Stem Cell Transplantation for Diamond–Blackfan Anemia. A Retrospective Study on Behalf of the Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)
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Stem Cell Transplantation for Diamond–Blackfan Anemia. A Retrospective Study on Behalf of the Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT). / Miano, Maurizio; Eikema, Dirk Jan; de la Fuente, Josu; Bosman, Paul; Ghavamzadeh, Ardeshir; Smiers, Frans; Sengeløv, Henrik; Yesilipek, Akif; Formankova, Renata; Bader, Peter; Díaz Pérez, Miguel Ángel; Bertrand, Yves; Niemeyer, Charlotte; Diallo, Safiatou; Ansari, Marc; Bykova, Tatiana A.; Faraci, Maura; Bonanomi, Sonia; Gozdzik, Jolanta; Satti, Tariq Mahmood; Bodova, Ivana; Wölfl, Matthias; Rocha, Vanderson G.; Mellgren, Karin; Rascon, Jelena; Holter, Wolfgang; Lange, Andrzej; Meisel, Roland; Beguin, Yves; Mozo, Yasmina; Kriván, Gergely; Sirvent, Anne; Bruno, Benedicte; Dalle, Jean Hugues; Onofrillo, Daniela; Giardino, Stefano; Risitano, Antonio M.; de Latour, Régis Peffault; Dufour, Carlo.
I: Transplantation and Cellular Therapy, Bind 27, Nr. 3, 03.2021, s. 274.e1-274.e5.Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
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TY - JOUR
T1 - Stem Cell Transplantation for Diamond–Blackfan Anemia. A Retrospective Study on Behalf of the Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT)
AU - Miano, Maurizio
AU - Eikema, Dirk Jan
AU - de la Fuente, Josu
AU - Bosman, Paul
AU - Ghavamzadeh, Ardeshir
AU - Smiers, Frans
AU - Sengeløv, Henrik
AU - Yesilipek, Akif
AU - Formankova, Renata
AU - Bader, Peter
AU - Díaz Pérez, Miguel Ángel
AU - Bertrand, Yves
AU - Niemeyer, Charlotte
AU - Diallo, Safiatou
AU - Ansari, Marc
AU - Bykova, Tatiana A.
AU - Faraci, Maura
AU - Bonanomi, Sonia
AU - Gozdzik, Jolanta
AU - Satti, Tariq Mahmood
AU - Bodova, Ivana
AU - Wölfl, Matthias
AU - Rocha, Vanderson G.
AU - Mellgren, Karin
AU - Rascon, Jelena
AU - Holter, Wolfgang
AU - Lange, Andrzej
AU - Meisel, Roland
AU - Beguin, Yves
AU - Mozo, Yasmina
AU - Kriván, Gergely
AU - Sirvent, Anne
AU - Bruno, Benedicte
AU - Dalle, Jean Hugues
AU - Onofrillo, Daniela
AU - Giardino, Stefano
AU - Risitano, Antonio M.
AU - de Latour, Régis Peffault
AU - Dufour, Carlo
N1 - Funding Information: The authors acknowledge ERG s.p.a. Rimorchiatori Riuniti-Genova, Cambiaso & Risso Marine-Genova, Saar Depositi Oleari Portuali-Genova, and Nicola Ferrari ONLUS for supporting the Haematology Unit of IRCCS Istituto Giannina Gaslini. Financial disclosure: The authors have nothing to disclose. Conflict of interest statement: There are no conflicts of interest to report. Authorship statement: M.M. designed the study, analyzed the data, and wrote the paper. D.J.E. and P.B. performed the statistical analyses. J.D.L.F. F.S. R.F. P.B. M.A.D.P. C.N. S.D. M.A. M.F. S.B. J.G. I.B. M.W. J.R. A.L. R.M. Y.B. Y.M. G.K. A.S. and B.B. contributed essential data and revised the manuscript. A.G. H.S. A.Y. Y.B. T.A.B. T.M.S. V.R. K.M. W.H. J.H.D. S.G. and D.O. contributed to analysis and interpretation of the data. C.D. P.DL. and A.M. coordinated the research and revised the manuscript. Publisher Copyright: © 2021
PY - 2021/3
Y1 - 2021/3
N2 - Data on stem cell transplantation (SCT) for Diamond–Blackfan Anemia (DBA) is limited. We studied patients transplanted for DBA and registered in the EBMT database. Between 1985 and 2016, 106 DBA patients (median age, 6.8 years) underwent hematopoietic stem cell transplantation from matched-sibling donors (57%), unrelated donors (36%), or other related donors (7%), using marrow (68%), peripheral blood stem cells (20%), both marrow and peripheral blood stem cells (1%), or cord blood (11%). The cumulative incidence of engraftment was 86% (80% to 93%), and neutrophil recovery and platelet recovery were achieved on day +18 (range, 16 to 20) and +36 (range, 32 to 43), respectively. Three-year overall survival and event-free survival were 84% (77% to 91%) and 81% (74% to 89%), respectively. Older patients were significantly more likely to die (hazard ratio, 1.4; 95% confidence interval, 1.06 to 1.23; P <.001). Outcomes were similar between sibling compared to unrelated-donor transplants. The incidence of acute grades II to IV of graft-versus-host disease (GVHD) was 30% (21% to 39%), and the incidence of extensive chronic GVHD was 15% (7% to 22%). This study shows that SCT may represent an alternative therapeutic option for transfusion-dependent younger patients.
AB - Data on stem cell transplantation (SCT) for Diamond–Blackfan Anemia (DBA) is limited. We studied patients transplanted for DBA and registered in the EBMT database. Between 1985 and 2016, 106 DBA patients (median age, 6.8 years) underwent hematopoietic stem cell transplantation from matched-sibling donors (57%), unrelated donors (36%), or other related donors (7%), using marrow (68%), peripheral blood stem cells (20%), both marrow and peripheral blood stem cells (1%), or cord blood (11%). The cumulative incidence of engraftment was 86% (80% to 93%), and neutrophil recovery and platelet recovery were achieved on day +18 (range, 16 to 20) and +36 (range, 32 to 43), respectively. Three-year overall survival and event-free survival were 84% (77% to 91%) and 81% (74% to 89%), respectively. Older patients were significantly more likely to die (hazard ratio, 1.4; 95% confidence interval, 1.06 to 1.23; P <.001). Outcomes were similar between sibling compared to unrelated-donor transplants. The incidence of acute grades II to IV of graft-versus-host disease (GVHD) was 30% (21% to 39%), and the incidence of extensive chronic GVHD was 15% (7% to 22%). This study shows that SCT may represent an alternative therapeutic option for transfusion-dependent younger patients.
KW - Bone Marrow Failure
KW - Congenital disorder
KW - Diamond-Blackfan Anemia
KW - Stem Cell Transplantation
U2 - 10.1016/j.jtct.2020.12.024
DO - 10.1016/j.jtct.2020.12.024
M3 - Journal article
C2 - 33781541
AN - SCOPUS:85101079973
VL - 27
SP - 274.e1-274.e5
JO - Transplantation and Cellular Therapy
JF - Transplantation and Cellular Therapy
SN - 2666-6375
IS - 3
ER -
ID: 302914928