An intensive multiagent chemotherapy regimen for brain tumours occurring in very young children

Standard treatment for the majority of malignant brain tumours consists of surgery and radiotherapy. This treatment has late morbidity which is accentuated in the very young child. As part of a strategy to improve quality of life and overall survival of young children with brain tumours, members of the United Kingdom Children's Cancer Study Group (UKCCSG) have piloted an intensive chemotherapy regimen which aims to avoid or delay radiotherapy following surgery. Twenty eight children with a variety of malignant brain tumours have received the regimen, which contains carboplatin, vincristine, cyclophosphamide, methotrexate, and cisplatin. The treatment is toxic, resulting in one death from infection. The bulk of the toxicity was associated with the administration of carboplatin. All but three children eventually required adjuvant radiotherapy and this was given between 1.5 and 27 months from diagnosis (median delay to radiotherapy, 12 months). Using this treatment regimen, overall survival at four years is 35% (confidence intervals 10% to 60%). While there is no evidence from this study that radiotherapy can be abandoned in the management of malignant brain tumours, its introduction may be delayed using suitable chemotherapy, thus allowing time for further CNS development. This treatment strategy has been taken forward as an international clinical trial run through the International Society for Paediatric Oncology, but using a smaller dose of carboplatin to reduce toxicity.