Arthritis & Rheumatism, Volume 63,
November 2011 Abstract Supplement
Abstracts of the American College of
Rheumatology/Association of Rheumatology Health Professionals
Annual Scientific Meeting
Chicago, Illinois November 4-9, 2011.
Long-Term Methotrexate Efficacy in Juvenile Localized Scleroderma.
Zulian1, Francesco, Vallongo1, Cristina, Vittadello1, Fabio, Patrizi2, Annalisa, Alessio3, Maria, Fortina1, Anna Belloni, Martino4, Silvana
Recent studies reported that methotrexate (MTX), appears beneficial in juvenile localized scleroderma (JLS) but little is known about its long-term efficacy. We assessed the long-term efficacy of MTX in a cohort of patients with JLS.
We prospectively followed a cohort of patients with JLS who were enrolled in a double-blind, randomized controlled trial1. Oral MTX was used at a dose of 15 mg/m2 once a week (max 20 mg) for at least 24 months; prednisone (1 mg/Kg/day, max 50 mg), as bridging therapy for 3 months was added. A target lesion was evaluated clinically, with infrared thermography and using a computerized scoring system with skin score rate (SSR) evaluation2. Response to treatment was defined as: no new lesions; SSR<1; decrease lesion temperature by at least 10% compared to baseline. Treatment failure was defined by appearance of new lesions, SSR>1, or increased lesion temperature. As far as outcome, partial remission (PR) was defined when the state of responder was maintained ON treatment for at least 6 months, complete remission (CR) when the state of responder was maintained OFF treatment for at least 6 months.
65 patients have been treated with MTX during the open-label phase of the study. Seven patients were lost to follow-up. Of the remaining 58 patients, after a mean follow-up of 43 months (median 36; range 672 months), 48 (82.8%) were responders, 10 (17.2%) relapsed by 24 months since MTX start. Among the responders, 35 (60.4%), after a 27.5 months of MTX treatment for (median 24, range 1830) maintained CR for 25 months after stopping treatment (median 24, range 648), none relapsed. 13 patients (22.4%), after a mean follow-up of 20.5 months (median 15.5, range 645), were in PR. Among the ten MTX-refractory patients, 4 had linear subtype (2 limb, 2 ECDS), 4 had mixed subtype (2 linear-circumscribed, 2 linear-pansclerotic) and 2 generalized morphea. MTX-related side-effects were seen in 28 patients (48.3%), were generally mild and rarely required treatment discontinuation.
Long-term methotrexate therapy is a safe and effective treatment for JLS. Three months of combined MTX and glucocorticoid bridging therapy and MTX duration of treatment longer than 24 months ensure prolonged disease remission.
To cite this abstract, please use the following information:
Zulian, Francesco, Vallongo, Cristina, Vittadello, Fabio, Patrizi, Annalisa, Alessio, Maria, Fortina, Anna Belloni, et al; Long-Term Methotrexate Efficacy in Juvenile Localized Scleroderma. [abstract]. Arthritis Rheum 2011;63 Suppl 10 :2018