Emerging New Therapeutics for Retinoblastoma

Raval, Vishal; Parulekar, Manoj; Singh, Arun D.

doi:10.1159/000524919

Cited by 5 publications

(1 citation statement)

References 47 publications

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“…In preclinical studies, the topical instillation of cell-penetrating peptide-conjugated melphalan has demonstrated efficacy comparable to intravitreal melphalan, with the added advantage of reducing the risk of metastasis [59]. Furthermore, the current trend in research has shifted toward exploring non-chemotherapeutic alternatives, including targeting specific molecular pathways such as MDMX-p53 and OTX2, immunotherapy, and genetic modulation through oncolytic adenoviruses [56,60,61]. These innovative approaches signify a broader exploration of treatment avenues beyond traditional chemotherapy.…”

Section: 2)mentioning

confidence: 99%

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

Lavasidis,

Papaioannou,

Anagnostou

et al. 2024

Ocul Oncol Pathol

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Background: Retinoblastoma, although rare, is one of the most common intraocular malignancies worldwide. Its prognosis has improved significantly in the past few decades, thanks to modern treatments, like systemic, intra-arterial, and intravitreal chemotherapy. However, regarding survival, there are significant differences between high- and low-income countries, eye salvage is still a challenge worldwide, and treatment-related toxicity needs to be carefully and sufficiently managed. Summary: To appraise the strength of supporting evidence, we performed a systematic review of randomized controlled trials investigating any therapeutic protocol for retinoblastoma. Four trials with 174 participants (188 eyes) were eligible, all pertaining to different intravenous chemotherapy regimens. Vincristine, etoposide, and carboplatin (VEC) appear superior to a 5-drug combination for stage III retinoblastoma. Moreover, etoposide and carboplatin as neoadjuvant chemotherapy followed by thermochemotherapy seem to offer better local control than vincristine and carboplatin. However, increasing carboplatin dose in the VEC protocol failed to improve treatment efficacy. Key Messages: Retinoblastoma is a success story of modern medicine. However, only intravenous chemotherapy has been studied through randomized trials, while evidence for the most novel retinoblastoma treatments has mainly stemmed from observational studies. International collaborations for multicenter randomized trials could overcome difficulties and increase certainty and precision in the field.

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Section: 2)mentioning

confidence: 99%

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

Lavasidis,

Papaioannou,

Anagnostou

et al. 2024

Ocul Oncol Pathol

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Survival and Health Care Burden of Children With Retinoblastoma in Europe

Virgili,

Capocaccia,

Botta

et al. 2024

JAMA Ophthalmol

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ImportanceStudies on the epidemiology of retinoblastoma (RB) could lead to improvement in management.ObjectiveTo estimate the incidence and survival of RB in European children and the occurrence of second primary tumors (other than RB) in these patients.Design, Setting, and ParticipantsThis cohort study used population-based data from 81 cancer registries in 31 European countries adhering to the European Cancer Registries (EUROCARE-6) project. Data collection took place between January 2000 and December 2013. European children aged 0 to 14 years diagnosed with RB were included. Data were analyzed from May to November 2023.ExposuresDiagnosis of RB with International Classification of Diseases for Oncology, Third Edition (ICD-O-3), morphology coded 9510-9514 (retinoblastoma) and malignant behavior (fifth digit of morphology code, 3).Main Outcome and MeasuresAnnual incidence (per million children aged 0-14 years), 5-year survival (%), and the standardized incidence ratio (SIR) of subsequent malignant neoplasms.ResultsThe study included 3262 patients (mean [SD] age, 1.27 [1.63] years; 1706 [52%] male and 1556 [48%] female) from 81 registries. Of these, 3098 patients were considered in trend analysis after excluding registries with incomplete time coverage: 940 in 2000 to 2003, 703 in 2004 to 2006, 744 in 2007 to 2009, and 856 in 2010 to 2013. The estimated overall European incidence rate was 4.0 (95% CI, 3.9-4.1). Rates among countries varied from less than 2 million to greater than 6 million per year. No time trend of incidence was observed in any area. The overall European 5-year survival was 97.8% (95% CI, 95.5-98.9; 3180 cases). Five-year survival was lower in Estonia and Bulgaria (&lt;80%) and 100% in several countries. Twenty-five subsequent malignant neoplasms were recorded during follow-up (up to 14 years), with an SIR of 8.2 and with cases occurring at mean ages between 1.3 and 8.9 years across different sites. An increased risk was found for hematological tumors (SIR, 5) and bone and soft tissue sarcomas (SIR, 29).Conclusions and RelevanceThis study showed RB incidence remained stable at 4.0 per 1 000 000 European children aged 0 to 14 years from 2000 to 2013, but estimates varied among countries and differences in survival across countries persist. These data might be used to monitor RB management and occurrences of second tumors. The findings suggest future registry studies should aim to collect standardized RB stage at diagnosis and treatment to interpret disparities and potentially improve surveillance.

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