Improvement in Disease Activity in Refractory Juvenile Dermatomyositis Following Abatacept Therapy

Rodolfo V. Curiel, Division of Rheumatology, Department of Medicine, George Washington University School of Medicine and Health Sciences, Washington, DC.
William Nguyen, Division of Rheumatology, Department of Medicine, George Washington University School of Medicine and Health Sciences, Washington, DC.
Gulnara Mamyrova, Division of Rheumatology, Department of Medicine, George Washington University School of Medicine and Health Sciences, Washington, DC.
Derek Jones, Division of Rheumatology, Department of Medicine, George Washington University School of Medicine and Health Sciences, Washington, DC.
Alison Ehrlich, Department of Dermatology, George Washington University School of Medicine and Health Sciences, Washington, DC.
Kathleen A. Brindle, Department of Radiology, George Washington University School of Medicine and Health Sciences, Washington, DC.
Shahriar Haji-Momenian, Department of Radiology, George Washington University School of Medicine and Health Sciences, Washington, DC.
Robert Sheets, Division of Allergy, Immunology and Rheumatology, Department of Pediatrics, University of California San-Diego, Rady Children's Hospital, San Diego, CA.
Hanna Kim, Division of Rheumatology, Department of Medicine, George Washington University School of Medicine and Health Sciences, Washington, DC.
Olcay Y. Jones, Division of Rheumatology, Department of Medicine, George Washington University School of Medicine and Health Sciences, Washington, DC.
Lisa G. Rider, Division of Rheumatology, Department of Medicine, George Washington University School of Medicine and Health Sciences, Washington, DC.

Document Type

Journal Article

Publication Date

1-19-2023

Journal

Arthritis & rheumatology (Hoboken, N.J.)

DOI

10.1002/art.42450

Abstract

OBJECTIVE: An open-label 24-week study was conducted to evaluate the safety and efficacy of abatacept in patients with refractory juvenile dermatomyositis (JDM). METHODS: Ten patients >7 years of age with moderate disease activity were enrolled in a 24-week study to examine the safety and treatment response of subcutaneous abatacept. The primary endpoint was the International Myositis Assessment and Clinical Studies Group (IMACS) Definition of Improvement (DOI). Secondary endpoints included safety, change in core set activity measures (CSMs) of IMACS and Pediatric Rheumatology International Trials Organization (PRINTO), and the ACR-EULAR response criteria for JDM. Blinded radiologists assessed thigh magnetic resonance imaging (MRI). Interferon gene score (IFNGS) was performed on whole-blood RNA by NanoString and cytokines were assessed by Luminex. RESULTS: Five patients achieved DOI at week 12, and nine achieved DOI at week 24, including two with minimal, four moderate, and three with major improvement by ACR-EULAR response criteria using IMACS CSMs. All CSMs improved from baseline at weeks 12 and 24, except muscle enzymes. Daily corticosteroid dose decreased from a mean of 16.7 mg at baseline to 10.2 mg at week 24 (p=0.002). Average MRI muscle edema score decreased from baseline 5.3 to 2.3 at week 24 (p=0.01). Six patients had down-trending IFNGS and galectin-9 at week 24. Decreases in IFNGS, IP-10, galectin-9 and IL-2 correlated with improvement in disease activity and in MRI muscle edema. Eleven Grade 2 or 3 treatment-emergent adverse events were observed. CONCLUSIONS: This open-label study demonstrated abatacept may be beneficial for treatment-refractory JDM.

Department

Radiology

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