Efficacy and complications of super-selective intra-ophthalmic artery melphalan for the treatment of refractory retinoblastoma

Ophthalmology. 2012 Mar;119(3):611-6. doi: 10.1016/j.ophtha.2011.08.045. Epub 2011 Dec 22.


Purpose: To report the efficacy of super-selective intra-ophthalmic artery melphalan (IAM) for the treatment of refractory retinoblastoma and any associated complications of this treatment.

Design: A prospective case series.

Participants: Eyes with retinoblastoma that had been treated with systemic chemotherapy or local therapy and had a relapse of their condition.

Methods: All patients receiving IAM between May 2009 and September 2010 were included in the study. Intra-ophthalmic artery melphalan was offered to patients who had failed to respond adequately to systemic chemotherapy and local treatment where appropriate or because of a new recurrence of retinoblastoma that could not be treated with local therapies. None of the patients were excluded because of central nervous system abnormalities. Patients received 2 treatments of IAM given 4 weeks apart. All patients received an orthoptic assessment 3 weeks after each treatment and an examination under anesthesia (EUA). A third treatment was given if an unsatisfactory response was observed on EUA after 2 treatments.

Main outcome measures: The response of the retinoblastoma tumor(s) and any associated local side effects from the treatment.

Results: A total of 15 eyes in 14 patients were treated with IAM during the study period. The mean age at the time of IAM was 31.5 months (median 17.3, range 11.2-150.7 months), and the mean follow-up was 8.7 months (3-16.3 months). Tumor control was achieved in 12 eyes (80%), and 12 eyes (80%) had local side effects that included third cranial nerve palsy in 6 (40%), orbital edema in 3 (20%), permanent retinal detachment in 1 (7%), and vitreous hemorrhage in 4 (27%). Seven eyes (47%) developed significant retinal pigment epithelium changes.

Conclusions: Intra-ophthalmic artery melphalan is an effective treatment for retinoblastoma, achieving a high level of remission in refractory tumors. It can be associated with significant local side effects that can result in loss of vision and possible amblyogenesis. Clinicians and parents need to consider the benefits and potential local side effects before embarking on treatment.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antineoplastic Agents, Alkylating / administration & dosage*
  • Antineoplastic Agents, Alkylating / adverse effects
  • Child
  • Child, Preschool
  • Follow-Up Studies
  • Humans
  • Infant
  • Infusions, Intra-Arterial
  • Melphalan / administration & dosage*
  • Melphalan / adverse effects
  • Ophthalmic Artery / drug effects*
  • Prospective Studies
  • Retinal Neoplasms / drug therapy*
  • Retinal Neoplasms / pathology
  • Retinoblastoma / drug therapy*
  • Retinoblastoma / pathology
  • Treatment Outcome


  • Antineoplastic Agents, Alkylating
  • Melphalan