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Annals of Hematology
Published in: Annals of Hematology 3/2024

20-01-2024 | Paroxysmal Nocturnal Hemoglobinuria | Original Article

Relapse and transformation to myelodysplastic syndrome and acute myeloid leukemia following immunosuppressive therapy for aplastic anemia is more common as compared to allogeneic stem cell transplantation with a negative impact on survival

Authors: Sohini Chattopadhyay, Sharon Lionel, Sushil Selvarajan, Anup J. Devasia, Anu Korula, Uday Kulkarni, Fouzia N. Aboobacker, Kavitha M. Lakshmi, Alok Srivastava, Vikram Mathews, Aby Abraham, Biju George

Published in: Annals of Hematology | Issue 3/2024

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Abstract

We studied the incidence of relapse, transformation to myelodysplastic syndrome/acute myeloid leukemia, and survival in patients with aplastic anemia (AA) surviving more than 1 year after ATG/ALG-based immunosuppressive therapy (IST) between 1985 and 2020. Four-hundred seventy patients (413 adults and 57 children) were studied, and data were compared with 223 patients who underwent matched sibling donor transplant (MSD HSCT). Median follow-up is 50 months (12–359). Relapse occurred in 21.9% at a median time of 33.5 months (5–228) post IST. Twenty-six (5.5%) patients progressed to PNH, while 20 (4.3%) evolved to MDS/AML. Ten-year estimated overall survival (OS) is 80.9 ± 3% and was significantly better in patients without an event (85.1 ± 4%) compared to relapse (74.6% ± 6.2%) or clonal evolution (12.8% ± 11.8%) (p = 0.024). While the severity of AA (p = 0.011) and type of ATG (p = 0.028) used predicted relapse, only age at IST administration influenced clonal evolution (p = 0.018). Among HSCT recipients, relapse rates were 4.9% with no clonal evolution, and the 10-year OS was 94.5 ± 2%. In patients who survived 1 year following IST, outcomes were good except with clonal evolution to MDS/AML. These outcomes, however, were still inferior compared to matched sibling donor HSCT.
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Metadata
Title
Relapse and transformation to myelodysplastic syndrome and acute myeloid leukemia following immunosuppressive therapy for aplastic anemia is more common as compared to allogeneic stem cell transplantation with a negative impact on survival
Authors
Sohini Chattopadhyay
Sharon Lionel
Sushil Selvarajan
Anup J. Devasia
Anu Korula
Uday Kulkarni
Fouzia N. Aboobacker
Kavitha M. Lakshmi
Alok Srivastava
Vikram Mathews
Aby Abraham
Biju George
Publication date
20-01-2024
Publisher
Springer Berlin Heidelberg
Published in
Annals of Hematology / Issue 3/2024
Print ISSN: 0939-5555
Electronic ISSN: 1432-0584
DOI
https://doi.org/10.1007/s00277-024-05621-2

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