Arthritis & Rheumatism, Volume 62,
November 2010 Abstract Supplement
Abstracts of the American College of
Rheumatology/Association of Rheumatology Health Professionals
Annual Scientific Meeting
Atlanta, Georgia November 6-11, 2010.
Methotrexate in Children with Juvenile Localized Scleroderma: A Randomized, Double-Blind, Placebo-Controlled Trial.
Zulian7, Francesco, Vallongo8, Cristina, Martini8, Giorgia, Falcini5, Fernanda, Patrizi4, Annalisa, Alessio3, Maria, Torre6, Francesco La
Children's Hospital of Mestre
Dermatology, IDI IRCCS, Rome
University Federico II Naples
University of Bologna
University of Florence, Florence, Italy
University of Messina
University of Padua, Padua, Italy
University of Padua
University of Turin
Juvenile localized scleroderma (JLS) is a chronic progressive fibrotic process of the skin causing permanent disability and aesthetic damage. Although no universally accepted effective treatment is available, recent studies seem to support the use of methotrexate (MTX).
We aimed to assess the safety and efficacy of methotrexate in patients with JLS.
We performed a double-blind, randomised controlled trial. Patients with active JLS, with linear, generalized or deep subtypes, were randomly assigned to receive oral MTX, at a dose of 15 mg/m2 once a week (max 20 mg), for 12 months or until flare of the disease (MTX arm), or placebo at the same dose and timing (placebo arm). Oral prednisone (1 mg/Kg/day, max 50 mg), in a single morning dose for 3 months then tapered down until stopping in one month, was added to both groups. The randomization rate MTX/PLAC was 2:1. The extension of the skin lesions was evaluated by a computerized scoring system1 and changes were quantified by the skin score rate (SSR) (skin score at timen/skin score at time0). Clinical examination and serial thermographies monitored the changes of active lesions2. The primary endpoint was the rate of response to treatment. Responders were defined those patients who satisfied the following 3 criteria: SSR<=1; decrease of the temperature at thermography of at least 10% compared to baseline; absence of new lesions. Disease relapse was defined when was present at least one of the following: SSR>1; unchanged or increased lesion temperature; appearance of new lesions. All analyses were intention-to-treat.
85 patients entered the screening phase and 70, aged 617 years, from 13 centres in Italy, were enrolled. 46 patients were randomized in the MTX arm and 24 in the placebo arm. Groups were homogeneous as far as clinical and immunological features, mean disease duration was 2,1 years in both groups. After an initial response in all patients, disease relapse occurred in 15 MTX patients (32,6%) and 17 placebo patients (70,8%) (p<0.005), mean SSR value decreased from 1 to 0.79 with MTX vs 1,1 with placebo. The mean target lesion temperature decreased 44% vs 12.1%. New lesions appeared in 3 MTX patients (6.5%) vs 4 on placebo (16.7%). 26 patients (56,5%) of MTX group and 11 patients of the placebo group (45,8%) presented mild side effects related to treatment. None was severe enough to stop treatment.
MTX is an effective and well tolerated treatment for patients with JLS.
To cite this abstract, please use the following information:
Zulian, Francesco, Vallongo, Cristina, Martini, Giorgia, Falcini, Fernanda, Patrizi, Annalisa, Alessio, Maria, et al; Methotrexate in Children with Juvenile Localized Scleroderma: A Randomized, Double-Blind, Placebo-Controlled Trial. [abstract]. Arthritis Rheum 2010;62 Suppl 10 :1439