Comparing efficacy and side effects of two systemic chemotherapy regimens for eye‐preserving therapy in children with retinoblastoma

Abstract

Background: Eye-preserving therapy in retinoblastoma comprises systemic chemotherapy, but studies analyzing the efficacy of different chemotherapy regimens are scarce. Methods:The efficacy and side effects of two different eye-preserving chemotherapy regimens containing either vincristine, etoposide, and carboplatin (VEC) or cyclophosphamide, vincristine, etoposide, and carboplatin (CyVEC) were compared in a prospective non-interventional observational study including children diagnosed

Evidence in Focus: The Sparse Landscape of Randomized Trials on Retinoblastoma Treatment

“…This falls in the 40–82% range previously reported [35‒39], although a direct comparison is difficult because of differences in the examined populations and treatment protocols. Regarding eye salvage, Lumbroso-Le Rouic et al [25] found an event-free and an overall eye survival rate of 75% and 78%, respectively, which came in accordance with the findings of a recent German and Austrian study [31]. The 62.5% event-free eye survival rate in patients with group D/E retinoblastoma found by Muhammed et al [27] was higher than previously reported [29, 31], but this could be explained by the shorter follow-up in this study.…”

“…Few associations were nominally statistically significant at the p = 0.05 level, and all of them pertained to the comparison between two drug combinations performed by Chawla et al [24]. The VEC protocol, the most used chemotherapeutic protocol for retinoblastoma [12, 13, 31], was associated with lower disease progression and mortality rates as well as less adverse events compared to a 5-drug combination consisting of carboplatin and etoposide, alternating with cyclophosphamide, idarubicin, and vincristine. A further comparison between different protocols showed that etoposide and carboplatin might offer better tumor control than vincristine and carboplatin when used as neoadjuvant chemotherapy, without an increase in severe toxicity [25].…”

“…Current indications for intravenous chemotherapy include bilateral or germline disease, positive family history, optic nerve, choroidal or scleral invasion, and extraocular retinoblastoma [12,13]. Multiple combinations of 2-5 chemotherapeutic agents including vincristine, carboplatin, etoposide, cyclophosphamide, doxorubicin, and idarubicin have been used as neoadjuvant or adjuvant treatment 31,36,42]. Although no definitive consensus has been reached, triple therapy with VEC is the combination of choice in most centers [12,43], having shown better results compared to other protocols in terms of efficacy and safety [24,31].…”

“…Multiple combinations of 2-5 chemotherapeutic agents including vincristine, carboplatin, etoposide, cyclophosphamide, doxorubicin, and idarubicin have been used as neoadjuvant or adjuvant treatment 31,36,42]. Although no definitive consensus has been reached, triple therapy with VEC is the combination of choice in most centers [12,43], having shown better results compared to other protocols in terms of efficacy and safety [24,31]. The standard doses are: vincristine 1.5 mg/m 2 , day 1 of each cycle; etoposide 150 mg/m 2 , days 1 and 2; carboplatin 560 mg/m 2 , day 1 [43].…”

Long-Term Results after Intraocular Surgery in Treated Retinoblastoma Eyes

“…Long-term results included gain in visual acuity, defined as an improvement of 2 or more lines, local recurrences, development of second primary malignancies (SPM), depending on the treatment modalities, and metastatic disease. Systemic chemotherapy was administered as a combination of vincristine, etoposide, and carboplatin and, in some cases, supplemented by cyclophosphamide, whereby 75% of the patients got more than 5 treatment cycles [8]. The apex dose for brachytherapy with 106 ruthenium plaques was initially selected at 138 Gy, followed by a reduction to 88 Gy at the tumor apex from 2004.…”