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Updated: May 7, 2025
Reconstruct Human Retinoblastoma In Vitro
Published on: October 11, 2022
Retinoblastoma with and without Extraocular Tumor Extension: A Global Comparative Study of 3435 Patients
Swathi Kaliki1, Vijitha S Vempuluru1, Ido Didi Fabian2,3
1The Operation Eyesight Institute for Eye Cancer, L.V. Prasad Eye Institute, Hyderabad, Telangana, India.
Insights
Children with retinoblastoma (RB) and extraocular tumor extension (RB-EOE) face significantly higher metastasis and death risks. Trimodal treatment, including chemotherapy, surgery, and radiotherapy, offers better outcomes for RB-EOE patients compared to other protocols.
Area of Science:
- Ophthalmology
- Pediatric Oncology
- Cancer Research
Background:
- Retinoblastoma (RB) is the most common intraocular malignancy in children.
- Extraocular tumor extension (RB-EOE) represents a more advanced stage of the disease.
- Understanding the treatment and outcomes of RB-EOE is crucial for improving patient survival.
Purpose of the Study:
- To investigate the treatment strategies and clinical outcomes for children diagnosed with retinoblastoma exhibiting extraocular tumor extension (RB-EOE).
- To compare the outcomes of RB-EOE cases with those of retinoblastoma without extraocular tumor extension (RB-w/o-EOE).
- To identify prognostic factors influencing metastasis and mortality in RB-EOE.
Main Methods:
- A prospective, multicenter, intercontinental collaborative study conducted from 2017 to 2020.
- Inclusion criteria for RB-EOE involved overt orbital tumor extension in treatment-naive patients; microscopic extension postenucleation was excluded.
- Data analysis included Kaplan-Meier and Cox regression to assess metastasis-related death and survival rates.
Main Results:
- RB-EOE cases showed a significantly higher incidence of systemic metastasis (32% vs. 4%) and metastasis-related death (63% vs. 6%) compared to RB-w/o-EOE.
- Multimodal treatment, particularly intravenous chemotherapy combined with enucleation, was the most common approach for RB-EOE.
- Trimodal treatment (chemotherapy, surgery, and external beam radiotherapy) was associated with a lower risk of death compared to unimodal or bimodal treatments.
Conclusions:
- Retinoblastoma with extraocular tumor extension is a critical risk factor for metastasis and mortality.
- Trimodal treatment, incorporating chemotherapy, surgery, and external beam radiotherapy, is recommended for patients with RB-EOE.
- Treatment protocols for RB-EOE should prioritize comprehensive approaches to improve survival rates.
Purpose:
To study the treatment and outcomes of children with retinoblastoma (RB) with extraocular tumor extension (RB-EOE) and compare them with RB without extraocular tumor extension (RB-w/o-EOE).
Design:
Multicenter intercontinental collaborative prospective study from 2017 to 2020. RB-EOE cases included those with overt orbital tumor extension in treatment-naive patients. Cases with microscopic orbital extension detected postenucleation were excluded from the study.
Participants:
A total of 319 children with RB-EOE and 3116 children with RB-w/o-EOE.
Intervention:
Chemotherapy, enucleation, exenteration, radiotherapy.
Main Outcome Measures:
Systemic metastasis and death.
Results:
Of the 3435 RB patients included in this study, 309 (9%) were from low-income countries (LIC), 1448 (42%) from lower-middle income, 1012 (29%) from upper-middle income, and 666 (19%) patients from high-income countries. There was an inverse relationship between the percentage of RB-EOE and national income level, with 96 (31%) patients from LIC, 197 (6%) lower-middle income, 20 (2%) upper-middle income, and 6 (1%) patients from high-income countries (P = 0.0001). The outcomes were statistically significant for RB-EOE compared with RB-w/o-EOE: systemic metastasis (32% vs. 4% respectively; P = 0.0001) and metastasis-related death (63% vs. 6% respectively; P = 0.0001). Multimodal treatment was the most common form of treatment (n = 177; 54%) for RB-EOE, with most cases undergoing a combination of intravenous chemotherapy and enucleation (n = 97; 30%). Adjuvant external beam radiotherapy (EBRT) after surgery (enucleation/orbital exenteration) was given in only 68 (21%) cases. Kaplan-Meier analysis for systemic metastasis and metastasis-related death in RB-EOE was 28% and 57% at 1 year, 29% and 60% at 2 years, and 29% and 61% at 3 years, respectively. Cox regression analysis revealed that the risk of death from RB-EOE was greater in patients aged >4 years than <2 years (hazard ratio, 2.912; P < 0.001) and for unimodal (surgery or intravenous chemotherapy) and bimodal (surgery and intravenous chemotherapy) treatment than trimodal treatment (surgery, intravenous chemotherapy, and EBRT) (hazard ratio, 2.023; P = 0.004 and hazard ratio, 1.819; P = 0.027, respectively).
Conclusions:
Retinoblastoma with extraocular tumor extension is associated with a higher risk of metastasis and death. Patients with RB-EOE are likely to benefit from trimodal treatment (intravenous chemotherapy, surgery, and EBRT) rather than treatment protocols excluding EBRT.
Financial Disclosures:
The authors have no proprietary or commercial interest in any materials discussed in this article.
