MMF-Based Regimen Shows Potential as Treatment Option for juvenile dermatomyositis: JAMA

A new study published in the Journal of the American Medical Association showed that for juvenile dermatomyositis (JDM), a combination of intermittent intravenous methylprednisolone pulse and mycophenolate mofetil (MMF) may be beneficial.

For individuals with JDM, oral glucocorticoids (GCs) combined with immunosuppressants continue to be the first line of therapy. Intermittent intravenous methylprednisolone pulse (IVMP) treatment has been utilized in autoimmune illnesses, such as JDM, and may have benefits versus daily high-dose oral GCs. For JDM, mycophenolate mofetil (MMF) is a substitute for methotrexate. This center has been using intermittent IVMP with MMF as first-line treatment for more than 10 years.

This study assessed 28 JDM patients who satisfied the EULAR/ACR (2017) or Bohan and Peter (2006) criteria and had at least 4 years of follow-up. Each got 7 to 9 IVMP treatments (20–30 mg/kg/day for 3 days each) in addition to MMF (500–600 mg/m² twice day). For greater skin disease activity scores (DASs) (≥5), hydroxychloroquine was administered, and IVIG was given for severe early symptoms, prolonged rash, or insufficient muscle improvement.

Every patient got vitamin D and calcium every day. There was no usage of biologics or other immunosuppressants. DAS, Childhood Myositis Assessment Scale (CMAS), bone mineral density (BMD) z scores, growth metrics, laboratory results, and adverse events were among the clinical outcomes. Wilcoxon tests, Generalized estimating equations models, and imputation for missing data were used in analyses that adhered to STROBE criteria.

Over a median of 61 months, none of the 28 children (median diagnosis age: 7 years) were lost to follow-up. The average dose of prednisone was 7.5 mg; all drugs were terminated by 29.5 months, and glucocorticoids by 23.5 months. Within 3 months, muscle enzyme levels returned to normal, DAS scores decreased, and CMAS considerably improved. 18% of patients relapsed, whereas the majority (93%) experienced full clinical response and remission.

There were no fatalities, ICU hospitalizations, calcinosis, or ILD. There were few serious infections. During treatment, height and BMD z scores declined. Triglycerides significantly decreased. A few individuals experienced temporary steroid-related ocular side effects, but there were no reports of osteonecrosis or fractures. Overall, the findings of this study suggest that the usage of MMF can be used to treat JDM.

Reference:

Guo, L., Liu, J., Xin, M., Yu, G., Zhu, C., Zhao, X., & Yang, S. (2025). Intermittent glucocorticoid pulse combined with mycophenolate mofetil in juvenile dermatomyositis. JAMA Dermatology (Chicago, Ill.). https://doi.org/10.1001/jamadermatol.2025.4483