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1. Outcomes of patients undergoing allogeneic haematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia; a study on behalf of the PDWP of the EBMT

Author

Aldebert, Clémence, Fahd, Mony, Galimard, Jacques-Emmanuel, Ghemlas, Ibrahim A., Zecca, Marco, Silva, Juliana, Mohseny, Alexander, Kupesiz, Alphan, Hamladji, Rose-Marie, Miranda, Nuno, Güngör, Tayfun, Wynn, Robert F., Merli, Pietro, Sundin, Mikael, Faraci, Maura, Diaz-de-Heredia, Cristina, Burkhardt, Birgit, Bordon, Victoria, Angoso, Marie, Bader, Peter, Ifversen, Marianne, Herrera Arroyo, Concepcion, Maximova, Natalia, Riesco, Susana, Stein, Jerry, Dalissier, Arnaud, Locatelli, Franco, Kalwak, Krzysztof, Dalle, Jean-Hugues, and Corbacioglu, Selim

Published
2024
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2. Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM

Author

Kalwak, Krzysztof, Moser, Laura M., Pötschger, Ulrike, Bader, Peter, Kleinschmidt, Katharina, Meisel, Roland, Dalle, Jean-Hugues, Yesilipek, Akif, Balduzzi, Adriana, Krivan, Gergely, Goussetis, Evgenios, Staciuk, Raquel, Sedlacek, Petr, Pichler, Herbert, Svec, Peter, Gabriel, Melissa, Güngör, Tayfun, Bilic, Ernest, Buechner, Jochen, Renard, Marleen, Vettenranta, Kim, Ifversen, Marianne, Diaz-de-Heredia, Cristina, Stein, Jerry, Toporski, Jacek, Bierings, Marc, Peters, Christina, Ansari, Marc, and Locatelli, Franco

Published
2025
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3. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter, Pötschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M., Balduzzi, Adriana, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco, and Peters, Christina

Published
2024
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4. Outcome of haematopoietic cell transplantation in children with lysosomal acid lipase deficiency: a study on behalf of the EBMT Inborn Errors Working Party

Author

Lum, Su Han, Minkov, Milen, Jones, Simon A., Hazelaar, Sheree, Sirait, Tiarlan, Potter, Jane E., Stepensky, Polina, Garban, Frederic, Pichler, Herbert, Stein, Jerry, Kaya, Zuhre, Schulz, Ansgar, Mellgren, Karin, Diaz de Heredia, Cristina, Pochon, Cecile, Riesco, Susana, Diaz, Miguel Angel, Michel, Gérard, Lindemans, Caroline, Gruhn, Bernd, Albert, Michael H., Lankester, Arjan C., Neven, Bénédicte, and Wynn, Robert

Published
2023
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9. Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia: A prospective multicentre trial of the I‐BFM Study Group

Author

Pichler, Herbert, primary, Sedlacek, Petr, additional, Meisel, Roland, additional, Beier, Rita, additional, Faraci, Maura, additional, Kalwak, Krzysztof, additional, Ifversen, Marianne, additional, Müller, Ingo, additional, Stein, Jerry, additional, Vettenranta, Kim, additional, Kropshofer, Gabriele, additional, Kolenova, Alexandra, additional, Karlhuber, Susanne, additional, Glogova, Evgenia, additional, Poetschger, Ulrike, additional, Peters, Christina, additional, Suttorp, Meinolf, additional, Matthes‐Leodolter, Susanne, additional, and Balduzzi, Adriana, additional

Published
2024
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10. Low rate of nonrelapse mortality in under 4-year-olds with ALL given chemo-conditioning for HSCT: Phase III FORUM study

Author

Bader, P, Poetschger, U, Dalle, J, Moser, L, Balduzzi, A, Ansari, M, Buechner, J, Güngör, T, Ifversen, M, Kriván, G, Pichler, H, Renard, M, Staciuk, R, Sedlacek, P, Stein, J, Heusel, J, Truong, T, Wachowiak, J, Yeşilipek, M, Locatelli, F, Peters, C, Bader, Peter, Poetschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M, Balduzzi, Adriana Cristina, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Kriván, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yeşilipek, M Akif, Locatelli, Franco, Peters, Christina, Bader, P, Poetschger, U, Dalle, J, Moser, L, Balduzzi, A, Ansari, M, Buechner, J, Güngör, T, Ifversen, M, Kriván, G, Pichler, H, Renard, M, Staciuk, R, Sedlacek, P, Stein, J, Heusel, J, Truong, T, Wachowiak, J, Yeşilipek, M, Locatelli, F, Peters, C, Bader, Peter, Poetschger, Ulrike, Dalle, Jean-Hugues, Moser, Laura M, Balduzzi, Adriana Cristina, Ansari, Marc, Buechner, Jochen, Güngör, Tayfun, Ifversen, Marianne, Kriván, Gergely, Pichler, Herbert, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr, Stein, Jerry, Heusel, Jan Robert, Truong, Tony, Wachowiak, Jacek, Yeşilipek, M Akif, Locatelli, Franco, and Peters, Christina

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUMstudy (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBIfree conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median followup: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFSwas 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM)and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95%CI: [removed]1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/ Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure.

Published
2024

11. Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study

Author

Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, Peters, Christina; https://orcid.org/0000-0003-0369-8515, Bader, Peter; https://orcid.org/0000-0003-4554-0265, Pötschger, Ulrike, Dalle, Jean-Hugues; https://orcid.org/0000-0002-8406-0793, Moser, Laura M, Balduzzi, Adriana; https://orcid.org/0000-0002-5879-0610, Ansari, Marc; https://orcid.org/0000-0002-9649-6498, Buechner, Jochen; https://orcid.org/0000-0001-5848-4501, Güngör, Tayfun; https://orcid.org/0000-0002-3261-1186, Ifversen, Marianne, Krivan, Gergely, Pichler, Herbert; https://orcid.org/0000-0001-5107-5123, Renard, Marleen, Staciuk, Raquel, Sedlacek, Petr; https://orcid.org/0000-0001-7895-2886, Stein, Jerry, Heusel, Jan Robert; https://orcid.org/0000-0002-5753-0817, Truong, Tony, Wachowiak, Jacek, Yesilipek, Akif, Locatelli, Franco; https://orcid.org/0000-0002-7976-3654, and Peters, Christina; https://orcid.org/0000-0003-0369-8515

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBI-free conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median follow-up: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFS was 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM) and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95% CI: <0.01-0.09) (P = .406) and 0.42 (95% CI, 0.31-0.52) vs 0.45 (95% CI, 0.34-0.56) (P = .920), respectively. Grade >1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure. This trial was registered at www.clinicaltrials.gov as #NCT01949129.

Published
2024

12. 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, Simone, Pillon, Marta, Sauer, Martin, Smiers, Frans, Faraci, Maura, de Heredia, Cristina Diaz, Wynn, Robert, Greil, Johann, Locatelli, Franco, Veys, Paul, Uyttebroeck, Anne, Ljungman, Per, Chevalier, Patrice, Ansari, Marc, Badell, Isabel, Güngör, Tayfun, Salim, Rahuman, Tischer, Johanna, Tecchio, Cristina, Russell, Nigel, Chybicka, Alicja, Styczynski, Jan, Krivan, Gergely, Smith, Owen, Stein, Jerry, Afanasyev, Boris, Pochon, Cécile, Menconi, Maria Cristina, Bosman, Paul, Mauro, Margherita, Tridello, Gloria, de Latour, Regis Peffault, and Dufour, Carlo

Published
2020
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14. 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, 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, Buechner, 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, Meisel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, and Bader, Peter

Published
2020
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17. Bone marrow failure unresponsive to bone marrow transplant is caused by mutations in thrombopoietin

Author

Seo, Aaron, Ben-Harosh, Miri, Sirin, Mehtap, Stein, Jerry, Dgany, Orly, Kaplelushnik, Joseph, Hoenig, Manfred, Pannicke, Ulrich, Lorenz, Myriam, Schwarz, Klaus, Stockklausner, Clemens, Walsh, Tom, Gulsuner, Suleyman, Lee, Ming K., Sendamarai, Anoop, Sanchez-Bonilla, Marilyn, King, Mary-Claire, Cario, Holger, Kulozik, Andreas E., Debatin, Klaus-Michael, Schulz, Ansgar, Tamary, Hannah, and Shimamura, Akiko

Published
2017
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19. A Need for a Novel Survival Risk Scoring System for Intensive Care Admissions Due to Sepsis in Pediatric Hematology/Oncology Patients.

Author

Wittmann Dayagi, Talya, Nirel, Ronit, Avrahami, Galia, Amar, Shira, Elitzur, Sarah, Fisher, Salvador, Gilead, Gil, Gilad, Oded, Goldberg, Tracie, Izraeli, Shai, Kadmon, Gili, Kaplan, Eytan, Krauss, Aviva, Michaeli, Orli, Stein, Jerry, Steinberg-Shemer, Orna, Tamary, Hannah, Tausky, Osnat, Toledano, Helen, and Weissbach, Avichai

Subjects
PEDIATRIC hematology, SEPSIS, HEMATOLOGIC malignancies, STEM cell transplantation, PEDIATRIC intensive care, C-reactive protein
Abstract

Background: Children with hemato-oncological diseases or following stem cell transplantation (SCT) are at high risk for life-threatening infections; sepsis in this population constitutes a substantial proportion of pediatric intensive care unit (PICU) admissions. The current pediatric prognostic scoring tools to evaluate illness severity and mortality risk are designed for the general pediatric population and may not be adequate for this vulnerable subpopulation. Methods: Retrospective analysis was performed on all PICU admissions for sepsis in children with hemato-oncological diseases or post-SCT, in a single tertiary pediatric hospital between 2008 and 2021 (n = 233). We collected and analyzed demographic, clinical, and laboratory data and outcomes for all patients, and evaluated the accuracy of two major prognostic scoring tools, the Pediatric Logistic Organ Dysfunction-2 (PELOD-2) and the Pediatric Risk of Mortality III (PRISM III). Furthermore, we created a new risk-assessment model that contains additional parameters uniquely relevant to this population. Results: The survival rate for the cohort was 83%. The predictive accuracies of PELOD-2 and PRISM III, as determined by the area under the curve (AUC), were 83% and 78%, respectively. Nine new parameters were identified as clinically significant: age, SCT, viral infection, fungal infection, central venous line removal, vasoactive inotropic score, bilirubin level, C-reactive protein level, and prolonged neutropenia. Unique scoring systems were established by the integration of these new parameters into the algorithm; the new systems significantly improved their predictive accuracy to 91% (p = 0.01) and 89% (p < 0.001), respectively. Conclusions: The predictive accuracies (AUC) of the PELOD-2 and PRISM III scores are limited in children with hemato-oncological diseases admitted to PICU with sepsis. These results highlight the need to develop a risk-assessment tool adjusted to this special population. Such new scoring should represent their unique characteristics including their degree of immunosuppression and be validated in a large multi-center prospective study. [ABSTRACT FROM AUTHOR]

Published
2024
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21. Effective treatment of steroid and therapy-refractory acute graft-versus-host disease with a novel mesenchymal stromal cell product (MSC-FFM)

Author

Bader, Peter, Kuçi, Zyrafete, Bakhtiar, Shahrzad, Basu, Oliver, Bug, Gesine, Dennis, Michael, Greil, Johann, Barta, Aniko, Kállay, Krisztián M., Lang, Peter, Lucchini, Giovanna, Pol, Raj, Schulz, Ansgar, Sykora, Karl-Walter, von Luettichau, Irene, Herter-Sprie, Grit, Uddin, Mohammad Ashab, Jenkin, Phil, Alsultan, Abdulrahman, Buechner, Jochen, Stein, Jerry, Kelemen, Agnes, Jarisch, Andrea, Soerensen, Jan, Salzmann-Manrique, Emilia, Hutter, Martin, Schäfer, Richard, Seifried, Erhard, Klingebiel, Thomas, Bonig, Halvard, and Kuçi, Selim

Published
2018
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22. Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis

Author

Chiesa, Robert, Ruggeri, Annalisa, Paviglianiti, Annalisa, Zecca, Marco, Gónzalez-Vicent, Marta, Bordon, Victoria, Stein, Jerry, Lawson, Sarah, Dupont, Sophie, Lanino, Edoardo, Abecasis, Manuel, Al-Seraihy, Amal, Kenzey, Chantal, Bierings, Marc, Locatelli, Franco, Gluckman, Eliane, Schulz, Ansgar, Gennery, Andrew, Page, Kristin, Kurtzberg, Joanne, and Rocha, Vanderson

Published
2016
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24. 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)

Author

Cesaro, Simone, Pillon, Marta, Sauer, Martin, Smiers, Frans, Faraci, Maura, de Heredia, Cristina Diaz, Wynn, Robert, Greil, Johann, Locatelli, Franco, Veys, Paul, Uyttebroeck, Anne, Ljungman, Per, Chevalier, Patrice, Ansari, Marc, Badell, Isabel, Güngör, Tayfun, Salim, Rahuman, Tischer, Johanna, Tecchio, Cristina, Russell, Nigel, Chybicka, Alicja, Styczynski, Jan, Krivan, Gergely, Smith, Owen, Stein, Jerry, Afanasyev, Boris, Pochon, Cécile, Menconi, Maria Cristina, Bosman, Paul, Mauro, Margherita, Tridello, Gloria, de Latour, Regis Peffault, and Dufour, Carlo

Published
2020
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25. 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)

Author

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, Buechner, 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, Meisel, Roland, Bakhtiar, Shahrzad, Cesaro, Simone, Willasch, Andre, Corbacioglu, Selim, and Bader, Peter

Published
2020
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26. Combined Kidney and Hematopoietic Cell Transplantation for Tolerance Induction between HLA Matched Sibling Donor-Recipient Pairs

Author

Yeshurun, Moshe, primary, Rachamimov, Ruth, additional, Shargian, Liat, additional, Fenig, Eyal, additional, Pasvolsky, Oren, additional, Raanani, Pia, additional, Sela-Navon, Michal, additional, Pearl-Yafe, Michal, additional, Stein, Jerry, additional, Mor, Eitan, additional, Israeli, Moshe, additional, and Nesher, Evyatar, additional

Published
2023
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29. Relapse Is the Most Common Treatment Failure Post HSCT in Children with ALL below 4 Years of Age Given a Chemo-Based Conditioning Regimen. Results from the Prospective Multinational Forum-Trial

Author

Peters, Christina, primary, Poetschger, Ulrike, additional, Dalle, Jean-Hugues, additional, Yesilipek, Akif, additional, Stein, Jerry, additional, Pichler, Herbert, additional, Balduzzi, Adriana, additional, Locatelli, Franco, additional, Sedlacek, Petr, additional, Wachowiak, Jacek, additional, Güngör, Tayfun, additional, Ifversen, Marianne, additional, Renard, Marleen, additional, Truong, Tony H, additional, Staciuk, Raquel, additional, Shaw, Peter J., additional, Kriván, Gergely, additional, Büchner, Jochen, additional, and Bader, Peter, additional

Published
2022
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30. Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Amegakaryocytic Thrombocytopenia, a PDWP/EBMT Study

Author

Aldebert, Clemence, primary, Fahd, Mony, additional, Galimard, Jacques-Emmanuel, additional, Ghemlas, Ibrahim A., additional, Zecca, Marco, additional, Silva, Juliana, additional, Mohseny, Alexander, additional, Kupesiz, Alphan, additional, Hamladji, Rose-Marie, additional, Miranda, Nuno, additional, Gungor, Tayfun, additional, Wynn, Robert F, additional, Merli, Pietro, additional, Sundin, Mikael, additional, Faraci, Maura, additional, Díaz-de-Heredia, Cristina, additional, Burkhardt, Birgit, additional, Bordon, Victoria, additional, Jubert, Charlotte, additional, Bader, Peter, additional, Ifversen, Marianne, additional, Herrera Arroyo, Concepcion, additional, Maximova, Natalia, additional, Riesco, Susana, additional, Stein, Jerry, additional, Dalissier, Arnaud, additional, Locatelli, Franco, additional, Kalwak, Krzysztof, additional, Dalle, Jean-Hugues, additional, and Corbacioglu, Selim, additional

Published
2022
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31. DPP9 deficiency: An inflammasomopathy that can be rescued by lowering NLRP1/IL-1 signaling

Author

Harapas, Cassandra R., primary, Robinson, Kim S., additional, Lay, Kenneth, additional, Wong, Jasmine, additional, Moreno Traspas, Ricardo, additional, Nabavizadeh, Nasrin, additional, Rass-Rothschild, Annick, additional, Boisson, Bertrand, additional, Drutman, Scott B., additional, Laohamonthonkul, Pawat, additional, Bonner, Devon, additional, Xiong, Jingwei Rachel, additional, Gorrell, Mark D., additional, Davidson, Sophia, additional, Yu, Chien-Hsiung, additional, Fleming, Mark D., additional, Gudera, Jonas, additional, Stein, Jerry, additional, Ben-Harosh, Miriam, additional, Groopman, Emily, additional, Shimamura, Akiko, additional, Tamary, Hannah, additional, Kayserili, Hülya, additional, Hatipoğlu, Nevin, additional, Casanova, Jean-Laurent, additional, Bernstein, Jonathan A., additional, Zhong, Franklin L., additional, Masters, Seth L., additional, and Reversade, Bruno, additional

Published
2022
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33. Analysis of risk factors influencing outcomes after cord blood transplantation in children with juvenile myelomonocytic leukemia: a EUROCORD, EBMT, EWOG-MDS, CIBMTR study

Author

Locatelli, Franco, Crotta, Alessandro, Ruggeri, Annalisa, Eapen, Mary, Wagner, John E., MacMillan, Margaret L., Zecca, Marco, Kurtzberg, Joanne, Bonfim, Carmem, Vora, Ajay, Díaz de Heredia, Cristina, Teague, Lochie, Stein, Jerry, O'Brien, Tracey A., Bittencourt, Henrique, Madureira, Adrienne, Strahm, Brigitte, Peters, Christina, Niemeyer, Charlotte, Gluckman, Eliane, and Rocha, Vanderson

Published
2013
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34. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling

Author

Locatelli, Franco, Kabbara, Nabil, Ruggeri, Annalisa, Ghavamzadeh, Ardeshir, Roberts, Irene, Li, Chi Kong, Bernaudin, Françoise, Vermylen, Christiane, Dalle, Jean-Hugues, Stein, Jerry, Wynn, Robert, Cordonnier, Catherine, Pinto, Fernando, Angelucci, Emanuele, Socié, Gérard, Gluckman, Eliane, Walters, Mark C., and Rocha, Vanderson

Published
2013
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37. Transplant results in adults with Fanconi anaemia

Author

Bierings, Marc, Bonfim, Carmem, Peffault De Latour, Regis, Aljurf, Mahmoud, Mehta, Parinda A., Knol, Cora, Boulad, Farid, Tbakhi, Abdelghani, Esquirol, Albert, McQuaker, Grant, Sucak, Gulsan A., Othman, Tarek B., Halkes, Constantijn J. M., Carpenter, Ben, Niederwieser, Dietger, Zecca, Marco, Kröger, Nicolaus, Michallet, Mauricette, Risitano, Antonio M., Ehninger, Gerhard, Porcher, Raphael, Dufour, Carlo, Bonfirm, Carmem, Socié, Gerard, Gurman, Gunham, Ghavamzadeh, Ardesir, Hamladji, Rise‐Marie, van Lint, M.T., Stepensky, Polina, Koh, Mickey, Ozkurt, Zubeyde Nur, Veelken, Joan Hendrik, Bunjes, Donald, Beelen, Dietrich, Campos, Antonio, Robinson, Stephen, Alessandrino, E. Paolo, Unal, Ali, Fernandez Navarro, José Maria, Mufti, G.J., Velardi, Andrea, Passweg, Jakob, Apperley, Jane, Sengeloev, Henrik, Ljungman, Per, Foá, Roberto, Alegre, Adrián, Espiga, Carlos Richard, Cornelissen, J.J., Di Bartolomeo, Paolo, Cordonnier, Catherine, Browne, Paul, Jubert, Charlotte, Gastl, Günther, Pierelli, Luca, Johansson, Jan‐Erik, Fagioli, Franca, Moraleda, José, Zuckerman, Tsila, Bazarbachi, Ali, Sedlacek, Petr, Rössig, Claudia, Wynn, R.F., Hallek, Michael, Toren, Amos, Zudaire, Teresa, Clausen, Joahannes, Spencer, Andrew, Grazon Lopez, Sebastian, Schots, Rik, Komarnicki, M., González Muniz, Soledad, Vitek, Antonin, Rambaldi, Alessandro, Merli, Francesco, Rubio, Marie Thérese, Rossig, Claudia, Cabrera Marín, José Rafael, Porto, Fulvio, Kerre, Tessa, Metzner, Bernd, Stein, Jerry, Bertrand, Yves, Ciceri, Fabio, Chybicka, Alicja, Diez‐Martin, J.L., Bayoumy, Mohamed, de la Fuente, Josu, and Fegueux, Nathalie

Published
2018
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38. Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial

Author

Corbacioglu, Selim, Cesaro, Simone, Faraci, Maura, Valteau-Couanet, Dominique, Gruhn, Bernd, Rovelli, Attilio, Boelens, Jaap J, Hewitt, Annette, Schrum, Johanna, Schulz, Ansgar S, Müller, Ingo, Stein, Jerry, Wynn, Robert, Greil, Johann, Sykora, Karl-Walter, Matthes-Martin, Susanne, Führer, Monika, O'Meara, Anne, Toporski, Jacek, Sedlacek, Petr, Schlegel, Paul G, Ehlert, Karoline, Fasth, Anders, Winiarski, Jacek, Arvidson, Johan, Mauz-Körholz, Christine, Ozsahin, Hulya, Schrauder, Andre, Bader, Peter, Massaro, Joseph, D'Agostino, Ralph, Hoyle, Margaret, Iacobelli, Massimo, Debatin, Klaus-Michael, Peters, Christina, and Dini, Giorgio

Published
2012
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39. Total Body Irradiation or Chemotherapy Conditioning in Childhood ALL: A Multinational, Randomized, Noninferiority Phase III Study

Author

Peters, Christina, Dalle, Jean-Hugues, Locatelli, Franco, Poetschger, Ulrike, Sedlacek, Petr, Buechner, Jochen, Shaw, Peter J, Staciuk, Raquel, Ifversen, Marianne, Pichler, Herbert, Vettenranta, Kim, Svec, Peter, Aleinikova, Olga, Stein, Jerry, Güngör, Tayfun, Toporski, Jacek, Truong, Tony H, Diaz-de-Heredia, Cristina, Bierings, Marc, Ariffin, Hany, Essa, Mohammed, Burkhardt, Birgit, Schultz, Kirk, Meisel, Roland, Lankester, Arjan, Ansari Djaberi, Marc Georges, Schrappe, Martin, von Stackelberg, Arend, Balduzzi, Adriana, Corbacioglu, Selim, Bader, Peter, IBFM Study Group, IntReALL Study Group, I-BFM SCT Study Group, EBMT Paediatric Diseases Working Party, Peters, C, Dalle, J, Locatelli, F, Poetschger, U, Sedlacek, P, Buechner, J, Shaw, P, Staciuk, R, Ifversen, M, Pichler, H, Vettenranta, K, Svec, P, Aleinikova, O, Stein, J, Güngör, T, Toporski, J, Truong, T, Diaz-de-Heredia, C, Bierings, M, Ariffin, H, Essa, M, Burkhardt, B, Schultz, K, Meisel, R, Lankester, A, Ansari, M, Schrappe, M, von Stackelberg, A, Balduzzi, A, Corbacioglu, S, Bader, P, HUS Children and Adolescents, University Management, Lastentautien yksikkö, and Children's Hospital

Subjects
Male, Oncology, Cancer Research, Lymphoblastic Leukemia, medicine.medical_treatment, Hematopoietic stem cell transplantation, law.invention, 0302 clinical medicine, Randomized controlled trial, 3123 Gynaecology and paediatrics, Busulfan / analogs & derivatives, law, Antineoplastic Combined Chemotherapy Protocols, TBI, Child, Childhood all, Etoposide, Busulfan / administration & dosage, Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy, ddc:618, Precursor Cell Lymphoblastic Leukemia-Lymphoma / pathology, Chemoradiotherapy, ORIGINAL REPORTS, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Total body irradiation, Prognosis, 3. Good health, Survival Rate, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Child, Preschool, 030220 oncology & carcinogenesis, HSCT, hematopoietic stem cell transplantation, Chemoradiotherapy / mortality, Female, Vidarabine, Whole-Body Irradiation, medicine.medical_specialty, Adolescent, 3122 Cancers, Vidarabine / analogs & derivatives, acute lymphoblastic leukemia, Equivalence Trials as Topic, 03 medical and health sciences, Internal medicine, Antineoplastic Combined Chemotherapy Protocols / therapeutic use, Hematologic Malignancy, medicine, Humans, Busulfan, Survival rate, childhood, Chemotherapy, business.industry, International Agencies, Thiotepa / administration & dosage, Clinical trial, Etoposide / administration & dosage, Vidarabine / administration & dosage, Whole-Body Irradiation / mortality, business, Literatur Kommentiert, total body irradiation, Thiotepa, Follow-Up Studies, 030215 immunology
Abstract

PURPOSE Total body irradiation (TBI) before allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with acute lymphoblastic leukemia (ALL) is efficacious, but long-term side effects are concerning. We investigated whether preparative combination chemotherapy could replace TBI in such patients. PATIENTS AND METHODS FORUM is a randomized, controlled, open-label, international, multicenter, phase III, noninferiority study. Patients ≤ 18 years at diagnosis, 4-21 years at HSCT, in complete remission pre-HSCT, and with an HLA-compatible related or unrelated donor were randomly assigned to myeloablative conditioning with fractionated 12 Gy TBI and etoposide versus fludarabine, thiotepa, and either busulfan or treosulfan. The noninferiority margin was 8%. With 1,000 patients randomly assigned in 5 years, 2-year minimum follow-up, and one-sided alpha of 5%, 80% power was calculated. A futility stopping rule would halt random assignment if chemoconditioning was significantly inferior to TBI (EudraCT: 2012-003032-22; ClinicalTrials.gov: NCT01949129 ). RESULTS Between April 2013 and December 2018, 543 patients were screened, 417 were randomly assigned, 212 received TBI, and 201 received chemoconditioning. The stopping rule was applied on March 31, 2019. The median follow-up was 2.1 years. In the intention-to-treat population, 2-year overall survival (OS) was significantly higher following TBI (0.91; 95% CI, 0.86 to 0.95; P < .0001) versus chemoconditioning (0.75; 95% CI, 0.67 to 0.81). Two-year cumulative incidence of relapse and treatment-related mortality were 0.12 (95% CI, 0.08 to 0.17; P < .0001) and 0.02 (95% CI, < 0.01 to 0.05; P = .0269) following TBI and 0.33 (95% CI, 0.25 to 0.40) and 0.09 (95% CI, 0.05 to 0.14) following chemoconditioning, respectively. CONCLUSION Improved OS and lower relapse risk were observed following TBI plus etoposide compared with chemoconditioning. We therefore recommend TBI plus etoposide for patients > 4 years old with high-risk ALL undergoing allogeneic HSCT.

Published
2021
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44. Decreasing Burnout and Improving Work Environment: The Impact of Firgun on a Pediatric Hematopoietic Cell Transplant Team.

Author

Stein, Jerry, Madni, Arshia, Moody, Karen, Kramer, Deborah, Vaughn, Dagny, Bhatia, Shalini, Sellers, Janet, Snyder, Angela, Zhaohua Lu, Baker, Justin N., and Levine, Deena R.

Subjects
PSYCHOLOGICAL burnout, WORK environment, PILOT projects, COMPUTER software, STATISTICS, FRUSTRATION, RESEARCH methodology, JOB stress, INTERVIEWING, HEALTH outcome assessment, SURVEYS, PSYCHOLOGICAL tests, PEARSON correlation (Statistics), HEALTH care teams, QUESTIONNAIRES, DESCRIPTIVE statistics, HEMATOPOIETIC stem cell transplantation, FATIGUE (Physiology), DATA analysis, DATA analysis software, LONGITUDINAL method
Abstract

PURPOSE Oncology teams are challenged by BO, which may be alleviated by meaningful recognition. In this study, firgun—altruistic acknowledgment—was implemented on a pediatric hematopoietic cell transplant unit to evaluate its impact on staff and work environment. METHODS In this longitudinal, mixed-methods pilot study, interdisciplinary inpatient hematopoietic cell transplant providers received web-based firgun education. Electronic administration of validated surveys occurred at baseline and 8 weeks, including Perceived Stress Scale, Professional Quality of Life Scale, Maslach Burnout Inventory, Workplace Civility Index, Areas of Work Life Survey, and WHO-5. Weekly e-mails reminded participants to practice and log firgun. Wilcoxon signed test for paired data compared pre/post results. Interviews conducted at project completion were coded using MaxQDA software. RESULTS Forty-two participants enrolled; 25 completed pre/post surveys; eight were interviewed. At study end, participants reported feeling less nervous and stressed (P = .008), and less difficulty coping (P = .01; Perceived Stress Scale), while noting increased acknowledgment of others' work (P = .04) and seeking constructive feedback (P = .04; Workplace Civility Index). Marked BO was not evident overall on the Maslach Burnout Inventory; however, emotional exhaustion subscale mean (SD) scores improved from pre (19.4 [8.6]) to post (16 [6.3; P = .02]) and individual items illustrated decreased fatigue (P = .008), frustration (P = .04), and feeling “at the end of my rope” (P = .001). Postintervention participants noted increased receipt of recognition (P = .02; Areas of Work Life Survey), decreased feeling “bogged down” (P = .02), decreased affective stress (P = .04), and negative pre-occupations (P = .04; Professional Quality of Life Scale). Qualitative analysis revealed themes of improved confidence at work and enhanced feelings of trust and teamwork. CONCLUSION Firgun is a tool that can potentially reduce BO and stress in interdisciplinary providers, facilitate teamwork, and promote positive work environments in clinical oncology and beyond. [ABSTRACT FROM AUTHOR]

Published
2023
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48. Allogeneic hematopoietic stem cell transplantation in children and adolescents with recurrent and refractory Hodgkin lymphoma: an analysis of the European Group for Blood and Marrow Transplantation

Author

Claviez, Alexander, Canals, Carme, Dierickx, Daan, Stein, Jerry, Badell, Isabel, Pession, Andrea, Mackinnon, Stephen, Slavin, Shimon, Dalle, Jean-Hugues, Chacón, Manuel Jurado, Sarhan, Mahmoud, Wynn, Robert Francis, Suttorp, Meinolf, Dini, Giorgio, Sureda, Anna, and Schmitz, Norbert

Published
2009
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