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IMMUNOTHERAPY

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

Leukemia volume 36, pages 1351–1360 (2022)Cite this article

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Abstract

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.

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Fig. 1: Response to sirolimus.
Fig. 2: Key laboratory parameter changes for individual patient prior and after sirolimus treatment at the end of follow-up.
Fig. 3: Recurrence of sirolimus treatment.

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Funding

This study was supported by grants from CAMS Innovation Fund for Medical Sciences (CIFMS 2021-I2M-1-003), National Natural Science Foundation of China (NSFC 81970106).

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Authors and Affiliations

  1. Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China

    Yuzhou Huang, Miao Chen, Chen Yang, Jing Ruan, Shuqing Wang & Bing Han

  2. Department of Gastroenterology, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, 510120, China

    Yuzhou Huang

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  1. Yuzhou Huang
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Contributions

All authors made a significant contribution to the work reported, whether that is in the conception, study design, execution, acquisition of data, analysis and interpretation, or in all these areas; took part in drafting, revising or critically reviewing the article; gave final approval of the version to be published; have agreed on the journal to which the article has been submitted; and agree to be accountable for all aspects of the work.

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Correspondence to Bing Han.

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The authors declare no competing interests.

Ethics approval and consent to participate

This prospective study was approved by the Ethics Committee of PUMCH and registered at clinicaltrial.gov (NCT03364764). All eligible patients were fully informed of this study and signed written informed consent to agree to their participation.

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Huang, Y., Chen, M., Yang, C. et al. Sirolimus is effective for refractory/relapsed/intolerant acquired pure red cell aplasia: results of a prospective single-institutional trial. Leukemia 36, 1351–1360 (2022). https://doi.org/10.1038/s41375-022-01532-1

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