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174 results on '"Wynn, R F"'

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. Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation

Author

Kunin-Batson, A. S., Shapiro, E. G., Rudser, K. D., Lavery, C. A., Bjoraker, K. J., Jones, S. A., Wynn, R. F., Vellodi, A., Tolar, J., Orchard, P. J., Wraith, J. E., Baumgartner, Matthias R., Series editor, Patterson, Marc, Series editor, Rahman, Shamima, Series editor, Peters, Verena, Series editor, Morava, Eva, Editor-in-chief, Zschocke, Johannes, Series editor, and Baumgartner, Matthias, editor

Published
2016
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5. Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation

Author

Kunin-Batson, A. S., primary, Shapiro, E. G., additional, Rudser, K. D., additional, Lavery, C. A., additional, Bjoraker, K. J., additional, Jones, S. A., additional, Wynn, R. F., additional, Vellodi, A., additional, Tolar, J., additional, Orchard, P. J., additional, and Wraith, J. E., additional

Published
2015
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11. Allogeneic hematopoietic stem cell transplantation in leukocyte adhesion deficiency type I and III

Author

Bakhtiar, S., Salzmann-Manrique, E., Blok, H. -J., Eikema, D. -J., Hazelaar, S., Ayas, M., Toren, A., Goldstein, G., Moshous, D., Locatelli, Franco, Merli, P., Michel, G., Ozturk, G., Schulz, A., Heilmann, C., Ifversen, M., Wynn, R. F., Aleinikova, O., Bertrand, Y., Tbakhi, A., Veys, P., Karakukcu, M., Kupesiz, A., Ghavamzadeh, A., Handgretinger, R., Unal, E., Perez-Martinez, A., Gokce, M., Porta, F., Aksu, T., Karasu, G., Badell, I., Ljungman, P., Skorobogatova, E., Yesilipek, A., Zuckerman, T., Bredius, R. R. G., Stepensky, P., Shadur, B., Slatter, M., Gennery, A. R., Albert, M. H., Bader, P., Lankester, A., Locatelli F. (ORCID:0000-0002-7976-3654), Bakhtiar, S., Salzmann-Manrique, E., Blok, H. -J., Eikema, D. -J., Hazelaar, S., Ayas, M., Toren, A., Goldstein, G., Moshous, D., Locatelli, Franco, Merli, P., Michel, G., Ozturk, G., Schulz, A., Heilmann, C., Ifversen, M., Wynn, R. F., Aleinikova, O., Bertrand, Y., Tbakhi, A., Veys, P., Karakukcu, M., Kupesiz, A., Ghavamzadeh, A., Handgretinger, R., Unal, E., Perez-Martinez, A., Gokce, M., Porta, F., Aksu, T., Karasu, G., Badell, I., Ljungman, P., Skorobogatova, E., Yesilipek, A., Zuckerman, T., Bredius, R. R. G., Stepensky, P., Shadur, B., Slatter, M., Gennery, A. R., Albert, M. H., Bader, P., Lankester, A., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Type I and III leukocyte adhesion deficiencies (LADs) are primary immunodeficiency disorders resulting in early death due to infections and additional bleeding tendency in LAD-III. The curative treatment of LAD-I and LAD-III is allogeneic hematopoietic stem cell transplantation (allo-HSCT). In this retrospective multicenter study, data were collected using the European Society for Blood and Marrow Transplantation registry; we analyzed data from 84 LAD patients from 33 centers, all receiving an allo-HSCT from 2007 to 2017. The 3-year overall survival estimate (95% confidence interval [CI]) was 83% (74-92) for the entire cohort: 84% (75-94) and 75% (50-100) for LAD-I and LAD-III, respectively. We observed cumulative incidences (95% CI) of graft failure (GF) at 3 years of 17% (9%-26%) and grade II to IV acute graft-versus-host disease (aGVHD) at 100 days of 24% (15%-34%). The estimate (95% CI) at 3 years for GF- and GVHD-II to IV-free survival as event-free survival (EFS) was 56% (46-69) for the entire cohort; 58% (46-72) and 56% (23-88) for LAD-I and LAD-III, respectively. Grade II to IV acute GVHD was a relevant risk factor for death (hazard ratio 3.6; 95% CI 1.4-9.1; P 5.006). Patients' age at transplant $13 months, transplantation from a nonsibling donor, and any serological cytomegalovirus mismatch in donor-recipient pairs were significantly associated with severe acute GVHD and inferior EFS. The choice of busulfan- or treosulfan-based conditioning, type of GVHD prophylaxis, and serotherapy did not impact overall survival, EFS, or aGVHD. An intrinsic inflammatory component of LAD may contribute to inflammatory complications during allo-HSCT, thus providing the rationale for considering anti-inflammatory therapy pretreatment.

Published
2021

28. Transplant results in adults with Fanconi anaemia

Author

Bierings M., Bonfim C., Peffault De Latour R., Aljurf M., Mehta P. A., Knol C., Boulad F., Tbakhi A., Esquirol A., McQuaker G., Sucak G. A., Othman T. B., Halkes C. J. M., Carpenter B., Niederwieser D., Zecca M., Kroger N., Michallet M., Risitano A. M., Ehninger G., Porcher R., Dufour C., Bonfirm C., Socie G., Gurman G., Ghavamzadeh A., Hamladji R. -M., van Lint M. T., Stepensky P., Koh M., Ozkurt Z. N., Veelken J. H., Bunjes D., Beelen D., Campos A., Robinson S., Alessandrino E. P., Unal A., Fernandez Navarro J. M., Mufti G. J., Velardi A., Passweg J., Apperley J., Sengeloev H., Ljungman P., Foa R., Alegre A., Espiga C. R., Cornelissen J. J., Di Bartolomeo P., Cordonnier C., Browne P., Jubert C., Gastl G., Pierelli L., Johansson J. -E., Fagioli F., Moraleda J., Zuckerman T., Bazarbachi A., Sedlacek P., Rossig C., Wynn R. F., Hallek M., Toren A., Zudaire T., Clausen J., Spencer A., Grazon Lopez S., Schots R., Komarnicki M., Gonzalez Muniz S., Vitek A., Rambaldi A., Merli F., Rubio M. T., Cabrera Marin J. R., Porto F., Kerre T., Metzner B., Stein J., Bertrand Y., Ciceri F., Chybicka A., Diez-Martin J. L., Bayoumy M., de la Fuente J., Fegueux N., Bierings, M., Bonfim, C., Peffault De Latour, R., Aljurf, M., Mehta, P. A., Knol, C., Boulad, F., Tbakhi, A., Esquirol, A., Mcquaker, G., Sucak, G. A., Othman, T. B., Halkes, C. J. M., Carpenter, B., Niederwieser, D., Zecca, M., Kroger, N., Michallet, M., Risitano, A. M., Ehninger, G., Porcher, R., Dufour, C., Bonfirm, C., Socie, G., Gurman, G., Ghavamzadeh, A., Hamladji, R. -M., van Lint, M. T., Stepensky, P., Koh, M., Ozkurt, Z. N., Veelken, J. H., Bunjes, D., Beelen, D., Campos, A., Robinson, S., Alessandrino, E. P., Unal, A., Fernandez Navarro, J. M., Mufti, G. J., Velardi, A., Passweg, J., Apperley, J., Sengeloev, H., Ljungman, P., Foa, R., Alegre, A., Espiga, C. R., Cornelissen, J. J., Di Bartolomeo, P., Cordonnier, C., Browne, P., Jubert, C., Gastl, G., Pierelli, L., Johansson, J. -E., Fagioli, F., Moraleda, J., Zuckerman, T., Bazarbachi, A., Sedlacek, P., Rossig, C., Wynn, R. F., Hallek, M., Toren, A., Zudaire, T., Clausen, J., Spencer, A., Grazon Lopez, S., Schots, R., Komarnicki, M., Gonzalez Muniz, S., Vitek, A., Rambaldi, A., Merli, F., Rubio, M. T., Cabrera Marin, J. R., Porto, F., Kerre, T., Metzner, B., Stein, J., Bertrand, Y., Ciceri, F., Chybicka, A., Diez-Martin, J. L., Bayoumy, M., de la Fuente, J., Fegueux, N., Clinical sciences, Hematology, Bierings, Marc, Bonfim, Carmem, Peffault De Latour, Regi, 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, Nicolau, Michallet, Mauricette, Risitano, Antonio M., Ehninger, Gerhard, Porcher, Raphael, Dufour, Carlo, Bonfirm, Carmem, Socié, Gerard, Gurman, Gunham, Ghavamzadeh, Ardesir, Hamladji, Rise-Marie, 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, Velardi, Andrea, Passweg, Jakob, Apperley, Jane, Sengeloev, Henrik, Ljungman, Per, Foá, Roberto, Alegre, Adrián, Espiga, Carlos Richard, 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, Hallek, Michael, Toren, Amo, Zudaire, Teresa, Clausen, Joahanne, Spencer, Andrew, Grazon Lopez, Sebastian, Schots, Rik, 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, Yve, Ciceri, Fabio, Chybicka, Alicja, Bayoumy, Mohamed, de la Fuente, Josu, and Fegueux, Nathalie

Subjects
Transplantation Conditioning, Graft vs Host Disease, Disease, Gastroenterology, 0302 clinical medicine, Retrospective Studie, Risk Factors, Inborn bone marrow failure syndrome, Neoplasms, Cause of Death, risk factors, inborn bone marrow failure syndrome, Transplantation, Homologou, Medicine(all), allogeneic transplant, Fanconi anaemia, myelodysplasia, Adolescent, Adult, Fanconi Anemia, Humans, Middle Aged, Neoplasms, Second Primary, Prognosis, Retrospective Studies, Survival Analysis, Tissue Donors, Transplantation, Homologous, Treatment Outcome, Young Adult, Hematopoietic Stem Cell Transplantation, Hematology, Incidence (epidemiology), Fludarabine, Second Primary, medicine.anatomical_structure, surgical procedures, operative, 030220 oncology & carcinogenesis, young adult, Survival Analysi, Human, medicine.drug, Homologous, medicine.medical_specialty, Cyclophosphamide, Prognosi, Myelodysplasia, Tissue Donor, Graft vs Host Disease/etiology, 03 medical and health sciences, Internal medicine, medicine, Hematopoietic Stem Cell Transplantation/adverse effects, Sibling, Transplantation, business.industry, Risk Factor, Neoplasms, Second Primary/etiology, Confidence interval, Allogeneic transplant, Surgery, Bone marrow, business, Fanconi Anemia/diagnosis, 030215 immunology
Abstract

The outcomes of adult patients transplanted for Fanconi anaemia (FA) have not been well described. We retrospectively analysed 199 adult patients with FA transplanted between 1991 and 2014. Patients were a median of 16years of age when diagnosed with FA, and underwent transplantation at a median age of 23years. Time between diagnosis and transplant was shortest (median 2years) in those patients who had a human leucocyte antigen identical sibling donor. Fifty four percent of patients had bone marrow (BM) failure at transplantation and 46% had clonal disease (34% myelodysplasia, 12% acute leukaemia). BM was the main stem cell source, the conditioning regimen included cyclophosphamide in 96% of cases and fludarabine in 64%. Engraftment occurred in 82% (95% confidence interval [CI] 76–87%), acute graft-versus-host disease (GvHD) grade II–IV in 22% (95% CI 16–28%) and the incidence of chronic GvHD at 96months was 26% (95% CI 20–33). Non-relapse mortality at 96months was 56% with an overall survival of 34%, which improved with more recent transplants. Median follow-up was 58months. Patients transplanted after 2000 had improved survival (84% at 36months), using BM from an identical sibling and fludarabine in the conditioning regimen. Factors associated with improved outcome in multivariate analysis were use of fludarabine and an identical sibling or matched non-sibling donor. Main causes of death were infection (37%), GvHD (24%) and organ failure (12%). The presence of clonal disease at transplant did not significant impact on survival. Secondary malignancies were reported in 15 of 131 evaluable patients.

Published
2018

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