Bibliometric and clinical trial landscape of hepatoblastoma (2000-2024): a multi-database analysis of global research
Abstract
Background: Hepatoblastoma (HB) is the most common childhood liver malignancy, yet its research trajectory and trial landscape remain uncharacterized.
Methods: We analyzed 219 trials from 16 international registries and 2,635 PubMed publications (2005-2024) using joinpoint regression, Gini coefficient, and diversity indices.
Results: Trial activity peaked in 2013 and 2016, driven by risk-stratified protocols and the emergence of targeted therapies. Phase III/IV trials were scarce. Cisplatin dominated drug utilization, with 54-80% resistance after 4-5 cycles. Targets concentrated on VEGFR/TOP1/RET/BRAF, while Hippo/YAP and immune checkpoints were underrepresented despite mechanistic relevance. AFP (n=607), CTNNB1 (>80% mutation frequency), TP53, AKT1, and MYC were top-reported genes. Geographic inequality correlated with healthcare infrastructure rather than disease burden.
Conclusions: The HB research ecosystem exhibits robust early-phase activity but constrained late-stage validation, narrow target diversity, and geographic inequity. Multi-omics integration and pediatric-specific preclinical platforms are urgently needed to bridge translational gaps.
How strong is the evidence?
Evidence Hub reads this as a systematic review, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid. Reported size: 607 participants or records.
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