Current and emerging therapy for improving outcomes in patients with intraocular retinoblastoma

Rachel C. Brennan, Eleanor M. Pritchard, R. Kiplin Guy, Michael A. Dyer, Matthew W. Wilson

Research output: Contribution to journalReview articlepeer-review


Introduction: Survival for patients with intraocular retinoblastoma (RB) is excellent. Over the past two decades, the focus of therapy has shifted to emphasize ocular salvage and vision preservation. The combination of chemoreduction and aggressive focal therapies has formed the new standard approach to most patients with advanced intraocular RB. However, seeding of the vitreous cavity continues to be the primary reason for treatment failure in these patients.Areas covered: Local delivery methods, including periocular injections, intra-ophthalmic artery infusions, and intravitreal injections, have been developed in the hope of controlling disease in advanced cases. This review highlights the current approach to therapy for patients and discusses these new local delivery techniques and the discovery of novel therapeutics that may help to further improve ocular outcomes for patients with intraocular RB.Expert opinion: Due to the rarity of this childhood disease, clinical efforts to improve ocular salvage and vision preservation must occur in parallel with translational research efforts that identify novel strategies for therapy of RB. A dedicated RB team provides guidance for a coordinated approach to address the key therapeutic, biologic, and long-term challenges in our patients.

Original languageEnglish
Pages (from-to)1155-1166
Number of pages12
JournalExpert Opinion on Orphan Drugs
Issue number10
StatePublished - Oct 3 2015

Bibliographical note

Funding Information:
This work has been supported in part by the American Lebanese Syrian Associated Charities (ALSAC) and National Institutes of Health grant numbers CA21765 and CA23099. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Publisher Copyright:
© 2015 Informa UK, Ltd.


  • chemoreduction
  • intraocular retinoblastoma
  • local delivery of ocular therapy
  • novel therapy
  • ocular salvage
  • targeted therapy

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)


Dive into the research topics of 'Current and emerging therapy for improving outcomes in patients with intraocular retinoblastoma'. Together they form a unique fingerprint.

Cite this