A 10‐year experience of outcome in chemotherapy‐treated hereditary retinoblastoma

Abstract

ABSTRACT.Purpose: The aim is to report the 10-year retrospective experience of systemic chemotherapy for a population-based group of patients with hereditary retinoblastoma at a national referral centre. The outcomes include control rates, treatment side-effects, adjuvant therapy, failure rate, survival, secondary cancers and visual acuity. Methods: All patients (n = 24, 46 eyes) diagnosed with retinoblastoma and treated with systemic chemotherapy at a national referral centre during 2001-2011 were included. Data were extracted from medical records. Results: The patients were followed for a mean of 60 months (range 13-144). Four-six cycles of VEC was administered to all newly diagnosed group B/C/D/ E eyes with bilateral disease and 83% (38 of 46) responded to the treatment. None of the patients discontinued chemotherapy because of adverse reactions. Altogether 26% (12 of 46) of the eyes received second-line therapy (other than thermotherapy, cryotherapy and chemotherapy). The failure rate was 35% (16 of 46) and mortality rate 0%. None of the patients developed CNS manifestations (metastases or trilateral retinoblastoma). One of the patients developed a second primary tumour (osteosarcoma) 4 years following retinoblastoma diagnosis. Altogether 17% (4 of 24) patients received radiation therapy, 28% (13 of 46) of the eyes had to be enucleated, and one patient underwent bilateral enucleation. The age-correlated visual acuity was mean of 73% of expected visual acuity. Conclusion: Group A/B retinoblastomas have a distinct chemotherapy response, while group C/D/E tumours do not respond as well. The success rate was 65%; while patients have a good prognosis for life, approximately one-third of all hereditary cases received radiation therapy or underwent enucleation.

Integrated Treatment during the Intravitreal Melphalan Era: Concurrent Intravitreal Melphalan and Systemic Chemoreduction

“…It is important to note that unlike intra-arterial chemotherapy [20], systemic chemotherapy alone has not been associated with retinal toxicity [21]. Intravitreal delivery of chemotherapy is not without its own set of side effects, including retinal toxicity as described by Munier [14].…”

Immunoreactivity of the 14F7 Mab raised against N‐Glycolyl GM3 Ganglioside in retinoblastoma tumours

“…(2) As a target-specific delivery of therapeutic agents for the treatment of retinoblastoma: In recent years, increased interest in local drug delivery was evident for retinoblastoma, including subconjunctival, intravitreal routes and intra-arterial chemotherapy (Abramson et al 2008;Bartuma et al 2013;Venturi et al 2013;Parareda et al 2014). To our knowledge, only conventional chemotherapy agents were given by these routes for retinoblastoma in the clinic.…”

Classification of Vitreous Seeds in Retinoblastoma

“…For the past several decades, over half of the retinoblastoma eyes with vitreous seeds would have been enucleated due to inadequate response rates to historical treatments 6,7 . However, the increased use of OAC alone, or in conjunction with intravitreal chemotherapy, now enables us to salvage a vast majority of eyes with vitreous seeds 8–11 .…”