Abstract
Purpose
Intraocular retinoblastoma treatments often combine chemotherapy and focal treatments. A first prospective protocol of conservative treatments in our institution showed the efficacy of the use of two courses of chemoreduction with etoposide and carboplatin, followed by chemothermotherapy using carboplatin as a single agent and diode laser. In order to decrease the possible long-term toxicity of chemotherapy due to etoposide, a randomized neoadjuvant phase II protocol was conducted using vincristine–carboplatin vs etoposide–carboplatin.
Patients and methods
The study was proposed when initial tumor characteristics did not allow front-line local treatments. Patients included in this phase II noncomparative randomized study of neoadjuvant chemotherapy received vincristin–carboplatin (new arm) vs etoposide–carboplatin (our reference arm). They were subsequently treated by local treatments and chemothermotherapy. Primary end point was the need for secondary enucleation or external beam radiotherapy (EBRT) not exceeding 40% at 2 years.
Results
A total of 65 eyes in 55 children were included in the study (May 2004 to August 2009). Of these, 32 eyes (27 children) were treated in the arm etoposide–carboplatin and 33 eyes (28 children) in the arm vincristin–carboplatin. At 2 years after treatment, 23/33 (69.7%) eyes were treated and salvaged without EBRT or enucleation in the arm vincristin–carboplatin and 26/32 (81.2%) in the arm etoposide–carboplatin.
Conclusion
Even if the two treatment arms could be considered as sufficiently active according to the study decision rules, neoadjuvant chemotherapy by two cycles of vincristine–carboplatin followed by chemothermotherapy appear to offer less optimal local control than the etoposide–carboplatin combination.
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Acknowledgements
We thank the parents and the children who participated to the study and the parents and patients association RETINOSTOP (www.retinostop.org) for their help in the preparation of the information and consent forms. We thank the pediatric oncologists of the SFCE (French society of cancer in children) who actively participated in the treatment of these patients. This study received a grant from the PHRC (Programme Hospitalier de Recherche Clinique) supported by the French Ministry of Health and the French National Cancer Institute. The study was declared as clinical trial at INCA (Institut National Cancer: French Institute for Cancer), as clinical study RECF 0151, and at the AFSSAPS (Agence Française de sécurité sanitaire des produits de santé: French agency for the security of drugs) as no. 040188.
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Lumbroso-Le Rouic, L., Aerts, I., Hajage, D. et al. Conservative treatment of retinoblastoma: a prospective phase II randomized trial of neoadjuvant chemotherapy followed by local treatments and chemothermotherapy. Eye 30, 46–52 (2016). https://doi.org/10.1038/eye.2015.179
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DOI: https://doi.org/10.1038/eye.2015.179
