Childhood phaeochromocytoma and paraganglioma: 100% incidence of genetic mutations and 100% survival

J Pediatr Surg. 2010 Feb;45(2):383-6. doi: 10.1016/j.jpedsurg.2009.10.082.

Abstract

Introduction: The aim is to identify the incidence of genetic mutations and outcome of children presenting with phaeochromocytoma/paraganglioma (PGL) to a single paediatric surgical service to determine the need for genetic counselling in associated kindreds.

Methods: A retrospective case note review was undertaken of all cases treated between 1998 and 2008 with particular reference to presentation, management, and predisposing genetic conditions.

Results: Seven cases (4 male, 3 female) were identified (median age, 13 years; interquartile range, 9-16). Three cases had a family history of phaeochromocytoma/PGL. All presented with neurologic symptoms related to hypertension, including headaches (n = 5), hemiparesis (n = 2), facial palsy, and hemianopia. All underwent surgical resection. Five patients had meta-iodobenzylguanidine (MIBG) therapy for apparently malignant features. All cases were found to have a predisposing genetic mutation: von Hippel-Lindau (n = 3), succinate dehydrogenase mutations (n = 3), and multiple endocrine neoplasia (n = 1). All patients are alive after a median follow-up of 5 (interquartile range, 2-7) years.

Conclusions: All 7 cases had a familial genetic mutation identified, and none arose de novo. We advocate genetic counselling for all families of children diagnosed with phaeochromocytoma/PGL with lifelong surveillance tailored to the underlying syndrome because of the increased risk of synchronous and metachronous tumours associated with these genetic syndromes.

MeSH terms

  • Adolescent
  • Adrenal Gland Neoplasms / genetics*
  • Adrenal Gland Neoplasms / surgery
  • Child
  • Female
  • Follow-Up Studies
  • Genetic Counseling
  • Genetic Predisposition to Disease
  • Humans
  • Incidence
  • Longitudinal Studies
  • Male
  • Multiple Endocrine Neoplasia / genetics
  • Multiple Endocrine Neoplasia / surgery
  • Mutation / genetics*
  • Neoplasms, Second Primary / epidemiology
  • Neoplasms, Second Primary / genetics
  • Paraganglioma / genetics*
  • Paraganglioma / surgery
  • Pheochromocytoma / genetics*
  • Pheochromocytoma / surgery
  • Proto-Oncogene Proteins c-ret / genetics
  • Retrospective Studies
  • Risk Factors
  • Succinate Dehydrogenase / genetics
  • Syndrome
  • von Hippel-Lindau Disease / genetics
  • von Hippel-Lindau Disease / surgery

Substances

  • Succinate Dehydrogenase
  • Proto-Oncogene Proteins c-ret