Safety and efficacy of low-dose sirolimus in the PIK3CA-related overgrowth spectrum

2018155 citationsJournal Articlehybrid Open Access

Authors

Victoria Parker · University of Cambridge

Kim M. Keppler‐Noreuil · National Human Genome Research Institute

Laurence Faivre · Université de Bourgogne

Maxime Luu · Centre Hospitalier Universitaire Dijon Bourgogne

Neal L. Oden · Emmes (United States)

Leena De Silva · University of Cambridge

Julie C. Sapp

Abstract

This study suggests that low-dose sirolimus can modestly reduce overgrowth, but cautions that the side-effect profile is significant, mandating individualized risk-benefit evaluations for sirolimus treatment in PROS.

Topics & Keywords

Vascular Malformations and Hemangiomas

UN Sustainable Development Goals

Affordable and clean energy

Publication Details

Published in: Genetics in Medicine

Volume 21, Issue 5, pp. 1189-1198

DOI: 10.1038/s41436-018-0297-9

Field-Weighted Citation Impact: 16.10

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