西罗莫司治疗成人复发/难治性自身免疫性溶血性贫血的临床疗效

颜红菊; 王平; 全瑶; 高蕾; 刘耀; 张诚; 高力; 孔佩艳; 冯一梅; 张曦

doi:10.16016/j.1000-5404.202005102

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西罗莫司治疗成人复发/难治性自身免疫性溶血性贫血的临床疗效

临床医学 | 更新时间：2026-01-09

- 西罗莫司治疗成人复发/难治性自身免疫性溶血性贫血的临床疗效
- Clinical efficacy of sirolimus in treatment of relapsed and refractory autoimmune hemolytic anemia in adults
- 第三军医大学学报 2020年42卷第17期页码：1757-1762
- 作者机构：
- 作者简介：
- 基金信息：
  
  陆军军医大学临床科研项目与人才培养计划(2019XLC3014);军事医学基础前沿领域专项(2018YQYLY002);国家重点研究计划重点专项(2017YFA0105502)
- DOI：10.16016/j.1000-5404.202005102
  中图分类号：
- 收稿：2020-05-14，
  
  纸质出版：2020-09-15
- 稿件说明：
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颜红菊, 王平, 全瑶, 等. 西罗莫司治疗成人复发/难治性自身免疫性溶血性贫血的临床疗效[J]. 第三军医大学学报, 2020,42(17):1757-1762.

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.
颜红菊, 王平, 全瑶, 等. 西罗莫司治疗成人复发/难治性自身免疫性溶血性贫血的临床疗效[J]. 第三军医大学学报, 2020,42(17):1757-1762. DOI： 10.16016/j.1000-5404.202005102.

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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