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Topotecan and vincristine combination is effective against advanced bilateral intraocular retinoblastoma and has manageable toxicity†
Version of Record online: 19 APR 2012
Copyright © 2012 American Cancer Society
Volume 118, Issue 22, pages 5663–5670, 15 November 2012
How to Cite
Qaddoumi, I., Billups, C. A., Tagen, M., Stewart, C. F., Wu, J., Helton, K., McCarville, M. B., Merchant, T. E., Brennan, R., Free, T. M., Given, V., Haik, B. G., Rodriguez-Galindo, C. and Wilson, M. W. (2012), Topotecan and vincristine combination is effective against advanced bilateral intraocular retinoblastoma and has manageable toxicity. Cancer, 118: 5663–5670. doi: 10.1002/cncr.27563
This work was presented in part at the annual meeting of the American Academy of Ophthalmology, October 2009, San Francisco, CA; the American Society of Clinical Oncology, June 2011, Chicago, IL; and at the International Society of Ocular Oncology, November 2011; Buenos Aires, Argentina.
- Issue online: 30 OCT 2012
- Version of Record online: 19 APR 2012
- Manuscript Accepted: 22 FEB 2012
- Manuscript Revised: 27 JAN 2012
- Manuscript Received: 22 DEC 2011
- first year of life;
New, effective chemotherapeutic agents are needed for intraocular retinoblastoma.
This institutional clinical trial sought to estimate the rate of response to 2 courses of vincristine and topotecan (VT) window therapy in patients with bilateral retinoblastoma and advanced disease (Reese-Ellsworth group IV or V) in at least 1 eye. The topotecan dose started at 3 mg/m2/day for 5 days and was adjusted to target a systemic exposure of 140 ± 20 ng/mL · hour. The vincristine dose was 0.05 mg/kg for patients <12 months of age and 1.5 mg/m2 for those >12 months of age at diagnosis.
From February 2005 to June 2010, 27 patients received VT window therapy. Median age at enrollment was 8.1 months (range, 0.7-22.1 months). Twenty-four patients (88.9%) responded to window therapy (95% confidence interval = 71.3%-96.9%). Hematologic toxicity comprised grade 4 neutropenia (n = 27), grade 3 anemia (n = 19), and grade 3/4 thrombocytopenia (n = 16). Thirteen patients had grade 3 nonhematologic toxicity. Granulocyte colony-stimulating factor support was added after 10 patients had been treated, and it significantly reduced the duration of grade 4 neutropenia (median, 7 vs 24 days; P < .001). Pharmacokinetic studies showed rapid changes in topotecan clearance rates during the first year of life.
The combination of topotecan and vincristine is effective for the treatment of advanced intraocular retinoblastoma. Granulocyte colony-stimulating factor treatment alleviates the duration of grade 4 neutropenia. Appropriate topotecan starting doses for patients 0-3, 3-6, 6-9, 9-12, and >12 months of age are specified. Cancer 2012. © 2012 American Cancer Society.