Methotrexate and corticosteroids in the treatment of localized scleroderma: a standardized prospective longitudinal single-center study.

OBJECTIVE: To evaluate the effectiveness of a uniform single-center treatment protocol composed of high-dose methotrexate (MTX) and oral corticosteroids in a pediatric localized scleroderma (LS) cohort.
METHODS: Thirty-six patients with LS were recruited. Patients with active disease, defined as erythematous lesions and/or new lesions, or expansion of existing lesions, were started on oral prednisone 2 mg/kg/day (maximum 60 mg/day) and subcutaneous (SC) MTX at 1 mg/kg/week (maximum 25 mg/week). Prednisone was tapered and kept at 0.25 mg/kg/day for 12 months. MTX SC was continued for 24 months, and then switched to oral administration to complete 36 months of therapy. Modified LS Skin Severity Index (mLoSSI) and the physician global assessment of disease activity (PGA-A) were used as outcome measures.
RESULTS: Twenty-five patients with LS were female with a median age at onset of 7.86 years [interquartile range (IQR) 4.63-11.91]. Median disease duration from onset until start of this treatment regimen was 19.2 months (IQR 8.96-35.35). Median duration of followup was 36.40 months (IQR 29.39-45.36). All patients demonstrated significant improvement in mLoSSI at median 1.77 months (IQR 0.76-2.37, 95% CI 1.54, 2.01). PGA-A followed the same trend. No significant adverse reactions or flares were observed during therapy.
CONCLUSION: This single-center LS treatment protocol was effective and well tolerated. Clinical outcome in LS is affected by dose and route of administration of immunosuppressive regimens. Daily tapering dose of corticosteroids and parenteral MTX were effective in controlling LS activity without significant adverse reaction. This regimen should be considered as one of the therapies for LS clinical trials.