Rilonacept maintains long-term inflammatory remission in patients with deficiency of the IL-1 receptor antagonist.

ABSTRACT:

Background

Deficiency of IL-1 receptor antagonist (DIRA) is a rare autoinflammatory disease that presents with life-threatening systemic inflammation, aseptic multifocal osteomyelitis, and pustulosis responsive to IL-1-blocking treatment. This study was performed (a) to investigate rilonacept, a long-acting IL-1 inhibitor, in maintaining anakinra-induced inflammatory remission in DIRA patients, (b) to determine doses needed to maintain remission, and (c) to evaluate the safety and pharmacokinetics of rilonacept in young children (<12 years).

Methods

Six mutation-positive DIRA patients (children, ages 3-6 years), treated with daily anakinra, were enrolled into an open-label pilot study of subcutaneous rilonacept for 24 months. Clinical symptoms and inflammatory blood paramete

SUBMITTER: Garg M 

PROVIDER: S-EPMC5621891 | biostudies-literature | 2017 Aug

REPOSITORIES: biostudies-literature