Pediatric Clinical Year in Review: 2023

Updates in pediatric rheumatology presented at ACR Convergence 2023

Stacy Ardoin, MD

SAN DIEGO—Too many excellent pediatric rheumatology studies to squeeze into one talk? What a good problem to have. This year at ACR Convergence 2023, Stacy Ardoin, MD, MSc, professor, Division of Rheumatology and Immunology, Nationwide Children’s Hospital and The Ohio State University College of Medicine, Columbus, presented an excellent overview of clinical updates in pediatric rheumatology from November 2022–November 2023.

In selecting studies to present, she sought to strike a balance between those that could affect patient outcomes, those that included practical information for the clinician and those that shared novel information.

Clinical Trials

Dr. Ardoin first highlighted some key clinical trials. She touched on baricitinib in juvenile idiopathic arthritis (JIA), which, in a phase 3 trial, decreased the time to disease flare compared with placebo (HR 0.241 [95% confidence interval 0.128–0.453]). As expected, treatment-emergent infections were significantly higher in the baricitinib group, but only two were classified as serious. One patient in the baricitinib group (n=82) developed a pulmonary embolism that was attributed to the drug.¹

Next, she discussed emapalumab in the treatment of patients with systemic JIA or adult-onset Still’s disease who develop macrophage activation syndrome (MAS). Emapalumab is an anti-interferon-gamma monoclonal antibody that is approved by the U.S. Food & Drug Administration (FDA) for the treatment of primary hemophagocytic lymphohistiocytosis (HLH). In this prospective study, emapalumab led to MAS remission in 13 of 14 patients at a median time of 25 days in patients whose high-dose glucocorticoids (GC) had failed to help.²

Last, she mentioned a small study that showed that abatacept led to improvement in disease activity in nine of 10 patients with refractory juvenile dermatomyositis. “This study was important to include [because] we have so few clinical trials in juvenile dermatomyositis,” Dr. Ardoin said.³

Practice Guidance: Vaccines

This year also saw some key updates to vaccine recommendations for pediatric patients with autoimmune and inflammatory rheumatic diseases from both the European Alliance of Associations for Rheumatology/Paediatric Rheumatology European Society (EULAR/PRES) and the ACR.^4,5 “EULAR/PRES really focused on expanded allowing for the use of the measles mumps rubella (MMR) booster and varicella zoster vaccine, both of which we know are live virus vaccines,” Dr. Ardoin explained. “Recent studies have shown that these vaccines can be used safely in patients taking methotrexate. Use can also be considered in those taking tumor necrosis factor-alpha inhibitors, interleukin-1 or interleukin-6 inhibitors, and low-dose glucocorticoids.”