Persistence of retinal function after intravitreal melphalan injection for retinoblastoma

Doc Ophthalmol. 2013 Feb;126(1):79-84. doi: 10.1007/s10633-012-9358-6. Epub 2012 Nov 11.


Background: The risk/benefit profile of intravitreal melphalan injection for treatment of active vitreous seeds in retinoblastoma remains uncertain. We report clinical and electroretinography results after 6 months of one patient who has shown a favorable initial clinical response to intravitreal melphalan injections for treatment of refractory vitreous seeds.

Methods: Clinical case report.

Patient: The patient presented at age 17 months with bilateral retinoblastoma [OD: International Classification (ICRB) group E, Reese-Ellsworth (R-E) class Vb; OS: ICRB D, R-E Vb] with no known prior family history. The right eye was enucleated primarily. The patient received systemic chemotherapy and extensive local treatment to the left eye. Ten months later, she presented with recurrent disease, including fine, diffuse vitreous seeds. Tumor control was established with intra-arterial chemotherapy and local treatment. Subsequent recurrence was treated with further intra-arterial chemotherapy, local treatment, and plaque radiotherapy with iodine-125. Persistent free-floating spherical vitreous seeds were treated with 4 cycles of intravitreal melphalan injection via the pars plana, with doses of 30, 30, 30, and 20 μg.

Results: After 6 months of follow-up, the left eye remained free of active tumor. Visual acuity was 20/40. Photopic ERGs amplitudes were unchanged compared with those recorded prior to the intravitreal injection treatments.

Conclusions: Intravitreal melphalan injection for refractory spherical vitreous seeds of retinoblastoma with favorable tumor response is compatible with good central visual acuity and preservation of retinal function as indicated by photopic ERG recordings.

Publication types

  • Case Reports

MeSH terms

  • Antineoplastic Agents, Alkylating / administration & dosage
  • Electroretinography / drug effects*
  • Female
  • Follow-Up Studies
  • Humans
  • Infant
  • Intravitreal Injections
  • Melphalan / administration & dosage*
  • Ophthalmoscopy
  • Retina / drug effects
  • Retina / pathology
  • Retina / physiopathology*
  • Retinal Neoplasms / drug therapy
  • Retinal Neoplasms / pathology
  • Retinoblastoma / drug therapy*
  • Retinoblastoma / pathology
  • Retinoblastoma / physiopathology


  • Antineoplastic Agents, Alkylating
  • Melphalan