June 2013
Volume 54, Issue 15
ARVO Annual Meeting Abstract  |   June 2013
Shifting Treatment Paradigms in Retinoblastoma: Dilemmas for the clinician in evaluating the use of novel therapies
Author Affiliations & Notes
  • Timothy Murray
    Ophthalmology, Murray Ocular Oncology and Retina, Miami, FL
    Ophthalmology, Miami Childrens Hospital, Miami, FL
  • Footnotes
    Commercial Relationships Timothy Murray, None
  • Footnotes
    Support None
Investigative Ophthalmology & Visual Science June 2013, Vol.54, 3975. doi:
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      Timothy Murray; Shifting Treatment Paradigms in Retinoblastoma: Dilemmas for the clinician in evaluating the use of novel therapies. Invest. Ophthalmol. Vis. Sci. 2013;54(15):3975.

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      © ARVO (1962-2015); The Authors (2016-present)

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To evaluate shifting treatment paradigms in the management of children with retinoblastoma over the last three decades. Focus on the integration of evolving pilot clinical data and basic/translational research data on a clinicians choice of primary therapy.


IRB approved, retrospective review of children treated between 1990 and 2012 with retinoblastoma by a single surgical ocular oncologist. Demographics included age at presentation, staging of disease, unilaterality versus bilaterality, familial versus non-familial and date of institution of primary therapy by the treating surgeon. All children underwent pre-treatment ocular and CNS imaging, examination under anesthesia and ongoing followup including serial EUA and awake examinations. Survival status, anatomic globe status and visual function were serially obtained. Treatments were grouped in three time intervals to evaluate treatment trends during the study window reported as 1990's, 2000's and 2010's.


272 children were primarily treated with retinoblastoma, 150 boys and 122 girls. Mean age at presentation was 14 months (range birth to 18 years). 108 children presented with bilateral disease (39%) with virtually all children presenting prior to three months developing bilateral eye involvement. In the initial review, Treatment evolution for primary therapy: 1990's, 85% of children treated with external beam radiotherapy (EBRT) and 25% enucleated, 2000's 85% of children treated with systemic chemotherapy and 10% enucleated, and 2010's 90 of children treated with focal chemotherapy (intra-arterial/peri-ocular) and less then 5% enucleated (p<.01). Primary retinoblastoma mortality rates were less then 1% for the entire study window (no deaths within the last 15 years (p<.005).


Retinoblastoma treatment has undergone significant shifts related to our ongoing understanding of molecular genomics, pathophysiology, treatment options, and treatment related morbidity. Over three decades treatment has moved from enucleation to external beam radiotherapy, to systemic chemotherapy to focal chemotherapy (including intra-arterial chemotherapy, peri-ocular chemotherapy and intra-vitreal chemotherapy). Ultimately, the evolution of treatment for retinoblastoma has remarkably improved survival outcomes, globe preservation, and visual function.

Keywords: 703 retinoblastoma • 462 clinical (human) or epidemiologic studies: outcomes/complications • 744 tumors  

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