Retinoblastoma: Advances in Diagnosis, Treatment, and Global Disparities

Retinoblastoma is the most common primary intraocular malignancy in children, affecting roughly 1 in 15,000 to 20,000 live births worldwide. Rooted in the developmental biology of the retina, it serves as a paradigm for understanding how disrupted embryological programmes drive pediatric cancer. Over recent decades, management has shifted from life-salvage to globe-salvage and vision preservation. Targeted therapies (intra-arterial, intravitreal, and intracameral chemotherapy) have markedly improved globe salvage in advanced intraocular disease. In parallel, liquid biopsies are refining prognostication and artificial intelligence is entering screening and diagnosis. At the same time, international collaborations have exposed wide disparities in presentation, treatment, and outcomes across high- and low-resource settings.

This Research Topic aims to capture the full spectrum of retinoblastoma care, from molecular markers and novel therapeutics to enabling technologies and health policy, and to set recent scientific advances alongside the global equity challenge. The goal is a collection that supports clinicians in day-to-day management, points scientists toward open research questions, and informs policymakers working toward uniform, high-quality care for children worldwide.

We welcome Original Research and comprehensive Reviews. Themes of interest include:

- Tumor biology and molecular drivers of progression, including liquid biopsies
- Genetic testing and the mutational landscape (RB1, MYCN, and beyond)
- Targeted drug delivery: refinements in intra-arterial and intravitreal chemotherapy, and preclinical/clinical studies of novel delivery systems
- Imaging for tumor staging, treatment response assessment, and prognostication in the oncologic setting
- Artificial intelligence for screening, diagnosis, and prognostication
- National and global collaborative studies of clinical presentation, high-risk features, and outcomes
- Socioeconomic factors in care: access, sociodemographic divides, and treatment abandonment
- Quality of life and long-term survivorship outcomes
- Screening programs, awareness campaigns, and policy reform

Please note: manuscripts consisting solely of bioinformatics, computational analysis, or predictions of public databases which are not accompanied by validation (independent clinical or patient cohort, or biological validation in vitro or in vivo, which are not based on public databases) are not suitable for publication in this journal.

Article types and fees

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Clinical Trial
• Editorial
• FAIR² Data
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• ... View all formats

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Article types

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Clinical Trial
• Editorial
• FAIR² Data
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• Original Research
• Perspective
• Review
• Systematic Review
• Technology and Code

Keywords: Retinoblastoma, Pediatric intraocular tumor, Globe salvage, Intra-arterial chemotherapy, Intravitreal chemotherapy, Liquid biopsy, RB1 gene mutation, Targeted drug delivery, Global health disparities, Ocular imaging, Vision preservation, Tumor biology

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.