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Systematic Review and Model-Based Meta-Analysis of Targeted Drugs for Systemic Sclerosis.

Created on 14 Sep 2026

Authors

Marina Vaskeikina, Yaroslav Ugolkov, Boris Kireev, Kirill Peskov, Alina Volkova

Published in

Pharmaceutics. Volume 18. Issue 2. Feb 18, 2026. Epub Feb 18, 2026.

Abstract

Background: Systemic sclerosis (SSc) is a complex autoimmune fibrotic disorder marked by heterogeneous clinical features and multiple pathophysiological mechanisms. The rapid emergence of targeted therapies, aimed at selectively modulating molecular targets, has expanded treatment options; however, making direct efficacy comparisons remains challenging due to the variability in trial designs, endpoints, and patient populations. Methods: A systematic search of PubMed and ClinicalTrials.gov identified randomized controlled trials (RCTs) evaluating targeted therapies in SSc. A longitudinal mixed-effect meta-model incorporating Emax structural functions characterized treatment response trajectories for the modified Rodnan skin score (mRSS) and forced vital capacity (FVC). Between-study and between-treatment-arm variability were explicitly modeled to account for heterogeneity. Results: A total of 32 RCTs with 2036 patients and 23 targeted agents were analyzed. Guselkumab, an anti-IL-23 antibody, showed the greatest effect on mRSS, followed by tofacitinib, inebilizumab, and baricitinib. For FVC, B-cell-targeted therapies, with belimumab and rituximab, demonstrated the highest efficacy, while tocilizumab and nintedanib had more moderate effects. Time to 50% maximal response was approximately 27.5 weeks, indicating a 6.3-month period for half treatment response development. Conclusions: This model-based meta-analysis provides a broad comparison of targeted therapies in SSc, highlighting distinct efficacy patterns for skin versus lung involvement and offering hypothesis-generating insights that may support treatment selection and the design of future clinical trials.

PMID:
41754991
Bibliographic data and abstract were imported from PubMed on 14 Sep 2026.

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