Familial autoinflammation with neutrophilic dermatosis reveals a regulatory mechanism of pyrin activation

SL Masters, V Lagou, I Jéru, PJ Baker… - Science translational …, 2016 - science.org
SL Masters, V Lagou, I Jéru, PJ Baker, L Van Eyck, DA Parry, D Lawless, D De Nardo
Science translational medicine, 2016science.org
Pyrin responds to pathogen signals and loss of cellular homeostasis by forming an
inflammasome complex that drives the cleavage and secretion of interleukin-1β (IL-1β).
Mutations in the B30. 2/SPRY domain cause pathogen-independent activation of pyrin and
are responsible for the autoinflammatory disease familial Mediterranean fever (FMF). We
studied a family with a dominantly inherited autoinflammatory disease, distinct from FMF,
characterized by childhood-onset recurrent episodes of neutrophilic dermatosis, fever …
Pyrin responds to pathogen signals and loss of cellular homeostasis by forming an inflammasome complex that drives the cleavage and secretion of interleukin-1β (IL-1β). Mutations in the B30.2/SPRY domain cause pathogen-independent activation of pyrin and are responsible for the autoinflammatory disease familial Mediterranean fever (FMF). We studied a family with a dominantly inherited autoinflammatory disease, distinct from FMF, characterized by childhood-onset recurrent episodes of neutrophilic dermatosis, fever, elevated acute-phase reactants, arthralgia, and myalgia/myositis. The disease was caused by a mutation in MEFV, the gene encoding pyrin (S242R). The mutation results in the loss of a 14-3-3 binding motif at phosphorylated S242, which was not perturbed by FMF mutations in the B30.2/SPRY domain. However, loss of both S242 phosphorylation and 14-3-3 binding was observed for bacterial effectors that activate the pyrin inflammasome, such as Clostridium difficile toxin B (TcdB). The S242R mutation thus recapitulated the effect of pathogen sensing, triggering inflammasome activation and IL-1β production. Successful therapy targeting IL-1β has been initiated in one patient, resolving pyrin-associated autoinflammation with neutrophilic dermatosis. This disease provides evidence that a guard-like mechanism of pyrin regulation, originally identified for Nod-like receptors in plant innate immunity, also exists in humans.
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