ORAL METHOTREXATE AND ALTERNATE-DAY PREDNISONE FOR LOW-RISK LANGERHANS CELL HISTIOCYTOSIS

Many treatments for low-risk Langerhans cell histiocytosis (LCH) invol ve unpleasant side-effects or risks of late effects. To provide treatm ent with minimal toxicity and no known risk of late effects, we have u sed oral alternate-day prednisone (PDN, 40 mg/sq.m./day) and weekly me thotrexate (MTX, 20 mg/sq.m. once weekly) in a series of 13 children w ith 17 episodes of LCH. Patients were monitored with monthly physical examinations, blood counts and chemistry panels, and radiographs and s cans obtained at the treating physician's discretion. Patients who res ponded had the prednisone tapered and MTX discontinued after three mon ths of treatment. Recurrences while treatment was being tapered, or af ter its discontinuation, were managed with resumption of MTX and PDN. Treatment was successful in 16 of the 17 episodes, meaning that sympto ms resolved and abnormal physical or radiographic findings improved. S ymptomatic relief occurred in two weeks or less in 14 of 17 episodes; objective improvement generally occurred within one month, and in all cases by three months. The median duration of treatment was 5 months. Toxicity was limited to slight, transient elevations in hepatic enzyme s in three patients. We conclude that oral chemotherapy with alternate -day PDN and weekly MTX is effective and non-toxic in patients with lo w-risk LCH. (C) 1995 Wiley-Liss, Inc.