Abstract

Juvenile dermatomyositis (DM) is a chronic disease that, like its adult equivalent, primarily affects skin and muscles. Despite improved disease outcomes with treatment strategies used over the last few decades, it is still associated with significant morbidity and mortality. Treatment for both children and adults is based on case reports and retrospective studies since very few controlled trials, conducted in small patient populations, have been performed

There is strong clinical consensus that corticosteroids represent the first-line treatment of choice for juvenile DM. In steroid resistant or steroid dependent cases, an immunosuppressive drug is added as steroid sparing agent. The choice of the immunosuppressive agent relies mostly on the experience of the physician and varies widely between countries. Two of the most common immunosuppressors used in the treatment of juvenile DM are methotrexate and cyclosporine. It has however been suggested that a more aggressive therapy combining steroids and an immunosuppressive drug at disease onset could result in a better outcome.

Recently the Paediatric Rheumatology International Trials Organisation (PRINTO) completed a randomized clinical trial in order to establish if, in newly diagnosed juvenile DM cases, combined treatment with steroids and methotrexate or steroids and cyclosporine has a safety and an efficacy profile which is superior to steroid monotherapy. The trial showed that combined therapy with prednisone and either cyclosporine or methotrexate was more effective than prednisone alone. The safety profile and the steroid sparing effect favored the combination of prednisone + methotrexate.

The current evidence from the literature for the treatment of juvenile DM will be revised.