This article is more than 5 years old. Information may no longer be current.
Topotecan/vincristine improved outcomes for children with intraocular retinoblastoma
Click Here to Manage Email Alerts
The combination of topotecan and vincristine for the treatment of intraocular retinoblastoma in children resulted in a high rate of response with manageable toxicity, according to the results of a prospective phase 2 study.
Researchers chose to examine topotecan in this disease state because of its efficacy on other central nervous system cancers, and the similarities between the blood-brain barrier and the blood-ocular barrier.
The study included 27 patients with bilateral retinoblastoma in at least one eye. The median age of patients was 8.1 months.
Treatment included topotecan started at a dose of 3 mg/m2 per day for 5 days. Dose was adjusted to meet a target systemic exposure. Patients aged younger than 12 months received vincristine 0.05 mg/kg, while those older than 12 months received vincristine 1.5 mg/m2. All but one patient completed the entire course of therapy. The median time required to complete the therapy was 47 days.
Ibrahim Qaddoumi
After treatment, 23 patients had sustained, confirmed partial response in both eyes and one patient had a partial response in one eye, for an overall response rate of 88.9% (95% CI, 71.3-96.9). All study patients experienced hematologic toxicity during therapy. The toxicities included grade-4 neutropenia (n=27), grade-3 anemia (n=19) and grade-3/grade-4 thrombocytopenia (n=16).
Although the study tested the combination of topotecan and vincristine, the researchers said they “believe that most of the observed response is due to topotecan.”
This data, which provides a toxicity profile of topotecan in the youngest age group yet reported, should be helpful in guiding treatment in patients of this age group, the researchers added.
References:
- Qaddoumi I. Cancer. 2012;doi:10.1002/cncr.27563.