阿伐曲泊帕联合环孢素治疗输血依赖非重型再生障碍性贫血研究OA北大核心CSTPCD

Objective To evaluate the efficacy and safety of Avatrombopag treatment combined with Cyclosporin A(CsA)therapy in patients with transfusion-dependent non-Severe aplastic anaemia(TD-NSAA).Methods Prospective registration from October 2020 to October 2023,21 patients with TD-NSAA treated with CsA combined with Avatrombopag.Results The median duration of treatment with Avatrombopag was 7.5(1.0-27.0)months,and eighteen patients(85.71％)obtained trilineage hemalogical response:3 cases of complete treatment response(CR)(14.29％),4 case of good treatment response(GPR)(19.05％)and 11 cases of partial treatment response(PR)(52.38％),3 case of partial no treatment response(NR)(14.29％),9 patients(42.86％)achived transfusion dependent.17 cases(80.90％)and 18 cases(85.71％)achived remission at 6 months and 12 months,the median time to get response was 1.6 months(range 0.1-7.4 months).The white blood cell count(2.21 × 109/L vs.3.82 × 109/L,P=0.029),absolute neutrophil count(0.92×109/L vs.1.96×109/L,P=0.001),platelet count(13× 109/L vs.57× 109/L,P=0.002),and reticulocyte(1.84％vs.2.63％,P=0.011)improved markedly.No Avatrombopag-related grade 2 or higher adverse events occurred during treatment.Conclusion The combination of Avatrombopag and CsA might be an appropriate approach for TD-NSAA with good safety.