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Published on: October 27, 2014
Targeted chemotherapy in retinoblastoma: A step forward from patient survival to globe survival
Masood Naseripour1, Ahad Sedaghat1, Reza Mirshahi1
1Eye Research Center, The Five Senses Health Institute, 48492Rassoul Akram Hospital, Iran University, of Medical Sciences, Tehran, Iran.
Insights
New targeted treatments significantly improved globe survival for retinoblastoma (Rb) patients in a developing country. Early enucleation remains crucial even with advanced therapies for this common childhood intraocular malignancy.
Area of Science:
- Ophthalmology
- Pediatric Oncology
- Cancer Research
Background:
- Retinoblastoma (Rb) is the most common intraocular malignancy in children.
- Significant disparities exist in patient and globe survival rates between developing and developed nations.
- The study addresses survival rates before and after novel treatments in a developing country.
Purpose of the Study:
- To evaluate patient and globe survival rates in retinoblastoma (Rb) patients.
- To compare survival outcomes before and after the introduction of targeted therapies.
- To assess the impact of intra-arterial and intravitreal chemotherapy on Rb treatment in Iran.
Main Methods:
- Retrospective review of 350 retinoblastoma (Rb) patient records (516 eyes) from a tertiary referral center in Tehran, Iran.
- Patients were divided into two groups based on diagnosis period: Group 1 (2001-2007) and Group 2 (2008-2018).
- Comparison of patient and globe survival rates between the two groups, considering the introduction of intra-arterial and intravitreal chemotherapy.
Main Results:
- Overall 5-year patient survival was 97%. Group 2 showed a trend towards higher overall survival (99%) compared to Group 1 (96%).
- Primary enucleation rates decreased significantly in Group 2 (35%) versus Group 1 (50%) (P < 0.001).
- Globe survival significantly improved for advanced International Classification of Retinoblastoma Groups D and E in Group 2 compared to Group 1 (P < 0.001).
Conclusions:
- Introduction of targeted treatments like intra-arterial and intravitreal chemotherapy has significantly improved globe survival in retinoblastoma (Rb) patients, mirroring outcomes in developed countries.
- Despite advances, timely enucleation remains a critical decision in managing retinoblastoma.
- The study highlights the successful integration of novel therapies in improving pediatric cancer outcomes in resource-limited settings.
Background/ Objectives:
Despite being the most common intraocular malignancy in childhood, there is a substantial disparity between developing and developed countries in terms of patient and globe survival in retinoblastoma (Rb). The current study intends to determine patient and globe survival before and after the introduction of the new targeted treatment modalities in a developing country.
Methods:
Medical records of 350 patients (516 eyes) with retinoblastoma referred to a tertiary referral center for Rb in Tehran, Iran, were reviewed. In order to compare patient and globe survival before and after the availability of the new treatment modalities, including intra-arterial and intravitreal chemotherapy, the patients were divided into group 1 (2001-2007) and group 2 (2008-2018) based on the calendar period of diagnosis.
Results:
Two-hundred-twenty-three eyes of 149 patients and 293 eyes of 201 patients were categorized into groups 1 and 2, respectively. The 5-year patient survival was 97% across the current survey, and the overall survival rate was 96% in group 1 and 99% in group 2 (P = 0.08). Overall, 50% of eyes with retinoblastoma underwent enucleation, which was the primary in 63% (116/184) of the unilateral and 30% (99/322) of the bilateral cases. Primary enucleation was significantly lower in group 2 (35%) in contrast to group 1 (50%) (P < 0.001). In addition, globe survival improved significantly in the International Classification of Retinoblastoma Groups D (17% in group 1 vs. 66% in group 2, P < 0.001) and E (1% in group 1 vs. 23% in group 2 P < 0.001) during the two timelines. In enucleated eyes, despite the increased rate of prelaminar involvement in group 2 (13% vs. 2% in group 1, P = 0.003), the rate of high-risk histopathologic findings was similar between the two groups.
Conclusion:
Similar to developed countries, the application of new targeted treatment modalities, including intra-arterial and intravitreal chemotherapy, has been associated with significantly improved globe survival in Rb patients. However, it should be noted that even with the availability of these novel treatment options, the decision for on-time enucleation should not be deferred.
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