title: High-dose chemotherapy with carboplatin, etoposide and cyclophosphamide followed by a haematopoietic stem cell rescue in patients with high-risk retinoblastoma: a SFOP and SFGM study.
abstract: This study investigates the role of high-dose chemotherapy with haematopoietic stem cell rescue as consolidation treatment in high-risk retinoblastoma (extraocular disease at diagnosis or relapse or invasion of cut end of optic nerve). 25 patients received high-dose chemotherapy including carboplatin (250 mg/m2/day from day 1 to day 5 for the 6 first patients and 350 mg/m2/day from day 1 to day 5 for the other patients), etoposide (350 mg/m2/day from day 1 to day 5) and cyclophosphamide (1.6 g/m2/day from day 2 to day 5) (CARBOPEC) followed by autologous haematopoietic stem cell rescue. 19 patients received this drug combination for chemosensitive extraocular relapse. The other 6 patients with histological high-risk factors were given this treatment as consolidation after enucleation and conventional chemotherapy. The three year disease-free survival was 67.1%. In 7 of the 9 relapsing patients, the first site of relapse was the central nervous system. All patients with central nervous system disease died except one. The main toxicity was haematological and digestive (mucositis and diarrhoea). 2 of the 13 evaluable patients had grade III and IV ototoxicity. One patient experienced an acute grade I reversible cardiotoxicity. The CARBOPEC regimen seems to be a promising therapeutic strategy in patients with high-risk retinoblastoma, especially those with bone and/or bone marrow involvement. This treatment did not improve the outcome of patients with central nervous system disease.
authors.affiliation: Paediatric Oncology Unit, Institut Curie, Paris, France.
authors.forename: F
authors.initials: F
authors.lastname: Namouni
authors.forename: F
authors.initials: F
authors.lastname: Doz
authors.forename: M L
authors.initials: ML
authors.lastname: Tanguy
authors.forename: E
authors.initials: E
authors.lastname: Quintana
authors.forename: J
authors.initials: J
authors.lastname: Michon
authors.forename: H
authors.initials: H
authors.lastname: Pacquement
authors.forename: E
authors.initials: E
authors.lastname: Bouffet
authors.forename: J C
authors.initials: JC
authors.lastname: Gentet
authors.forename: D
authors.initials: D
authors.lastname: Plantaz
authors.forename: P
authors.initials: P
authors.lastname: Lutz
authors.forename: J P
authors.initials: JP
authors.lastname: Vannier
authors.forename: P
authors.initials: P
authors.lastname: Validire
authors.forename: S
authors.initials: S
authors.lastname: Neuenschwander
authors.forename: L
authors.initials: L
authors.lastname: Desjardins
authors.forename: J M
authors.initials: JM
authors.lastname: Zucker
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage / Q000009:adverse effects; D016543:Central Nervous System Neoplasms / Q000188:drug therapy / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000009:adverse effects; D018572:Disease-Free Survival; D005047:Etoposide / Q000008:administration & dosage / Q000009:adverse effects; D005260:Female; D019650:Hematopoietic Stem Cell Mobilization / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D007674:Kidney Diseases / Q000139:chemically induced; D008297:Male; D009503:Neutropenia / Q000139:chemically induced; D019572:Retinal Neoplasms / Q000188:drug therapy / Q000628:therapy*; D012175:Retinoblastoma / Q000188:drug therapy / Q000628:therapy*
chemical_list: D005047:Etoposide; D003520:Cyclophosphamide; D016190:Carboplatin