Journal
BONE MARROW TRANSPLANTATION
Volume 40, Issue 8, Pages 741-746Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.bmt.1705809
Keywords
neuroblastoma; autologous transplantation; late effects
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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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