TOPLINE:

In a study involving children with juvenile localized scleroderma, mycophenolate mofetil (MMF) and methotrexate (MTX) were associated with comparable reductions in disease activity and similar flare rates, but MMF was better tolerated.

METHODOLOGY:

• Researchers conducted a retrospective cohort study of 114 patients (68% female; median age at onset, 8.3 years; 92% White) with juvenile localized scleroderma from the National Registry for Childhood Onset Scleroderma at the Pediatric Scleroderma Center at Children’s Hospital of Pittsburgh, University of Pittsburgh Medical Center, from January 2010 to January 2023.
• Linear scleroderma of the trunk and limb was the most common disease subtype (56%); 68 patients (59.6%) received MTX alone, 28 (24.6%) received MMF alone, and 18 (15.8%) received both.
• The median disease duration was 2.6 years, and the median follow-up duration was 4.3 years.
• Study outcomes included cutaneous disease activity and damage.

TAKEAWAY:

• Patients demonstrated significant decreases in disease activity across all treatment groups, with no differences in the rate of reduction in the modified Localized Scleroderma Skin Severity Index (mLoSSI) between the groups.
• At 3 months, all treatment groups achieved a clinically meaningful reduction in disease activity. By 12 months of treatment, each group achieved a median mLoSSI score of 0.
• Overall, 29 of the 114 patients (25.3%) experienced a disease flare (mLoSSI score > 0): 29.4% in the MTX group, 17.9% in the MMF group, and 22.2% in the combination treatment group. The mean time to flare was 44.7, 10.1, and 23.3 months, respectively.
• Patients treated with MTX alone vs MMF alone experienced significantly higher rates of adverse events, including nausea (60.3% vs 7.1%, P = .001), fatigue (47.1% vs 10.7%, P = .001), anxiety (29.4% vs 7.1%; P = .02), anticipatory vomiting (17.7% vs 0%; P = .02), and alopecia (14.7% vs 0%; P = .03). Rates among those on combination therapy were 55.6%, 22.2%, 5.6%, 11.1%, and 0%, respectively.

IN PRACTICE:

“MMF demonstrated comparable clinical response with MTX in reducing disease activity and maintaining remission, with fewer associated adverse effects,” the authors wrote, noting that to their knowledge, the study included the largest cohort of patients with localized scleroderma “treated de novo with MMF to date.” The results, they added, “are promising and underscore the need for future trials to further inform evidence-based guidelines and optimize treatment strategies for this rare but debilitating pediatric disease.”

SOURCE:

The study was led by Elena C. de Rosas, University of Pittsburgh, and was published online on January 28 in JAMA Dermatology.

LIMITATIONS:

This study was limited by retrospective, nonrandomized design, and unequal group sizes. Adverse effects were captured through clinical interviews rather than validated patient-reported outcome measures.

DISCLOSURES:

The study was supported in part by the National Institutes of Health (NIH), the Nancy Taylor Foundation for Chronic Diseases, and the Natali Foundation. One author reported receiving grants from NIH, the US FDA, Lucile Packard Foundation, and the Childhood Arthritis and Rheumatology Research Alliance, and consulting fees from Emmes/Casimir outside of the submitted work.

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