西罗莫司治疗六例难治性免疫性血细胞减少症并文献复习

Treatment for 6 cases of refractory autoimmune cytopenias by sirolimus and literature review

目的探讨西罗莫司治疗难治性免疫性血细胞减少症的疗效。方法回顾性分析郑州大学第一附属医院2017年10月至2018年10月收治的6例难治性免疫性血细胞减少症患者的临床资料,男4例,女2例,中位年龄38(23~55)岁,病程4个月~9年;其中免疫相关性全血细胞减少症3例,Evans综合征2例,免疫性血小板减少症1例。6例患者均经常规治疗效果欠佳,后给予西罗莫司0.2~1.0 mg/d口服治疗,其中5例患者同时联合糖皮质激素或环孢素治疗,血象上升后减少激素、环孢素用量,分析其临床疗效。结果6例难治性免疫性血细胞减少症患者经西罗莫司口服治疗后,达到完全缓解3例,部分缓解2例,无效1例。结论西罗莫司治疗难治性免疫性血细胞减少症安全有效,但仍需要多中心、大样本的临床研究来进一步验证,其对血液病的治疗有着广阔的前景。

Objective To discuss the efficacy of sirolimus in the treatment of refractory autoimmune cytopenias. Methods Clinical data of 6 patients with refractory autoimmune cytopenias admitted to the First Affiliated Hospital of Zhengzhou University from October 2017 to October 2018 were retrospectively analyzed. The 6 patients included 4 males and 2 females, with a median age of 38 (23- 55) years old, disease duration of 4 months to 9 years. Three cases of the 6 patients had immune-related pancytopenia, 2 cases had Evans syndrome, and 1 case had immune thrombocytopenia. Six patients were treated by conventional treatment, but did not get good results, so that they were given sirolimus 0.2 to 1.0 mg/d orally;of them, 5 patients were treated with glucocorticoid or cyclosporine, and the dosages of hormones and cyclosporine were reduced after the blood loss increased. And the efficacy was analyzed. Results Among the 6 patients with refractory autoimmune cytopenias, 3 cases achieved complete remission, 2 cases had partial remission, and 1 case had ineffective result after oral treatment with sirolimus. Conclusions Sirolimus is effective and safe in the treatment of refractory autoimmune cytopenias, but its effect needs to be clinically validated by multi-center and large sample research. The treatment method has broad prospects for treatment of blood diseases.