High-dose intravenous pulse methotrexate in patients with eosinophilic fasciitis

IMPORTANCE Eosinophilic fasciitis (EF) is a connective tissue disorder in which conventional treatment leads to disappointing results in a proportion of patients. Therefore, we investigated high-dose intravenous (IV) pulse methotrexate (MTX) as a treatment for EF. OBJECTIVE To examine safety and effects of monthly high-dose IV pulse MTX in EF. DESIGN, SETTING, AND PARTICIPANTS For this prospective single-Arm study,we recruited 12 patients diagnosed with biopsy specimen-proven EF between 2006 and 2009 from the Department of Dermatology and Rheumatology at the Radboud University Medical Centre. INTERVENTIONS Intravenous MTX (4mg/kg) monthly for 5 months with folinic acid rescue 24 hours after MTX administration. MAIN OUTCOMES AND MEASURES The primary outcomewas improvement of the modified skin score at month 5 vs baseline. Secondary outcomes were durometry, range of motion, visual analog scale scores for disease activity, and 36-Item Short Form Survey health questionnaires. RESULTS Overall, 12 patients (11 women between 37-69 years old) received a median (range) monthly dose of 288 (230-336)mg MTX. Median (range) modified skin score improved from 17.5 (8.0-24.0) at baseline to 8.5 (1.0-20.0) at month 5 (P = .001). Secondary outcome measures improved significantly, except for durometer scores and range of motion of the elbows. Adverse events included gastrointestinal symptoms (n = 9), mild stomatitis (n = 5), and alopecia (n = 4). CONCLUSIONS AND RELEVANCE High-dose IV pulse MTX is a safe and effective treatment option in EF.