Elderly Patients With Aplastic Anemia: Treatment Patterns and Outcomes in the Real World
- PMID: 39878088
- PMCID: PMC11886494
- DOI: 10.1002/ajh.27611
Elderly Patients With Aplastic Anemia: Treatment Patterns and Outcomes in the Real World
Abstract
We retrospectively analyzed a large international cohort of 1113 patients with aplastic anemia to evaluate treatment choice and outcome in elderly patients as compared with a younger population. Overall, 319 (29%) patients were > 60 years old at diagnosis (60-64 years (n = 85), 106 65-69 years (n = 106), and 128 > 70 years (n = 128)). Elderly patients showed a more severe thrombocytopenia at onset and a significantly lower overall response (complete plus partial) to first-line therapy at 6 months as compared to younger patients (47% vs. 65%, p < 0.0001), irrespective of treatment modality (ATG or CyA combinations, eltrombopag, or androgens); 27 (8%) received transplant as second line therapy and 11 (41%) died, mainly due to transplant complications. The rate of evolution to MDS was greater in elderly patients (12% vs. 7% in younger AA, p = 0.002), whilst the rate of evolution to AML was similar (1.8 vs. 1.3%). By multivariable analysis, older age remained the main factor associated with mortality [HR 1.64 (95% CI 1.5-1.7), p < 0.001], followed by disease severity by Camitta classification [HR 2.24 (95% CI 1.6-3.1) for severe AA; HR 3.8 (95% CI 2.4-6) for very severe AA], and male gender [1.45 (95% CI 1.1-1.8), p < 0.001]. In this large study, elderly AA was associated with inferior outcome even in the TPO-RA era, highlighting the need for further optimization of clinical management.
Keywords: anti‐thymocyte globulin; aplastic anemia; cyclosporine; elderly; eltrombopag.
© 2025 The Author(s). American Journal of Hematology published by Wiley Periodicals LLC.
Conflict of interest statement
The authors declare no conflicts of interest.
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References
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- Scheinberg P., Finelli C., Montaňo‐Figueroa E. H., et al., “Activity and Safety of Eltrombopag in Combination With Cyclosporin A as First‐Line Treatment of Adults With Severe Aplastic Anaemia (SOAR): A Phase 2, Single‐Arm Study,” Lancet Haematology 11, no. 3 (March 2024): e206–e215, 10.1016/S2352-3026(23)00395-2. - DOI - PubMed
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