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Autografting

Long-term outcomes in children with high-risk neuroblastoma treated with autologous stem cell transplantation

Bone Marrow Transplantation volume 40, pages 741–746 (2007)Cite this article

Abstract

We retrospectively analysed the outcomes of children transplanted for high-risk neuroblastoma (NB) at a single institution predominantly transplanted with total body irradiation and chemotherapy. The aims of this study were to determine the prognostic impact of clinical and biological features and to document long-term health outcomes. Forty patients were transplanted with a single unpurged autograft. Fourteen patients died from disease progression and two from late complications of treatment. Twenty-three patients are alive at a median of 4.6 years from diagnosis. Kaplan–Meier estimates of overall survival at 2, 5 and 10 years are 76±7.0, 60.2±8.4 and 54.7±9.3% following transplant. Response to induction therapy was significantly associated with survival (P<0.01). Long-term complications included growth (100%) and pubertal failure (83%), hearing impairment (73%), orthopaedic complications (63%), renal impairment (47%) and thyroid abnormalities (36%). Intrinsic and acquired resistance to chemotherapy remains the major obstacle to improving outcomes in high-risk NB. Although patients with chemo-sensitive disease are less likely to experience a relapse, substantial therapy-related toxicities result in poor long-term health outcomes for survivors.

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Acknowledgements

TN Trahair is a recipient of an Australian National Health and Medical Research Council Peter Doherty Australian Biomedical Fellowship (359301) and a Cancer Institute NSW Clinical Research Fellowship.

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Authors and Affiliations

  1. Centre for Children's Cancer and Blood Disorders, Sydney Children's Hospital, Randwick, New South Wales, Australia

    T N Trahair, M R Vowels, K Johnston, R J Cohn, S J Russell & G M Marshall

  2. School of Women's and Children's Health, University of NSW, Sydney, New South Wales, Australia

    T N Trahair, R J Cohn, S J Russell, K A Neville & G M Marshall

  3. Department of Endocrinology, Sydney Children's Hospital, Randwick, New South Wales, Australia

    K A Neville

  4. Department of Radiation Oncology, Prince of Wales Hospital, Randwick, New South Wales, Australia

    S Carroll

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Correspondence to T N Trahair.

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Trahair, T., Vowels, M., Johnston, K. et al. Long-term outcomes in children with high-risk neuroblastoma treated with autologous stem cell transplantation. Bone Marrow Transplant 40, 741–746 (2007). https://doi.org/10.1038/sj.bmt.1705809

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