Jakinibs for the Treatment of Immune Dysregulation in Patients With Gain-Of-Function Signal Transducer and Activator of Transcription 1 (STAT1) or STAT3 Mutations

J Allergy Clin Immunol. 2018 Nov;142(5):1665-1669. doi: 10.1016/j.jaci.2018.07.020. Epub 2018 Aug 6.

Abstract

Treatment of the autoimmune and immune-dysregulatory features of patients with STAT1 GOF or STAT3 GOF disease remains challenging. Jakinibs have been used to treat the severe immune-dysregulation in patients with either STAT1 GOF or STAT3 GOF mutations.

Publication types

  • Letter
  • Multicenter Study
  • Research Support, N.I.H., Intramural

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Female
  • Gain of Function Mutation
  • Humans
  • Immunologic Deficiency Syndromes / drug therapy
  • Immunologic Deficiency Syndromes / genetics*
  • Janus Kinases / antagonists & inhibitors*
  • Male
  • Piperidines / therapeutic use
  • Protein Kinase Inhibitors / therapeutic use
  • Pyrazoles / therapeutic use
  • Pyrimidines / therapeutic use
  • Pyrroles / therapeutic use
  • STAT1 Transcription Factor / genetics*
  • STAT3 Transcription Factor / genetics*
  • Treatment Outcome

Substances

  • INCB018424
  • Piperidines
  • Protein Kinase Inhibitors
  • Pyrazoles
  • Pyrimidines
  • Pyrroles
  • STAT1 Transcription Factor
  • STAT1 protein, human
  • STAT3 Transcription Factor
  • STAT3 protein, human
  • tofacitinib
  • Janus Kinases