Efficacy and safety of immunosuppressive therapy in connective tissue disease-related immune thrombocytopenia: a systematic review and meta-analysis

Yupei Zhang; Xiaxiao Yan; Alyssa A Grimshaw; Yanhong Wang; Mengtao Li; Xiaofeng Zeng; Evelyn Hsieh; Nan Jiang

doi:10.1177/20406207261422963

Efficacy and safety of immunosuppressive therapy in connective tissue disease-related immune thrombocytopenia: a systematic review and meta-analysis

Ther Adv Hematol. 2026 Feb 28:17:20406207261422963. doi: 10.1177/20406207261422963. eCollection 2026.

Authors

Yupei Zhang^{1

2}, Xiaxiao Yan³, Alyssa A Grimshaw⁴, Yanhong Wang⁵, Mengtao Li¹, Xiaofeng Zeng¹, Evelyn Hsieh⁶, Nan Jiang⁷

Affiliations

¹ Department of Rheumatology and Clinical Immunology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, National Clinical Research Center for Dermatologic and Immunologic Diseases (NCRC-DID), Ministry of Science and Technology, State Key Laboratory of Complex Severe and Rare Diseases, Key Laboratory of Rheumatology and Clinical Immunology, Ministry of Education, Beijing, China.
² Department of Internal Medicine, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
³ Emergency Department, State Key Laboratory of Complex Severe and Rare Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
⁴ Harvey Cushing/John Hay Whitney Medical Library, Yale University, New Haven, CT, USA.
⁵ Department of Epidemiology and Bio-Statistics, Institute of Basic Medical Sciences, China Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
⁶ Section of Rheumatology, Allergy & Immunology, Yale School of Medicine, New Haven, CT, USA.
⁷ Department of Rheumatology and Clinical Immunology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, National Clinical Research Center for Dermatologic and Immunologic Diseases (NCRC-DID), Ministry of Science and Technology, State Key Laboratory of Complex Severe and Rare Diseases, Key Laboratory of Rheumatology and Clinical Immunology, Ministry of Education, No. 1 Wangfujing Avenue, Beijing, China.

Abstract

Background: Connective tissue diseases (CTDs) are a group of autoimmune disorders in which immune thrombocytopenia (ITP) represents a common and important manifestation. Current CTD-ITP management mainly relies on glucocorticoids and immunosuppressive agents.

Objectives: This systematic review and meta-analysis aims to evaluate the efficacy and safety of immunosuppressive agents in patients with CTD-ITP.

Design: Systematic review and meta-analysis.

Methods: This study was conducted using eight databases up to July 22, 2024. Observational studies and experimental trials with relevant efficacy and safety data were included. Methodological quality were evaluated using the Newcastle-Ottawa Scale and the ROBINS-I tool. The Mantel-Haenszel formula with a random effect model was employed to estimate the overall effect size. Subgroup analyses were performed based on the study characteristics, clinical features, and treatment regimen.

Results: Through integrating 24 studies (1 single-arm clinical trial, 1 case-control, and 22 cohort studies) involving 775 CTD-ITP patients, the pooled estimates of the optimal overall and complete response rate for immunosuppressive therapy were 82% (95% CI: 75-88) and 64% (95% CI: 56-72), with stable results after sensitivity analysis. The combined side-effect incidence was 31% (95% CI: 24-39). The pooled relapse rate was 30% (95% CI: 18-43) among 403 immunosuppressive therapy responders.

Conclusion: Immunosuppressive treatments exhibited favorable efficacy and safety in CTD-ITP patients. Future larger-scale multicenter studies are needed.

Keywords: connective tissue disease; immune thrombocytopenia; immunosuppressant.