Article
Defining vascular anomaly phenotypes in children based on a systematic literature search: A critical step in developing a single severity score for interventional clinical trials.
Pediatric blood & cancer - 1 Oct 2022
Gariépy-Assal Laurence, Dubois Josée, Zwicker Kelley, Pincivy Alix, Powell Julie, Zhang Yang, Breakey Vicky, Price Victoria, Brandão Leonardo R, Carcao Manuel, Kleiber Niina
Abstract excerpt
INTRODUCTION: Genetically targeted drugs in vascular anomalies (VA) are used despite the absence of a validated severity score. The aim of this study was to evaluate the feasibility of grouping phenotypic VA clinical characteristics into a single severity score. METHODS: A systematic literature review including children treated with sirolimus accompanied by a detailed description of phenotype and management was...
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