Phase II study of vinorelbine and continuous low doses cyclophosphamide in children and young adults with a relapsed or refractory malignant solid tumour: good tolerance profile and efficacy in rhabdomyosarcoma--a report from the Société Française des Cancers et leucémies de l'Enfant et de l'adolescent (SFCE)

摘要: Abstract Aim This phase II study evaluated efficacy, safety and pharmacokinetics (PK) profile of combination intravenous vinorelbine (VNL) continuous low doses oral cyclophosphamide (CPM) in children young adults with a recurrent or refractory solid tumour. Methods A total 117 patients (median age, 12 years) within six disease strata received VNL 25 mg/m 2 on days 1, 8 15 each 28-day cycle combined daily CPM . Tumour response was assessed every two cycles according to WHO (World Health Organisation) criteria. PK investigated subset 18 aged 4–15 years. Results In rhabdomyosarcoma (RMS) ( n  = 50), the best overall rate (ORR) 36% four complete (8%) 14 partial responses (28%). The ORR 13% Ewing’s sarcoma  = 15), 6% non-RMS soft tissue  = 16) neuroblastoma  = 16). No observed osteosarcoma  = 10) medulloblastoma  = 7). main grade 3/4 toxicity neutropenia (38%). Other severe toxicities were limited 3% peripheral neuropathy no haemorrhagic cystitis. analysis revealed equivalent blood exposure between >4 years adult series when dose based body surface area-based dosing. Concluding statement heavily pre-treated children, showed an interesting RMS acceptable supporting further evaluation these agents III trials.