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)

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) Publisher Pubmed

Miano M¹ ; Eikema DJ² ; De La Fuente J³ ; Bosman P² ; Ghavamzadeh A⁴ ; Smiers F⁵ ; Sengelov H⁶ ; Yesilipek A⁷ ; Formankova R⁸ ; Bader P⁹ ; Diaz Perez MA¹⁰ ; Bertrand Y¹¹ ; Niemeyer C¹² ; Diallo S¹³ Show All Authors

Miano M¹
Eikema DJ²
De La Fuente J³
Bosman P²
Ghavamzadeh A⁴
Smiers F⁵
Sengelov H⁶
Yesilipek A⁷
Formankova R⁸
Bader P⁹
Diaz Perez MA¹⁰
Bertrand Y¹¹
Niemeyer C¹²
Diallo S¹³
Ansari M¹⁴
Bykova TA¹⁵
Faraci M¹⁶
Bonanomi S¹⁷
Gozdzik J¹⁸
Satti TM¹⁹
Bodova I²⁰
Wolfl M²¹
Rocha VG²²
Mellgren K²³
Rascon J²⁴
Holter W²⁵
Lange A²⁶
Meisel R²⁷
Beguin Y²⁸
Mozo Y²⁹
Krivan G³⁰
Sirvent A³¹
Bruno B³²
Dalle JH³³
Onofrillo D³⁴
Giardino S¹⁶
Risitano AM³⁵
De Latour RP³⁶
Dufour C¹

Show Affiliations

1. Haematology Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy
2. EBMT Statistics, EBMT Data Office, Leiden, Netherlands
3. Centre for Haematology, Imperial College London, London, United Kingdom
4. Hematology-Oncology and Stem Cell Transplantation Research Center, Shariati Hospital, Tehran, Iran
5. Department of Pediatrics, Leiden University Medical Center, Leiden, Netherlands
6. Rigshospitalet Copenhagen, Copenhagen, Denmark
7. Medical Park Antalya Hospital, Antalya, Turkey
8. University Hospital Motol, Prague, Czech Republic
9. Division for Stem Cell Transplantation and Immunology, Department for Children and Adolescents, University Hospital Frankfurt, Frankfurt, Germany
10. Department of Pediatrics, Hematology/Oncology and Hematopoietic Stem Cell Transplant Unit, Hospital Infantil Universitario Nino Jesus, Madrid, Spain
11. Institute of Pediatric Hematology and Oncology, Civil Hospital of Lyon, Lyon, France
12. Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany
13. Department of Hematology, Jules Bordet Institute, Brussels, Belgium
14. Pediatric Oncology and Hematology, Department of Paediatrics, Gynaecology, and Obstetrics, Geneva University Hospital, Switzerland, Geneva, Switzerland
15. Raisa Gorbacheva Memorial Scientific Institute of Children Oncology, Hematology and Transplantation, First Pavlov State Medical University of St. Petersburg, St. Petersburg, Russian Federation
16. BMT Unit, Istituto Giannina Gaslini, Genova, Italy
17. MBBM Foundation, University of Milano-Bicocca, San Gerardo Hospital, Monza, Italy
18. Jagiellonian University, Medical Collage, Krakow, Poland
19. Armed Forces Bone Marrow Transplant Centre, Rawalpindi, Pakistan
20. National Institute of Children's Diseases, Bratislava, Slovakia
21. Pediatric Blood and Marrow Transplantation Program, Children's Hospital, University Hospital of Wurzburg, Wurzburg, Germany
22. Churchill Hospital, Oxford, United Kingdom
23. Sahlgrenska University Hospital, Gothenburg, Sweden
24. Center for Pediatric Oncology and Hematology, Vilnius University, Vilnius, Lithuania
25. St. Anna Kinderspital, Vienna, Austria
26. Lower Silesian Center for Cellular Transplantation, Ludwik Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of Sciences, Wroclaw, Poland
27. Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology and Clinical Immunology, Heinrich-Heine-University, Dusseldorf, Germany
28. CHU de Liege, University of Liege, Liege, Belgium
29. Hospital Universitario La Paz, Madrid, Spain
30. Department of Paediatric Haematology and Stem Cell Transplantation, Central Hospital of Southern Pest, National Institute of Hematology and Infectious Diseases, Budapest, Hungary
31. Onco-Hematologie Pediatrique, CHU de Montpellier, Montpellier, France
32. CHU Lille, Hematogie pediatrique, Lille, France
33. Hematology and Immunology Department, Hopital Robert-Debre, universite de Paris, Paris, France
34. Haematology Unit, Ospedale Civile, Pescara, Italy
35. Department of Clinical Medicine and Surgery, University of Naples, Naples, Italy
36. Bone Marrow Transplantation Unit, Saint Louis Hospital, APHP, Paris, France

Source: Transplantation and Cellular Therapy Published:2021

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. © 2021

2. Outcome of Allogeneic Hematopoietic Stem Cell Transplantation on Diamond-Blackfan Anemia Using Busulfan-Based Myeloablative Regimen, Turkish Journal of Pediatrics (2019)

Experts (# of related papers)

Amir Ali Hamidieh (2)

Maryam Behfar (1)

Style	Citing Format
MLA	Miano M, et al.. "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, vol. 27, no. 3, 2021, pp. 274.e1-274.e5.
APA	Miano M, Eikema DJ, De La Fuente J, Bosman P, Ghavamzadeh A, Smiers F, Sengelov H, Yesilipek A, Formankova R, Bader P, Diaz Perez MA, Bertrand Y, Niemeyer C, Diallo S, Ansari M, Bykova TA, 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.
Chicago	Miano M, Eikema DJ, De La Fuente J, Bosman P, Ghavamzadeh A, Smiers F, Sengelov H, et al.. "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, no. 3 (2021): 274.e1-274.e5.
Harvard	Miano M et al. (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), pp. 274.e1-274.e5.
Vancouver	Miano M, Eikema DJ, De La Fuente J, Bosman P, Ghavamzadeh A, Smiers F, et al.. 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;27(3):274.e1-274.e5.
BibTex	@article{ author = {Miano M and Eikema DJ and De La Fuente J and Bosman P and Ghavamzadeh A and Smiers F and Sengelov H and Yesilipek A and Formankova R and Bader P and Diaz Perez MA and Bertrand Y and Niemeyer C and Diallo S and Ansari M and Bykova TA and Faraci M and Bonanomi S and Gozdzik J and Satti TM and Bodova I and Wolfl M and Rocha VG and Mellgren K and Rascon J and Holter W and Lange A and Meisel R and Beguin Y and Mozo Y and Krivan G and Sirvent A and Bruno B and Dalle JH and Onofrillo D and Giardino S and Risitano AM and De Latour RP and Dufour C}, 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)}, journal = {Transplantation and Cellular Therapy}, volume = {27}, number = {3}, pages = {274.e1-274.e5}, year = {2021} }
RIS	TY - JOUR AU - Miano M AU - Eikema DJ AU - De La Fuente J AU - Bosman P AU - Ghavamzadeh A AU - Smiers F AU - Sengelov H AU - Yesilipek A AU - Formankova R AU - Bader P AU - Diaz Perez MA AU - Bertrand Y AU - Niemeyer C AU - Diallo S AU - Ansari M AU - Bykova TA AU - Faraci M AU - Bonanomi S AU - Gozdzik J AU - Satti TM AU - Bodova I AU - Wolfl M AU - Rocha VG AU - Mellgren K AU - Rascon J AU - Holter W AU - Lange A AU - Meisel R AU - Beguin Y AU - Mozo Y AU - Krivan G AU - Sirvent A AU - Bruno B AU - Dalle JH AU - Onofrillo D AU - Giardino S AU - Risitano AM AU - De Latour RP AU - Dufour C TI - 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) JO - Transplantation and Cellular Therapy VL - 27 IS - 3 SP - 274.e1 EP - 274.e5 PY - 2021 ER -

Science Communicator Platform

Authors

Authors Affiliations

Abstract