June 2015
Volume 56, Issue 7
ARVO Annual Meeting Abstract  |   June 2015
Ocular Toxicity of Intravitreal Melphalan for Retinoblastoma
Author Affiliations & Notes
  • Stephen J Smith
    Kellogg Eye Center, Univeristy of Michigan, Ann Arbor, MI
  • Brian Smith
    Hematology/Oncology, University of Rochester, Rochester, NY
  • Footnotes
    Commercial Relationships Stephen Smith, None; Brian Smith, None
  • Footnotes
    Support None
Investigative Ophthalmology & Visual Science June 2015, Vol.56, 76. doi:
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      Stephen J Smith, Brian Smith; Ocular Toxicity of Intravitreal Melphalan for Retinoblastoma. Invest. Ophthalmol. Vis. Sci. 2015;56(7 ):76.

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

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Purpose: To describe the ocular side effects in patients receiving melphalan intravitreal injection therapy (IViT) for retinoblastoma.

Methods: PUBMED (1946-present), SCOPUS (all years), Science Citation Index (1900 - present) and Conference Proceedings Citation Index - Science (1990 - present) electronic databases were searched to identify all published reports of therapeutic intravitreal injections for retinoblastoma in humans.

Results: Eleven studies with original melphalan IViT ocular side effect data were included in this systematic review. In these combined reports, a total of 1311 intravitreal injections were given to 327 eyes of 317 patients. Melphalan IViT doses ranged from 8 to 50 micrograms. Ocular side effects occurred in 50 patients, with 37 patients experiencing side effects likely secondary to melphalan toxicity. The proportion of patients experiencing drug related ocular side effects following melphalan IViT regiments was 0.117 (37/317). Some patients experienced more than one side effect. Drug related side effects in patients receiving doses ranging from 8 to 40 micrograms included salt and pepper retinopathy, ERG reduction, iris atrophy, chorioretinal atrophy, and peripheral lens opacity. Four patients received a single dose of 50 micrograms of melphalan and developed retinal necrosis and gliosis, choroidal congestion, optic nerve atrophy, cataract, and retinal neovascularization.

Conclusions: Ocular toxicity following melphalan IViT occurs in a dose dependent fashion, and can be severe. There appears to be significant variability in assessing and reporting ocular side effects, potentially underestimating drug related toxicity in these patients. Care must be taken in the dosing of intravitreal melphalan treatments to avoid potentially irreversible vision loss.


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