Intra-arterial chemotherapy for the management of retinoblastoma: four-year experience

Arch Ophthalmol. 2011 Jun;129(6):732-7. doi: 10.1001/archophthalmol.2011.5. Epub 2011 Feb 14.


Objective: To determine whether intra-arterial chemotherapy is safe and effective in advanced intraocular retinoblastoma. Retinoblastoma often presents with advanced intraocular disease and, despite conventional treatment with intravenous chemotherapy and external beam radiation therapy, may still require enucleation.

Design: Single-arm, prospective registry from May 30, 2006, to May 30, 2010, at an ophthalmic oncology referral center with ambulatory care. A total of 95 eyes of 78 patients with unilateral or bilateral retinoblastoma were treated. The intervention was selective catheterization of the ophthalmic artery and injection of chemotherapy, usually melphalan with or without topotecan. Drug dosage was determined by age and angioanatomy. The main outcome measures were procedural success, event-free (enucleation or radiotherapy) ocular survival, and ocular and extraocular complications.

Results: Catheterization succeeded in 98.5% of procedures. There were 289 chemotherapy injections (median, 3 per eye). The Kaplan-Meier estimates of ocular event-free survival rates at 2 years were 70.0% (95% confidence interval, 57.9%-82.2%) for all eyes, 81.7% (95% confidence interval, 66.8%-96.6%) for eyes that received intra-arterial chemotherapy as primary treatment, and 58.4% (95% confidence interval, 39.5%-77.2%) for eyes that had previous treatment failure with intravenous chemotherapy and/or external beam radiation therapy. There were no permanent extraocular complications.

Conclusion: Our experience suggests that intra-arterial chemotherapy is safe and effective in the treatment of advanced intraocular retinoblastoma.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Comparative Study
  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Antineoplastic Agents / administration & dosage*
  • Carboplatin / administration & dosage*
  • Child
  • Child, Preschool
  • Dose-Response Relationship, Drug
  • Female
  • Follow-Up Studies
  • Humans
  • Infant
  • Infant, Newborn
  • Injections, Intra-Arterial
  • Male
  • Ophthalmic Artery
  • Prospective Studies
  • Retina / drug effects
  • Retina / pathology*
  • Retinal Neoplasms / drug therapy*
  • Retinal Neoplasms / pathology
  • Retinoblastoma / drug therapy*
  • Retinoblastoma / pathology
  • Treatment Outcome
  • Young Adult


  • Antineoplastic Agents
  • Carboplatin