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)

被引：21

作者：

Miano, Maurizio ^{[1
]}

Eikema, Dirk-Jan ^{[2
]}

de la Fuente, Josu ^{[3
]}

Bosman, Paul ^{[2
]}

Ghavamzadeh, Ardeshir ^{[4
]}

Smiers, Frans ^{[5
]}

Sengelov, Henrik ^{[6
]}

Yesilipek, Akif ^{[7
]}

Formankova, Renata ^{[8
]}

Bader, Peter ^{[9
]}

Diaz Perez, Miguel Angel ^{[10
]}

Bertrand, Yves ^{[11
]}

Niemeyer, Charlotte ^{[12
]}

Diallo, Safiatou ^{[13
]}

Ansari, Marc ^{[14
]}

Bykova, Tatiana A. ^{[15
]}

Faraci, Maura ^{[16
]}

Bonanomi, Sonia ^{[17
]}

Gozdzik, Jolanta ^{[18
]}

Satti, Tariq Mahmood ^{[19
]}

Bodova, Ivana ^{[20
]}

Woelfl, Matthias ^{[21
]}

Rocha, Vanderson G. ^{[22
]}

Mellgren, Karin ^{[23
]}

Rascon, Jelena ^{[24
]}

Holter, Wolfgang ^{[25
]}

Lange, Andrzej ^{[26
]}

Meisel, Roland ^{[27
]}

Beguin, Yves ^{[28
]}

Mozo, Yasmina ^{[29
]}

Krivan, Gergely ^{[30
]}

Sirvent, Anne ^{[31
]}

Bruno, Benedicte ^{[32
]}

Dalle, Jean Hugues ^{[33
]}

Onofrillo, Daniela ^{[34
]}

Giardino, Stefano ^{[16
]}

Risitano, Antonio M. ^{[35
]}

de Latour, Regis Peffault ^{[36
]}

Dufour, Carlo ^{[1
]}

机构：

[1] IRCCS Ist Giannina Gaslini, Haematol Unit, Largo G Gaslini 5, I-16148 Genoa, Italy

[2] EBMT Data Off, EBMT Stat, Leiden, Netherlands

[3] Imperial Coll London, Ctr Haematol, London, England

[4] Shariati Hosp, Hematol Oncol & Stem Cell Transplantat Res Ctr, Tehran, Iran

[5] Leiden Univ, Dept Pediat, Med Ctr, Leiden, Netherlands

[6] Rigshosp Copenhagen, Copenhagen, Denmark

[7] Med Pk Antalya Hosp, Antalya, Turkey

[8] Univ Hosp Motol, Prague, Czech Republic

[9] Univ Hosp Frankfurt, Dept Children & Adolescents, Div Stem Cell Transplantat & Immunol, Frankfurt, Germany

[10] Hosp Infantil Univ Nino Jesus, Dept Pediat, Hematol Oncol & Hematopoiet Stem Cell Transplant, Madrid, Spain

[11] Civil Hosp Lyon, Inst Pediat Hematol & Oncol, Lyon, France

[12] Univ Freiburg, Fac Med, Med Ctr, Dept Pediat & Adolescent Med,Div Pediat Hematol &, Freiburg, Germany

[13] Jules Bordet Inst, Dept Hematol, Brussels, Belgium

[14] Geneva Univ Hosp, Dept Paediat Gynaecol & Obstet, Pediat Oncol & Hematol, Geneva, Switzerland

[15] First Pavlov State Med Univ St Petersburg, Raisa Gorbacheva Mem Sci Inst Children Oncol Hema, St Petersburg, Russia

[16] Ist Giannina Gaslini, BMT Unit, Genoa, Italy

[17] Univ Milano Bicocca, San Gerardo Hosp, MBBM Fdn, Monza, Italy

[18] Jagiellonian Univ, Med Collage, Krakow, Poland

[19] Armed Forces Bone Marrow Transplant Ctr, Rawalpindi, Pakistan

[20] Natl Inst Childrens Dis, Bratislava, Slovakia

[21] Univ Hosp Wurzburg, Childrens Hosp, Pediat Blood & Marrow Transplantat Program, Wurzburg, Germany

[22] Churchill Hosp, Oxford, England

[23] Sahlgrens Univ Hosp, Gothenburg, Sweden

[24] Vilnius Univ, Ctr Pediat Oncol & Hematol, Vilnius, Lithuania

[25] St Anna Childrens Hosp, Vienna, Austria

[26] Polish Acad Sci, Lower Silesian Ctr Cellular Transplantat, Ludwik Hirszfeld Inst Immunol & Expt Therapy, Wroclaw, Poland

[27] Heinrich Heine Univ, Dept Pediat Oncol Hematol & Clin Immunol, Div Pediat Stem Cell Therapy, Dusseldorf, Germany

[28] Univ Liege, CHU Liege, Liege, Belgium

[29] Hosp Univ La Paz, Madrid, Spain

[30] Cent Hosp Southern Pest, Natl Inst Hematol & Infect Dis, Dept Paediat Haematol & Stem Cell Transplantat, Budapest, Hungary

[31] CHU Montpellier, Oncohematol Pediat, Montpellier, France

[32] CHU Lille, Hemat Pediat, Lille, France

[33] Univ Paris, Hematol & Immunol Dept, Hop Robert Debre, Paris, France

[34] Osped Civile, Haematol Unit, Pescara, Italy

[35] Univ Naples Federico II, Dept Clin Med & Surg, Naples, Italy

[36] St Louis Hosp, AP HP, Bone Marrow Transplantat Unit, Paris, France

来源：

TRANSPLANTATION AND CELLULAR THERAPY | 2021年 / 27卷 / 03期

关键词：

Diamond-Blackfan Anemia; Bone Marrow Failure; Stem Cell Transplantation; Congenital disorder; PEDIATRIC HEMATOLOGY; ITALIAN ASSOCIATION; RECOMMENDATIONS; SOCIETY;

D O I：

10.1016/j.jtct.2020.12.024

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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. (C) 2021 Published by Elsevier Inc. on behalf of The American Society for Transplantation and Cellular Therapy.

引用

页码：274.e1 / 274.e5

页数：5

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