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Updated: Oct 25, 2025
Reconstruct Human Retinoblastoma In Vitro
Published on: October 11, 2022
Natural History of Untreated Retinoblastoma
Junyang Zhao1, Zhaoxun Feng2, Brenda L Gallie3,4,5,6
1Department of Ophthalmology, Chongqing Aier Children's Eye Hospital, Chongqing 400020, China.
Insights
Untreated retinoblastoma, a childhood eye cancer, progresses rapidly, leading to death within months. This study highlights the critical need for prompt diagnosis and treatment to prevent fatal outcomes in affected children.
Area of Science:
- Ophthalmology
- Pediatric Oncology
- Cancer Research
Background:
- Treatment abandonment is a primary cause of mortality in pediatric retinoblastoma globally.
- Understanding the natural history of untreated retinoblastoma is crucial for emphasizing treatment urgency.
Purpose of the Study:
- To delineate the natural history of retinoblastoma in children who abandoned treatment upfront at diagnosis.
- To provide real-world evidence on the progression and survival rates of untreated retinoblastoma.
Main Methods:
- Retrospective study of 44 children diagnosed with retinoblastoma between 2007-2017 across 29 Chinese centers who received no treatment.
- Data collected via medical chart reviews and family interviews.
- Analysis of clinical and radiologic evidence for orbital extension and systemic metastasis.
Main Results:
- Median progression times: intraocular tumor to orbital disease (13.7 months), orbital disease to metastasis (2.6 months), metastasis to death (2.0 months).
- Children with brain metastasis had significantly shorter survival (1.0 month) compared to other sites (3.1 months; p=0.015).
- Mortality rates: 36% within 12 months, 77% within 24 months, 95% within 36 months, and 100% within 48 months of diagnosis.
Conclusions:
- Untreated retinoblastoma follows a rapid and fatal natural history.
- Real-world evidence underscores retinoblastoma's life-threatening nature, advocating for immediate treatment initiation upon diagnosis.
- Informing clinicians and parents about these outcomes can improve treatment adherence and survival rates.
Abstract:
Treatment abandonment is a leading cause of death in children with retinoblastoma worldwide. We studied children who abandoned treatment upfront at diagnosis to delineate the natural history of untreated retinoblastoma. Studied were children who received no treatment, diagnosed between 2007 and 2017 at 29 Chinese centers. Data were retrospectively collected from medical chart reviews and interviews with each patient's family. During the study period, 44 children received no treatment after diagnosis of retinoblastoma. Clinical or radiologic evidence of orbital extension was available for 25 children, and radiologic evidence of systemic metastasis was available for 12 children. Median times from diagnosis of intraocular tumor to orbital disease was 13.7 months, orbital disease to metastasis was 2.6 months, and metastasis to death was 2.0 months. Children with brain metastasis had shorter survival than those with metastasis to other sites (median 1.0 vs. 3.1 months; p = 0.015). Overall, 36% of patients died within 12 months of diagnosis, 77% within 24 months, 95% within 36 months and 100% within 48 months. While multiple factors influence refusal of treatment, insights into the natural history of retinoblastoma derived from real-world evidence can inform clinicians and parents that retinoblastoma is life-threatening and encourage urgent treatment at diagnosis.
