Novel application for the JAK inhibitor baricitinib in the treatment of Vogt–Koyanagi–Harada disease: a prospective cohort study

TL;DR

This study shows that baricitinib, a JAK inhibitor, effectively treats both early and late stages of Vogt–Koyanagi–Harada disease with minimal side effects.

Contribution

The novel use of baricitinib as a treatment for VKH disease is demonstrated, including its efficacy in monotherapy and combination therapy.

Findings

Baricitinib monotherapy significantly improved visual acuity, reduced inflammation, and resolved subretinal fluid in VKH patients.

No severe adverse drug reactions or disease recurrences were observed during the follow-up period.

Baricitinib allows for a reduction in corticosteroid use while maintaining effective disease control.

Abstract

The purpose of this study was to prospectively evaluate the clinical efficacy and safety of baricitinib, a selective Janus kinase (JAK)1/JAK2 inhibitor, in the treatment of patients with Vogt–Koyanagi–Harada (VKH) disease.

The study was designed as a prospective, open-label, longitudinal, single-center cohort study.

We enrolled 38 patients (76 eyes) with VKH disease (including both initial-onset and recurrent/chronic cases) at Tongji Hospital, Wuhan, China, between 2022 and 2024. Patients were assigned to one of two treatment protocols based on disease severity and prior treatment history: group A received oral baricitinib (4 mg/day) combined with systemic methylprednisolone, and group B received oral baricitinib monotherapy. Clinical assessments were performed at baseline and at 1, 3, 6, 12, and 18 months. Primary outcome measures included best-corrected visual acuity (BCVA), anterior chamber (AC) cell and flare grades, subfoveal choroidal thickness (SFCT), subretinal fluid (SRF) height, and angiographic inflammatory scores (fluorescein angiography [FA]/indocyanine green angiography [ICGA]). Secondary outcomes included quality-of-life scores (25-item version of the Visual Function Questionnaire [VFQ-25], SF-36), adverse drug reactions (ADRs), and recurrence rates.

The cohort included 44 eyes with early-stage (initial-onset) and 32 eyes with late-stage (recurrent) VKH disease (mean age: 43.1 years ± 12.3 years; follow-up: 6–18 months). Inflammation control was achieved in all patients in both groups. In group A, the combination therapy led to rapid resolution of inflammation. In group B (monotherapy), significant improvements were observed from baseline to the final visit, including improved BCVA (0.12 to 0.68, p < 0.001), reduced AC cell grade (1.54 to 0.05, p = 0.007), decreased SFCT (537.6 to 252.4 µm, p = 0.007), and complete resolution of SRF (p < 0.001). FA and ICGA scores significantly decreased (p < 0.05), and VFQ-25 scores improved (82.6 to 92.9, p < 0.001). No severe ADRs or disease recurrences were observed during the follow-up period.

This study demonstrates that baricitinib is a promising therapeutic option for both early- and late-stage VKH disease. It effectively controls choroidal inflammation, improves visual function, and allows for a substantial reduction in corticosteroid burden with a favorable safety profile.

http://www.chictr.org.cn, identifier ChiCTR2100048030.

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