Blood Cancer J. 2025 Jul 5;15(1):119. doi: 10.1038/s41408-025-01328-3.
ABSTRACT
This trial compared antithymocyte globulin (ATG) + cyclosporine A (CsA) + avatrombopag (AVA) and CsA + AVA in older adults with severe aplastic anemia (SAA). The patients were randomized to receive either ATG + CsA + AVA or CsA + AVA. Of 84 included patients, 42 were treated with ATG + CsA + AVA and 42 with CsA + AVA. With a median follow-up of 13 (0.3-17) months, the objective response rates (ORRs) at 3, 6, and 12 months and the end of follow-up were 53.7%, 65.9%, 80.6%, and 71.4% in the ATG + CsA + AVA group and 61.9%, 73.2%, 77.4%, and 64.3% in the CsA + AVA group, respectively (P > 0.05 at any time point). Three-month ORR was an independent predictor of 6-month complete response rates (P = 0.019). Patients in the ATG + CsA + AVA group showed a higher incidence of adverse events than those in the CsA + AVA group (64.3% vs. 35.7%, P = 0.009). The rates of relapse (P = 0.667), mortality (P = 1.000) and clonal evolution (P = 1.000) were comparable between the groups. The combination of CsA + AVA achieved comparable efficacy with superior safety compared to the combination of ATG + CsA + AVA in older adults newly diagnosed with SAA.
PMID:40617806 | DOI:10.1038/s41408-025-01328-3
