PurposeTo report the results of a prospective, nonrandomized European study on infants with neuroblastomaand MYCN gene amplification.Patients and MethodsInfants with neuroblastoma (stage 2, 3, 4, and 4s) and MYCN gene amplification who werediagnosed between 1999 and 2004 were eligible for enrollment onto the study. After diagnosis,staging, and mandatory biologic studies, induction chemotherapy (IC) with conventional drugs wasadministered, followed by delayed surgery, megatherapy (busulfan-melphalan as a conditioningregimen), and local radiotherapy.ResultsOf the 46 infants enrolled onto the study, 35 infants were eligible; of these 35 infants, 97% hadmetastatic spread (24 infants had stage 4, and 10 infants had stage 4s). Two-year overall survival(OS) was 30% (SE, 0.08), with median survival time of 12 months, and 23 deaths due to disease.Two-year, event-free survival (EFS) was 29% (SE, 0.07). The treatment was well tolerated with nodeaths as a result of toxicity or severe toxicity. Despite protocol adherence, 30% of the patientswho were assessable for response to IC experienced disease progression or did not respond.Stage and high lactate dehydrogenase reached significance in the univariate analysis (P .028 and.039, respectively for OS; and P .05 and .031 respectively, for EFS). Ten of 16 patients whoreceived megatherapy are still alive.ConclusionAlthough treatment was well tolerated, survival was poor and our IC failed to achieve a satisfactoryresponse in 30% of our patients. New therapeutic approaches and more intense world-widecollaboration are needed to achieve a cure in this population.
|Titolo:||Poor survival for infants with MYCN amplified metastatic neuroblastoma despite intensified treatment: The International Society of Paediatric Oncology European Neuroblastoma experience|
|Data di pubblicazione:||2009|
|Appare nelle tipologie:||1.1 Articolo in rivista|