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Neuroblastoma in Infants: Long Term Survival from INES Protocols - A SIOPEN Study
  • +19
  • J. Savagner,
  • Caroline Munzer,
  • Gudrun Schleiermacher,
  • Alberto Garaventa,
  • Riccardo Haupt,
  • Stefania Sorrentino,
  • Katia Mazzocco,
  • Andrea Di Cataldo,
  • Adela Cañete,
  • Vanessa Segura,
  • Désirée Ramal,
  • Kate Wheeler,
  • R. Ladenstein,
  • Ana Lacerda,
  • Benedicte Brichard,
  • Maja Beck-Popovic,
  • Dominique Valteau-Couanet,
  • Claudia Pasqualini,
  • C. Coze,
  • Benoît Dumont,
  • Anne-Sophie Defachelles,
  • Marion Gambart
J. Savagner
Hopital des Enfants

Corresponding Author:[email protected]

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Caroline Munzer
Hopital des Enfants
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Gudrun Schleiermacher
Institut Curie
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Alberto Garaventa
Istituto Giannina Gaslini
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Riccardo Haupt
Istituto Giannina Gaslini
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Stefania Sorrentino
Istituto Giannina Gaslini
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Katia Mazzocco
Istituto Giannina Gaslini
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Andrea Di Cataldo
Universita degli Studi di Catania Dipartimento di Medicina Clinica e Sperimentale
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Adela Cañete
Hospital Universitari i Politecnic La Fe
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Vanessa Segura
Instituto de Investigacion Sanitaria La Fe
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Désirée Ramal
Instituto de Investigacion Sanitaria La Fe
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Kate Wheeler
University of Oxford Radcliffe Department of Medicine
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R. Ladenstein
St Anna Kinderspital GmbH
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Ana Lacerda
Instituto Portugues de Oncologia de Lisboa Francisco Gentil EPE
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Benedicte Brichard
Cliniques universitaires Saint-Luc Departement de Pediatrie
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Maja Beck-Popovic
Centre Hospitalier Universitaire Vaudois Hopital de l'enfance de Lausanne
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Dominique Valteau-Couanet
Gustave Roussy
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Claudia Pasqualini
Gustave Roussy
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C. Coze
Hopital de la Timone Service de Pediatrie Specialisee et de Medecine Infantile
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Benoît Dumont
Institut d'Hematologie et d'Oncologie Pediatrique
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Anne-Sophie Defachelles
Centre Oscar Lambret
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Marion Gambart
Hopital des Enfants
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Abstract

Purpose: Neuroblastoma is the most frequent extracranial solid tumors in infants, with a characteristic presentation and a possibility of spontaneous regression even in disseminated disease. Despite an overall good prognosis, relapse can occur in some patients, leading to a poorer outcome in a subgroup of patients. A long-term analysis of these patients assessing survival at 10 years from diagnosis is relevant in estimating the risks of late relapse, progression or long-term toxicity due to very young exposure to treatment. Methods: Estimation of the 10-year event-free and overall survivals in 750 infants under 12 months of age with Neuroblastoma, enrolled in the prospective INES protocols between 1999 and 2004. Follow-up data from INES patients were updated, and survival analyses were performed in order to determine prognostic factors such as: age, stage, genomic profile or MYCN amplification. Results: Overall, 10-year OS was 91.1% ± 1.0%, and 10-year EFS was 82.4% ±1.4%, with significantly better outcomes in infants under 6 months compared to those aged 6 to 12 months, even considering the MYCN-amplified tumors only. MYCN amplification was the strongest prognostic factor and was correlated with substantially lower survival rates among patients with metastatic disease. Discussion: Survival in infancy remains excellent and stable even at long term, as a 10-year follow-up did not change the number of events. However, survival in MYCN-amplified tumors remained poor, this population needs to be treated more aggressively. Patients with metastatic tumors require accurate risk stratification, especially for younger infants under 6 months. For each treatment group, there was no significant difference in long-term outcomes compared to previous publications from INES. No lethal toxicity affecting long-term survival occurred.