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Emerging Place of JAK Inhibitors in the Treatment of Inborn Errors of Immunity
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Emerging Place of JAK Inhibitors in the Treatment of Inborn Errors of Immunity
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Journal Article
Emerging Place of JAK Inhibitors in the Treatment of Inborn Errors of Immunity
2021
Overview
Among inborn errors of immunity (IEIs), some conditions are characterized by inflammation and autoimmunity at the front line and are particularly challenging to treat. Monogenic diseases associated with gain-of-function mutations in genes critical for cytokine signaling through the JAK-STAT pathway belong to this group. These conditions represent good candidates for treatment with JAK inhibitors. Type I interferonopathies, a group of recently identified monogenic auto-inflammatory diseases characterized by excessive secretion of type I IFN, are also good candidates with growing experiences reported in the literature. However, many questions remain regarding the choice of the drug, the dose (in particular in children), the efficacy on the various manifestations, the monitoring of the treatment, and the management of potent side effects in particular in patients with infectious susceptibility. This review will summarize the current experiences reported and will highlight the unmet needs.
Publisher
Frontiers Media SA,Frontiers Media S.A
Subject
/ Drug Evaluation, Preclinical
/ Genetic Diseases, Inborn - drug therapy
/ Genetic Diseases, Inborn - etiology
/ Genetic Diseases, Inborn - metabolism
/ Humans
/ Immune System Diseases - drug therapy
/ Immune System Diseases - etiology
/ Immune System Diseases - metabolism
/ Immunity
/ inborn errors of immunity (IEIs)
/ Jakinib
/ Janus Kinase Inhibitors - administration & dosage
/ Janus Kinase Inhibitors - adverse effects
/ Janus Kinase Inhibitors - therapeutic use
/ Kinases
/ Leukemia
/ Mutation
/ STAT 1 and STAT3 gain of function
