Conservative treatment of retinoblastoma: a prospective phase II randomized trial of neoadjuvant chemotherapy followed by local treatments and chemothermotherapy

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 longterm toxicity of chemotherapy due to etoposide, a randomized neoadjuvant phase II protocol was conducted using vincristinecarboplatin 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 etoposidecarboplatin combination.

Treatment of retinoblastoma in Sub‐Saharan Africa: Experience of the paediatric oncology unit at Gabriel Toure Teaching Hospital and the Institute of African Tropical Ophthalmology, Bamako, Mali

“…Indications of different conservative treatments were decided according to medical evaluation of each eye and the presence or absence of vitreous seeding. [38][39][40] We did not observe any orbital relapse after intravitreous injection in our patients. Bilateral intraocular Rb accounted for 27.3% of patients in our cohort (n = 24) with a median age of 1 year, similar to the age observed in industrialised countries.…”

“…We used thermo‐chemotherapy and intravitreous melphalan. Indications of different conservative treatments were decided according to medical evaluation of each eye and the presence or absence of vitreous seeding . We did not observe any orbital relapse after intravitreous injection in our patients.…”

Looking for the Most Suitable Orthotopic Retinoblastoma Mouse Model in Order to Characterize the Tumoral Development

“…3 Nevertheless, in order to minimize the risk of complications due to treatment, the development of new therapeutic approaches is still required. For this purpose, studies on animal models are a compulsory step before considering clinical trials.…”

Combination of Carboplatin and Bevacizumab Is an Efficient Therapeutic Approach in Retinoblastoma Patient-Derived Xenografts

“…3 In contrast, children with less advanced unilateral Rb or bilateral Rb undergo anticancer therapy to avoid enucleation or blindness, such as intravenous chemotherapy combined with local treatments (e.g., laser cryotherapy) or intra-arterial/ intravitreous chemotherapy, or very rarely today, external beam radiotherapy (EBRT). 4,5 Currently, carboplatin is a standard of care in Rb patients treated with intravenous chemotherapy and is included in main chemotherapy protocols. [5][6][7][8] However, patients with constitutional Rb gene abnormality who survive an ocular tumor have a higher risk of secondary cancers, especially sarcomas, mostly occurring in the irradiated field.…”

“…4,5 Currently, carboplatin is a standard of care in Rb patients treated with intravenous chemotherapy and is included in main chemotherapy protocols. [5][6][7][8] However, patients with constitutional Rb gene abnormality who survive an ocular tumor have a higher risk of secondary cancers, especially sarcomas, mostly occurring in the irradiated field. 9,10 Hence, considerable efforts have been made to avoid external beam irradiation as much as possible.…”