Cryptococcal pneumonia in an adolescent with a gain-of-function variant in signal transduction and activator of transcription 1 (STAT1)

BMJ Case Rep. 2020 Apr 22;13(4):e234120. doi: 10.1136/bcr-2019-234120.

Abstract

An adolescent male with a history of autoimmune enteropathy, autoimmune hypothyroidism, aphthous stomatitis and recurrent oral Candida infections only in the setting of curative antibiotic courses presented with cryptococcal pneumonia and perihilar adenitis, which was successfully treated with antifungal therapy. The patient had a complex history with several immunological anomalies. Whole exome sequencing revealed a known STAT1 pathogenic variant, associated with gain of function (GOF). This case expands our understanding of the broad clinical phenotype manifested by STAT1 GOF and emphasises the importance of consideration of this diagnosis in patients presenting with opportunistic infections and autoimmunity.

Keywords: Cryptococcus; drugs: respiratory system; immunological products and vaccines; infections; radiology.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Anti-Infective Agents / therapeutic use
  • Antifungal Agents / therapeutic use
  • Cryptococcosis / drug therapy
  • Cryptococcosis / genetics*
  • Cryptococcosis / microbiology
  • Diagnosis, Differential
  • Drug Therapy, Combination
  • Exome Sequencing
  • Gain of Function Mutation*
  • Humans
  • Immunocompromised Host
  • Immunoglobulins, Intravenous / therapeutic use
  • Male
  • Phenotype
  • Pneumonia / drug therapy
  • Pneumonia / genetics*
  • Pneumonia / microbiology
  • STAT1 Transcription Factor / genetics*
  • Signal Transduction
  • Tomography, X-Ray Computed

Substances

  • Anti-Infective Agents
  • Antifungal Agents
  • Immunoglobulins, Intravenous
  • STAT1 Transcription Factor
  • STAT1 protein, human