Efficacy and Safety of Different TPO-RAs Combined with Cyclosporine in the Treatment of NSAA

TPO-RA has significantly improved the outcomes of patients with aplastic anemia. Currently, three small-molecule TPO-RAs, eltrombopag, hetrombopag, and avatrombopag are available for clinical use and work very well. This article compares the efficacy and safety of these TPO-RAs in the treatment of non-severe aplastic anemia (NSAA).Out of 67 NSAA patients, 31were male and 36 female, with a median age of 50 years (9-81 years). All patients received CsA combined with TPO-RAs (10 patients received avatrombopag, 15 patients received hetrombopag, and 42 patients received eltrombopag). The 3-month efficacy of CsA combined with TPO-RAs: the ORR of the avatrombopag group was 90.00% (CR 20.00%, PR 70.00%), the hetrombopag group was 73.33% (CR 13.33%, PR 60.00%), and the eltrombopag group was 66.67% (CR 4.76%, PR 61.90%); the 6-month efficacy, the ORR of the avatrombopag group was 90.00% (CR 20.00%, PR 70.00%), the hetrombopag group was 80.00% (CR 26.67%, PR 53.33%), and the eltrombopag group was 80.95% (CR 9.52%, PR 71.43%), with no significant difference among the three groups. There was no significant difference in the incidence of new liver damage, infection, and mortality among the three groups during treatment. Age(r=0.258, P=0.038) and infection(r=0.253,P=0.042) were independent factors affecting the length of the first hospitalization. In conclusion, eltrombopag, hetrombopag, and avatrombopag combined with CsA have comparable efficacy in the treatment of NSAA. Avatrombopag has advantages in patients with underlying liver diseases. Bone marrow residual hematopoiesis and adequate TPO-RAs dosage are independent factors for achieving good efficacy at 6 months.

No relevant conflicts of interest to declare.