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Solid Tumours

28 years of high-dose therapy and SCT for neuroblastoma in Europe: lessons from more than 4000 procedures

Bone Marrow Transplantation volume 41, pages S118–S127 (2008)Cite this article

Abstract

Between 1978 and 2006, the European Group for Blood and Marrow Transplantation registered 4098 high-dose therapy (HDT) procedures followed by stem cell rescue (SCR) (3974 autologous/124 allogeneic) in patients with neuroblastoma. The 5-year rates for overall (OS) and event-free survival are 37 and 32%, respectively. The median age at diagnosis is 3.9 years (0.3–62 years) with 76 patients older than 18 years. Patients above 10 years carry a 2.5-fold higher risk. Younger patients cure significantly (<0.001) better with OS rates of 40 and 30% for age groups 2–4 years and 4–10 years, respectively. Their risks are about twofold higher than that of patients below 2 years with OS rates of 60%. The better the quality of remission status before HDT/SCT the better are the observed OS rates: 43% in CR1 (1199 patients) and 42% for CR2 (140 patients), and 36% for those in very good partial or partial remission (1413 patients) and 21% for those with sensitive relapse (134 patients). Patients reported with stable disease in first remission still had an OS rate of 30%. Multivariate analysis shows significantly better OS in the age group of less than 2 years (<0.0001), as well as a better quality of remission status before HDT/SCT (P<0.0001), with the use of peripheral stem cells (P=0.014), autologous SCT (P=0.031) and busulphan/melphalan HDT (P<0.001). Busulphan/melphalan HDT/SCT in first remission achieves an OS of 48%, while it is only 35% with other regimens (P<0.001), including melphalan alone, other melphalan-containing regimens, a variety of other drugs given as a single HDT as well as the addition of TBI or sequential HDT/SCT procedures. Further progress in the field may only be expected from large-scale international randomized trials.

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Authors and Affiliations

  1. St Anna Kinderspital, Kinderspitalgasse, Vienna, Austria

    R Ladenstein

  2. Unit for Studies and Statistics of Integrated Research and Projects, Children's Cancer Research Institute—St Anna Kinderkrebsforschung, Kinderspitalgasse, Vienna, Austria

    R Ladenstein & U Pötschger

  3. Institut Gustave Roussy, Rue Camille Desmoulins, Villejuif, France

    O Hartman

  4. Royal Marsden Hospital, The Institute of Cancer Research, Surrey, Sutton, UK

    A D J Pearson

  5. Klinikum der Johann-Wolfgang Goethe Universitaet, Klinik für Kinderheilkunde und Jugendmedizin, Frankfurt am Main, Germany

    T Klingebiel

  6. Hospital Universitario Infantil La Fe, Oncologia Pediatrica, Avenida de Campanar, Valencia, Spain

    V Castel

  7. Schneider Children's Medical Center of Israel, Petach-Tikva, Israel

    I Yaniv

  8. Ankara University Faculty of Medicine,

    T Demirer

  9. Cebeci Campus Dirimevi, Ankara, Turkey

    T Demirer

  10. Istituto Giannina Gaslini, Largo G Gaslini, Genova, Italy

    G Dini

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Ladenstein, R., Pötschger, U., Hartman, O. et al. 28 years of high-dose therapy and SCT for neuroblastoma in Europe: lessons from more than 4000 procedures. Bone Marrow Transplant 41 (Suppl 2), S118–S127 (2008). https://doi.org/10.1038/bmt.2008.69

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