Cyclosporine plus avatrombopag versus cyclosporine monotherapy for first-line treatment of elderly patients with transfusion-dependent non-severe aplastic anemia: a single center retrospective study.

Background: Elderly patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA) face limited treatment options owing to transplantation incompatibility and safety concerns.

Methods: Data from older patients (≥60 years) newly diagnosed with TD-NSAA and had been treated with either avatrombopag (AVA) + cyclosporine (CsA) or CsA monotherapy at our center were retrospectively collected.

Results: In total, 52 patients were enrolled, with 26 receiving AVA + CsA and 26 receiving CsA monotherapy. The overall response (OR) rate was higher in the AVA + CsA group than in the CsA group at the 1, 2, 3, 6 months, and at the end of the follow-up (all < 0.05). The complete response (CR) rate was higher in the AVA + CsA group than in the CsA group at the 6 month and at the end of follow-up ( = 0.017 and 0.039). Patients receiving AVA + CsA had a shorter time to achieve the first response ( = 0.008) and CR ( = 0.010) than those receiving CsA monotherapy. Furthermore, patients receiving AVA + CsA exhibited a higher rate of platelet transfusion independence at the 3 ( = 0.041), 6 ( = 0.008) months, and likewise at the end of the follow-up ( = 0.097).

Conclusions: The addition of AVA to CsA can significantly improve the OR or CR rate and platelet levels with acceptable side effects in elderly patients, which implies that the combination is a viable alternative for elderly patients with TD-NSAA.