STAT3 gain-of-function mutation presenting with adult-onset recurrent abdominal pain, fever and lymphadenopathy, managed by JAK inhibitor therapy.
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.
Authors
- Samir Khwaja (ORCID: https://orcid.org/0000-0002-2481-6800)
- John I. Bell (ORCID: https://orcid.org/0000-0003-4071-6460)
- Rofaida AbdAllah Shalabi Desoki
- Stephany Barreda
Publication Details
- Journal
- Apollo (University of Cambridge)
- Published
- 2026-01-08
- DOI
- https://doi.org/10.17863/cam.124558
- Primary Topic
- Immunodeficiency and Autoimmune Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00