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

Hongju YAN; Ping WANG; Yao QUAN; Lei GAO; Yao LIU; Cheng ZHANG; Li GAO; Peiyan KONG; Yimei FENG; Xi ZHANG

doi:10.16016/j.1000-5404.202005102

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

更新时间：2026-01-09

- Clinical efficacy of sirolimus in treatment of relapsed and refractory autoimmune hemolytic anemia in adults
- Journal of Third Military Medical University Vol. 42, Issue 17, Pages: 1757-1762(2020)
- 作者机构：
- 作者简介：
- 基金信息：
  
  Clinical Research Project and Talent Training Plan of Army Medical University(2019XLC3014);the Special Project of Basic Frontier of Military Medicine(2018YQYLY002);the Key Special Project of National Key R & D Program(2017YFA0105502)
- DOI：10.16016/j.1000-5404.202005102
  CLC：
- Received：14 May 2020，
  
  Published：15 September 2020
- 稿件说明：
移动端阅览
Hongju YAN, Ping WANG, Yao QUAN, et al. Clinical efficacy of sirolimus in treatment of relapsed and refractory autoimmune hemolytic anemia in adults[J]. Journal of Third Military Medical University, 2020, 42(17): 1757-1762.
DOI：

Hongju YAN, Ping WANG, Yao QUAN, et al. Clinical efficacy of sirolimus in treatment of relapsed and refractory autoimmune hemolytic anemia in adults[J]. Journal of Third Military Medical University, 2020, 42(17): 1757-1762. DOI： 10.16016/j.1000-5404.202005102.

摘要

Abstract

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.

关键词

Keywords

references

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