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Pathologic Risk-based Adjuvant Chemotherapy for Unilateral Retinoblastoma Following Enucleation

Sullivan, Erin M. MD*; Wilson, Matthew W. MD†,‡,§; Billups, Catherine A. MS; Wu, Jianrong PhD; Merchant, Thomas E. DO, PhD; Brennan, Rachel C. MD*,§,#; Haik, Barrett G. MD‡,§; Shulkin, Barry MD; Free, Tammy M. CCRP**; Given, Vickie CRA-RN**; Rodriguez-Galindo, Carlos MD*,#; Qaddoumi, Ibrahim MD, MS*,#

Journal of Pediatric Hematology/Oncology: August 2014 - Volume 36 - Issue 6 - p e335–e340
doi: 10.1097/MPH.0000000000000141
Online Articles: Original Articles

Background: There are no standardized diagnostic or treatment guidelines for patients with advanced unilateral retinoblastoma.

Materials and Methods: Patients with advanced unilateral retinoblastoma were prospectively treated after enucleation using a risk-based protocol. Patients were assigned to low risk (LR), intermediate risk (IR), or high risk (HR) based on pathology. LR patients underwent observation. IR patients received 4 courses of chemotherapy with vincristine, doxorubicin, and cyclophosphamide (VDC). In the HR group, patients received 3 courses of VDC alternating with 3 courses of vincristine, carboplatin, and etoposide (VCE) and irradiation when indicated.

Results: Fifty patients with advanced unilateral retinoblastoma were treated (LR, n=36; IR, n=7; HR, n=7). All eyes were Reese-Ellsworth group V. All bone scans (n=81), lumbar punctures (n=16), and bone marrow aspirates (n=16) were negative. Chemotherapy was well tolerated. Grades 3/4 hematologic toxicities were seen in all patients; grades 3/4 nonhematologic toxicities were seen in half the patients. Only one patient in the HR group received radiation therapy. All patients were alive at the time of analysis with no signs of disease recurrence. Median follow-up was 3.4 years (range, 0.8 to 6.4 y).

Conclusions: Patients with nonmetastatic unilateral retinoblastoma undergoing primary enucleation can be cured with a graduated intensity approach based on pathology.

*Departments of Oncology




Radiological Sciences

**Cancer Center Administration, St. Jude Children’s Research Hospital

§Departments of Ophthalmology

#Pediatrics, University of Tennessee Health Sciences Center, Memphis, TN

This work was presented at the American Society of Pediatric Hematology Oncology (ASPHO) in Baltimore, MD 2011 and at the International Society of Ocular Oncology (ISOO) in Buenos Aires, Argentina 2011.

This work was supported by grants CA21765 and CA23099 from the National Institutes of Health, by the American Lebanese Syrian Associated Charities (ALSAC), and by Research to Prevent Blindness Inc., and St. Giles Foundation.

The authors declare no conflict of interest.

Reprints: Ibrahim Qaddoumi, MD, MS, St. Jude Children’s Research Hospital, 262 Danny Thomas Place, Memphis, TN 38105 (e-mail:

Received April 16, 2013

Accepted February 4, 2014

© 2014 by Lippincott Williams & Wilkins.