Juvenile RA/Pediatric Rheumatology
Sangeeta Sule, M.D.
Abstract 718: Preliminary Evidence for Bioactivity of IL-1 Trap (Rilonacept), A Long Acting IL-1 Inhibitor, in Systemic Juvenile Idiopathic Arthritis (SJIA)
Authors: Daniel J. Lovell1, Edward H. Giannini1, Yukiko Kimura2, Suzanne Li2, Philip J. Hashkes3, Andreas O. Reiff4, Carol A. Wallace, Dr.5, Karen B. Onel6, Douglas R. Nadler7, Jeffrey R. Rosenberg8, K. Gregory Osgood8, Joanne H. Papadopoulos8, Allen R. Radin8. 1Children's Hospital Medical Center, Cincinnati, OH; 2Hackensack University Medical Center, Hackensack, NJ; 3Cleveland Clinic Foundation, Cleveland, OH; 4Children's Hospital of LA, Los Angeles, CA; 5Children's Hospital and Regional Medical Center, Seattle, WA; 6La Rabida Children's Hospital, Chicago, IL; 7Regeneron Pharmaceuticals, Inc, Tarrytown, NY; 8Regeneron Pharmaceuticals, Inc., Tarrytown, NY
Purpose: Obtain preliminary data from an ongoing double-blind placebo-controlled study of IL-1 Trap in SJIA
Methods: 11 children aged 5-18 years old were randomized to receive either 2.2 mg/kg IL- 1 Trap or placebo as subcutaneous injections once a week for 4 weeks as part of the double-blind phase of the study. Patients were eligible for an open-label phase after 4 weeks in the double-blind phase or a minimum of 2 weeks if rescue in double-blind was clinically indicated.
Results:
Core Set Measures and Additional Variables |
Day 0, pre-DB Baseline mean n=11 |
Wk 2,
OL median % change from baseline n=9 |
Wk 4, OL median % change from baseline n=9 |
| Phys VAS (0-10) | 5.7 | -62.5% | -80.0% |
| Par VAS (0-10) | 5.5 | -47.3% | -68.6% |
| No. of active joints | 17.5 | -42.9% | --57.1% |
| No. of joints with limited motion | 15.1 | -38.5% | -61.5% |
| CHAQ (0-3) | 1.4 | -48.4% | -78.1% |
|
hs-CRP (mg/L) nl< 8.4 mg/L |
92.4 | -95.4% | -92.2% |
| WBC (K/mm3) | 13.8 | -34.4% | -20.6% |
| D-dimer (ng/mL) | 3888.2 | -76.6% | -71.4% |
| Hb (g/dL) | 11.1 | +6.8% | +5.4% |
| Platelet (K/mm3) | 577.2 | -6.5% | -20.2% |
|
Fever and/or rash % of n reported |
100 | 0 | 0 |
| ACR Ped 30 n (%) | 0 | 7 (77.8%) | 7 (77.8%) |
| ACR Ped 50 n (%) | 0 | 5 (55.6%) | 7 (77.8%) |
| ACR Ped 70 n (%) | 0 | 2 (22.2%) | 4 (44.4%) |
| ACR Ped 30, 50 and 70 are cumulative | |||
Conclusions: Improvement was noted in 6 of the ACR Pediatric core set variables after treatment with IL-1 Trap for 2 weeks and 4 weeks. Fever and/or rash in all subjects resolved. Preliminary evidence indicates that IL-1 Trap may be beneficial in children with systemic onset JIA.
Editorial Comments: This study reports preliminary data from an ongoing double-blind study of IL-1 Trap in the treatment of systemic onset JIA. Anakinra, an IL-1 antagonist, has also been shown to be effective in systemic onset JIA. However, with the IL-1 Trap, injections are given once a week, as opposed to anakinra, a daily injection. This is an important difference, particularly in the pediatric population. It is impressive that the fever and rash completely resolved in those treated. However, these results are very preliminary and treatment was conducted over a very short time. The long-term safety effects of IL-suppression in children need further evaluation.