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

Harvard

Miano, M, Eikema, DJ, de la Fuente, J, Bosman, P, Ghavamzadeh, A, Smiers, F, Sengeløv, H, Yesilipek, A, Formankova, R, Bader, P, Díaz Pérez, MÁ, Bertrand, Y, Niemeyer, C, Diallo, S, Ansari, M, Bykova, TA, Faraci, M, Bonanomi, S, Gozdzik, J, Satti, TM, Bodova, I, Wölfl, M, Rocha, VG, Mellgren, K, Rascon, J, Holter, W, Lange, A, Meisel, R, Beguin, Y, Mozo, Y, Kriván, G, Sirvent, A, Bruno, B, Dalle, JH, Onofrillo, D, Giardino, S, Risitano, AM, de Latour, RP & Dufour, C 2021, '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)', Transplantation and Cellular Therapy, bind 27, nr. 3, s. 274.e1-274.e5. https://doi.org/10.1016/j.jtct.2020.12.024

APA

Miano, M., Eikema, D. J., de la Fuente, J., Bosman, P., Ghavamzadeh, A., Smiers, F., Sengeløv, H., Yesilipek, A., Formankova, R., Bader, P., Díaz Pérez, M. Á., Bertrand, Y., Niemeyer, C., Diallo, S., Ansari, M., Bykova, T. A., Faraci, M., Bonanomi, S., Gozdzik, J., ... Dufour, C. (2021). 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). Transplantation and Cellular Therapy, 27(3), 274.e1-274.e5. https://doi.org/10.1016/j.jtct.2020.12.024

Vancouver

Miano M, Eikema DJ, de la Fuente J, Bosman P, Ghavamzadeh A, Smiers F o.a. 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). Transplantation and Cellular Therapy. 2021 mar.;27(3):274.e1-274.e5. https://doi.org/10.1016/j.jtct.2020.12.024

Author

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. / 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). I: Transplantation and Cellular Therapy. 2021 ; Bind 27, Nr. 3. s. 274.e1-274.e5.

Bibtex

@article{117eaf07034541ae89d2976a6e6dee62,

title = "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)",

abstract = "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.",

keywords = "Bone Marrow Failure, Congenital disorder, Diamond-Blackfan Anemia, Stem Cell Transplantation",

author = "Maurizio Miano and Eikema, {Dirk Jan} and {de la Fuente}, Josu and Paul Bosman and Ardeshir Ghavamzadeh and Frans Smiers and Henrik Sengel{\o}v and Akif Yesilipek and Renata Formankova and Peter Bader and {D{\'i}az P{\'e}rez}, {Miguel {\'A}ngel} and Yves Bertrand and Charlotte Niemeyer and Safiatou Diallo and Marc Ansari and Bykova, {Tatiana A.} and Maura Faraci and Sonia Bonanomi and Jolanta Gozdzik and Satti, {Tariq Mahmood} and Ivana Bodova and Matthias W{\"o}lfl and Rocha, {Vanderson G.} and Karin Mellgren and Jelena Rascon and Wolfgang Holter and Andrzej Lange and Roland Meisel and Yves Beguin and Yasmina Mozo and Gergely Kriv{\'a}n and Anne Sirvent and Benedicte Bruno and Dalle, {Jean Hugues} and Daniela Onofrillo and Stefano Giardino and Risitano, {Antonio M.} and {de Latour}, {R{\'e}gis Peffault} and Carlo Dufour",

note = "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: {\textcopyright} 2021",

year = "2021",

month = mar,

doi = "10.1016/j.jtct.2020.12.024",

language = "English",

volume = "27",

pages = "274.e1--274.e5",

journal = "Transplantation and Cellular Therapy",

issn = "2666-6375",

publisher = "Elsevier",

number = "3",

}

RIS

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