VEXAS syndrome: favourable clinical and partial haematological responses to subcutaneous abatacept therapy with 30-month follow-up.

Abstract

Dear Editor, The clinical spectrum of VEXAS (vacuoles, E-1 ubiquitin activating enzyme, X-linked, autoinflammatory and somatic) syndrome continues to expand since the original report by Beck et al. [1]. Despite increased recognition, there remains a distinct lack of treatment options with multiple immunosuppressive agents deemed ineffective [2, 3]. Initial literature suggested a high mortality rate approaching 50%, however subsequent reports have described a broader phenotypic spectrum and prognostic profile [3, 4]. In this report, the response to abatacept is described in a patient who exhibited significant haematological abnormalities, major systemic manifestations and moderate dose corticosteroid dependence. Abatacept introduction led to a sustained period of clinical stability, reduction in inflammatory symptoms including chondritis and fever, and improvement in haematological indices while just using 5 mg prednisolone daily. A 71-year-old male presented with a generalized rash, fatigue and night sweats in 2013. Initially,...