JAK1/2 inhibition with baricitinib in the treatment of autoinflammatory interferonopathies.

ABSTRACT:

Background

Monogenic IFN-mediated autoinflammatory diseases present in infancy with systemic inflammation, an IFN response gene signature, inflammatory organ damage, and high mortality. We used the JAK inhibitor baricitinib, with IFN-blocking activity in vitro, to ameliorate disease.

Methods

Between October 2011 and February 2017, 10 patients with CANDLE (chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperatures), 4 patients with SAVI (stimulator of IFN genes-associated [STING-associated] vasculopathy with onset in infancy), and 4 patients with other interferonopathies were enrolled in an expanded access program. The patients underwent dose escalation, and the benefit was assessed by reductions in daily disease symptoms and corticosteroid requirem

SUBMITTER: Sanchez GAM 

PROVIDER: S-EPMC6026004 | biostudies-literature | 2018 Jul

REPOSITORIES: biostudies-literature