Background: To determine the safety and efficacy of methotrexate as a steroid-sparing agent in patients with symptomatic sarcoidosis, a nonrandomized interventional study of patients with chronic sarcoidosis treated with methotrexate for at least 2 years was performed. Efficacy was assessed for all patients after 2 years of treatment. Toxicity was assessed for all patients receiving therapy for the entire time (a total of 150 patient-years).
Methods: Patients were treated in a subspecialty ambulatory clinic at a university hospital. Patients with biopsy-confirmed sarcoidosis who had persistent symptoms and who were eager to avoid or reduce corticosteroid therapy were selected for study. A total of 50 patients completed at least 2 years of methotrexate therapy. Patients were treated with oral methotrexate once a week. Dosage was adjusted based on the patient's white blood cell count. Clinical response was measured in the affected organ, including the lung (measurement of vital capacity), skin (regression of skin lesions), and central nervous system (magnetic resonance imaging). Also noted was the initial and subsequent dosage of prednisone used as therapy for sarcoidosis.
Results: Improvement in vital capacity or other affected symptomatic organ was noted in 33 of 50 treated patients. Corticosteroids were discontinued in an additional six patients who remained stable with clinical or symptomatic improvement. The major toxic effects noted in 150 patient-years of therapy were hepatic (six patients), leukopenia requiring hospitalization (one patient), and cough (one patient). Forty-one liver biopsy procedures were performed in 33 patients. Of these, six demonstrated significant changes related to methotrexate that led to drug discontinuation.
Conclusion: Methotrexate is a well-tolerated therapeutic agent with significant steroid sparing and efficacy for the treatment of chronic symptomatic sarcoidosis.