Survival and late effects in children with stage 4 neuroblastoma

Pediatr Blood Cancer. 2011 Oct;57(4):629-35. doi: 10.1002/pbc.23036. Epub 2011 Feb 11.

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.

Procedure: 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.

MeSH terms

  • Antineoplastic Agents / therapeutic use
  • Child
  • Child, Preschool
  • Disease-Free Survival
  • Female
  • Humans
  • Incidence
  • Infant
  • Kaplan-Meier Estimate
  • Male
  • Neoplasm Staging
  • Neoplasms, Second Primary / epidemiology
  • Neuroblastoma / complications*
  • Neuroblastoma / mortality*
  • Neuroblastoma / pathology
  • Neuroblastoma / therapy
  • Radiotherapy / adverse effects
  • Retrospective Studies
  • Survivors

Substances

  • Antineoplastic Agents