The use and effectiveness of temozolomide in children with central nervous system tumours: a survey from the Canadian Paediatric Brain Tumour Consortium

Objective To describe the use of temozolomide (rmz) in Canadian children treated for brain tumours and to evaluate survival and predictors of survival for children treated with this agent. Methods A survey was conducted within the Canadian Paediatric Brain Tumour Consortium (CPBTC), a group of tertiary care centres in pediatric neurooncology (n = 16) in Canada that are involved in the treatment of children with central nervous system tumours. Results In 10 of the 16 participating pediatric oncology centres of the CPBTC, 137 children with brain tumours were treated with TMZ between January 2000 and March 2006. Although 33% of the children were enrolled into a clinical trial, 67% were treated outside open studies. Most patients (72%) received TMZ treatment on recurrence of their brain tumour (first or subsequent). The most commonly administered regimen was single-agent TMZ 150-200 mg/m(2) administered on 5 consecutive days every 28 days. The median duration of TMZ treatment was 141 days (range: 4-11102 days). Response data were provided for 127 of the 1.37 patients, of whom 6 showed a complete response. Sixteen patients experienced a minor or partial response, 53 had stable disease, and 52 had progressive disease. Of 32 patients alive at last follow-up, 19 had a diagnosis of low-grade glioma. Conclusions Temozolomide is used in a variety of pediatric brain tumours, often at the time of recurrence. The lack of insight into clear indications for this agent in pediatric brain tumours used either alone or in combination therapy may be a result of suboptimal design of phase I and II studies and a lack of phase HI trials in the pediatric brain tumour population.