Sirolimus is effective for refractory/relapsed/intolerant acquired pure red cell aplasia: results of a prospective single-institutional trial.

Some patients with acquired pure red cell aplasia (aPRCA) have no response or are intolerant to cyclosporine A. From April 2017 to August 2020, patients diagnosed with aPRCA at Peking Union Medical College Hospital who were refractory/recurrent/intolerant to at least 6 months of full-dose cyclosporin A (CsA) with/without steroids were recruited and treated with sirolimus for at least 6 months. Finally, a total of 64 patients were enrolled. The overall response rate and complete response rate after 3, 6 and 12 months of sirolimus were 60.9%, 84.4%, and 73.5% and 50.0%, 65.6%, and 66.0%, respectively. At a median of 14.5 (6-47) months of follow-up, 14.8% (8/54) of the patients relapsed. Apart from haemoglobin improvement, patients had decreased creatine levels and serum ferritin levels at the end of the follow-up compared with the baseline (169.3 μmol/L vs. 146.4 μmol/L, p = 0.041; 2121.5 ng/mL vs. 1018.3 ng/mL, p = 0.013). Adverse events were recorded in 19 patients, including infections and increase of creatine. Secondary aPRCA with stable underlying diseases had similar results as those with primary aPRCA. In summary, sirolimus is effective for patients with refractory/recurrent/intolerant aRPCA with a low recurrence rate and toxicities.