ACR open rheumatology · 2026
Evaluating the Efficacy of Cytokine-Directed Therapies in the Management of VEXAS Syndrome.
СтатьяНазвание и аннотация: Перевод в очереди
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Montes Daniel, Langenfeld Hannah E, Hanson Andrew C, Crowson Cynthia S, Patnaik Mrinal M, Go Ronald S, Kalantari Kambiz, Kusne Yael, Lasho Terra, Mangaonkar Abhishek A, Olteanu Horatiu, Reichard Kaaren K, Sullivan Megan M, Viswanatha David S, Warrington Kenneth J, Koster Matthew J
Аннотация
OBJECTIVE: To evaluate the efficacy of cytokine-directed therapies in VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory somatic) syndrome using previously proposed treatment response criteria.
METHODS: All patients with genetically confirmed VEXAS syndrome who received targeted therapies with JAK inhibitors (JAKi), tumor necrosis factor α inhibitors (TNFαi), or interleukin-1 (IL-1) or IL-6 inhibitors were identified. Clinical and biochemical data were recorded for patients at time of targeted therapy initiation, and at 3-, 6-, and 12-month follow-up time points. Treatment response was labeled as complete response (CR; normalization of C-reactive protein [CRP], prednisone <10 mg/day, and resolution of disease activity), partial response (PR; >50% improvement in CRP, >50% prednisone dose reduction, improvement or resolution of disease features), treatment failure (TF; worsening CRP, increasing prednisone, worsening of disease features), or no response (not meeting criteria for CR, PR, or TF).
RESULTS: Overall, 52 patients received an IL-1i, IL-6i, or JAKi therapy. At six-month follow-up, 3 of 19 IL-6i patients experienced CR, 4 of 19 experienced PR, and 7 of 19 experienced TF. 2 of 17 patients receiving JAKi experienced CR, 3 of 17 experienced PR, and 6 of 17 experienced TF. No patients receiving IL-1i or TNFαi experienced CR, and only 1 of 5 IL-1i patients experienced PR. Among those still receiving biologic therapy at 12 months, only 1 of 11 receiving IL-6i and 0 of 7 receiving JAKi experienced CR.
CONCLUSION: IL-6i and JAKi therapies demonstrated limited, unsustained efficacy for management of VEXAS syndrome. IL-1i and TNFαi therapies were not efficacious. Useful application of previously reported treatment response criteria was demonstrated, but future incorporation of hematologic parameters may be beneficial.
METHODS: All patients with genetically confirmed VEXAS syndrome who received targeted therapies with JAK inhibitors (JAKi), tumor necrosis factor α inhibitors (TNFαi), or interleukin-1 (IL-1) or IL-6 inhibitors were identified. Clinical and biochemical data were recorded for patients at time of targeted therapy initiation, and at 3-, 6-, and 12-month follow-up time points. Treatment response was labeled as complete response (CR; normalization of C-reactive protein [CRP], prednisone <10 mg/day, and resolution of disease activity), partial response (PR; >50% improvement in CRP, >50% prednisone dose reduction, improvement or resolution of disease features), treatment failure (TF; worsening CRP, increasing prednisone, worsening of disease features), or no response (not meeting criteria for CR, PR, or TF).
RESULTS: Overall, 52 patients received an IL-1i, IL-6i, or JAKi therapy. At six-month follow-up, 3 of 19 IL-6i patients experienced CR, 4 of 19 experienced PR, and 7 of 19 experienced TF. 2 of 17 patients receiving JAKi experienced CR, 3 of 17 experienced PR, and 6 of 17 experienced TF. No patients receiving IL-1i or TNFαi experienced CR, and only 1 of 5 IL-1i patients experienced PR. Among those still receiving biologic therapy at 12 months, only 1 of 11 receiving IL-6i and 0 of 7 receiving JAKi experienced CR.
CONCLUSION: IL-6i and JAKi therapies demonstrated limited, unsustained efficacy for management of VEXAS syndrome. IL-1i and TNFαi therapies were not efficacious. Useful application of previously reported treatment response criteria was demonstrated, but future incorporation of hematologic parameters may be beneficial.