Treatment of intraocular retinoblastoma with vincristine and carboplatin

J Clin Oncol. 2003 May 15;21(10):2019-25. doi: 10.1200/JCO.2003.09.103.


Purpose: To evaluate the efficacy of chemoreduction using vincristine and carboplatin in preventing or delaying external-beam radiotherapy (EBRT) or enucleation in patients with intraocular retinoblastoma.

Patients and methods: Twenty-five patients (43 eyes) with newly diagnosed intraocular retinoblastoma received primary treatment with eight courses of vincristine and carboplatin. Focal treatments were delayed until documentation of disease progression. Outcome measures for each eye were length of time to disease progression, avoidance or delay of EBRT, and globe survival. Event-free survival was defined as the length of time to EBRT or enucleation.

Results: Disease in all eyes responded to chemotherapy and progressed in only two patients before completion of the eight courses of therapy. Disease in all but four eyes progressed and required focal treatments. Event-free survival estimates at 2 years were 59.2% +/- 12.0% for Reese-Ellsworth group I, II, and III eyes and 26.3% +/- 9.2% for group IV and V eyes. Nineteen eyes (44.2%) required EBRT and 13 eyes (30.2%) were enucleated. The ocular salvage rate was 83.3% for Reese-Ellsworth group I to III eyes and 52.6% for group IV and V eyes. For those patients receiving EBRT, the median time from enrollment to EBRT was 9.5 months (median age at EBRT, 21 months).

Conclusion: In combination with appropriate early intensive focal treatments, chemoreduction with vincristine and carboplatin, without etoposide, may be an alternative treatment for patients with early-stage intraocular retinoblastoma, although additional studies are needed. Patients with advanced intraocular disease require more aggressive treatments.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Carboplatin / administration & dosage
  • Child, Preschool
  • Disease Progression
  • Disease-Free Survival
  • Eye Enucleation
  • Female
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Neoplasm Staging
  • Prospective Studies
  • Radiotherapy, Adjuvant
  • Retinal Neoplasms / drug therapy*
  • Retinal Neoplasms / pathology
  • Retinal Neoplasms / radiotherapy
  • Retinal Neoplasms / surgery
  • Retinoblastoma / drug therapy*
  • Retinoblastoma / pathology
  • Retinoblastoma / radiotherapy
  • Retinoblastoma / surgery
  • Tennessee
  • Treatment Outcome
  • Vincristine / administration & dosage


  • Vincristine
  • Carboplatin