Efficacy and safety of avatrombopag for thrombocytopenia following allogeneic hematopoietic stem cell transplantation: A real-world data evaluation on 14 cases.

OBJECTIVES: Thrombocytopenia following allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a common and serious complication that leads to an increased risk of bleeding and poor prognosis. Traditional strategies consist of platelet transfusion, glucocorticoid therapy, intravenous human immunoglobulin, recombinant human thrombopoietin injection, and CD34+ -selected hematopoietic stem cell transplantation, but the effects of these treatments are not satisfactory and the treatment continues to be challenged. This study aims to determine the treating efficacy of avatrombopag, a novel thrombopoietin receptor agonist, on thrombocytopenia after allo-HSCT, and to increase the evidence-based medical evidence for the clinical use of this drug.

METHODS: Fourteen patients with thrombocytopenia after allo-HSCT underwent avatrom-bopag treatment from September 2020 to September 2021 were retrospectively studied. Of these patients, 8 patients had delayed platelet engraftment (DPE) and 6 cases had secondary failure of platelet recovery (SFPR). The efficacy and safety of the treatment and the survival of the patients were assessed.

RESULTS: The median treatment time of avatrombopag was 34 days, and no patients stopped treatment due to adverse reactions or drug intolerance. Compared with the treatment before, the levels of platelet count, megakaryocytes, and hemoglobin in patients were significantly increased ( P =0.000 1, P =0.001 0, and P =0.001 7, respectively). The optimal platelet count of 13 patients reached the complete response standard after drug withdrawal. The median follow-up time of 14 patients was 371 days, and the 2-year overall survival rate was 78.6%.

CONCLUSIONS: Avatrombopag is effective on increasing platelet counts in patients with thrombocytopenia after allo-HSCT, with a good safety profile. It is a suitable therapeutic option for thrombocytopenia after allo-HSCT.