title: Conservative treatment of retinoblastoma: a prospective phase II randomized trial of neoadjuvant chemotherapy followed by local treatments and chemothermotherapy.
abstract: PurposeIntraocular retinoblastoma treatments often combine chemotherapy and focal treatments. A first prospective protocol of conservative treatments in our institution showed the efficacy of the use of two courses of chemoreduction with etoposide and carboplatin, followed by chemothermotherapy using carboplatin as a single agent and diode laser. In order to decrease the possible long-term toxicity of chemotherapy due to etoposide, a randomized neoadjuvant phase II protocol was conducted using vincristine-carboplatin vs etoposide-carboplatin.Patients and methodsThe study was proposed when initial tumor characteristics did not allow front-line local treatments. Patients included in this phase II noncomparative randomized study of neoadjuvant chemotherapy received vincristin-carboplatin (new arm) vs etoposide-carboplatin (our reference arm). They were subsequently treated by local treatments and chemothermotherapy. Primary end point was the need for secondary enucleation or external beam radiotherapy (EBRT) not exceeding 40% at 2 years.ResultsA total of 65 eyes in 55 children were included in the study (May 2004 to August 2009). Of these, 32 eyes (27 children) were treated in the arm etoposide-carboplatin and 33 eyes (28 children) in the arm vincristin-carboplatin. At 2 years after treatment, 23/33 (69.7%) eyes were treated and salvaged without EBRT or enucleation in the arm vincristin-carboplatin and 26/32 (81.2%) in the arm etoposide-carboplatin.ConclusionEven if the two treatment arms could be considered as sufficiently active according to the study decision rules, neoadjuvant chemotherapy by two cycles of vincristine-carboplatin followed by chemothermotherapy appear to offer less optimal local control than the etoposide-carboplatin combination.
authors.affiliation: Department of Ocular Oncology, Institut Curie, Paris, France.
authors.forename: L
authors.initials: L
authors.lastname: Lumbroso-Le Rouic
authors.affiliation: Department of Pediatric, Adolescent and Young Adults Oncology, Institut Curie, Paris, France.
authors.forename: I
authors.initials: I
authors.lastname: Aerts
authors.affiliation: Department of Biostatistics, Institut Curie, Paris, France.
authors.forename: D
authors.initials: D
authors.lastname: Hajage
authors.affiliation: Department of Ocular Oncology, Institut Curie, Paris, France.
authors.forename: C
authors.initials: C
authors.lastname: Lévy-Gabriel
authors.affiliation: Department of Biostatistics, Institut Curie, Paris, France.
authors.forename: A
authors.initials: A
authors.lastname: Savignoni
authors.affiliation: Department of Biostatistics, Institut Curie, Paris, France.
authors.forename: N
authors.initials: N
authors.lastname: Algret
authors.affiliation: Department of Ocular Oncology, Institut Curie, Paris, France.
authors.forename: N
authors.initials: N
authors.lastname: Cassoux
authors.affiliation: Department of Pediatric Oncology, CHU Toulouse, Hôpital des Enfants, Toulouse, France.
authors.forename: A-I
authors.initials: AI
authors.lastname: Bertozzi
authors.affiliation: Department of Anaesthesiology, Institut Cure, Paris, France.
authors.forename: M
authors.initials: M
authors.lastname: Esteve
authors.affiliation: Department of Pediatric, Adolescent and Young Adults Oncology, Institut Curie, Paris, France.|University Paris Descartes, Sorbonne Paris Cité, Paris, France.
authors.forename: F
authors.initials: F
authors.lastname: Doz
authors.affiliation: Department of Ocular Oncology, Institut Curie, Paris, France.
authors.forename: L
authors.initials: L
authors.lastname: Desjardins
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D006801:Humans; D006979:Hyperthermia, Induced; D007223:Infant; D008297:Male; D020360:Neoadjuvant Therapy; D011446:Prospective Studies; D019572:Retinal Neoplasms / Q000145:classification / Q000473:pathology / Q000628:therapy*; D012175:Retinoblastoma / Q000145:classification / Q000473:pathology / Q000628:therapy*; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D005047:Etoposide; D016190:Carboplatin