Originally published as JCO Early Release 10.1200/JCO.2007.14.5839 on January 26 2009

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Poor Survival for Infants With MYCN-Amplified Metastatic Neuroblastoma Despite Intensified Treatment: The International Society of Paediatric Oncology European Neuroblastoma Experience

From La Fe Children's Hospital and Biostatistics Department, Universidad de Valencia, Valencia, Spain; Sheffield Children's Hospital, Sheffield; The Royal Marsden Hospital, Sutton, Surrey, United Kingdom; Hôpital des Enfants, Toulouse and Institute Curie, Paris, France; Centro di Riferimento Regionali di Ematologia ed Oncologia Pediatrica, Catania; G Gaslini Children's Hospital, Genoa, Italy; St Anna's Children's Hospital, Vienna, Austria; and Saint Luc University Hospital, Catholic University of Louvain, Brussels, Belgium.

Corresponding author: Adela Canete, PhD, Unidad de Oncología Pediátrica, Hospital Infantil La Fe, Avda de Campanar 21, 46009 Valencia, Spain; e-mail: canyete_ade{at}gva.es.

Purpose To report the results of a prospective, nonrandomized European study on infants with neuroblastoma and MYCN gene amplification.

Patients and Methods Infants with neuroblastoma (stage 2, 3, 4, and 4s) and MYCN gene amplification who were diagnosed between 1999 and 2004 were eligible for enrollment onto the study. After diagnosis, staging, and mandatory biologic studies, induction chemotherapy (IC) with conventional drugs was administered, followed by delayed surgery, megatherapy (busulfan-melphalan as a conditioning regimen), and local radiotherapy.

Results Of the 46 infants enrolled onto the study, 35 infants were eligible; of these 35 infants, 97% had metastatic 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 no deaths as a result of toxicity or severe toxicity. Despite protocol adherence, 30% of the patients who 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 who received megatherapy are still alive.

Conclusion Although treatment was well tolerated, survival was poor and our IC failed to achieve a satisfactory response in 30% of our patients. New therapeutic approaches and more intense world-wide collaboration are needed to achieve a cure in this population.

Supported by the Association pour la Recherche sur le Cancer, the Institute Curie, 3Es; the Association Hubert Gavin, France; and the European Neuroblastoma Quality Assurance Group.

Presented in part at the 37th Congress of the International Society of Paediatric Oncology (SIOP), September 2005, Vancouver, Canada; and Advances in Neuroblastoma Research XII Conference, (EEUU), June 2006, Los Angeles, CA.

Authors' disclosures of potential conflicts of interest and author contributions are found at the end of this article.

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