Elderly Patients With Aplastic Anemia: Treatment Patterns and Outcomes in the Real World
Fattizzo B, Gurnari C, Giammarco S, Ricchiuti A, Awada H, Bortolotti M, Galli N, Pedone GL, Versino F, Consonni D, Trikha R, Gandhi S, Sica S, Maciejewski JP, Kulasekararaj A, Barcellini W
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.
© 2025 The Author(s). American Journal of Hematology published by Wiley Periodicals LLC.
American journal of hematology, 2025-04-03