Hematopoietic stem cell transplantation is effective in achieving long-term survival for post-aplastic anemia myeloid neoplasms - the EBMT Severe Aplastic Anemia Report

Sep 29, 2026

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Aplastic anemia transformation into myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) carries a poor prognosis, and hematopoietic stem cell transplant is the only curative option, though long-term survival data are limited. This retrospective EBMT analysis included 270 patients transplanted for MDS, AML, or isolated cytogenetic abnormalities after AA or paroxysmal nocturnal hemoglobinuria. Five-year overall survival was 64%, with non-relapse mortality varying by disease and higher rates after myeloablative conditioning; relapse rates were 12% for MDS and 22% for AML.

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ARTICLE-Bone Marrow Failure Hematopoietic stem cell transplantation is effective in achieving long-term survival for post-aplastic anemia myeloid neoplasms: the EBMT Severe Aplastic Anemia Report Pedro Henrique Prata,1 Dirk-Jan Eikema,2 Brian Piepenbroek,2 Austin Kulasekararaj,3 Beatrice Drexler,4 Jennifer M-L. Tjon,5 Krista Vaht,6 Mahmoud Aljurf,7 Patrice Chevallier,8 Tobias GeddeDahl,9 Ain Kaare,10 Urpu Salmenniemi,11 Thomas Schroeder,12 Jenny Byrne,13 Jérôme Cornillon,14 Fiorenza Barraco,15 Johan Maertens,16 Eleni Tholouli,17 Uwe Platzbecker,18 Nicolaus Kröger,19 Didier Blaise,20 Simona Sica,21 Robert Zeiser,22 Antonio Maria Risitano23 and Régis Peffault de Latour24 1CHRU de Limoges, Limoges, France; 2 EBMT Leiden Study Unit, Leiden, the Netherlands; 3 Kings College Hospital, London, UK; 4 University Hospital, Basel, Switzerland; 5 Leiden University Hospital, Leiden, the Netherlands; 6Sahlgrenska University Hospital, Goetenborg, Sweden; 7 King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia; 8CHU de Nantes, Nantes, France; 9Oslo University Hospital, Rikshospitalet, Oslo, Norway; 10Tartu University Hospital, Tartu, Estonia; 11 HUCH Comprehensive Cancer Center, Helsinki, Finland; 12 University Hospital, Essen, Germany; 13 Nottingham City Hospital, Nottingham, UK; 14CHU de Saint-Etienne, Saint-Etienne, France; 15Centre Hospitalier Lyon Sud, Lyon, France; 16 University Hospital Gasthuisberg, Leuven, Belgium; 17 Manchester Royal Infirmary, Manchester, UK; 18 Medical Clinic and Policinic 1, Leipzig, Germany; 19 University Hospital Eppendorf, Hamburg, Germany; 20 Institut Paoli-Calmettes, Marseille, France; 21 Università Cattolica S. Cuore, Rome, Italy; 22 University of Freiburg, Freiburg, Germany; 23A.O. R. N.‘San Giuseppe Moscati’, Avellino, Italy and 24Saint-Louis Hospital, Paris, France Abstract Correspondence: P. H. Prata [pedro.prata@chu-limoges.fr](pedro.prata@chu-limoges.fr) Received: December 18, 2024. Accepted: June 25, 2025. Early view: July 3, 2025. [https://doi.org/10.3324/haematol.2024.287205](https://doi.org/10.3324/haematol.2024.287205) ©2026 Ferrata Storti Foundation Published under a CC BY-NC license Aplastic anemia (AA) transformation into myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) is associated with a dismal prognosis. Hematopoietic stem cell transplant offers the sole possibility of cure, but data on long-term survival are scarce. We retrospectively analyzed 270 patients transplanted for MDS, AML, or an isolated cytogenetic abnormality after a diagnosis of AA or paroxysmal nocturnal hemoglobinuria reported to the European Society for Blood and Marrow Transplantation (EBMT) . The median age at transplantation was 39 years. The 5-year overall survival rate was 64%, and was unaffected by chromosome 7 abnormalities, age at transplant, sex, interval from clonal evolution to transplant, and intensity of conditioning regimen. The 5-year non-relapse mortality rates were 34%(95% Confidence Interval [CI]: 25-42%) for MDS patients and 19%(95% CI: 7-31%) for AML patients, and were higher following a myeloablative conditioning regimen. The 5-year relapse rate was 12%(95% CI: 6-19%) for MDS and 22%(95% CI: 9-35%) for AML. Our study’s survival estimates reflect a younger cohort of patients, considering the bimodal distribution of AA. Conditioning regimen intensity did not affect relapse. For MDS patients, pretreating before transplant did not improve survival nor reduce relapse. Transplantation is feasible and effective in achieving long-term survival for transplant-eligible post-AA myeloid neoplasm patients. MDS patients may benefit from upfront reduced intensity conditioning transplant, limiting toxicity without higher rates of relapse. Post-transplant maintenance therapies to reduce the relapse incidence among AML patients might be warranted. Introduction Clonal evolution into myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) repres

FAQs

What is the main clinical problem addressed by the study?

The study focuses on post-aplastic anemia myeloid neoplasms, where aplastic anemia can transform into MDS or AML, a complication linked to poor prognosis.

What did the study find about long-term overall survival after transplant?

In 270 transplanted patients, the 5-year overall survival rate was 64% and was unaffected by factors including chromosome 7 abnormalities, transplant age, sex, interval, and conditioning regimen intensity.

How did relapse and non-relapse mortality differ between MDS and AML patients?

Five-year relapse rates were 12% for MDS and 22% for AML. Five-year non-relapse mortality was 34% for MDS and 19% for AML, with higher non-relapse mortality after myeloablative conditioning.

Sep 29, 2026

Uploaded by Rowan