Tocilizumab Shown to Be Safe and Effective as Novel Treatment for Children with Juvenile Idiopathic Arthritis
Children with polyarticular juvenile idiopathic arthritis (JIA) can now be treated safely and effectively with tocilizumab, an interleukin-6 receptor inhibitor drug, according to a 58-center global study coordinated and co-designed at Cincinnati Children’s.
An extended report on the results of the Phase III CHERISH study was first published online May 16, 2014, in the Annals of the Rheumatic Diseases. The U.S. Food and Drug Administration (FDA) has used positive results of this randomized clinical trial to expand the indication for tocilizumab, which had previously been approved to treat moderate to severe active rheumatoid arthritis and systemic JIA.
Tocilizumab is the first new treatment for polyarticular JIA in five years and significantly enhances options for treating joint pain and swelling in children with JIA.
Hermine Brunner, MD, MSc, MBA, director of the Division of Rheumatology at Cincinnati Children’s and scientific director of the multinational Pediatric Rheumatology Collaborative Study Group (PRCSG), led the study. Daniel Lovell, MD, MPH, chairman of the PRCSG and clinical director of Rheumatology, also participated in study design and coordination.
The CHERISH study showed that 89 percent of study participants treated with tocilizumab markedly improved within 16 weeks. The drug also significantly decreased the number of JIA exacerbations or flares.
The study enrolled 188 patients with active polyarticular JIA, ages 2 to 17 years. All received monthly infusions of tocilizumab for 16 weeks. Those whose symptoms improved by week 16 were entered into a 24-week, double-blind phase of the study in which half of the patients received a placebo and half continued receiving the drug.
Arthritis symptoms flared in 48 percent of placebo patients versus 26 percent in the tocilizumab group. Side effects, primarily upper respiratory infections, occurred in 8.5 percent of participants.
“The overall pediatric safety profile of tocilizumab was consistent with that seen in adults with rheumatoid arthritis,” Brunner says. “Most importantly, tocilizumab provides sustained and clinically meaningful improvement of polyarticular JIA.”
