Long-term safety and efficacy of rilonacept in patients with systemic juvenile idiopathic arthritis.

Abstract

OBJECTIVE:

To determine the long-term safety and efficacy of rilonacept, an anti-interleukin-1 fusion protein, in patients with active systemic juvenile idiopathic arthritis (JIA).

METHODS:

In patients with systemic JIA, ages 4-20 years, the efficacy of rilonacept was evaluated using 30%, 50%, and 70% levels of improvement according to the adapted American College of Rheumatology (ACR) Pediatric 30, 50, and 70 response criteria, respectively. Efficacy and safety were evaluated during 23 months of open-label treatment (3 phases) after a 4-week, double-blind, placebo-controlled phase. Following double-blind treatment with 2.2 mg/kg or 4.4 mg/kg of rilonacept, patients were eligible to receive open-label treatment at their prior dose, with adjustments. Reductions in the median daily dose of oral prednisone and improvements in laboratory parameters of disease activity (i.e., decreased levels of D-dimer and myeloid-related proteins [MRPs]) were also evaluated.

RESULTS:

Twenty-four patients entered the double-blind study and 23 entered the open-label period. Patients were predominantly white and female, and had a median age of 14.0 years at baseline. No significant differences in efficacy were observed between the rilonacept- and placebo-treated patients during the double-blind phase, but fever and rash completely resolved by month 3 in all patients during the open-label treatment period and did not recur. Adapted ACR Pediatric 30, 50, and 70 response rates at 3 months from the start of the study were 78.3%, 60.9%, and 34.8%, respectively; these responses were generally maintained over the study duration. Levels of D-dimer and MRP-8/MRP-14 dramatically improved during the study, and in 22 of 23 patients, the prednisone dose was decreased or prednisone therapy was discontinued. No serious treatment-related adverse events were observed.

CONCLUSION:

Sustained improvements in clinical and laboratory measures of the articular and systemic manifestations of systemic JIA were achieved in >50% of rilonacept-treated patients over 2 years. Treatment with rilonacept had a substantial steroid-sparing effect and was generally well-tolerated.

• Authors: Daniel J Lovell, Edward H Giannini, Andreas O Reiff, Yukiko Kimura, Suzanne Li, Philip J Hashkes, Carol A Wallace, Karen B Onel, Dirk Foell, Richard Wu, Stephanie Biedermann, Jennifer D Hamilton, Allen R Radin
• Journal: Arthritis and rheumatism (Arthritis Rheum) Vol. 65 Issue 9 Pg. 2486-96 (Sep 2013) ISSN: 1529-0131 [Electronic] United States
• PMID: 23754188 (Publication Type: Clinical Trial, Clinical Trial, Phase II, Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2013 by the American College of Rheumatology.

Chemical References

• Antirheumatic Agents
• Recombinant Fusion Proteins
• rilonacept

Topics

• Adolescent
• Antirheumatic Agents (adverse effects, therapeutic use)
• Arthritis, Juvenile (drug therapy)
• Child
• Child, Preschool
• Double-Blind Method
• Female
• Humans
• Male
• Recombinant Fusion Proteins (adverse effects, therapeutic use)
• Treatment Outcome
• Young Adult