Continuing with the topic of therapeutics in JIA, Dr. Becker discussed a phase 3b/4, open-label, randomized study on tapering canakinumab monotherapy in children with systemic JIA in clinical remission. In this international study, investigators looked at two different methods for tapering canakinumab. Patients were randomized into either a dose-reduction arm or a dose-interval prolongation arm. On both schedules, around 40% of children maintained complete remission, but only about one-third of all patients were able to completely discontinue canakinumab.11
Therapeutic Innovations
For her final theme, Dr. Becker discussed several innovative studies that are moving the field of pediatric rheumatology forward. She highlighted a study by Singh et al., that sought to examine the exposure-response relationships for biologic agents in rheumatoid arthritis and JIA. By looking at nine adult rheumatoid arthritis trials and four JIA trials, investigators found that in pediatric patients, drug exposures were generally comparable to those of adult patients. Further, for polyarticular JIA, response rates were just as favorable as adult responses.
These analyses, she explained, bridge a gap in clinical pharmacology and may allow the extrapolation of efficacy from adults with rheumatoid arthritis to children with JIA for medications with an established mechanism of action.12
The next innovative study she highlighted was a direct-to-family trial called iPERSONAL, in which pediatric lupus patients have access to an electronic pill bottle reminder to promote medication adherence. Impressively, this trial met enrollment within 10 days of opening and has a backup list of patients interested in participating.
This paper, by Randell et al., discusses how the researchers have implemented the study, including their use of in-home visits, how they navigated the direct-to-patient implementation and other lessons they learned during the trial. Dr. Becker applauded their unique and innovative approach, explaining that innovative strategies help advance the field.13
For her last three studies, Dr. Becker reviewed new therapies for children with rheumatic disease. Li et al. provided preliminary evidence on the safety and efficacy of abatacept in children with refractory juvenile localized scleroderma.14
A report by Zulian and colleagues discussed the researchers’ positive experience with rituximab for children with rapidly progressive systemic sclerosis involving the heart and/or lung.15
Dr. Becker concluded by highlighting a study by Li et al. showing significant and rapid improvement in global disease, skin manifestations, muscle strength and magnetic resonance imaging findings in four children with refractory juvenile dermatomyositis when treated with baricitinib, a Janus kinase inhibitor.16
