The modified antibody abatacept showed promise in controlling disease activity in a small study of 10 patients aged 7 years or older with refractory juvenile dermatomyositis. The primary endpoint of the 24-week trial was the International Myositis Assessment and Clinical Studies Group Definition of Improvement (DOI); at week 12, five patients had achieved DOI, and at the end of the study (week 24), nearly all patients—nine total—had achieved DOI. Corticosteroid use among the patient cohort decreased throughout the trial, and improvements were noted in secondary endpoints such as muscle strength, physical function, endurance, and rash, as well as in the amount of muscle inflammation on magnetic resonance imaging (MRI). These findings were published by Curiel et al in Arthritis & Rheumatology. In a companion press release on the findings from The George Washington University, senior study author Lisa G. Rider, MD, Head of the Environmental Autoimmunity Group at the National Institute of Environmental Health Sciences and Clinical Professor of Medicine at George Washington University School of Medicine & Health Sciences, also noted: “The majority of these treatment-refractory patients improved at least moderately in clinical disease activity measures, as well as in two objective blinded measures, interferon genes and protein markers, reflecting downregulation of a key cytokine signaling pathway in juvenile dermatomyositis…. These data strengthen the trial findings and promise of abatacept for juvenile dermatomyositis, although further studies are needed.”
May 01, 2023