STAT3 gain-of-function mutation presenting with adult-onset recurrent abdominal pain, fever and lymphadenopathy, managed by JAK inhibitor therapy.

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Authors: Rofaida Desoki, Stephany Barreda, Samir A. Khwaja, Miles Parkes
Cited by: 0

Abstract

Signal transducer and activator of transcription 3 (STAT3) gain-of-function (GOF) syndrome is a rare genetic immune dysregulation disorder that typically presents in infancy with autoimmunity, lymphoproliferation, susceptibility to infections and growth delay. We report a case of a man in his late 20s admitted to hospital multiple times with abdominal pain, fever and raised C-reactive protein, where imaging revealed progressive mesenteric lymphadenopathy. A definitive diagnosis remained elusive until genetic analysis highlighted a GOF heterozygous mutation in the STAT3 gene. Targeted therapy with Janus kinase inhibitor (tofacitinib) led to significant clinical and radiological improvement. This case underscores the value of a multidisciplinary assessment and early genome sequencing in diagnosing unexplained systemic manifestations, offering promising prospects for more effective management through targeted therapies.

Fields of Study & Subjects

Immunodeficiency and Autoimmune Disorders Cytokine Signaling Pathways and Interactions interferon and immune responses

Publication details

Journal / source
Apollo (University of Cambridge)
Publication date
8 January 2026
Language
EN