Evidence map›Paper›PMID 42010264›Full record

Trial reportSignal transduction and targeted therapy2026

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

Hong Wang, Qingyuan Wang, Shan Liu, Yanming Zhang, Ke Ding, Xiaoli Li, Wenjing Jiao, Qian Zhang, Jianhong Fu, Jing Cao and 16 more

Abstract readMulticenter StudyClinical Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Signal transduction and targeted therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

26 authors.

Hong Wang *National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China.
Qingyuan Wang *National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China.
Shan Liu *Sichuan Provincial Key Laboratory for Human Disease Gene Study, Department of Medical Genetics, Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, China.
Yanming ZhangDepartment of Hematology, The Affiliated Huai'an Hospital of Xuzhou Medical University and The Second People's Hospital of Huai'an, Huai'an, China.
Ke DingUniversity of Toronto Scarborough, Scarborough, ON, Canada.
Xiaoli LiSoochow Hopes Hematonosis Hospital, Suzhou, China.
Wenjing JiaoDepartment of Hematology, Xian Yang Central Hospital, Xianyang, China.
Qian ZhangSoochow Hopes Hematonosis Hospital, Suzhou, China.
Jianhong FuNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China.
Jing CaoDepartment of Hematology, Zhangjiagang First People's Hospital, Suzhou, China.
Weiyun JiaoDepartment of Hematology, Hefei First People's Hospital, Hefei, China.
Miao MiaoNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China.
Zefa LiuDepartment of Hematology, People Hospital of Xinghua, Xinghua, China.
Qiurong LiuDepartment of Hematology, Pingxiang Municipal People's Hospital of Jiangxi Province, Pingxiang, China.
Guanqun YangDepartment of Hematology, Changshu Hospital Affiliated to Soochow University, Changshu NO.1 People's Hospital, Changshu, China.
Xiao MaNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China.
Feng ChenNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China.
Jun WangNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China.
Lingzhi YanNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China.
Xiaohui ShangguanDepartment of Hematology, Longyan First Hospital, Affiliated to Fujian Medical University, Longyan, China.
Jinge XuDepartment of Hematology, The Second Affiliated Hospital of Xuzhou Medical University, Xuzhou, China.
Meiqing LeiDepartment of Hematology, Haikou Municipal People's Hospital, Affiliated Haikou Hospital of Xiangya Medical College, Central South University, Haikou, China.
Hongxia MaDepartment of Hematology, The Third People's Hospital of Zhengzhou, Zhengzhou, China.
Liansheng ZhangDepartment of Hematology, The Second Hospital of Lanzhou University, Lanzhou, China.
Limin LiuNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China. liminliu2006@163.com.
Depei WuNational Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation of Soochow University, Collaborative Innovation Center of Hematology, Suzhou, China. wudepei@suda.edu.cn.ORCID http://orcid.org/0000-0002-6312-3863

Funding

National Natural Science Foundation of China (National Science Foundation of China) 82020108003, 82330008
6 · The paper itself

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.

Indexed as

ErythropoiesisGlycineImmunosuppression TherapyIsoquinolinesRed-Cell Aplasia, PureSirolimusAdultAgedFemaleHumansMaleMiddle AgedGlycineIsoquinolinesroxadustatSirolimus

Identifiers

PMID42010264
PMCPMC13096638

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.