title: Methotrexate treatment in juvenile localized scleroderma: a randomized, double-blind, placebo-controlled trial.
abstract: OBJECTIVE
  Juvenile localized scleroderma is a chronic progressive fibrotic disorder of the skin that causes permanent disability and aesthetic damage. This study was undertaken to assess the safety and efficacy of methotrexate (MTX) in the treatment of juvenile localized scleroderma.
  
  
  METHODS
  In this double-blind study, patients with active juvenile localized scleroderma were randomized (2:1) to receive oral MTX (15 mg/m², maximum 20 mg) or placebo once weekly, for 12 months or until treatment failure. Both groups received oral prednisone (1 mg/kg/day, maximum 50 mg) for the first 3 months. A target lesion was evaluated clinically, with infrared thermography and using a computerized scoring system with skin score rate (SSR) evaluation. Response to treatment was defined as the absence of new lesions, SSR ≤ 1, and a decrease in lesion temperature of at least 10% compared to baseline. Treatment failure was defined as the occurrence of new lesions, SSR > 1, or increased lesion temperature. All analyses were done on the intent-to-treat population.
  
  
  RESULTS
  Of the 85 patients screened, 70 (ages 6-17 years) were randomized (46 to the MTX group, 24 to the placebo group). The mean disease duration was 2.3 years. After an initial response in all patients, disease relapsed in 15 MTX-treated patients (32.6%) and 17 placebo-treated patients (70.8%) (P < 0.005). New lesions appeared in 3 MTX-treated patients (6.5%) versus 4 placebo-treated patients (16.7%). The mean SSR decreased from 1 to 0.79 in the MTX group and increased from 1 to 1.1 in the placebo group, and the mean target lesion temperature decreased by 44.4% in the MTX group versus 12.1% in the placebo group. Twenty-six patients in the MTX group (56.5%) and 11 patients in the placebo group (45.8%) developed mild side effects related to treatment. None of the side effects were severe enough to necessitate treatment discontinuation.
  
  
  CONCLUSIONS
  Our findings indicate that MTX is efficacious in the treatment of juvenile localized scleroderma and is well tolerated.
authors.affiliation: University of Padua, Padua, Italy. zulian@pediatria.unipd.it
authors.forename: Francesco
authors.initials: F
authors.lastname: Zulian
authors.forename: Giorgia
authors.initials: G
authors.lastname: Martini
authors.forename: Cristina
authors.initials: C
authors.lastname: Vallongo
authors.forename: Fabio
authors.initials: F
authors.lastname: Vittadello
authors.forename: Fernanda
authors.initials: F
authors.lastname: Falcini
authors.forename: Annalisa
authors.initials: A
authors.lastname: Patrizi
authors.forename: Maria
authors.initials: M
authors.lastname: Alessio
authors.forename: Francesco
authors.initials: F
authors.lastname: La Torre
authors.forename: Rosa A
authors.initials: RA
authors.lastname: Podda
authors.forename: Valeria
authors.initials: V
authors.lastname: Gerloni
authors.forename: Mario
authors.initials: M
authors.lastname: Cutrone
authors.forename: Anna
authors.initials: A
authors.lastname: Belloni-Fortina
authors.forename: Mauro
authors.initials: M
authors.lastname: Paradisi
authors.forename: Silvana
authors.initials: S
authors.lastname: Martino
authors.forename: Giorgio
authors.initials: G
authors.lastname: Perilongo
mesh_terms: D000293:Adolescent; D018501:Antirheumatic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D003879:Dermatologic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D004311:Double-Blind Method; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D057194:Intention to Treat Analysis; D053208:Kaplan-Meier Estimate; D008297:Male; D008727:Methotrexate / Q000009:adverse effects / Q000627:therapeutic use*; D011446:Prospective Studies; D012594:Scleroderma, Localized / Q000188:drug therapy*; D012720:Severity of Illness Index; D016896:Treatment Outcome
chemical_list: D018501:Antirheumatic Agents; D003879:Dermatologic Agents; D008727:Methotrexate