Late effects in children treated with intensive multimodal therapy for high-risk neuroblastoma: high incidence of endocrine and growth problems

Bone Marrow Transplant. 2014 Apr;49(4):502-8. doi: 10.1038/bmt.2013.218. Epub 2014 Jan 20.


Due to the poor prognosis of high-risk (HR) neuroblastoma (NBL), scant data exist on late effects after treatment. Recently, protocols utilizing intense multimodal treatment have resulted in improved long-term survival. The objective of this study was to determine the prevalence of late effects in survivors of HR NBL. A retrospective review of clinical data for serial patients completing treatment between September 1994 and October 2007 and surviving for at least 1 year was performed. Therapy included aggressive chemotherapy, surgery, radiation and single or tandem SCT. Oncology follow-up was standard; clinical criteria were utilized for referrals to endocrinology and other services. Fifty-one eligible patients were identified. Median follow-up was 6.1 years (range 1.0-15.2). Height was significantly impacted (ΔZ-score -1.91 in those treated with TBI and -0.77 in those without). Pre-diabetes or diabetes, hypothyroidism and ovarian insufficiency were observed in 50, 59 and 75% of at-risk survivors, respectively. Hearing loss and dental issues were common. Nine patients had relapse of NBL; seven died of progressive disease. As there is a high prevalence of late effects in long-term survivors of HR NBL, close monitoring and further studies after treatment are indicated, and in particular after more modern, non-TBI regimens.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Cohort Studies
  • Combined Modality Therapy
  • Endocrine System Diseases / etiology*
  • Female
  • Growth Disorders / etiology*
  • Growth Hormone
  • Humans
  • Hypothyroidism / etiology
  • Incidence
  • Infant
  • Infant, Newborn
  • Insulin Resistance
  • Male
  • Neoplasm Recurrence, Local / metabolism
  • Neoplasm Recurrence, Local / therapy
  • Neuroblastoma / metabolism
  • Neuroblastoma / therapy*
  • Prognosis
  • Retrospective Studies
  • Survivors


  • Growth Hormone