TOPLINE:

Intermittent intravenous methylprednisolone pulse (IVMP) and mycophenolate mofetil (MMF) led to a complete clinical response in 93% of patients with juvenile dermatomyositis (JDM), with manageable adverse effects over a median of 61 months.

METHODOLOGY:

• Researchers analyzed 28 patients (median age, 7.0 years; 54% female) with JDM who met the 2006 Bohan and Peter criteria or the 2017 European League Against Rheumatism/American College of Rheumatology classification criteria, with at least 4 years of follow-up from 2014 to 2025, at the Children’s Medical Center in Changchun, China.
• Patients received seven to nine courses of IVMP pulses (20-30 mg/kg/d for 3 consecutive days per course) with MMF (500-600 mg/m² twice daily), a regimen used as first-line therapy for over a decade at the center.
• Hydroxychloroquine was added for patients with high skin activity (Disease Activity Score [DAS] ≥ 5), and intravenous (IV) immunoglobulin was added for poor muscle strength, rash, or severe initial symptoms. The median follow-up was 61 months.
• The study outcomes included Childhood Myositis Assessment Scale (CMAS) scores, DAS, height and BMI z-scores, bone mineral density (BMD) z-scores, laboratory parameters, and adverse events.

TAKEAWAY:

• With treatment, CMAS scores increased by 21.8 points in 12 months, and the total DAS decreased by 10.3 points from baseline (P < .001 for both).
• Of the 28 patients, 26 (93%) achieved a complete clinical response with a median time to response of 10 months and a median time to clinical remission of 35.5 months; five (18%) patients relapsed, and none developed calcinosis or interstitial lung disease.
• Height z-scores reached their lowest point at 24 months (0.7 points lower than the baseline), while BMD z-scores decreased significantly from 0.6 at baseline to -0.8 at a median of 48.5 months (P < .001 for both). Triglyceride levels dropped by 63.7 mg/dL in 12 months (P < .001), and muscle enzyme levels normalized within 3 months.
• The most common adverse events included transient steroid-induced ocular hypertension (six patients), including four who had transient glaucoma, and cataracts (one patient); no patient experienced osteonecrosis or fractures.

IN PRACTICE:

“This study suggests that the regimen, especially the use of MMF, is a therapeutic option for JDM,” the study authors wrote, noting that the adverse effects of treatment were “mild and manageable.”

SOURCE:

The study was led by Lishuang Guo, MM, Department of Pediatric Rheumatology, Immunology, & Allergy, Children’s Medical Center, the First Hospital of Jilin University, Changchun, China, and was published online on November 19 in JAMA Dermatology.

LIMITATIONS:

The study was limited by its retrospective design, absence of a control group, and small sample size. Additionally, the concurrent use of intravenous immunoglobulin and hydroxychloroquine could interfere with the interpretation of the treatment responses.

DISCLOSURES:

The study was funded by a grant from the Clinical Research Project of the First Hospital of Jilin University and the Department of Science and Technology of Jilin Province. The authors declared having no conflicts of interest.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.