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Mycophenolate Mofetil in severe or methotrexate refractory localized scleroderma

Juvenile Localized Scleroderma (JLS), is characterised by presence of areas of skin thickening, which is relatively benign, but if deeper tissues such as muscle and bone are involved severe deformities may develop. Mycophenolate Mofetil (MMF) is increasingly utilised both for treatment of systemic sclerosis and immune-mediated skin diseases such as psoriasis, graft-versus-host and lichen planus. Our aim was to evaluate efficacy and safety of MMF in the treatment of JLS. Seven patients entered the study (3 M, 4 F). The JLS clinical subtypes were Pansclerotic morphea (2 pts), Generalized Morphea (1), En coup de Sabre (ECDS) (1), Mixed (ECDS/Linear) (3), The age at onset of the disease was 7.5 yrs (range 3 – 16.9) and the disease duration at diagnosis was 1.9 years (range 3 months – 4 years). Previous treatments before starting MMF were oral steroids in 6 pts, IV steroids in 5, MTX in 5, while the patient with ECDS received no treatment. MMF was started because of steroids side effects in one patient and MTX resistance in 5. In ECDS MMF was chosen because of concomitant cerebral vasculitis. All treated patients presented clinical improvement which allowed withdrawal of steroids. Over the follow-up of 27 months (range 6–36 months) only mild abdominal discomfort in one patient was reported. In conclusion MMF seems to be effective in severe or MTX-refractory JLS and is generally well tolerated. Further controlled studies are necessary to confirm these data.

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Open Access This article is published under license to BioMed Central Ltd. This is an Open Access article is distributed under the terms of the Creative Commons Attribution 2.0 International License (, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Martini, G., Falcini, F., Girschick, H. et al. Mycophenolate Mofetil in severe or methotrexate refractory localized scleroderma. Pediatr Rheumatol 6 (Suppl 1), P229 (2008).

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