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Multicenter Study
. 2025 Apr;100(4):584-591.
doi: 10.1002/ajh.27611. Epub 2025 Jan 29.

Elderly Patients With Aplastic Anemia: Treatment Patterns and Outcomes in the Real World

Affiliations
Multicenter Study

Elderly Patients With Aplastic Anemia: Treatment Patterns and Outcomes in the Real World

Bruno Fattizzo et al. Am J Hematol. 2025 Apr.

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.

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Conflict of interest statement

The authors declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
Cumulative incidence of evolution into myeloid neoplasm and paroxysmal nocturnal hemoglobinuria. Survival of aplastic anemia patients according to age category. Upper panel shows cumulative data, and lower panel shows data stratified according to age at diagnosis.
FIGURE 2
FIGURE 2
Overall survival of aplastic anemia patients according to age category. Upper panel shows cumulative data over a 15 year follow up; lower panel shows the analysis restricted to those patients treated after 2014.
FIGURE 3
FIGURE 3
Overall survival of aplastic anemia patients according to Camitta criteria. Upper panel shows cumulative data, and lower panel shows data stratified according to age at diagnosis.
FIGURE 4
FIGURE 4
A proposed algorithm for the management of non‐severe, severe/very severe aplastic anemia (NSAA, SAA/VSAA) elderly patients. ATG, anti‐thymocyte globulin; BSC, best supportive care; CYA, cyclosporine A; EPAG, eltrombopag; NR, no response; R, response. [Color figure can be viewed at wileyonlinelibrary.com]

References

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