The management of retinoblastoma

Oncogene. 2018 Mar;37(12):1551-1560. doi: 10.1038/s41388-017-0050-x. Epub 2018 Jan 11.

Abstract

Retinoblastoma (Rb) is the most common primary intraocular malignancy of childhood, but an uncommon paediatric cancer, with a constant incidence worldwide of 1:15,000-1:20,000 live births. Despite its rarity, Rb has served as a cornerstone in the field of oncology in many of the aspects that comprise cancer management, including classification schemes, treatment modalities, genetic testing and screening. Until just over half a century ago, the major treatment for Rb was eye removal, and prognosis was poor with outcome fatal for most children. The dramatic evolution, in a short period of time across all fields of Rb management, as well as the development of specialized centres, better infrastructure and introduction of awareness campaigns, has resulted in nearly 100% survival in developed countries and allowed eye salvage in many of the cases. External beam radiotherapy was used as the main treatment choice for four decades, but replaced by chemotherapy at the turn of the century. Initially, and still in many centres, chemotherapy is administered intravenously, but recently is targeted directly into the eye by means of intra-ophthalmic artery and intravitreal chemotherapy. To date, a range of treatments is available to the Rb expert, including enucleation, but there is lack of consensus in a number of scenarios as to what to use and when. In such a rare cancer, treatment outcomes are reported usually via retrospective analyses, with few prospective randomized controlled trials. Classification schemes have also evolved following the introduction of new treatment modalities, but discrepancies exist among centres with respect to the preferred schema and its interpretation. Retinoblastoma management is a remarkable success story, but the future will require a collaborative effort in the form of multicentre randomized controlled trials in order to further improve the quality of care for this subset of young children with ocular cancer.

Publication types

  • Review

MeSH terms

  • Child
  • Eye Enucleation / methods
  • Humans
  • Incidence
  • Organ Sparing Treatments / methods
  • Prognosis
  • Retinal Neoplasms / diagnosis
  • Retinal Neoplasms / epidemiology
  • Retinal Neoplasms / genetics
  • Retinal Neoplasms / therapy*
  • Retinoblastoma / diagnosis
  • Retinoblastoma / epidemiology
  • Retinoblastoma / genetics
  • Retinoblastoma / therapy*
  • Treatment Outcome