The safety and efficacy of otilimab for rheumatoid arthritis: a systematic review and meta-analysis of randomized controlled trials

Abstract Background Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by joint inflammation and damage, affecting millions globally. Despite treatment advancements, achieving remission remains a challenge. Granulocyte–macrophage colony-stimulating factor (GM-CSF), elevated in R...

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Main Authors: Abdelrahman Mahmoud, Salem Elshenawy, Ahmed A. Ibrahim, Yasmine Adel, Yehya Khlidj, Ali M. Al-Khalaileh, Majd M. AlBarakat, Salma Allam, Mohamed Abuelazm
Format: Article
Language:English
Published: SpringerOpen 2025-05-01
Series:The Egyptian Journal of Internal Medicine
Online Access:https://doi.org/10.1186/s43162-025-00455-4
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author Abdelrahman Mahmoud
Salem Elshenawy
Ahmed A. Ibrahim
Yasmine Adel
Yehya Khlidj
Ali M. Al-Khalaileh
Majd M. AlBarakat
Salma Allam
Mohamed Abuelazm
author_facet Abdelrahman Mahmoud
Salem Elshenawy
Ahmed A. Ibrahim
Yasmine Adel
Yehya Khlidj
Ali M. Al-Khalaileh
Majd M. AlBarakat
Salma Allam
Mohamed Abuelazm
author_sort Abdelrahman Mahmoud
collection DOAJ
description Abstract Background Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by joint inflammation and damage, affecting millions globally. Despite treatment advancements, achieving remission remains a challenge. Granulocyte–macrophage colony-stimulating factor (GM-CSF), elevated in RA, is a promising therapeutic target. Otilimab, a GM-CSF-blocking antibody, showed promising potential in clinical trials. Methods We systematically searched PubMed, Web of Science, and Scopus databases until Mar 2, 2024. For dichotomous outcomes, we employed the risk ratio (RR). We used the mean difference (MD) with a 95% confidence interval (CI) for continuous outcomes, utilizing the random-effects model to pool the results (PROSPERO ID: CRD42024527342). Results Three publications reporting four trials were included, with 3933 patients. Moderate-certainty evidence demonstrated that otilimab significantly increased ACR responders compared to placebo, both in the 90 mg group (RR, 1.50; 95% CI 1.14, 1.98; P < 0.0001) and the 150 mg group (RR, 1.39; 95% CI 1.12; 1.73, P < 0.0001). High-certainty evidence suggests that otilimab, at both 90 mg and 150 mg doses, did not differ significantly from placebo in terms of experiencing any adverse events (90 mg: RR, 1.02; 95% CI [0.92, 1.13]; P = 0.71; 150 mg: RR, 1.03; 95% CI [0.89, 1.19]; P = 0.73). Conclusion Otilimab shows a favorable balance of efficacy and safety over placebo in RA treatment, but it is less effective than tofacitinib. These findings support GM-CSF inhibition as a viable therapeutic strategy and provide a basis for further development of more effective RA-targeted therapies.
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spelling doaj-art-9366128eca9f491ba7032513efd638db2025-08-20T03:22:13ZengSpringerOpenThe Egyptian Journal of Internal Medicine2090-90982025-05-0137111210.1186/s43162-025-00455-4The safety and efficacy of otilimab for rheumatoid arthritis: a systematic review and meta-analysis of randomized controlled trialsAbdelrahman Mahmoud0Salem Elshenawy1Ahmed A. Ibrahim2Yasmine Adel3Yehya Khlidj4Ali M. Al-Khalaileh5Majd M. AlBarakat6Salma Allam7Mohamed Abuelazm8Faculty of Medicine, Minia UniversityFaculty of Medicine, Alexandria UniversityFaculty of Medicine, Menoufia UniversityFaculty of Medicine, Assiut UniversityFaculty of Medicine, Algiers UniversityFaculty of Medicine, Jordan University of science and technologyFaculty of Medicine, Jordan University of science and technologyFaculty of Medicine, Galala UniversityFaculty of Medicine, Tanta UniversityAbstract Background Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by joint inflammation and damage, affecting millions globally. Despite treatment advancements, achieving remission remains a challenge. Granulocyte–macrophage colony-stimulating factor (GM-CSF), elevated in RA, is a promising therapeutic target. Otilimab, a GM-CSF-blocking antibody, showed promising potential in clinical trials. Methods We systematically searched PubMed, Web of Science, and Scopus databases until Mar 2, 2024. For dichotomous outcomes, we employed the risk ratio (RR). We used the mean difference (MD) with a 95% confidence interval (CI) for continuous outcomes, utilizing the random-effects model to pool the results (PROSPERO ID: CRD42024527342). Results Three publications reporting four trials were included, with 3933 patients. Moderate-certainty evidence demonstrated that otilimab significantly increased ACR responders compared to placebo, both in the 90 mg group (RR, 1.50; 95% CI 1.14, 1.98; P < 0.0001) and the 150 mg group (RR, 1.39; 95% CI 1.12; 1.73, P < 0.0001). High-certainty evidence suggests that otilimab, at both 90 mg and 150 mg doses, did not differ significantly from placebo in terms of experiencing any adverse events (90 mg: RR, 1.02; 95% CI [0.92, 1.13]; P = 0.71; 150 mg: RR, 1.03; 95% CI [0.89, 1.19]; P = 0.73). Conclusion Otilimab shows a favorable balance of efficacy and safety over placebo in RA treatment, but it is less effective than tofacitinib. These findings support GM-CSF inhibition as a viable therapeutic strategy and provide a basis for further development of more effective RA-targeted therapies.https://doi.org/10.1186/s43162-025-00455-4
spellingShingle Abdelrahman Mahmoud
Salem Elshenawy
Ahmed A. Ibrahim
Yasmine Adel
Yehya Khlidj
Ali M. Al-Khalaileh
Majd M. AlBarakat
Salma Allam
Mohamed Abuelazm
The safety and efficacy of otilimab for rheumatoid arthritis: a systematic review and meta-analysis of randomized controlled trials
The Egyptian Journal of Internal Medicine
title The safety and efficacy of otilimab for rheumatoid arthritis: a systematic review and meta-analysis of randomized controlled trials
title_full The safety and efficacy of otilimab for rheumatoid arthritis: a systematic review and meta-analysis of randomized controlled trials
title_fullStr The safety and efficacy of otilimab for rheumatoid arthritis: a systematic review and meta-analysis of randomized controlled trials
title_full_unstemmed The safety and efficacy of otilimab for rheumatoid arthritis: a systematic review and meta-analysis of randomized controlled trials
title_short The safety and efficacy of otilimab for rheumatoid arthritis: a systematic review and meta-analysis of randomized controlled trials
title_sort safety and efficacy of otilimab for rheumatoid arthritis a systematic review and meta analysis of randomized controlled trials
url https://doi.org/10.1186/s43162-025-00455-4
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