title: Survival and late effects in children with stage 4 neuroblastoma.
abstract: BACKGROUND
  Treatment of metastatic neuroblastoma (NB) demands aggressive oncological therapy, which may cause long-term sequelae in survivors. The aim of this retrospective single center study is to give an overview of survival in children with stage 4 NB and to describe the spectrum of late effects seen in survivors.
  
  
  METHODS
  Medical records of 31 patients with stage 4 NB treated between 1984 and 2009, who were included in a follow-up programme, were reviewed for information on tumor, treatment and late effects.
  
  
  RESULTS
  Five-year overall survival was 54.3 ± 9% and 5-year event-free survival was 44.9 ± 9%. Patients diagnosed after 1996 had a significantly better survival rate than those diagnosed before (74 ± 11.2% vs. 33.3 ± 12.2%, P = 0.011). In 15 of the 16 survivors (93.8%), numerous late effects were detected. The most common long-term sequelae were renal changes in 10 patients (62.5%) and endocrine disturbances in 9 patients (56.3%), including hypothyroidism with need of substitution in 50%, GH deficiency in 37.5% and hypogonadism in 12.5%. Sensorineural hearing loss occurred in 37.5% of survivors. Further observed late effects were hepatobiliary changes (31.3%), musculoskeletal problems, and pulmonary abnormalities (each 25%), as well as neurologic changes (18.8%), dental defects (12.5%), and unilateral blindness (6.3%). Second neoplasms appeared in 3 patients, 1 of whom died of hepatocellular carcinoma following infection with hepatitis B.
  
  
  CONCLUSIONS
  More than 50% of children with stage 4 NB may survive. The high incidence of severe long-term sequelae underlines the importance of careful follow-up in order to detect and treat late effects early enough.
authors.affiliation: Division of Paediatric Haematology/Oncology, Medical University of Graz, Graz, Austria. thomas.perwein@gmx.at
authors.forename: Thomas
authors.initials: T
authors.lastname: Perwein
authors.forename: Herwig
authors.initials: H
authors.lastname: Lackner
authors.forename: Petra
authors.initials: P
authors.lastname: Sovinz
authors.forename: Martin
authors.initials: M
authors.lastname: Benesch
authors.forename: Sandrin
authors.initials: S
authors.lastname: Schmidt
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Schwinger
authors.forename: Christian
authors.initials: C
authors.lastname: Urban
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D015994:Incidence; D007223:Infant; D053208:Kaplan-Meier Estimate; D008297:Male; D009367:Neoplasm Staging; D016609:Neoplasms, Second Primary / Q000453:epidemiology; D009447:Neuroblastoma / Q000150:complications* / Q000401:mortality* / Q000473:pathology / Q000628:therapy; D011878:Radiotherapy / Q000009:adverse effects; D012189:Retrospective Studies; D017741:Survivors
chemical_list: D000970:Antineoplastic Agents