A TRIAL OF ADJUVANT CHEMOTHERAPY IN PATIENTS WITH INTRAOCULAR RETINOBLASTOMA

Trial Information

A TRIAL OF ADJUVANT CHEMOTHERAPY IN PATIENTS WITH INTRAOCULAR RETINOBLASTOMA

OBJECTIVES: I. Estimate the response rate to adjuvant carboplatin (CBDCA) alone in children
with intraocular retinoblastoma. II. Assess the overall survival, disease-free survival, and
time to treatment failure in these patients. III. Attempt to maintain a 90% disease-free
survival rate with this treatment. IV. Estimate the percentage of disease-free survivors who
do not require the use of radiotherapy after adjuvant CBDCA. V. Assess the toxicity of this
treatment. VI. Estimate the percentage of functional eyes salvaged using this
radiation-sparing protocol and compare these results, in a nonrandomized manner, with
historical treatment results in this population.

OUTLINE: Patients are stratified according to vitreous disease (yes vs no). Patients receive
carboplatin IV over 1 hour on day 0. Treatment repeats every 2-3 weeks for 2 courses.
Patients achieving complete response (CR) proceed to local control measures consisting of
cryotherapy, laser photocoagulation, etc. (except brachytherapy). Patients not achieving CR
continue with carboplatin until local control measures feasible. Patients with vitreous
disease achieving CR receive 2 additional courses and then receive local control measures.
Patients with vitreous disease not achieving CR receive intensive chemotherapy. Patients
receive intensive chemotherapy consisting of vincristine IV on days 0, 7, and 14, cisplatin
IV over 6 hours on day 0, etoposide IV on days 1 and 2, followed by cyclophosphamide IV over
60 minutes on days 1 and 2. Patients receive filgrastim (G-CSF) subcutaneously beginning on
day 3 and until blood counts recover. Treatment repeats every 2-4 weeks for 2 courses beyond
CR. Patients are followed for 3 years.

PROJECTED ACCRUAL: A total of 50 patients will be accrued for this study over 5-6 years.