title: Long-term outcomes in children with high-risk neuroblastoma treated with autologous stem cell transplantation.
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.
authors.affiliation: Centre for Children's Cancer and Blood Disorders, Sydney Children's Hospital, Randwick, New South Wales, Australia. Toby.Trahair@sesiahs.health.nsw.gov.au
authors.forename: T N
authors.initials: TN
authors.lastname: Trahair
authors.forename: M R
authors.initials: MR
authors.lastname: Vowels
authors.forename: K
authors.initials: K
authors.lastname: Johnston
authors.forename: R J
authors.initials: RJ
authors.lastname: Cohn
authors.forename: S J
authors.initials: SJ
authors.lastname: Russell
authors.forename: K A
authors.initials: KA
authors.lastname: Neville
authors.forename: S
authors.initials: S
authors.lastname: Carroll
authors.forename: G M
authors.initials: GM
authors.lastname: Marshall
mesh_terms: D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009447:Neuroblastoma / Q000628:therapy*; D012189:Retrospective Studies; D033581:Stem Cell Transplantation / Q000009:adverse effects; D016019:Survival Analysis; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome