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

• A. Di Cataldo ^[1] ; A. Agodi ^[1] ; J. Balaguer ^[10] ; A. Garaventa ^[11] ; M. Barchitta ^[1] ; V. Segura ^[10] ; M. Bianchi ^[2] ; V. Castel ^[10] ; A. Castellano ^[12] ; S. Cesaro ^[13] ; J. M. Couselo ^[14] ; O. Cruz ^[15] ; P. D Angelo ^[16] ; B. De Bernardi ^[11] ; J. Donat ^[3] ; N. G. de Andoin ^[4] ; M. I. Hernandez ^[5] ; M. La Spina ^[1] ; M. Lillo ^[17] ; R. Lopez-Almaraz ^[18] ; R. Luksch ^[19] ; S. Mastrangelo ^[20] ; E. Mateos ^[6] ; J. Molina ^[7] ; C. Moscheo ^[19] ; R. Mura ^[8] ; F. Porta ^[21] ; G. Russo ^[1] ; A. Tondo ^[22] ; M. Torrent ^[23] ; S. Vetrella ^[24] ; J. A. Villegas ^[9] ; E. Viscardi ^[25] ; G. A. Zanazzo ^[26] ; A. Cañete ^[10]
  1. [1] University of Catania

     University of Catania

     Catania, Italia

     
  2. [2] Ospedale Regina Margherita

     Ospedale Regina Margherita

     Torino, Italia

     
  3. [3] Instituto de Investigación Sanitaria Fundación para la Investigación del Hospital Clínico de Valencia

     Instituto de Investigación Sanitaria Fundación para la Investigación del Hospital Clínico de Valencia

     Valencia, España

     
  4. [4] Hospital Universitario de Donostia

     Hospital Universitario de Donostia

     San Sebastián, España

     
  5. [5] Hospital Universitario Son Espases

     Hospital Universitario Son Espases

     Palma de Mallorca, España

     
  6. [6] Hospital Universitario Reina Sofia

     Hospital Universitario Reina Sofia

     Cordoba, España

     
  7. [7] Hospital Virgen del Camino

     Hospital Virgen del Camino

     Pamplona, España

     
  8. [8] Ospedale Microcitemico

     Ospedale Microcitemico

     Cagliari, Italia

     
  9. [9] Hospital Universitario Central de Asturias

     Hospital Universitario Central de Asturias

     Oviedo, España

     
  10. [10] Hospital Universitari i Politecnic La Fe, España
  11. [11] Giannina Gaslini Children’s Hospital, Italia
  12. [12] Ospedale Bambino Gesù, Italia
  13. [13] Ospedale Borgo Roma, Italia
  14. [14] Hospital Clínico Universitario de Santiago, España
  15. [15] Hospital San Joan de Deu, España
  16. [16] Civico e Benfratelli Hospital, Italia
  17. [17] Hospital General Universitario de Albacete, España
  18. [18] Hospital Universitariode Canarias, España
  19. [19] Fondazione IRCCS Istituto Nazionale dei Tumori, Italia
  20. [20] Policlinico Gemelli, Italia
  21. [21] Spedali Civili, Italia
  22. [22] Meyer Hospital, Italia
  23. [23] Hospital Santa Creui Sant Pau, España
  24. [24] Santobono Pausilipon Hospital, Italia
  25. [25] Padova Hospital, Italia
  26. [26] Burlo Garofolo Hospital, Italia

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• Localización: Clinical & translational oncology, ISSN 1699-048X, Vol. 19, Nº. 1 (January 2017), 2017, págs. 76-83
• Idioma: inglés
• Texto completo no disponible (Saber más ...)
• Resumen

  • 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.