Tofacitinib for Glucocorticoid-Resistant Moderate-to-Severe Thyroid Eye Disease

Summary

Thyroid Eye Disease (TED), also known as Graves' orbitopathy, is an autoimmune condition that causes inflammation and tissue expansion behind the eyes, leading to bulging eyes (proptosis), double vision, and pain. Currently, intravenous glucocorticoids (steroids) are the standard first-line treatment. However, approximately 20-30% of patients do not respond to steroids, or cannot tolerate their side effects. This study aims to evaluate the safety and efficacy of Tofacitinib, an oral medication known as a Janus kinase (JAK) inhibitor, as a rescue therapy for these difficult-to-treat cases. Tofacitinib works by blocking specific signaling pathways (JAK-STAT) that drive inflammation and fibrosis in the eye socket. In this study, patients with moderate-to-severe active TED who are resistant to or intolerant of steroids will receive Tofacitinib tablets (5 mg twice daily) for 24 weeks. The researchers will assess whether the treatment can effectively reduce eye bulging and improve clinical activity scores.

Timeline

Start

2026-04-24

Primary completion

2027-12-30

Completion

2027-12-30

Publications

• Background Kim W, Seo MK, Kim YJ, Choi SH, Ku CR, Kim S, Lee EJ, Yoon JS. Role of the suppressor of cytokine signaling-3 in the pathogenesis of Graves' orbitopathy. Front Endocrinol (Lausanne). 2025 Mar 4;16:1527275. doi: 10.3389/fendo.2025.1527275. eCollection 2025.
• Background Ghahvehchian H, Eshraghi B. Tofacitinib for Refractory Thyroid Eye Disease. JAMA Ophthalmol. 2025 Dec 1;143(12):1073-1075. doi: 10.1001/jamaophthalmol.2025.3974.
• Background Wiersinga WM, Eckstein AK, Zarkovic M. Thyroid eye disease (Graves' orbitopathy): clinical presentation, epidemiology, pathogenesis, and management. Lancet Diabetes Endocrinol. 2025 Jul;13(7):600-614. doi: 10.1016/S2213-8587(25)00066-X. Epub 2025 May 2.
• Background Bartalena L, Kahaly GJ, Baldeschi L, Dayan CM, Eckstein A, Marcocci C, Marino M, Vaidya B, Wiersinga WM; EUGOGO dagger. The 2021 European Group on Graves' orbitopathy (EUGOGO) clinical practice guidelines for the medical management of Graves' orbitopathy. Eur J Endocrinol. 2021 Aug 27;185(4):G43-G67. doi: 10.1530/EJE-21-0479.

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