk factor. After 2000, superior OS was achieved with matched donors. Use of myeloablative regimens and HSCT at 2 years or less from diagnosis associated with higher OS and DFS. EFS was best with matched sibling donors, myeloablative conditioning (MAC), and bone marrow-derived stem cells. Most rejections occurred after reduced-intensity or nonmyeloablative conditioning, which associated with poor donor cell engraftment. Mortality occurred mainly early after HSCT, predominantly from infections. Among survivors who ceased immunoglobulin replacement, T-lymphocyte chimerism was 50% or greater donor in 85.2%. CONCLUSION: HSCT is curative in patients with CD40L deficiency, with improved outcome if performed before organ damage development. MAC is associated with better OS, EFS, and DFS. Prospective studies are required to compare the risks of HSCT with those of lifelong supportive therapy. ispartof: JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY vol:143 issue:6 pages:2238-2253 ispartof: location:United States status: published
Published: 2019

25. "Tandem" autologous haemopoietic stem cell transplantation in multiple myeloma - a single-centre report

Author: Barta, A., Gopcsa, L., Torbagyi, E., Lengyel, L., Rasonyi, R., and Krivan, G.
Published: 2007

26. Allogeneic haemopoietic stem cell transplantation in severe aplastic anaemia - a single-centre result

Author: Barta, A., Torbagyi, E., Gopcsa, L., Lengyel, L., and Krivan, G.
Published: 2007

27. CD 34 positive selected peripheral blood stem cell transplantation in haematological malignancies - a single-centre experience

Author: Rasonyi, R., Barta, A., Reti, M., Tremmel, A., Torbagyi, E., Gopcsa, L., and Krivan, G.
Published: 2007

28. Outcomes and Treatment Strategies for Autoimmunity and Hyperinflammation in Patients with RAG Deficiency

Author: Farmer, J. R., Foldvari, Z., Ujhazi, B., De Ravin, S. S., Chen, K., Bleesing, J. J. H., Schuetz, C., Al-Herz, W., Abraham, R. S., Joshi, A. Y., Costa-Carvalho, B. T., Buchbinder, D., Booth, C., Reiff, A., Ferguson, P. J., Aghamohammadi, A., Abolhassani, H., Puck, J. M., Adeli, M., Cancrini, C., Palma, P., Bertaina, A., Locatelli, Franco, Di Matteo, G., Geha, R. S., Kanariou, M. G., Lycopoulou, L., Tzanoudaki, M., Sleasman, J. W., Parikh, S., Pinero, G., Fischer, B. M., Dbaibo, G., Unal, E., Patiroglu, T., Karakukcu, M., Al-Saad, K. K., Dilley, M. A., Pai, S. -Y., Dutmer, C. M., Gelfand, E. W., Geier, C. B., Eibl, M. M., Wolf, H. M., Henderson, L. A., Hazen, M. M., Bonfim, C., Wolska-Kusnierz, B., Butte, M. J., Hernandez, J. D., Nicholas, S. K., Stepensky, P., Chandrakasan, S., Miano, M., Westermann-Clark, E., Goda, V., Krivan, G., Holland, S. M., Fadugba, O., Henrickson, S. E., Ozen, A., Karakoc-Aydiner, E., Baris, S., Kiykim, A., Bredius, R., Hoeger, B., Boztug, K., Pashchenko, O., Neven, B., Moshous, D., de Villartay, J. -P., Bousfiha, A. A., Hill, H. R., Notarangelo, L. D., Walter, J. E., Locatelli F. (ORCID:0000-0002-7976-3654), Farmer, J. R., Foldvari, Z., Ujhazi, B., De Ravin, S. S., Chen, K., Bleesing, J. J. H., Schuetz, C., Al-Herz, W., Abraham, R. S., Joshi, A. Y., Costa-Carvalho, B. T., Buchbinder, D., Booth, C., Reiff, A., Ferguson, P. J., Aghamohammadi, A., Abolhassani, H., Puck, J. M., Adeli, M., Cancrini, C., Palma, P., Bertaina, A., Locatelli, Franco, Di Matteo, G., Geha, R. S., Kanariou, M. G., Lycopoulou, L., Tzanoudaki, M., Sleasman, J. W., Parikh, S., Pinero, G., Fischer, B. M., Dbaibo, G., Unal, E., Patiroglu, T., Karakukcu, M., Al-Saad, K. K., Dilley, M. A., Pai, S. -Y., Dutmer, C. M., Gelfand, E. W., Geier, C. B., Eibl, M. M., Wolf, H. M., Henderson, L. A., Hazen, M. M., Bonfim, C., Wolska-Kusnierz, B., Butte, M. J., Hernandez, J. D., Nicholas, S. K., Stepensky, P., Chandrakasan, S., Miano, M., Westermann-Clark, E., Goda, V., Krivan, G., Holland, S. M., Fadugba, O., Henrickson, S. E., Ozen, A., Karakoc-Aydiner, E., Baris, S., Kiykim, A., Bredius, R., Hoeger, B., Boztug, K., Pashchenko, O., Neven, B., Moshous, D., de Villartay, J. -P., Bousfiha, A. A., Hill, H. R., Notarangelo, L. D., Walter, J. E., and Locatelli F. (ORCID:0000-0002-7976-3654)
Abstract: Background: Although autoimmunity and hyperinflammation secondary to recombination activating gene (RAG) deficiency have been associated with delayed diagnosis and even death, our current understanding is limited primarily to small case series. Objective: Understand the frequency, severity, and treatment responsiveness of autoimmunity and hyperinflammation in RAG deficiency. Methods: In reviewing the literature and our own database, we identified 85 patients with RAG deficiency, reported between 2001 and 2016, and compiled the largest case series to date of 63 patients with prominent autoimmune and/or hyperinflammatory pathology. Results: Diagnosis of RAG deficiency was delayed a median of 5 years from the first clinical signs of immune dysregulation. Most patients (55.6%) presented with more than 1 autoimmune or hyperinflammatory complication, with the most common etiologies being cytopenias (84.1%), granulomas (23.8%), and inflammatory skin disorders (19.0%). Infections, including live viral vaccinations, closely preceded the onset of autoimmunity in 28.6% of cases. Autoimmune cytopenias had early onset (median, 1.9, 2.1, and 2.6 years for autoimmune hemolytic anemia, immune thrombocytopenia, and autoimmune neutropenia, respectively) and were refractory to intravenous immunoglobulin, steroids, and rituximab in most cases (64.7%, 73.7%, and 71.4% for autoimmune hemolytic anemia, immune thrombocytopenia, and autoimmune neutropenia, respectively). Evans syndrome specifically was associated with lack of response to first-line therapy. Treatment-refractory autoimmunity/hyperinflammation prompted hematopoietic stem cell transplantation in 20 patients. Conclusions: Autoimmunity/hyperinflammation can be a presenting sign of RAG deficiency and should prompt further evaluation. Multilineage cytopenias are often refractory to immunosuppressive treatment and may require hematopoietic cell transplantation for definitive management.
Published: 2019

29. Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation

Author: Guardiola, Ph., Pasquini, R., Dokal, I., Ortega, J.J., van Weel-Sipman, M., Marsh, J.C.W., Ball, S.E., Locatelli, F., Vermylen, C., Skinner, R., Ljungman, P., Miniero, R., Shaw, P.J., Souillet, G., Michallet, M., Bekassy, A.N., Krivan, G., Di Bartolomeo, P., Heilmann, C., Zanesco, L., Cahn, J.Y., Arcese, W., Bacigalupo, A., and Gluckman, E.
Published: 2000
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30. Haematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT Inborn Errors Working Party (IEWP) study

Author: Ferrua, F., Courteille, V., Janda, A., Slatter, M., Albert, M.H., Al-Mousa, H., Al-Saud, B., Balashov, D., Bertrand, Y., Booth, C., Bordon, V., Czogala, W., Dogu, F., Fasth, A., Formankova, R., Gozdzik, J., Heilmann, C., Honig, M., Ikinciogullari, A., Kalwak, K., Keogh, S.J., Krivan, G., Lange, A., Lankester, A.C., Ljungman, P., Meyts, I., Neven, B., Soncini, E., Suarez, F., Mahlaoui, N., Fischer, A., Davies, E.G., Gennery, A.R., and EBMT IEWP
Published: 2015

31. Innocuité et tolérabilité de l’immunoglobuline humaine G à 20 %, administrée par voie sous-cutanée (Ig20Gly) : analyse finale d’une étude de phase 2/3 portant sur des patients souffrant de déficits immunitaires primitifs (DIP) en Europe

Author: Borte, M., Kriván, G., Dérfalvi, B., Maródi, L., Dicső, F., Engl, W., Mccoy, B., Leibl, H., Boudjemia, K., and Yel, L.
Published: 2018
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32. P253 Safety and tolerability of SCIG 20% in patients with PIDD in europe and north america

Author: Suez, D., primary, Krivan, G., additional, Derfalvi, B., additional, Hussain, I., additional, Stein, M., additional, Gupta, S., additional, Paris, K., additional, Brodszki, N., additional, Leibl, H., additional, McCoy, B., additional, and Yel, L., additional
Published: 2016
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33. Pre-medication practices and incidence of infusion-related reactions in patients receiving amphotec : data from the patient registry of amphotericin B cholesteryl sulfate complex for injection clinical tolerability (PROACT) registry

Author: Paterson, D.L., David, K., Mrsić, Mirando, Cetkovsky, P., Weng, X.H., Sterba, J., Krivan, G., Bošković, D., Lu, M., Zhu, L.P., Duraković, Nadira, Guiot, H., Stefaniak, M., Thamlikitku, V., Govedarović, N., Sanchez, C., Matysiak, M., Soto-Malave, R., Santana-Bagur, J., Torosjan, T., Jaruratanasirikul, S., Marjanović, S., Salleh, S., and Savić, I.
Subjects: Adult, Male, Microbiology (medical), medicine.medical_specialty, Antifungal Agents, Adolescent, Anti-Inflammatory Agents, Histamine Antagonists, Adrenal Cortex Hormones, Amphotericin B, Internal medicine, Epidemiology, parasitic diseases, medicine, Humans, Pharmacology (medical), liposomal, tolerability, fungal infections, Infusions, Intravenous, Mycosis, Acetaminophen, Aged, Aged, 80 and over, Pharmacology, ABCD² score, business.industry, Incidence (epidemiology), Middle Aged, medicine.disease, Surgery, Drug Combinations, Infectious Diseases, Mycoses, Tolerability, population characteristics, Female, Chills, Cholesterol Esters, medicine.symptom, business, medicine.drug
Abstract: Clinical studies have suggested that rates of infusion-related reactions (IRRs) may be higher with amphotericin B colloidal dispersion (ABCD) versus other forms of amphotericin B. However, these studies did not permit the use of pre-medications upfront, which are now commonly used. Objectives were to describe the use of pre-medications and determine the rate of IRRs in the real-world setting. PRoACT, a multicentre, worldwide observational registry, captured real-world data about pre-medication practices and IRRs in patients receiving ABCD. Eligible patients were those beginning treatment with ABCD ; treatment was according to the site's standard treatment practice. Incidence of IRRs was collected during the first 10 days of ABCD therapy. Clinical response data were collected 12 weeks after treatment start. One hundred and seventy patients from 21 worldwide sites were included (median age 37 years ; 52% male). There were a total of 1230 ABCD infusions (mean dose 2.8 mg/kg/day) ; 90% of the infusions (1105/1230) had pre-medication. Common pre-medications included corticosteroids, antihistamines, paracetamol (acetaminophen) and metamizole. The overall IRR rate was 12% (147/1230) and was lower in infusions with pre-medication (11%) versus no pre-medication (22%), P < 0.001. Corticosteroids were associated with a decreased incidence of IRRs (P < 0.05), while paracetamol and antihistamines were not. The most common IRRs were chills (7%), fever (7%) and rigors (5%). Clearance of the fungal infection occurred in 52% of the participants. These data suggest a lower rate of IRRs with ABCD than previously reported. Pre-medication is associated with decreased IRR incidence. Corticosteroids in particular appear to decrease IRRs while paracetamol and antihistamines, though commonly used, do not.
Published: 2008

34. In a high-dose melphalan setting, palifermin compared with placebo had no effect on oral mucositis or related patient's burden.

Author: Blijlevens, N.M., Chateau, M. de, Krivan, G., Rabitsch, W., Szomor, A., Pytlik, R., Lissmats, A., Johnsen, H.E., Witte, T.J. de, Einsele, H., Ruutu, T., Niederwieser, D., Blijlevens, N.M., Chateau, M. de, Krivan, G., Rabitsch, W., Szomor, A., Pytlik, R., Lissmats, A., Johnsen, H.E., Witte, T.J. de, Einsele, H., Ruutu, T., and Niederwieser, D.
Abstract: 01 juli 2013, Item does not contain fulltext, This randomized-controlled trial studied the efficacy of palifermin in a chemotherapy-only, high-dose Melphalan (HDM) transplant setting, to reduce oral mucositis (OM) and its sequelae measured by patient-reported outcomes (PRO) and medical resource use. Palifermin, relative to placebo was given either pre-/post-HDM or pre-HDM in patients with multiple myeloma (MM) undergoing auto-SCT at 39 European centers. Oral cavity assessment (WHO) and PRO questionnaires (oral mucositis daily questionnaire (OMDQ) and EQ 5D) were used in 281 patients (mean age 56, +/-s.d.=8 years). 57 patients received placebo. One hundred and fifteen subjects were randomized to pre-/post-HDM receiving palifermin on 3 consecutive days before HDM and after auto-SCT and 109 patients were randomized to pre-HDM, receiving palifermin (60 mug/kg/day) i.v. for 3 consecutive days before HDM. There was no statistically significant difference in maximum OM severity. Severe OM occurred in 37% (placebo), 38% (pre-/post-HDM) and 24% (pre-HDM) of patients. No significant difference was observed with respect to PRO assessments or medical resource use, but more infections and fever during neutropenia were reported in pre-/post-HDM vs placebo (for example, 51 and 26%). To conclude, palifermin was unable to reduce OM or OM-related patient's burden in MM transplant patients.
Published: 2013

35. 1834 Subcutaneous Human Hepatits B Immunoglobulin (Fovepta) in Neonates of HBV-Carrier Mothers

Author: Schloesser, R., primary, Kamin, W., additional, Krivan, G., additional, Siklos, P., additional, Mendling, W., additional, and Buxmann, H., additional
Published: 2012
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36. Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation

Author: Guardiola, P, Pasquini, R, Dokal, I, Ortega, J J, van Weel-Sipman, M, Marsh, J C, Ball, S E, Locatelli, Franco, Vermylen, C, Skinner, R, Ljungman, P, Miniero, R, Shaw, P J, Souillet, G, Michallet, M, Bekassy, A N, Krivan, G, Di Bartolomeo, P, Heilmann, C, Zanesco, L, Cahn, J Y, Arcese, W, Bacigalupo, Andrea, Gluckman, E, Locatelli, F (ORCID:0000-0002-7976-3654), Bacigalupo, A (ORCID:0000-0002-9119-567X), Guardiola, P, Pasquini, R, Dokal, I, Ortega, J J, van Weel-Sipman, M, Marsh, J C, Ball, S E, Locatelli, Franco, Vermylen, C, Skinner, R, Ljungman, P, Miniero, R, Shaw, P J, Souillet, G, Michallet, M, Bekassy, A N, Krivan, G, Di Bartolomeo, P, Heilmann, C, Zanesco, L, Cahn, J Y, Arcese, W, Bacigalupo, Andrea, Gluckman, E, Locatelli, F (ORCID:0000-0002-7976-3654), and Bacigalupo, A (ORCID:0000-0002-9119-567X)
Abstract: Allogeneic stem cell transplantation is the only treatment that can restore a normal hematopoiesis in Fanconi anemia (FA). In this retrospective multicenter study, we analyzed the results of this approach using HLA-matched unrelated bone marrow donors, and tried to identify covariates predicting the outcome of the transplant. From January 1985 to June 1998, 69 FA patients were transplanted with unrelated HLA-matched donors. Patients' characteristics before and after transplant were provided by the European group blood and marrow transplant registry and were analyzed in collaboration with the European Fanconi Anemia Registry. The 3-year probability of survival was 33%. Extensive malformations, a positive recipient cytomegalovirus serology, the use of androgens before transplant, and female donors were associated with a worse outcome. Primary graft failures were observed more frequently when female donors were used, mainly because the grafts contained lower nucleated cell doses per kilogram of recipient body weight compared with grafts coming from male donors. The probability of grade III-IV acute graft-versus-host disease (GVHD) was 34%. Elevated serum alanine/aspartate transaminases before transplantation; limb, urogenital tract, or nephrologic malformations; and non-T-cell-depleted grafts were predictors of severe acute GVHD. This study shows the dramatic impact of preexisting congenital malformations on the outcome of FA patients transplanted with HLA-matched unrelated donors. If the use of T-cell depletion has led to a dramatic reduction of acute GVHD incidence, no significant outcome improvement was observed with this approach, mainly because of an increased risk of graft failure. (Blood. 2000;95:422-429)
Published: 2000

37. P186 Fatal fulminant herpes simplex virus-1 hepatitis 20 years after allogeneic bone marrow transplantation from acute myeloid leukemia case report

Author: Gopcsa, L., primary, Barta, A., additional, Torbagyi, E., additional, Lengyel, L., additional, Csomor, J., additional, Szalai, E., additional, Poros, A., additional, and Krivan, G., additional
Published: 2007
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38. P013 Complete resolution of Behcet's disease after HLA-mismatched allogeneic peripheral haematopoietic stem cell transplantation for myelodysplastic syndrome case report

Author: Gopcsa, L., primary, Barta, A., additional, Torbagyi, E., additional, Lengyel, L., additional, Kallay, K., additional, Rasonyi, R., additional, Tremmel, A., additional, Csomor, J., additional, Kadar, J., additional, Lueff, S., additional, and Krivan, G., additional
Published: 2007
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39. Allogeneic stem cell transplantation for Fanconi anaemia

Author: UCL - Cliniques universitaires Saint-Luc, UCL - MD/GYPE - Département de gynécologie, d'obstétrique et de pédiatrie, Guardiola, P, Vermylen, Christiane, Socie, G., Pasquini, R, Dokal, I, Ortega, JJ, van Weel-Sipman, M, Marsh, J, Locatelli, F., Souillet, G., Cahn, JY., Ljungman, P., Miniero, R, Shaw, J, Archimbaud, E., Bekassy, AN, Krivan, G, Di Bartolomeo, P, Bacigalupo, A., Gluckman, E., 1st Meeting of the Working-Party-on-Paediatric-Diseases of the European-Group-for-Blood-and-Marrow-Transplantation, UCL - Cliniques universitaires Saint-Luc, UCL - MD/GYPE - Département de gynécologie, d'obstétrique et de pédiatrie, Guardiola, P, Vermylen, Christiane, Socie, G., Pasquini, R, Dokal, I, Ortega, JJ, van Weel-Sipman, M, Marsh, J, Locatelli, F., Souillet, G., Cahn, JY., Ljungman, P., Miniero, R, Shaw, J, Archimbaud, E., Bekassy, AN, Krivan, G, Di Bartolomeo, P, Bacigalupo, A., Gluckman, E., and 1st Meeting of the Working-Party-on-Paediatric-Diseases of the European-Group-for-Blood-and-Marrow-Transplantation
Abstract: Fanconi anaemia is a hereditary disorder characterised by chromosomal breaks increased by cross-linking agents. Bone marrow transplantation is the treatment of choice when a HLA identical sibling donor has been identified. The use of low-dose cyclophosphamide with thoraco-abdominal irradiation for the conditioning regimen of FA patients has lead to a dramatic improvement of survival, with a long-term survival of 75% at our institution. However, if most patients are completely cured of their haematological disease, there is concern about an-increased frequency of secondary tumours, mostly head and neck squamous cell carcinomas of poor prognosis. Results of BMT using alternative donors (HLA mismatched related and unrelated donors) have also.improved during the last decade. A better selection of the donor via high-resolution techniques for class-II HLA matching, and more recently the use of T cell depleted grafts are probably the main explanations. Despite a short follow-up and the small number of patients analysed, transplants using HLA matched family cord blood give some promising results. On the other hand, first results with unrelated cord blood remind that this approach is clearly an experimental one that has to be evaluated through international registries and prospective studies. New approaches including autologous stem cell transplantations and gene therapy are currently explored.
Published: 1998

40. Allogeneic stem cell transplantation for Fanconi Anaemia. Severe Aplastic Anaemia Working Party of the EBMT and EUFAR. European Group for Blood and Marrow Transplantation

Author: Guardiola, P, Socié, G, Pasquini, R, Dokal, I, Ortega, J J, van Weel-Sipman, M, Marsh, J, Locatelli, Franco, Souillet, G, Cahn, J Y, Ljungman, P, Miniero, R, Shaw, J, Vermylen, C, Archimbaud, E, Bekassy, A N, Krivan, G, Di Bartolomeo, P, Bacigalupo, Andrea, Gluckman, E, Locatelli, F (ORCID:0000-0002-7976-3654), Bacigalupo, A (ORCID:0000-0002-9119-567X), Guardiola, P, Socié, G, Pasquini, R, Dokal, I, Ortega, J J, van Weel-Sipman, M, Marsh, J, Locatelli, Franco, Souillet, G, Cahn, J Y, Ljungman, P, Miniero, R, Shaw, J, Vermylen, C, Archimbaud, E, Bekassy, A N, Krivan, G, Di Bartolomeo, P, Bacigalupo, Andrea, Gluckman, E, Locatelli, F (ORCID:0000-0002-7976-3654), and Bacigalupo, A (ORCID:0000-0002-9119-567X)
Abstract: Fanconi anaemia is a hereditary disorder characterised by chromosomal breaks increased by cross-linking agents. Bone marrow transplantation is the treatment of choice when a HLA identical sibling donor has been identified. The use of low-dose cyclophosphamide with thoraco-abdominal irradiation for the conditioning regimen of FA patients has lead to a dramatic improvement of survival, with a long-term survival of 75% at our institution. However, if most patients are completely cured of their haematological disease, there is concern about an increased frequency of secondary tumours, mostly head and neck squamous cell carcinomas of poor prognosis. Results of BMT using alternative donors (HLA mismatched related and unrelated donors) have also improved during the last decade. A better selection of the donor via high-resolution techniques for class-II HLA matching, and more recently the use of T cell depleted grafts are probably the main explanations. Despite a short follow-up and the small number of patients analysed, transplants using HLA matched family cord blood give some promising results. On the other hand, first results with unrelated cord blood remind that this approach is clearly an experimental one that has to be evaluated through international registries and prospective studies. New approaches including autologous stem cell transplantations and gene therapy are currently explored.
Published: 1998

41. P-109: Autologous haematopoietic stem cell transplantation in two paediatric patients with refractory Crohn’s disease

Author: Boros, B.K., Müller, K.E., Kriván, G., Kállay, K., Szabó, D., Kovács, L., Krikovszky, D., and Veres, G.
Published: 2014
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42. Premedication Practices and Incidence of Infusion-related Reactions in Patients Receiving AMPHOTEC: Data from the PRoACT-Patient Registry of Amphotericin B Cholesteryl Sulfate Complex for Injection Clinical Tolerability

Author: Krivàn, G., Mrsic, M., David, K., and Paterson, D.
Published: 2008
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43. P016 Allogeneic haemopoietic stem cell transplantation in children with acute leukemias. A single centre experience in Hungary (1992 2007).

Author: Kriván, G., Kállay, K., Tremmel, A., Réti, M., Rásonyi, R., Reményi, P., Barta, A., Gopcsa, L., Torbágyi, É., Lengyel, L., and Masszi, T.
Published: 2007
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44. Allogeneic stem cell transplantation for Fanconi anaemia

Author: Guardiola, Ph, Socié, G., Pasquini, R., Inderjeet Dokal, Ortega, J. J., Weel-Sipman, M., Marsh, J., Locatelli, F., Souillet, G., Cahn, J. Y., Ljungman, P., Miniero, R., Shaw, J., Vermylen, Ch, Archimbaud, E., Bekassy, A. N., Krivan, G., Di Bartolomeo, P., Bacigalupo, A., and Gluckman, E.
Subjects: Secondary malignancies, Cord blood, Allogeneic bone marrow transplantation, Aplastic anaemia, Fanconi anaemia, Alternative donors, T cell depletion

45. Initial presenting manifestations in 16,486 patients with inborn errors of immunity include infections and noninfectious manifestations

Author: Tim Niehues, Catherine Waruiru, Conleth Feighery, Uwe Schauer, Virginie Courteille, Kai Lehmberg, Ingo Müller, I. Esteves, Henner Morbach, Michael Borte, Patrick Hundsdoerfer, Klaus Schwarz, Ewelina Gowin, Alessandro Aiuti, Andreas Holbro, Federica Barzaghi, João Farela Neves, Dagmar Graf, Hannah Tamary, Veneta Milenova, Benedikt Boetticher, Eleonora Gambineri, Vera Goda, Alia Eldash, Jan-Christian Wasmuth, Fabio Candotti, Svetlana O. Sharapova, Markus Metzler, Juergen Brunner, Anna Hilfanova, Brindusa Ruxandra Capilna, Pere Soler-Palacín, Arnau Antolí, Horst von Bernuth, Vassilios Lougaris, Maria Carrabba, Bernd H. Belohradsky, Julian Thalhammer, Nathalie de Vergnes, Peter Olbrich, Peter Kopač, Leif G. Hanitsch, Alexandra Nieters, Filomeen Haerynck, Juliana Gabzdilova, Sezin Aydemir, Rabab El Hawary, Patrick F.K. Yong, Maria Giovanna Danieli, Alberto Tommasini, Sandra Steinmann, Ulrich Baumann, Figen Dogu, Elisabeth Förster-Waldl, Carolina Marasco, Donato Amodio, Lorenzo Lodi, Xavier Solanich, Caterina Cancrini, Brigita Sitkauskiene, Torsten Witte, Clementina Vanessa, Nima Rezaei, Jean-Christophe Goffard, Kirsten Wittke, Emmanouil Liatsis, Helen Baxendale, Susana L. Silva, Bodo Grimbacher, Henrike Ritterbusch, Evangelia Farmaki, Safa Meshaal, Sujal Ghosh, Larysa Kostyuchenko, David Edgar, Simone Cesaro, R Zeuner, Nerea Salmón Rodríguez, Isabella Quinti, Stephan Ehl, Pauline Brosselin, Joerg C. Henes, Pilar Llobet Agulló, Rosa Maria Dellepiane, Andrea Meinhardt, Marina Kojić, Georgios Sogkas, Stephan Borte, Catharina Schuetz, Suheyla Ocak, Karin Marschall, Lukas M. Gasteiger, Stefan Raffac, Sofia Tantou, Sadia Noorani, Matthaios Speletas, Philippe Randrianomenjanahary, Ursula Holzer, Ayca Kiykim, Johannes G. Liese, Angelo Vacca, Gisela Fecker, Ekrem Unal, Koen J. van Aerde, Alba Parra-Martínez, Kaan Boztug, Sophie Stiehler, Sybille Landwehr-Kenzel, Claudio Pignata, Jennifer Neubert, Janine Reichenbach, Shahnaz Parvin, Sarah Goddard, Andrea Schroll, Dirk Holzinger, Asghar Aghamohammadi, Hassan Abolhassani, Johannes Trück, Estela Paz-Artal, Shereen M. Reda, Anna Shcherbina, Maria Raptaki, Jaroslava Orosova, Beata Wolska-Kuśnierz, Tessa Kerre, Gerrit Ahrenstorf, Ben Zion Garty, Dirk Foell, Benjamin Becker, Ulrike F. Demel, Androniki Kapousouzi, Abraham Rutgers, Klaus Warnatz, Gemma Rocamora Blanch, Stephan Rusch, Luis M. Allende, Dalia Abd Elaziz, Safa Baris, Jorisvan Montfrans, Dominik T. Schneider, Raphael Scheible, Juana Gil-Herrera, Gerhard Kindle, Annarosa Soresina, Giovanna Fabio, Uwe Wintergerst, Emilia Faria, Maria Fasshauer, Silvia Ricci, Aisha Elmarsafy, Barbara Pietrucha, Carsten Speckmann, Nizar Mahlaoui, Ulrich Heininger, Isabelle Meyts, Matthew Buckland, Efimia Papadopoulou-Alataki, Robin Kobbe, A Herwadkar, Sebastian F. N. Bode, Ali Sobh, László Maródi, Baldassarre Martire, Chiara Azzari, Maximilian Heeg, Katja Masjosthusmann, Michael H. Albert, Matteo Chinello, Juan Luis Santos-Pérez, Aarnoud Huissoon, Tanya I. Coulter, Hendrik Schulze-Koops, Norbert Graf, Radwa Alkady, Jolanta Bernatoniene, Seraina Prader, Alenka Gagro, Joachim Roesler, Taco W. Kuijpers, Ewa Więsik-Szewczyk, Maria Elena Maccari, Conrad Ferdinand Lippert, Miriam González-Amores, Johannes Dirks, Daniel E Pleguezuelo, Christof M. Kramm, Anders Fasth, Volker Schuster, Olov Ekwall, Nikolaus Rieber, Javier Carbone, Petra Kaiser-Labusch, Diana Ernst, Lucia Augusta Baselli, Luis Ignacio Gonzalez-Granado, Maria Kanariou, Stefanie S. V. Henriet, Sigune Goldacker, Kerstin Felgentreff, Oana Joean, Fine Roosens, Fabian Hauck, Eva C. Schwaneck, Milos Jesenak, Manfred Hoenig, Lenka Kapustova, Christoph Boesecke, Alain Fischer, Sara Pereira da Silva, Julia Körholz, Ansgar Schulz, Carolynne Schwarze-Zander, Mikko Seppänen, Nermeen Galal, Nora Naumann-Bartsch, Tomaz Garcez, Peter Ciznar, Klara M. Posfay-Barbe, Zelimir Pavle Eric, Reinhold E. Schmidt, Hermann J. Girschick, Sabine Heine, Anika-Kerstin Biegner, Annick A. J. M. van de Ven, Stefan Schreiber, J. Merlijn van den Berg, Nurit Assia Batzir, Alexandra Jablonka, Kim Stol, Gregor Dückers, Antonios G.A. Kolios, Ioannis Kakkas, Christian Klemann, Marina N. Guseva, Sofia Grigoriadou, Elif Karakoc-Aydiner, Antonio Marzollo, Peter D. Arkwright, Urs C. Steiner, Sara Sebnem Kilic, Romina Dieli-Crimi, Gergely Kriván, Monika Sparber-Sauer, Marco Cazzaniga, Fulvio Porta, Paraskevi Maggina, Tomas Milota, Robbert G. M. Bredius, Martine Pergent, Klaus Tenbrock, Jana Pachlopnik Schmid, Florentia Dimitriou, Cathal Laurence Steele, Helen Bourne, Anna Bobcakova, Gerd Horneff, Judith Potjewijd, Marc Schmalzing, Tobias Ankermann, Paul Ryan, Oksana Boyarchuk, Necil Kutukculer, Carl Friedrich Classen, Zita Chovancová, Moira Thomas, Cinzia Milito, Michaela Bitzenhofer-Grüber, Faranaz Atschekzei, Eva Hlaváčková, Viviana Moschese, Julie Smet, Hans-Hartmut Peter, Carla Teixeira, Sabine M El-Helou, Suzanne de Kruijf Bazen, Helmut Wittkowski, Donate Jakoby, Marina Garcia-Prat, Esther de Vries, Richard Herriot, Sven Kracker, Alessandro Plebani, Lisa Göschl, Laura Hora Marques, Anna Sediva, Jiri Litzman, Mark M. Gompels, Renate Krüger, Şefika İlknur Kökçü Karadağ, Nadine Binder, Anna Szaflarska, Peter Jandus, Lisa Ibberson, Johann Greil, Ulf Schulze-Sturm, Mehtap Sirin, Aydan Ikinciogullari, Edyta Heropolitańska-Pliszka, Michael E. Weiss, Alla Skapenko, Lukas Wisgrill, Hana Alachkar, Uta Behrends, Silvia Sánchez-Ramón, Maria N. Hatzistilianou, Otilia Petrovicova, Darko Richter, Zoreh Nademi, Jürgen K. Rockstroh, Sohilla Lotfy, Markus G. Seidel, Timothy Ronan Leahy, Audra Blažienė, Translational Immunology Groningen (TRIGR), Paediatric Infectious Diseases / Rheumatology / Immunology, AII - Inflammatory diseases, ARD - Amsterdam Reproduction and Development, University of Zurich, Ehl, Stephan, Thalhammer, J., Kindle, G., Nieters, A., Rusch, S., Seppanen, M. R. J., Fischer, A., Grimbacher, B., Edgar, D., Buckland, M., Mahlaoui, N., Ehl, S., Boztug, K., Brunner, J., Demel, U. F., Forster-Waldl, E., Gasteiger, L. M., Goschl, L., Kojic, M., Schroll, A., Seidel, M. G., Wintergerst, U., Wisgrill, L., Sharapova, S. O., Goffard, J. -C., Kerre, T., Meyts, I., Roosens, F., Smet, J., Haerynck, F., Eric, Z. P., Milenova, V., Gagro, A., Richter, D., Chovancova, Z., Hlavackova, E., Litzman, J., Milota, T., Sediva, A., Elaziz, D. A., Alkady, R. S., El Sayed El Hawary, R., Eldash, A. S., Galal, N., Lotfy, S., Meshaal, S. S., Reda, S. M., Sobh, A., Elmarsafy, A., Brosselin, P., Courteille, V., De Vergnes, N., Kracker, S., Pergent, M., Randrianomenjanahary, P., Ahrenstorf, G., Albert, M. H., Ankermann, T., Atschekzei, F., Baumann, U., Becker, B. C., Behrends, U., Belohradsky, B. H., Biegner, A. -K., Binder, N., Bode, S. F. N., Boesecke, C., Boetticher, B., Borte, M., Borte, S., Classen, C. F., Dirks, J., Duckers, G., El-Helou, S., Ernst, D., Fasshauer, M., Fecker, G., Felgentreff, K., Foell, D., Ghosh, S., Girschick, H. J., Goldacker, S., Graf, N., Graf, D., Greil, J., Hanitsch, L. G., Hauck, F., Heeg, M., Heine, S. I., Henes, J. C., Hoenig, M., Holzer, U., Holzinger, D., Horneff, G., Hundsdoerfer, P., Jablonka, A., Jakoby, D., Joean, O., Kaiser-Labusch, P., Klemann, C., Kobbe, R., Korholz, J., Kramm, C. M., Kruger, R., Landwehr-Kenzel, S., Lehmberg, K., Liese, J. G., Lippert, C. F., Maccari, M. E., Masjosthusmann, K., Meinhardt, A., Metzler, M., Morbach, H., Muller, I., Naumann-Bartsch, N., Neubert, J., Niehues, T., Peter, H. -H., Rieber, N., Ritterbusch, H., Rockstroh, J. K., Roesler, J., Schauer, U., Scheible, R., Schmalzing, M., Schmidt, R. E., Schneider, D. T., Schreiber, S., Schuetz, C., Schulz, A., Schulze-Koops, H., Schulze-Sturm, U., Schuster, V., Schwaneck, E. C., Schwarz, K., Schwarze-Zander, C., Sirin, M., Skapenko, A., Sogkas, G., Sparber-Sauer, M., Speckmann, C., Steinmann, S., Stiehler, S., Tenbrock, K., von Bernuth, H., Warnatz, K., Wasmuth, J. -C., Weiss, M., Witte, T., Wittke, K., Wittkowski, H., Zeuner, R. A., Farmaki, E., Hatzistilianou, M. N., Kakkas, I., Kanariou, M. G., Kapousouzi, A., Liatsis, E., Maggina, P., Papadopoulou-Alataki, E., Raptaki, M., Speletas, M., Tantou, S., Goda, V., Krivan, G., Marodi, L., Abolhassani, H., Aghamohammadi, A., Rezaei, N., Feighery, C., Leahy, T. R., Ryan, P., Batzir, N. A., Garty, B. Z., Tamary, H., Aiuti, A., Amodio, D., Azzari, C., Barzaghi, F., Baselli, L. A., Cancrini, C., Carrabba, M., Cazzaniga, M., Cesaro, S., Chinello, M., Danieli, M. G., Dellepiane, R. M., Fabio, G., Gambineri, E., Lodi, L., Lougaris, V., Marasco, C., Martire, B., Marzollo, A., Milito, C., Moschese, V., Pignata, C., Plebani, A., Porta, F., Quinti, I., Ricci, S., Soresina, A., Tommasini, A., Vacca, A., Vanessa, C., Blaziene, A., Sitkauskiene, B., Gowin, E., Heropolitanska-Pliszka, E., Pietrucha, B., Szaflarska, A., Wiesik-Szewczyk, E., Wolska-Kusnierz, B., Esteves, I., Faria, E., Marques, L. H., Neves, J. F., Silva, S. L., Teixeira, C., Pereira da Silva, S., Capilna, B. R., Guseva, M. N., Shcherbina, A., Bobcakova, A., Ciznar, P., Gabzdilova, J., Jesenak, M., Kapustova, L., Orosova, J., Petrovicova, O., Raffac, S., Kopac, P., Allende, L. M., Antoli, A., Blanch, G. R., Carbone, J., Dieli-Crimi, R., Garcia-Prat, M., Gil-Herrera, J., Gonzalez-Granado, L. I., Agullo, P. L., Olbrich, P., Parra-Martinez, A., Paz-Artal, E., Pleguezuelo, D. E., Rodriguez, N. S., Sanchez-Ramon, S., Santos-Perez, J. L., Solanich, X., Soler-Palacin, P., Gonzalez-Amores, M., Ekwall, O., Fasth, A., Bitzenhofer-Gruber, M., Candotti, F., Dimitriou, F., Heininger, U., Holbro, A., Jandus, P., Kolios, A. G. A., Marschall, K., Schmid, J. P., Posfay-Barbe, K. M., Prader, S., Reichenbach, J., Steiner, U. C., Truck, J., Bredius, R. G., de Kruijf- Bazen, S., de Vries, E., Henriet, S. S. V., Kuijpers, T. W., Potjewijd, J., Rutgers, A., Stol, K., van Aerde, K. J., Van den Berg, J. M., van de Ven, A. A. J. M., Montfrans, J., Aydemir, S., Baris, S., Dogu, F., Ikinciogullari, A., Karakoc-Aydiner, E., Kilic, S. S., Kiykim, A., Kokcu Karadag, S. I., Kutukculer, N., Ocak, S., Unal, E., Boyarchuk, O., Hilfanova, A., Kostyuchenko, L. V., Alachkar, H., Arkwright, P. D., Baxendale, H. E., Bernatoniene, J., Coulter, T. I., Garcez, T., Goddard, S., Gompels, M. M., Grigoriadou, S., Herriot, R., Herwadkar, A., Huissoon, A., Ibberson, L., Nademi, Z., Noorani, S., Parvin, S., Steele, C. L., Thomas, M., Waruiru, C., Yong, P. F. K., and Bourne, H.
Subjects: 0301 basic medicine, Male, Pediatrics, syndromic, Sex Factor, Disease, registry, medicine.disease_cause, Cohort Studies, 0302 clinical medicine, Primary Immunodeficiency Disease, inborn error of immunity, Immunology and Allergy, warning signs, Age Factor, Registries, Family history, presenting symptom, Child, Primary immunodeficiency, Granuloma, autoimmune, immune dysregulation, inflammatory, Adult, Autoimmune Diseases, Female, Humans, Infections, Lymphoproliferative Disorders, Middle Aged, Primary Immunodeficiency Diseases, Sex Factors, Age Factors, 10177 Dermatology Clinic, Infections/epidemiology, 3. Good health, Settore MED/02, Warning signs, Lymphoproliferative Disorder, 2723 Immunology and Allergy, Infection, Human, medicine.medical_specialty, Immunology, 610 Medicine & health, Malignancy, primary immunodeficiency, Autoimmune Disease, 03 medical and health sciences, Immunity, Autoimmune Diseases/epidemiology, medicine, 2403 Immunology, business.industry, warning sign, Common variable immunodeficiency, Granuloma/epidemiology, Immune dysregulation, medicine.disease, Primary Immunodeficiency Diseases/epidemiology, 030104 developmental biology, Lymphoproliferative Disorders/epidemiology, Cohort Studie, business, 030215 immunology
Abstract: BACKGROUND: Inborn errors of immunity (IEI) are rare diseases, which makes diagnosis a challenge. A better description of the initial presenting manifestations should improve awareness and avoid diagnostic delay. Although increased infection susceptibility is a well-known initial IEI manifestation, less is known about the frequency of other presenting manifestations.OBJECTIVE: We sought to analyze age-related initial presenting manifestations of IEI including different IEI disease cohorts.METHODS: We analyzed data on 16,486 patients of the European Society for Immunodeficiencies Registry. Patients with autoinflammatory diseases were excluded because of the limited number registered.RESULTS: Overall, 68% of patients initially presented with infections only, 9% with immune dysregulation only, and 9% with a combination of both. Syndromic features were the presenting feature in 12%, 4% had laboratory abnormalities only, 1.5% were diagnosed because of family history only, and 0.8% presented with malignancy. Two-third of patients with IEI presented before the age of 6 years, but a quarter of patients developed initial symptoms only as adults. Immune dysregulation was most frequently recognized as an initial IEI manifestation between age 6 and 25 years, with male predominance until age 10 years, shifting to female predominance after age 40 years. Infections were most prevalent as a first manifestation in patients presenting after age 30 years.CONCLUSIONS: An exclusive focus on infection-centered warning signs would have missed around 25% of patients with IEI who initially present with other manifestations.
Published: 2021

46. Supportive care during pediatric hematopoietic stem cell transplantation: beyond infectious diseases. A report from workshops on supportive care of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author: Krzysztof Kałwak, Peter Bader, Alice Bertaina, Riitta Niinimäki, Tayfun Güngör, Brenda Gibson, Marianne Ifversen, Marc Ansari, Christina Diaz de Heredia, Akif Yesilipek, Tiago Nava, Jerry Stein, Jochen Buechner, E. Trigoso, Jacek Wachowiak, Marco Deiana, Koray Yalcin, Christina Peters, Halvard Boenig, Simone Cesaro, Isaac Yaniv, Gergely Kriván, Kim Vettenranta, Toni Matic, Dominik Turkiewicz, Roland Meisel, Michaela Kuhlen, Giovanna Lucchini, Shahrzad Bakhtiar, Andre Willasch, Luisa Sisinni, Petr Sedlacek, Daphna Hutt, Thomas Lehrnbecher, Anita Lawitschka, Adriana Balduzzi, Jean Hugues Dalle, Tamara Diesch, Arnaud Dalissier, Selim Corbacioglu, Andrea Jarisch, Ulrike Falkenberg, Nava, T, Ansari, M, Dalle, J, de Heredia, C, Güngör, T, Trigoso, E, Falkenberg, U, Bertaina, A, Gibson, B, Jarisch, A, Balduzzi, A, Boenig, H, Krivan, G, Vettenranta, K, Matic, T, Büchner, J, Kalwak, K, Lawitschka, A, Yesilipek, A, Lucchini, G, Peters, C, Turkiewicz, D, Niinimäki, R, Diesch, T, Lehrnbecher, T, Sedlacek, P, Hutt, D, Dalissier, A, Wachowiak, J, Yaniv, I, Stein, J, Yalçin, K, Sisinni, L, Deiana, M, Ifversen, M, Kuhlen, M, Miesel, R, Bakhtiar, S, Cesaro, S, Willasch, A, Corbacioglu, S, and Bader, P
Subjects: HEPATIC VENOOCCLUSIVE DISEASE, medicine.medical_specialty, bone marrow transplantation, medicine.medical_treatment, MEDLINE, ENTERAL NUTRITION, Hematopoietic stem cell transplantation, ORAL MUCOSITIS, Communicable Diseases, 03 medical and health sciences, bone marrow transplantation, pediatric, supportive care, 0302 clinical medicine, Bone Marrow, Internal medicine, IRON OVERLOAD, Mucositis, Humans, Medicine, Child, Intensive care medicine, SYNDROME/VENO-OCCLUSIVE DISEASE, ANTICIPATORY NAUSEA, Transplantation, Hematology, business.industry, Marrow transplantation, Research, Hematopoietic Stem Cell Transplantation, SEVERITY CRITERIA, medicine.disease, supportive care, hematopoietic stem cell transplantation, pediatric, URSODEOXYCHOLIC ACID, Europe, supportive care, Leukemia, pediatric, ANTINEOPLASTIC MEDICATION, surgical procedures, operative, Parenteral nutrition, medicine.anatomical_structure, 030220 oncology & carcinogenesis, Bone marrow, business, CHEMOTHERAPY-INDUCED NAUSEA, 030215 immunology
Abstract: Hematopoietic stem cell transplantation (HSCT) is currently the standard of care for many malignant and nonmalignant blood diseases. As several treatment-emerging acute toxicities are expected, optimal supportive measurements critically affect HSCT outcomes. The paucity of good clinical studies in supportive practices gives rise to the establishment of heterogeneous guidelines across the different centers, which hampers direct clinical comparison in multicentric studies. Aiming to harmonize the supportive care provided during the pediatric HSCT in Europe, the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) promoted dedicated workshops during the years 2017 and 2018. The present paper describes the resulting consensus on the management of sinusoidal obstructive syndrome, mucositis, enteral and parenteral nutrition, iron overload, and emesis during HSCT.
Published: 2020

47. Supportive Care During Pediatric Hematopoietic Stem Cell Transplantation : Prevention of Infections. A Report From Workshops on Supportive Care of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Author: Marianne Ifversen, Roland Meisel, Petr Sedlacek, Krzysztof Kalwak, Luisa Sisinni, Daphna Hutt, Thomas Lehrnbecher, Adriana Balduzzi, Tamara Diesch, Andrea Jarisch, Tayfun Güngör, Jerry Stein, Isaac Yaniv, Halvard Bonig, Michaela Kuhlen, Marc Ansari, Tiago Nava, Jean-Hugues Dalle, Cristina Diaz-de-Heredia, Eugenia Trigoso, Ulrike Falkenberg, Mihaela Hartmann, Marco Deiana, Marta Canesi, Chiara Broggi, Alice Bertaina, Brenda Gibson, Gergely Krivan, Kim Vettenranta, Toni Matic, Jochen Buechner, Anita Lawitschka, Christina Peters, Akif Yesilipek, Koray Yalçin, Giovanna Lucchini, Shahrzad Bakhtiar, Dominik Turkiewicz, Riitta Niinimäki, Jacek Wachowiak, Simone Cesaro, Arnaud Dalissier, Selim Corbacioglu, Andre Manfred Willasch, Peter Bader, Children's Hospital, Lastentautien yksikkö, HUS Children and Adolescents, Ifversen, M, Meisel, R, Sedlacek, P, Kalwak, K, Sisinni, L, Hutt, D, Lehrnbecher, T, Balduzzi, A, Diesch, T, Jarisch, A, Gungor, T, Stein, J, Yaniv, I, Bonig, H, Kuhlen, M, Ansari, M, Nava, T, Dalle, J, Diaz-de-Heredia, C, Trigoso, E, Falkenberg, U, Hartmann, M, Deiana, M, Canesi, M, Broggi, C, Bertaina, A, Gibson, B, Krivan, G, Vettenranta, K, Matic, T, Buechner, J, Lawitschka, A, Peters, C, Yesilipek, A, Yalcin, K, Lucchini, G, Bakhtiar, S, Turkiewicz, D, Niinimaki, R, Wachowiak, J, Cesaro, S, Dalissier, A, Corbacioglu, S, Willasch, A, Bader, P, Institut Català de la Salut, [Ifversen M] Department of Paediatrics and Adolescent Medicine, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark. [Meisel R] Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich-Heine-University, Duesseldorf, Germany. [Sedlacek P] Department of Pediatric Hematology and Oncology, Hospital Motol, Charles University, Prague, Czechia. [Kalwak K] Department of Pediatric Hematology, Oncology and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland. [Sisinni L] Pediatric Hematology, Oncology and Hematopoietic Stem Cell Transplantation Unit, Hospital Santa Creu i Sant Pau, Barcelona, Spain. [Hutt D] Division of Pediatric Hematology, Oncology and Bone Marrow Transplantation, The Edmond and Lily Safra Children's Hospital, Tel Aviv, Israel. [Diaz-de-Heredia C] Servei d’Oncologia i Hematologia Pediàtriques, Vall d'Hebron Hospital Universitari, Barcelona, Spain, and Vall d'Hebron Barcelona Hospital Campus
Subjects: medicine.medical_treatment, allogeneic hematological stem cell transplantation, CHILDREN, Hematopoietic stem cell transplantation, Otros calificadores::Otros calificadores::/efectos adversos [Otros calificadores], GUIDELINES, Pediatrics, PROPHYLAXIS, 0302 clinical medicine, 3123 Gynaecology and paediatrics, Antibiotic prophylactic therapy, Young adult, Children, antibiotic prophylactic therapy, Original Research, COMPLICATIONS, Hematology, ddc:618, Therapeutics::Biological Therapy::Immunomodulation::Immunotherapy::Immunosuppression::Transplantation Conditioning [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT], terapéutica::terapia biológica::inmunomodulación::inmunoterapia::inmunosupresión::acondicionamiento para el trasplante [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS], Vaccination, Hematopoietic stem cell, allogeneic hematological stem cell transplantation, antibiotic prophylactic therapy, children, infection precaution, vaccination, CHEMOTHERAPY, CANCER, 3. Good health, Leukemia, medicine.anatomical_structure, surgical procedures, operative, 030220 oncology & carcinogenesis, Cohort, terapéutica::terapia biológica::tratamientos basados en células y tejidos::trasplante de células::trasplante de células madre::trasplante de células madre hematopoyéticas [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS], medicine.medical_specialty, Cirurgia - Complicacions, Isolation (health care), Pathological Conditions, Signs and Symptoms::Pathologic Processes::Postoperative Complications [DISEASES], RJ1-570, 03 medical and health sciences, Internal medicine, medicine, Other subheadings::Other subheadings::/adverse effects [Other subheadings], PARTICULATE AIR FILTRATION, infection precaution, ddc:610, NEUTROPENIC DIET, Intensive care medicine, Donor selection, business.industry, Cèl·lules mare hematopoètiques - Trasplantació, medicine.disease, vaccination, afecciones patológicas, signos y síntomas::procesos patológicos::complicaciones posoperatorias [ENFERMEDADES], RECIPIENTS, Infection precaution, Pediatrics, Perinatology and Child Health, Sang - Malalties, Therapeutics::Biological Therapy::Cell- and Tissue-Based Therapy::Cell Transplantation::Stem Cell Transplantation::Hematopoietic Stem Cell Transplantation [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT], business, Allogeneic hematological stem cell transplantation, LEUKEMIA, 030215 immunology
Abstract: Terapia profiláctica antibiótica; Niños; Vacunación Teràpia profilàctica antibiòtica; Nens; Vacunació Antibiotic prophylactic therapy; Children; Vaccination Specific protocols define eligibility, conditioning, donor selection, graft composition and prophylaxis of graft vs. host disease for children and young adults undergoing hematopoietic stem cell transplant (HSCT). However, international protocols rarely, if ever, detail supportive care, including pharmaceutical infection prophylaxis, physical protection with face masks and cohort isolation or food restrictions. Supportive care suffers from a lack of scientific evidence and implementation of practices in the transplant centers brings extensive restrictions to the child's and family's daily life after HSCT. Therefore, the Board of the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) held a series of dedicated workshops since 2017 with the aim of initiating the production of a set of minimal recommendations. The present paper describes the consensus reached within the field of infection prophylaxis.
Published: 2021

48. Treatment and Outcome Analysis of 639 Relapsed Non-Hodgkin Lymphomas in Children and Adolescents and Resulting Treatment Recommendations

Author: Adriana Balduzzi, G. A. Amos Burke, Stephanie Mueller, Birgit Burkhardt, Lisa Lyngsie Hjalgrim, Kristin Koeppen, Andishe Attarbaschi, Edita Kabickova, Felix Niggli, Mara Andrés, Heidi Herbrueggen, Nathalie Garnier, Alina Fedorova, Jan Loeffen, E. Bubanska, Monika Csóka, Anne Uyttebroeck, Marta Pillon, Volkan Hazar, Veronique Minard-Colin, Jelena Lazic, Mary Taj, Karin Mellgren, Wilhelm Woessmann, Tomoo Osumi, Anna Pieczonka, Alan K. S. Chiang, Jacek Wachowiak, Auke Beishuizen, Natalia Myakova, Martin Zimmermann, Svetlana Donska, Julia Palma, Jochen Buechner, Gergely Kriván, Jaime Verdu-Amoros, Burkhardt, Birgit [0000-0002-1151-829X], Taj, Mary [0000-0002-7107-618X], Osumi, Tomoo [0000-0001-5536-6788], Attarbaschi, Andishe [0000-0002-9285-6898], Chiang, Alan Kwok Shing [0000-0002-1089-5325], Wachowiak, Jacek [0000-0002-4680-603X], Uyttebroeck, Anne [0000-0001-5644-424X], Buechner, Jochen [0000-0001-5848-4501], Krivan, Gergely [0000-0003-4853-4354], Burke, GA Amos [0000-0003-2671-9972], Balduzzi, Adriana [0000-0002-5879-0610], Apollo - University of Cambridge Repository, Burkhardt, B, Taj, M, Garnier, N, Minard-Colin, V, Hazar, V, Mellgren, K, Osumi, T, Fedorova, A, Myakova, N, Verdu-Amoros, J, Andres, M, Kabickova, E, Attarbaschi, A, Chiang, A, Bubanska, E, Donska, S, Hjalgrim, L, Wachowiak, J, Pieczonka, A, Uyttebroeck, A, Lazic, J, Loeffen, J, Buechner, J, Niggli, F, Csoka, M, Krivan, G, Palma, J, Burke, G, Beishuizen, A, Koeppen, K, Mueller, S, Herbrueggen, H, Woessmann, W, Zimmermann, M, Balduzzi, A, and Pillon, M
Subjects: Oncology, Cancer Research, medicine.medical_specialty, medicine.medical_treatment, Outcome analysis, CHILDHOOD, Hematopoietic stem cell transplantation, ACUTE-LYMPHOBLASTIC-LEUKEMIA, Article, refractory and relapsed non-Hodgkin lymphoma, 03 medical and health sciences, RETROSPECTIVE ANALYSIS, 0302 clinical medicine, PROGNOSTIC-FACTORS, Refractory, immune system diseases, Internal medicine, hemic and lymphatic diseases, medicine, Overall survival, stem cell transplant, ALLOGENEIC TRANSPLANTATION, RITUXIMAB, Anaplastic large-cell lymphoma, RC254-282, Science & Technology, business.industry, Disease progression, Neoplasms. Tumors. Oncology. Including cancer and carcinogens, B-CELL LYMPHOMA, Children and adolescent, medicine.disease, 3. Good health, Lymphoma, Leukemia, HIGH-RISK, children and adolescents, 030220 oncology & carcinogenesis, TRIAL, BURKITT-LYMPHOMA, business, Life Sciences & Biomedicine, 030215 immunology
Abstract: Despite poor survival, controversies remain in the treatment for refractory or relapsed pediatric non-Hodgkin lymphoma (r/r NHL). The current project aimed to collect international experience on the re-induction treatment of r/r NHL, hematopoietic stem cell transplantation (HSCT), risk factors associated with outcome, and to suggest treatment recommendations. Inclusion criteria were (i) refractory disease, disease progression or relapse of any NHL subtype except anaplastic large cell lymphoma, (ii) age &lt, 18 years at initial diagnosis, (iii) diagnosis in/after January 2000. Data from 639 eligible patients were evaluable. The eight-year probability of overall survival was 34 ± 2% with highly significant differences according to NHL subtypes: 28 ± 3% for 254 Burkitt lymphoma/leukemia, 50 ± 6% for 98 diffuse large B-cell lymphomas, 57 ± 8% for 41 primary mediastinal large B-cell lymphomas, 27 ± 3% for 177 T-lymphoblastic lymphomas, 52 ± 10% for 34 precursor-B-cell lymphoblastic lymphomas and 30 ± 9% for 35 patients with rare NHL subtypes. Subtype-specific factors associated with survival and treatment recommendations are suggested. There were no survivors without HSCT, except in few very small subgroups. Conclusions: There is an urgent need to further improve survival in r/r NHL. The current study provides the largest real-world series, which underlines the role of HSCT and suggests treatment recommendations.
Published: 2021

49. ABO incompatibile graft management in pediatric transplantation

Author: Balduzzi, Adriana, Bönig, Halvard, Jarisch, Andrea, Nava, Tiago, Ansari Djaberi, Marc Georges, Cattoni, Alessandro, Prunotto, Giulia, Lucchini, Giovanna, Krivan, Gergely, Matic, Toni, Kalwak, Krzyzstof, Yesilipek, Akif, Ifversen, Marianne, Svec, Peter, Buechner, Jochen, Vettenranta, Kim, Meisel, Roland, Lawitschka, Anita, Peters, Christina, Gibson, Brenda, Dalissier, Arnaud, Corbacioglu, Selim, Willasch, André, Dalle, Jean-Hugues, Bader, Peter, EBMT Pediatric Diseases Working Party, Balduzzi, A, Bönig, H, Järisch, A, Nava, T, Ansari, M, Cattoni, A, Prunotto, G, Lucchini, G, Krivan, G, Matic, T, Kalwak, K, Yesilipek, A, Ifversen, M, Svec, P, Büchner, J, Vettenranta, K, Meisel, R, Lawitschka, A, Peters, C, Gibson, B, Dalissier, A, Corbacioglu, S, Willasch, A, Dalle, J, Bader, P, HUS Children and Adolescents, Lastentautien yksikkö, Children's Hospital, and Helsinki University Hospital Area
Subjects: medicine.medical_specialty, Erythrocytes, BONE-MARROW, medicine.medical_treatment, Pure red cell aplasia, Hematopoietic stem cell transplantation, Hemolysis, Isohemagglutinin, ABO Blood-Group System, ABO incompatibility, 03 medical and health sciences, 0302 clinical medicine, TRANSFUSION, 3123 Gynaecology and paediatrics, ABO blood group system, medicine, Humans, Child, Bone Marrow Transplantation, Transplantation, ddc:618, PLASMA, business.industry, Hematology, medicine.disease, 3. Good health, Surgery, REDUCTION, medicine.anatomical_structure, pediatric, Tolerability, Blood Group Incompatibility, 030220 oncology & carcinogenesis, hematopoietic stem cell transplantation, Erythropoiesis, PROGENITOR-CELL TRANSPLANTATION, Bone marrow, DEPLETION, business, 030215 immunology
Abstract: Up to 40% of donor-recipient pairs in SCT have some degree of ABO incompatibility, which may cause severe complications. The aim of this study was to describe available options and survey current practices by means of a questionnaire circulated within the EBMT Pediatric Diseases Working Party investigators. Major ABO incompatibility (donor's RBCs have antigens missing on the recipient's cell surface, towards which the recipient has circulating isohemagglutinins) requires most frequently an intervention in case of bone marrow grafts, as immediate or delayed hemolysis, delayed erythropoiesis and pure red cell aplasia may occur. RBC depletion from the graft (82%), recipient plasma-exchange (14%) were the most common practices, according to the survey. Graft manipulation is rarely needed in mobilized peripheral blood grafts. In case of minor incompatible grafts (donor has isohemagglutinins directed against recipient RBC antigens), isohemagglutinin depletion from the graft by plasma reduction/centrifugation may be considered, but acute tolerability of minor incompatible grafts is rarely an issue. According to the survey, minor ABO incompatibility was either managed by means of plasma removal from the graft, especially when isohemagglutinin titer was above a certain threshold, or led to no intervention at all (41%). Advantages and disadvantages of each method are discussed.
Published: 2021

50. P484 - The importance of non-HLA (TLX-B) alloantigens in related BMT

Author: Nyári, E., Padányi, Á., Gyódi, É., Rajczy, K., Dénes, R., Masszi, T., Kriván, G., and Petrányi, Gy.
Published: 1996
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