Sirolimus plus roxadustat synergistically enhances immunosuppression and erythropoiesis in pure red cell aplasia: a multicenter trial

Acquired pure red cell aplasia (aPRCA) is rare and challenging to treat. We investigated the efficacy and safety of sirolimus plus roxadustat in patients with aPRCA (Chinese Clinical Trial Register number, ChiCTR2200065107). We enrolled 82 patients with aPRCA in this prospective single-arm, open-label, multicenter trial between October 2022 and January 2024. Treatment response and safety files were evaluated. The median age was 63 years. Seven patients withdrew during the trial period. Sirolimus plus roxadustat produced an overall response (OR) in 65 patients (90.3%) 3 months after initiation, which included a complete response (CR) in 39 (54.2%) and a partial response in 26 (36.1%). The 3-month CR rate was significantly higher in the newly diagnosed group (65.9% vs. 38.7%; P = 0.022). The 6-month OR rate in the entire cohort was 93.0% (CR, 77.5%; PR, 15.5%). The mean hemoglobin concentration increased from 5.5 ± 1.6 g/dL at baseline to 11.6 ± 2.5 g/dL after 6 months of treatment. The proportions of patients who achieved transfusion independence within 1, 2, and 3 months of treatment were 57.4%, 76.6%, and 89.5%, respectively. The Functional Assessment of Chronic Illness Therapy-Fatigue Scale score and SF-36 survey score significantly improved after treatment. Treatment-related adverse events occurred in 24 patients (29.2%), and four events (4.9%) were grade ≥3. Sirolimus plus roxadustat is a promising treatment for aPRCA and has an acceptable safety profile, which warrants further investigation in a randomized setting.

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Publication Details

Journal
Signal Transduction and Targeted Therapy
Published
2026-04-21
DOI
https://doi.org/10.1038/s41392-026-02635-2
Primary Topic
Erythrocyte Function and Pathophysiology
Type
article
Field-Weighted Citation Impact
0.00

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article

Sirolimus plus roxadustat synergistically enhances immunosuppression and erythropoiesis in pure red cell aplasia: a multicenter trial

Ke Ding, Wenjing Jiao, Qiurong Liu, Weiyun Jiao et al.
Signal Transduction and Targeted Therapy
Erythrocyte Function and Pathophysiology
article

Sirolimus plus roxadustat synergistically enhances immunosuppression and erythropoiesis in pure red cell aplasia: a multicenter trial

Ke Ding, Wenjing Jiao, Qiurong Liu, Weiyun Jiao, Meiqing Lei, Zefa Liu, Feng Chen, Jun Wang, Qingyuan Wang, Hong Wang, Jinge Xu, Guanqun Yang, Xiaohui Shangguan, Limin Liu, Depei Wu, Miao Miao, Lingzhi Yan, Yanming Zhang, Liansheng Zhang, Jing Cao, Xiaoli Li, Shan Liu, Xiao Ma, Hongxia Ma, Jianhong Fu, Qian Zhang
article en

Abstract

Acquired pure red cell aplasia (aPRCA) is rare and challenging to treat. We investigated the efficacy and safety of sirolimus plus roxadustat in patients with aPRCA (Chinese Clinical Trial Register number, ChiCTR2200065107). We enrolled 82 patients with aPRCA in this prospective single-arm, open-label, multicenter trial between October 2022 and January 2024. Treatment response and safety files were evaluated. The median age was 63 years. Seven patients withdrew during the trial period. Sirolimus plus roxadustat produced an overall response (OR) in 65 patients (90.3%) 3 months after initiation, which included a complete response (CR) in 39 (54.2%) and a partial response in 26 (36.1%). The 3-month CR rate was significantly higher in the newly diagnosed group (65.9% vs. 38.7%; P = 0.022). The 6-month OR rate in the entire cohort was 93.0% (CR, 77.5%; PR, 15.5%). The mean hemoglobin concentration increased from 5.5 ± 1.6 g/dL at baseline to 11.6 ± 2.5 g/dL after 6 months of treatment. The proportions of patients who achieved transfusion independence within 1, 2, and 3 months of treatment were 57.4%, 76.6%, and 89.5%, respectively. The Functional Assessment of Chronic Illness Therapy-Fatigue Scale score and SF-36 survey score significantly improved after treatment. Treatment-related adverse events occurred in 24 patients (29.2%), and four events (4.9%) were grade ≥3. Sirolimus plus roxadustat is a promising treatment for aPRCA and has an acceptable safety profile, which warrants further investigation in a randomized setting.

Signal Transduction and Targeted TherapyVol. 11(1)
Fujian Medical University (CN), Central South University (CN), Xuzhou Medical College (CN), Scarborough Health Network (CA), Soochow University (CN), Haikou City People's Hospital (CN), Zhangjiagang First People's Hospital (CN), Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital (CN), First Affiliated Hospital of Fujian Medical University (CN), Jiangxi Pingxiang People's Hospital (CN), Lanzhou University Second Hospital (CN), Second People’s Hospital of Huai’an (CN), Changshu No.1 People's Hospital (CN), Xintai People's Hospital (CN), Xian Yang Central Hospital (CN), Zhengzhou People's Hospital (CN), First Affiliated Hospital of Soochow University (CN), Hefei First People's Hospital (CN), Lanzhou University (CN)
National Natural Science Foundation of China, Priority Academic Program Development of Jiangsu Higher Education Institutions
Zero hunger
Openalex Percentile: Top 17%
Erythrocyte Function and Pathophysiology
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