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

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
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article

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

Samir Khwaja, John I. Bell, Rofaida AbdAllah Shalabi Desoki, Stephany Barreda
Apollo (University of Cambridge)
Immunodeficiency and Autoimmune Disorders
article

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

Samir Khwaja, John I. Bell, Rofaida AbdAllah Shalabi Desoki, Stephany Barreda
article en

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.

Apollo (University of Cambridge)
Good health and well-being
Openalex Percentile: Top 93%
Immunodeficiency and Autoimmune Disorders
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STAT3 gain-of-function mutation presenting with adult-onset recurrent abdominal pain, fever and lymphadenopathy, managed by JAK inhibitor therapy. — Samir Khwaja, John I. Bell, et al. · Apollo (University of Cambridge) (2026) | TGRS Research Map | TGRS