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

A. Di Cataldo, Antonella Agodi, J. Balaguer, Alberto Garaventa, M. Barchitta, V. Segura, M. Bianchi, V. Castel, Aurora Castellano, S. Cesaro, J. M. Couselo, O. Cruz, P. D’Angelo, B. De Bernardi, J. Donat, N. G. de Andoin, M. I. Hernandez, Milena La Spina, M. Lillo, Ricardo López-AlmarazRoberto Luksch, S. Mastrangelo, E. Mateos, J. Molina, Carla Moscheo, Rossella Mura, F. Porta, Giovanna Russo, Annalisa Tondo, M. Torrent, Simona Vetrella, J. A. Villegas, E. Viscardi, Giulio Andrea Zanazzo, Adela Cañete

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)1-8
Number of pages8
JournalClinical and Translational Oncology
DOIs
Publication statusAccepted/In press - Apr 4 2016

Fingerprint

Neuroblastoma
Disease-Free Survival
Survival Rate
Clinical Trials
Survival
Population
Survival Analysis
Registries
Multivariate Analysis

Keywords

  • Eligibility
  • Infant
  • Metastatic
  • Neuroblastoma
  • Survival

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Metastatic neuroblastoma in infants : are survival rates excellent only within the stringent framework of clinical trials? / Di Cataldo, A.; Agodi, Antonella; Balaguer, J.; Garaventa, Alberto; Barchitta, M.; Segura, V.; Bianchi, M.; Castel, V.; Castellano, Aurora; Cesaro, S.; Couselo, J. M.; Cruz, O.; D’Angelo, P.; De Bernardi, B.; Donat, J.; de Andoin, N. G.; Hernandez, M. I.; La Spina, Milena; Lillo, M.; López-Almaraz, Ricardo; Luksch, Roberto; Mastrangelo, S.; Mateos, E.; Molina, J.; Moscheo, Carla; Mura, Rossella; Porta, F.; Russo, Giovanna; Tondo, Annalisa; Torrent, M.; Vetrella, Simona; Villegas, J. A.; Viscardi, E.; Zanazzo, Giulio Andrea; Cañete, Adela.

In: Clinical and Translational Oncology, 04.04.2016, p. 1-8.

Research output: Contribution to journalArticle

Di Cataldo, A, Agodi, A, Balaguer, J, Garaventa, A, Barchitta, M, Segura, V, Bianchi, M, Castel, V, Castellano, A, Cesaro, S, Couselo, JM, Cruz, O, D’Angelo, P, De Bernardi, B, Donat, J, de Andoin, NG, Hernandez, MI, La Spina, M, Lillo, M, López-Almaraz, R, Luksch, R, Mastrangelo, S, Mateos, E, Molina, J, Moscheo, C, Mura, R, Porta, F, Russo, G, Tondo, A, Torrent, M, Vetrella, S, Villegas, JA, Viscardi, E, Zanazzo, GA & Cañete, A 2016, 'Metastatic neuroblastoma in infants: are survival rates excellent only within the stringent framework of clinical trials?', Clinical and Translational Oncology, pp. 1-8. https://doi.org/10.1007/s12094-016-1505-1
Di Cataldo, A. ; Agodi, Antonella ; Balaguer, J. ; Garaventa, Alberto ; Barchitta, M. ; Segura, V. ; Bianchi, M. ; Castel, V. ; Castellano, Aurora ; Cesaro, S. ; Couselo, J. M. ; Cruz, O. ; D’Angelo, P. ; De Bernardi, B. ; Donat, J. ; de Andoin, N. G. ; Hernandez, M. I. ; La Spina, Milena ; Lillo, M. ; López-Almaraz, Ricardo ; Luksch, Roberto ; Mastrangelo, S. ; Mateos, E. ; Molina, J. ; Moscheo, Carla ; Mura, Rossella ; Porta, F. ; Russo, Giovanna ; Tondo, Annalisa ; Torrent, M. ; Vetrella, Simona ; Villegas, J. A. ; Viscardi, E. ; Zanazzo, Giulio Andrea ; Cañete, Adela. / Metastatic neuroblastoma in infants : are survival rates excellent only within the stringent framework of clinical trials?. In: Clinical and Translational Oncology. 2016 ; pp. 1-8.
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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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T1 - Metastatic neuroblastoma in infants

T2 - are survival rates excellent only within the stringent framework of clinical trials?

AU - Di Cataldo, A.

AU - Agodi, Antonella

AU - Balaguer, J.

AU - Garaventa, Alberto

AU - Barchitta, M.

AU - Segura, V.

AU - Bianchi, M.

AU - Castel, V.

AU - Castellano, Aurora

AU - Cesaro, S.

AU - Couselo, J. M.

AU - Cruz, O.

AU - D’Angelo, P.

AU - De Bernardi, B.

AU - Donat, J.

AU - de Andoin, N. G.

AU - Hernandez, M. I.

AU - La Spina, Milena

AU - Lillo, M.

AU - López-Almaraz, Ricardo

AU - Luksch, Roberto

AU - Mastrangelo, S.

AU - Mateos, E.

AU - Molina, J.

AU - Moscheo, Carla

AU - Mura, Rossella

AU - Porta, F.

AU - Russo, Giovanna

AU - Tondo, Annalisa

AU - Torrent, M.

AU - Vetrella, Simona

AU - Villegas, J. A.

AU - Viscardi, E.

AU - Zanazzo, Giulio Andrea

AU - Cañete, Adela

PY - 2016/4/4

Y1 - 2016/4/4

N2 - 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.

AB - 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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KW - Infant

KW - Metastatic

KW - Neuroblastoma

KW - Survival

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