Management of advanced retinoblastoma with intravenous chemotherapy then intra-arterial chemotherapy as alternative to enucleation

Retina. 2013 Nov-Dec;33(10):2103-9. doi: 10.1097/IAE.0b013e318295f783.


Purpose: To determine the efficacy of primary intravenous chemotherapy (IVC) plus secondary intraarterial chemotherapy (IAC) for patients with advanced retinoblastoma.

Methods: Retrospective, nonrandomized interventional case series of 14 patients with retinoblastoma managed with primary systemic IVC (vincristine, etoposide, and carboplatin for 6 cycles) followed by secondary IAC (melphalan for 1-6 cycles).

Results: Fourteen patients with advanced retinoblastoma classified by the International Classification of Retinoblastoma as Group D (n = 6, 43%) or Group E (n = 8, 57%) were treated with IVC as primary treatment and subsequent secondary IAC as rescue or consolidation therapy. The IAC was given for recurrent retinoblastoma and/or subretinal/vitreous seeds in 13 eyes (93%) and for persistent viable retinoblastoma in 1 eye (7%). Enucleation was the alternative option. The mean interval between IVC completion and IAC start was 40 weeks (median, 11 weeks; range, 2-170 weeks) and the mean number of IAC cycles was 3 (median, 3; range, 1-6). After primary IVC plus secondary IAC, globe salvage was achieved in 8 patients (57%) at mean 2-year follow-up. There was no evidence of retinoblastoma metastasis or death and no sign of second cancer or life-threatening complication.

Conclusion: For advanced retinoblastoma (Groups D and E) in which enucleation is the alternative option, primary systemic IVC followed by secondary focal IAC provides globe salvage in 57% of the eyes and with no metastatic event.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents, Alkylating / therapeutic use*
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Carboplatin / therapeutic use
  • Child, Preschool
  • Etoposide / therapeutic use
  • Eye Enucleation*
  • Female
  • Humans
  • Infant
  • Infusions, Intra-Arterial
  • Infusions, Intravenous
  • Male
  • Melphalan / therapeutic use*
  • Retinal Neoplasms / drug therapy*
  • Retinal Neoplasms / pathology
  • Retinoblastoma / drug therapy*
  • Retinoblastoma / pathology
  • Retrospective Studies
  • Treatment Outcome
  • Vincristine / therapeutic use


  • Antineoplastic Agents, Alkylating
  • Vincristine
  • Etoposide
  • Carboplatin
  • Melphalan

Supplementary concepts

  • CEV regimen