The common use of off-label treatments and high failure with available biologic therapies shows the need for new medications for children in the United States with juvenile idiopathic arthritis (JIA), a study using a population-based group of patients and a nationwide registry reports.
The research, “New Medications Are Needed for Children with Juvenile Idiopathic Arthritis,” was presented at the 2019 American College of Rheumatology and Association of Rheumatology Professionals (ACR/ARP) Annual Meeting, held in Atlanta.
However, many children with JIA continue to have active disease despite treatment, and are given other medications off-label. These include Kineret (anakinra, by Sobi) and Simponi (golimumab, by Janssen) — most commonly treatments approved for adults with rheumatoid arthritis, but still requiring testing in young patients with JIA.
To better understand the use of biologics in children with this disorder in the U.S., researchers analyzed two datasets: the health records of 1,599 children treated at Cincinnati Children’s Hospital Medical Center (CCHMC) since 2008 and data from 7,379 children enrolled in the Childhood Arthritis & Rheumatology Research Alliance (CARRA) registry.
Among the group from CCHMC, 40% of the patients had polyarticular JIA, 17% had juvenile psoriatic arthritis/enthesitis-related arthritis (jPSA/ERA), 16% had persistent-oligoarticular JIA, and 9% had systemic JIA.
In the registry, 46% of children had polyarticular disease, 8% systemic JIA, and 18% jPSA/ERA.
Results showed that the use of biologics was common in both datasets. These medications were used by 829 children (53%) at CCHMC, and 4,766 (65%) of those in the CARRA registry. Among these children, 304 (37%) at CCHMC and 1,122 (24%) in the registry had been treated with biologics off-label.
In addition, a substantial proportion of children still had active disease despite treatment (as assessed by three or more active joints, as well as physician- and patient-reported measures). In both datasets, approximately 5% of the children had failed treatment with five or more different biologics irrespective of disease duration.
“The approved treatment options for JIA have expanded tremendously, but there are still significant proportions of children who do not respond to available therapies or who are receiving medications that have not been approved for JIA,” Timothy Beukelman, MD, a professor at The University of Alabama and a co-investigator in the study, said in a press release.
“We must demand that newly developed medications are studied for safety and effectiveness in children,” Beukelman added.
Hermine I. Brunner, a pediatric rheumatologist at CCHMC, added: “Only if [the U.S. Food and Drug Administration] demands studies from the pharmaceutical companies as part of their drug development program will pediatric rheumatologists have valid information about the proper dosing, efficacy, and preliminary safety of new medications. Further, FDA approval greatly increases access of JIA patients to new medications.”
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