ReviewJournal of Indian Association of Pediatric Surgeons
Genotype-directed Targeted Therapy for Pediatric Vascular Malformations: A Systematic Review.
Review in Journal of Indian Association of Pediatric Surgeons. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
0 citing papers in PubMed.
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Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: Pediatric vascular malformations are rare, heterogeneous disorders increasingly driven by somatic mutations affecting key molecular pathways, particularly the PI3K-AKT-mTOR and RAS-MAPK signaling cascades. Advances in genomic profiling have enabled the use of targeted pharmacological therapies, but the available evidence remains fragmented. Objective: To systematically review the clinical outcomes and safety of genotype-directed targeted systemic therapies in pediatric patients with genetically characterized vascular malformations. Methods: A systematic literature search was conducted in PubMed, Embase, and Scopus from database inception to November 21, 2025. Eligible studies included case reports, case series, cohort studies, and prospective investigations reporting genotype-directed targeted therapy in patients aged 0-18 years. Data on genetic alterations, targeted therapies, clinical and radiologic outcomes, and adverse events were extracted and synthesized qualitatively. Results: Fifty-two studies were included, reporting pediatric patients from the neonatal period to 18 years of age. The most frequently identified genetic alterations involved the PI3K-AKT-mTOR and RAS-MAPK pathways, predominantly Conclusions: Genotype-directed targeted therapies appear to provide clinical and radiologic benefit in pediatric vascular malformations with acceptable safety profiles. However, the current evidence is largely derived from observational studies and requires prospective validation through standardized investigations.
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