Clinical efficacy of sirolimus in treatment of relapsed and refractory autoimmune hemolytic anemia in adults

oleh: YAN Hongju, WANG Ping, QUAN Yao, GAO Lei, LIU Yao

Format: Article
Diterbitkan: Editorial Office of Journal of Third Military Medical University 2020-09-01

Deskripsi

Objective To evaluate the efficacy and safety of sirolimus (SRL) in the treatment of relapsed/refractory (R/R) primary warm-antibody autoimmune hemolytic anemia (AIHA) in adults. Methods A total of 16 R/R AIHA patients admitted in our medical center from January 2016 to January 2019 were recruited in this study. They were all given a dose of SRL 2~4 mg/d to maintain the plasma concentration of 5~15 ng/mL. The total efficacy, complete remission (CR)+partial remission (PR) and side effects after administration were observed. Results The 16 patients (available for efficacy assessment, medication for over 2 months) were 2 males and 14 females, and at a median age of 50 (35~72) years. The median onset of SRL action was 29 d (10~41 d). For the patients taking the agent for 3 months, SRL was still effective for 11 (68.75%) patients. After the treatment for 12 months, it was still effective for 9 patients (56.25%), with 7 patients achieving CR and 2 PR. Till the end of follow-up (median 12 months, ranging from 2 to 42 months) in February 2020, 8 patients still kept continued remission (7 CR and 1 PR). Of note, 5 of them had been discontinued with no relapse, and the longest withdrawal time was as long as 24 months. The side effects of SRL were mild and tolerable to the patients. Conclusion SRL is effective for R/R AIHA in adults, with mild side effects, and recommended as priority choice for treatment of the disease