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Janus-kinase inhibitor use in immune-mediated inflammatory diseases beyond licensed indications: A scoping review

Challoumas, Dimitris; Simpson, Cameron; Arnold, Matthew; Mease, Philip; Moots, Robert; Ndosi, Mwidimi; Locher, Zoe Rutter

Janus-kinase inhibitor use in immune-mediated inflammatory diseases beyond licensed indications: A scoping review Thumbnail


Authors

Dimitris Challoumas

Cameron Simpson

Matthew Arnold

Philip Mease

Robert Moots

Profile image of Mwidimi Ndosi

Dr Mwidimi Ndosi Mwidimi.Ndosi@uwe.ac.uk
Associate Professor in Nursing Rheumatology

Zoe Rutter Locher



Abstract

Introduction
The use of Janus kinase inhibitors (JAKis) in immune-mediated inflammatory diseases (IMIDs) beyond licence is expanding rapidly. The aim of this scoping review was to identify and present the available evidence on the efficacy of JAKis in all conditions without marketing authorisation.

Methods
Through a systematic literature search we identified studies including 5 or more patients that assessed the use of any JAKi for any efficacy outcome. Quantitative analyses in the form of pairwise meta-analyses were performed for eligible data from randomised controlled trials (RCTs) only.

Results
Eighty-three (n = 83) studies in total were included in our review, assessing efficacy of JAKis in 34 IMIDs. In most conditions, JAKis exhibited generally positive effects, though the majority of evidence came from observational, non-comparative studies.

Pairwise meta-analyses were possible for hidradenitis suppurativa and systemic lupus erythematosus (SLE). For hidradenitis suppurativa, we found a clear benefit of treatment with JAKis compared with placebo in achieving clinical response (OR 2.35, 95 % CI 1.24 to 4.46). For treatment-resistant SLE, the results were equivocal; JAKis showed some benefit over placebo but statistical significance was only reached for one of the two meta-analysed outcome measures (SLE Responder Index 4, OR 1.41, 95 % CI 1.01 to 1.98; SLE Disease Activity Index 2000; OR 1.36, 95 % CI 0.99 to 1.88).

Conclusions
There is a rapidly increasing use of JAKis beyond current licensing in most IMIDs. Large comparative trials are necessary to confirm efficacy and guide future licensing decisions.

Journal Article Type Article
Acceptance Date Dec 20, 2024
Online Publication Date Dec 30, 2024
Publication Date Feb 28, 2025
Deposit Date Jan 3, 2025
Publicly Available Date Jan 7, 2025
Journal Autoimmunity Reviews
Print ISSN 1568-9972
Publisher Elsevier
Peer Reviewed Peer Reviewed
Volume 24
Issue 3
Article Number 103736
DOI https://doi.org/10.1016/j.autrev.2024.103736
Keywords Autoimmune, Rheumatology, Dermatology, Gastroenterology, Multi-organ
Public URL https://uwe-repository.worktribe.com/output/13591494
Publisher URL https://www.sciencedirect.com/science/article/pii/S1568997224002271?via%3Dihub
Related Public URLs https://doi.org/10.1016/j.autrev.2024.103736
Additional Information This article is maintained by: Elsevier; Article Title: Janus-kinase inhibitor use in immune-mediated inflammatory diseases beyond licensed indications: A scoping review; Journal Title: Autoimmunity Reviews; CrossRef DOI link to publisher maintained version: https://doi.org/10.1016/j.autrev.2024.103736; Content Type: article; Copyright: © 2024 The Authors. Published by Elsevier B.V.
This output contributes to the following UN Sustainable Development Goals:

SDG 3 - Good Health and Well-Being

Ensure healthy lives and promote well-being for all at all ages

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