High-dose chemotherapy in childhood brain tumours

New therapeutic strategies are required to improve the prognosis of malignant brain tumours in children, in terms of survival and quality of life. During the last 10 years, high-dose chemotherapy (HDCT) with autologous haematopoietic stem cell rescue has been studied in different types of paediatric brain tumours. The most frequently used combined regimens were busulfan-thiotepa and etoposide-thiotepa along with carboplatin or BCNU. High response rates have been reported in medulloblastoma and germ cell tumours, and HDCT has been further developed as salvage therapy or for consolidation in these diseases. Interesting objective tumour responses have been obtained in supratentorial high-grade glioma, but HDCT has not so far been effective either in ependymoma or in diffuse pontine brain stem tumours. This article reviews the rationale for HDCT in brain tumours and the current clinical results obtained in each tumour type. The place of HDCT in the therapeutic strategy for paediatric brain tumours, especially in young children, is discussed.