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.
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We thank all the patients who consented to disclose their medical records and answered our review calls. Their cooperation and helpful comments throw light upon our data collection and statistical analysis.
This study was supported by grants from Beijing Natural Science Foundation (7192168), the Chinese Academy of Medical Sciences (CAMS) innovation fund for medical sciences (2016-I2M-3-004), and the National Key Research and Development Program of China (2016YFC0901500).
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Chen, Z., Liu, X., Chen, M. et al. Successful sirolimus treatment of patients with pure red cell aplasia complicated with renal insufficiency. Ann Hematol (2020). https://doi.org/10.1007/s00277-020-03946-2
- Pure red cell aplasia
- Renal insufficiency
- Cyclosporine A