Intracystic bleomycin therapy for craniopharyngioma in children: the Canadian experience.

BACKGROUND: Surgical removal and radiation therapy are associated with significant risk for morbidity in the pediatric population with craniopharyngioma. Intracystic therapies have been utilized in some centers to potentially decrease morbidity associated with cystic craniopharyngioma. The aim of the study was to review the Canadian experience with intracystic bleomycin therapy (ICB).

METHODS: All centers in the Canadian Pediatric Brain Tumor Consortium (CPBTC) were invited to participate in a retrospective review of this treatment. A questionnaire was sent to each center. The data were analyzed at British Columbia's Children's Hospital.

RESULTS: In all, 6 of 17 centers utilized ICB and submitted data. A total of 17 of 19 patients with the intention to treat received bleomycin. Twelve were treated at the time of diagnosis, and 5 at the time of recurrence. Five patients achieved a complete response, 6 achieved a partial response, and 5 achieved a minor response to bleomycin. One patient was stable for 2.8 years. At the time of last follow-up, 8 patients have not required further intervention. Complications included transient symptomatic peritumoral edema (2 patients), precocious puberty (1 patient) and panhypopituitarism (2 patients). The median follow-up was 4 years (range, 0.5-10.2 years). The median progression-free survival was 1.8 years (range, 0.3-6.1 years). One patient died of a massive infarct secondary to radiation-induced moyamoya syndrome.

CONCLUSIONS: ICB was found to be well tolerated in this group of children. ICB may be a feasible and effective therapy for certain children with craniopharyngioma. Bleomycin may delay the need for aggressive surgery or radiation therapy for several years. Prospective multiinstitutional clinical trials are required to further evaluate the feasibility, effectiveness, and dose schedules of this treatment.