A Prospective Observational Study Comparing the Non-inferiority of Mycophenolate Mofetil to Methotrexate for the Treatment of Juvenile Localized Scleroderma

Project Period:

Grant Category:
Small Grant

Disease Area:
Scleroderma, SVARD

Juvenile Localized Scleroderma (jLS) is an inflammatory disease affecting the skin and other organs of children, caused by an impaired function of their immune system. It can be associated with body disfigurement and disability due to growth disturbance. Standard treatment for jLS includes the use of medications that control the excessive inflammation to prevent irreversible damage. The most widely used treatment regimens include methotrexate (MTX), either alone or in combination with corticosteroids. While many respond to this regimen, at least 25% of patients do not tolerate or fail to respond to MTX. A few studies have showed improvement of disease in patients with severe LS that were treated with mycophenolate mofetil (MMF). However, the evidence for its use is limited. In this study, we intend to investigate how effective MMF is in treating jLS as compared to MTX, with the goal to improve the outcome of these patients.


Dawn Wahezi, MD, MS

The Children's Hospital at Montefiore