A Canadian paediatric brain tumour consortium (CPBTC) phase II molecularly targeted study of imatinib in recurrent and refractory paediatric central nervous system tumours.

摘要: Abstract Purpose To evaluate the safety, efficacy and pharmacokinetics of imatinib in children with recurrent or refractory central nervous system (CNS) tumours expressing KIT and/or PDGFRA. Methods Nineteen patients aged 2–18years, CNS either target receptors PDGFRA (by immunohistochemistry) were eligible. Participants received orally at a dose 440mg/m 2 /day toxicities tumour responses monitored. Serial blood cerebrospinal fluid samples for obtained subset consenting patients. Frozen analysed retrospectively gene amplification whom available. Results Common lymphopaenia, neutropaenia, leucopaenia, elevated serum transaminases vomiting. No intratumoural haemorrhages observed. Although there no objective to imatinib, four had long-term stable disease (SD) (38–104weeks). Our results suggest possible relationship between expression maintenance SD treatment; immunopositivity was seen only 58% (11/19) study participants overall, but 100% 38weeks. All patient showed expression. Pharmacokinetic data high interpatient variability, corresponded previously reported values. Conclusions Imatinib is relatively safe tumours, induced responses. Demonstration progressing (KIT-expressing) suggests cytostatic activity imatinib.