Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Department of Hematology, Institute of Hematology of Henan Province, Affiliated Cancer Hospital of Zhengzhou University, Henan Cancer Hospital, 127 Dongming Road, Zhengzhou, China.
Ann Hematol. 2020 Apr;99(4):737-741. doi: 10.1007/s00277-020-03946-2. Epub 2020 Feb 6.
For patients with pure red cell aplasia (PRCA), cyclosporine (CsA) is the first line therapy. Occasionally, some patients who suffer from renal insufficiency cannot tolerate CsA. To explore the efficacy and tolerance of sirolimus treatment for those patients, twelve PRCA patients with renal insufficiency from May 2014 to May 2018 in Peking Union Medical College Hospital were enrolled, treated with sirolimus, and followed up at the median time of 16 (10-50) months. Eleven patients (91.7%) responded to sirolimus, with 58.3% complete response (CR) and 41.7% partial response (PR). The median time to achieve the optimum effect was 4 (1-7) months. The serum creatinine level remained stable or even reduced during the treatment period for eleven patients. Seven patients (58.3%) reported adverse events during sirolimus therapy, including increased blood glucose, infection, skin rash, elevated triglyceride or total cholesterol, and elevated serum creatinine compared with baseline. No treatment-related death was noticed during the follow-up time. Three patients relapsed with an overall response rate of 75.0% at 1 year. These results suggested that sirolimus was effective and tolerable for patients with PRCA complicated with renal insufficiency.
对于纯红细胞再生障碍性贫血（PRCA）患者，环孢素（CsA）是一线治疗药物。偶尔，一些患有肾功能不全的患者不能耐受 CsA。为了探索西罗莫司治疗这些患者的疗效和耐受性，我们纳入了 2014 年 5 月至 2018 年 5 月期间北京协和医院的 12 例肾功能不全的 PRCA 患者，给予西罗莫司治疗，并在中位数 16（10-50）个月时进行随访。11 例患者（91.7%）对西罗莫司有反应，其中 58.3%完全缓解（CR）和 41.7%部分缓解（PR）。达到最佳疗效的中位数时间为 4（1-7）个月。11 例患者在治疗期间血清肌酐水平保持稳定或甚至降低。7 例患者（58.3%）在西罗莫司治疗期间出现不良反应，包括血糖升高、感染、皮疹、甘油三酯或总胆固醇升高和血清肌酐升高与基线相比。在随访期间未观察到与治疗相关的死亡。3 例患者在 1 年内复发，总反应率为 75.0%。这些结果表明，西罗莫司对伴有肾功能不全的 PRCA 患者有效且耐受良好。
