1. ¹Centre for Children's Cancer and Blood Disorders, Sydney Children's Hospital, Randwick, New South Wales, Australia
2. ²School of Women's and Children's Health, University of NSW, Sydney, New South Wales, Australia
3. ³Department of Endocrinology, Sydney Children's Hospital, Randwick, New South Wales, Australia
4. ⁴Department of Radiation Oncology, Prince of Wales Hospital, Randwick, New South Wales, Australia

Correspondence: Dr TN Trahair, Centre for Children's Cancer and Blood Disorders, Level 1, Sydney Children's Hospital, High Street, Randwick, New South Wales 2031, Australia. E-mail: Toby.Trahair@sesiahs.health.nsw.gov.au

Received 4 October 2006; Revised 23 May 2007; Accepted 24 May 2007; Published online 27 August 2007.

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 767.0, 60.28.4 and 54.79.3% 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.