Phase II evaluation of interferon-alpha-2a for progressive or recurrent craniopharyngiomas

Object. Craniopharyngiomas originate from the same cells as squamous cell s kin carcinoma, which can be treat ed successfully with interferon-alpha (IF N alpha)-2a. The authors evaluated the activity and toxicity of systemic IF N in young patients with craniopharyngiomas. Methods. Fifteen patients between the ages of 4.2 and 19.8 years who had pr ogressive or recurrent craniopharyngiomas were enrolled in this study. Nine of these patients had never received external-beam radiation therapy. Ther apy consisted of 8,000,000 U/m(2) IFN alpha-2a administered daily for 16 we eks (induction phase) followed by the same dose three times per week fur an additional 32 weeks (maintenance phase). Of the 12 patients who could be e valuated, radiological studies demonstrated a response to treatment in thre e with predominantly cystic tumors tone minor response, one partial respons e, and one complete response); one of these patients also showed improvemen t in visual fields. The size of the cystic component of the tumors often in creased temporarily during the first several months of therapy. Three patie nts met the criteria for progressive disease during therapy. The median tim e to progression was 25 months. The need for radiation therapy in patients treated with IFN was delayed for 18 to 35 month?, (median 25 months) in six patients. All patients developed transient flulike symptoms shortly after receiving the first dose of IFN. Other toxicities (predominantly hepatic. n eurological, and cutaneous) were seen in nine (60%) of the 15 patients duri ng the first 8 weeks of treatment but resolved after temporary discontinuat ion and/or dose reduction. Conclusions. Interferon-alpha-2a is active against some childhood craniopha ryngiomas; its toxicity precludes administration of high daily doses, and t he optimum dose level and schedule remain to be defined.