阿伐曲泊帕转换治疗TPO受体激动剂难治性再生障碍性贫血疗效及安全性

OBJECTIVE: Short-term efficacy and safety of afatrombopag conversion therapy in patients with aplastic anemia（AA）who were previously ineffectively treated with intense immunosuppressive therapy（IST）combined with TPO receptor Agonist（TPO-RA）or who were unable to tolerate the side effects of TPO-RA. METHODS: Analysis of patients with severe aplastic anemia（SAA）treated in Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College from January 2021 to December 2021 who received IST combined with TPO-RA（eltrombopag/hatrombopag）for at least 6 months, but was ineffective for at least 3 months or patients who cannot continue to use TPO-RA due to side effects, and switched from TPO-RA to avatrombopag（APAG）, and treated for at least 6 months. This study analyzed its short-term efficacy and evaluated its safety. RESULTS: The median age was 54（14–68）years old among the 16 patients with AA（8 male and eight female）. A total of ten patients（refractory group）who did not respond to IST combined with TPO-RA were converted to APAG median therapy for 6（6–10）months. Only seven patients（70％）obtained trilineage HR [four cases of complete treatment response（CTR）, one case of good treatment response（GPR）, and two cases of partial treatment response（PR）], all of which began to take effect at 3 months of APAG treatment. There were six patients with TPO-RA intolerance, and APAG was treated for 6（2 to 8）months. About four patients（67％）received HR（three GPR cases and one PR case）, of which two patients received PR at 3 months and four patients received HR at 6 months of APAG treatment. No APAG-related grade 2 or more adverse events occurred during the APAG therapy, no thrombotic events occurred, no fibrologic tissue hyperplasia was found in the bone marrow pathology reexamination at 6 months of treatment, and none of the patients discontinued the drug due to adverse events. CONCLUSION: APAG may be a better switching treatment option for patients with AA who are refractory or are intolerant to TPO-RA.