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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

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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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Data availability

Primary data is available with the first and corresponding author on appropriate request.

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Authors and Affiliations

  1. Department of Haematology, Christian Medical College, Vellore, India

    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

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  1. Sohini Chattopadhyay
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Contributions

SC and BG designed the study, performed the research, analyzed the data, and wrote the paper; KM.L performed the statistical analysis, analyzed the data, and edited the paper; SL, SS, AJ.D, AK, UK, FN.A, AS, VM, and AA recruited and treated the patients, provided critical advice, and reviewed the paper. All authors have read and approved the final manuscript.

Corresponding author

Correspondence to Biju George.

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Ethical approval

All procedures followed were in accordance with the ethical standards of the responsible committee on human experimentation (institutional and national) and with the Helsinki Declaration of 1975, as revised in 2008. This study was approved by the Institutional Review Board of Christian Medical College, Vellore, India.

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Since it is a retrospective study, consent requirement was waived for retrospective participants by the above-named ethics committee.

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The authors declare no competing interests.

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Chattopadhyay, S., Lionel, S., Selvarajan, S. et al. 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. Ann Hematol 103, 749–758 (2024). https://doi.org/10.1007/s00277-024-05621-2

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