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Metastatic neuroblastoma in infants: are survival rates excellent only within the stringent framework of clinical trials?

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Abstract

Introduction

SIOPEN INES protocol yielded excellent 5-year survival rates for MYCN-non-amplified metastatic neuroblastoma. Patients deemed ineligible due to lack or delay of MYCN status or late registration were treated, but not included in the study. Our goal was to analyse survival at 10 years among the whole population.

Materials and methods

Italian and Spanish metastatic INES patients’ data are reported. SPSS 20.0 was used for statistical analysis.

Results

Among 98 infants, 27 had events and 19 died, while 79 were disease free. Five- and 10-year event-free survival (EFS) were 73 and 70 %, and overall survival (OS) was 81 and 74 %, respectively. MYCN status was significant for EFS, but not for OS in multivariate analysis.

Conclusions

The survival rates of patients who complied with all the inclusion criteria for INES trials are higher compared to those that included also not registered patients. Five-year EFS and OS for INES 99.2 were 87.8 and 95.7 %, while our stage 4s population obtained 78 and 87 %. Concerning 99.3, 5-year EFS and OS were 86.7 and 95.6 %, while for stage 4 we registered 61 and 68 %. MYCN amplification had a strong impact on prognosis and therefore we consider it unacceptable that many patients were not studied for MYCN and probably inadequately treated. Ten-year survival rates were shown to decrease: EFS from 73 to 70 % and OS from 81 to 74 %, indicating a risk of late events, particularly in stage 4s. Population-based registries like European ENCCA WP 11-task 11 will possibly clarify these data.

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Acknowledgments

We wish to thank the Italian Neuroblastoma Foundation for financial support, Dr Desiree Ramal for managing data, Ms Daniela Bonfiglio for her precious assistance in English language, and Mr. Giuseppe Auteri for secretarial work. We wish to thank all the physicians and nurses working in the following hospitals where the patients of this study were hospitalized:

Spanish hospitals: Hospital Universitarii Politecnic La Fe–Valencia; Hospital Universitario Infantil Niño Jesus–Madrid; Hospital Universitario de Cruces–Bilbao; Hospital Universitario Virgen Arrixaca–Murcia; Hospital Universitario 12 de Octubre–Madrid; Hospital Universitario Virgen del Rocio–Sevilla; Hospital General Universitario Gregorio Marañon - Madrid; Hospital MaternoInfantil Carlos Haya–Malaga; Hospital UniversitarioMonteprincipe–Madrid; Hospital UniversitariValld’Hebron–Barcelona; Hospital General Universitario de Alicante–Alicante; Hospital de Torrecardenas –Almeria; Hospital Universitario Miguel Servet–Zaragoza; Hospital Universitario Reina Sofia–Murcia; Hospital Virgen del Camino–Pamplona; Hospital Universitario de Canarias–Santa Cruz de Tenerife; Hospital UniversitarioDonostia–Donostia; Hospital San Joan de Deu–Barcelona; Hospital Santa CreuiSant Pau–Barcelona; Hospital Universitari Son Espases–Palma Mallorca; Hospital ClinicoUniversitario de Santiago–Santiago de Compostela; Hospital Universitario Central de Asturias–Oviedo; Hospital General Universitario de Albacete–Albacete; Hospital Clinico de Valencia–Valencia.

Italian hospitals: Policlinico–Catania; Istituto Gaslini–Genova; Ospedale Santa Chiara–Pisa; Ospedale Bambin Gesù–Roma; Policlinico–Bari; Ospedale Maggiore–Milano; Policlinico–Modena; Ospedale Casa Sollievo della Sofferenza–San Giovanni Rotondo; Ospedale Infermi–Rimini; Ospedale G.Salesi–Ancona; Istituto Nazionale dei Tumori–Milano; Azienda Ospedaliera Seconda Università–Napoli; Ospedale Sant’Orsola Malpighi, Bologna; Ospedale Papa Giovanni XXIII–Bergamo; Ospedale Sant’Anna–Ferrara; Policlinico San Matteo–Pavia; Ospedale Regina Margherita–Torino; Ospedale Borgo Roma–Verona; Ospedale Civico e Benfratelli–Palermo; Policlinico Gemelli–Roma; Ospedale Microcitemico–Cagliari; Spedali Civili–Brescia; Ospedale Meyer–Firenze; Ospedale SantobonoPausillipon–Napoli; Ospedale di Padova–Padova; Ospedale Burlo Garofolo–Trieste.

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Correspondence to J. Balaguer.

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All procedures performed in the studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki Declaration and its later amendments or comparable ethical standards.

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Di Cataldo, A., Agodi, A., Balaguer, J. et al. Metastatic neuroblastoma in infants: are survival rates excellent only within the stringent framework of clinical trials?. Clin Transl Oncol 19, 76–83 (2017). https://doi.org/10.1007/s12094-016-1505-1

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