Safety and efficacy of low-dose PI3K inhibitor taselisib in adult patients with CLOVES and Klippel-Trenaunay syndrome (KTS): the TOTEM trial, a phase 1/2 multicenter, open-label, single-arm study.
Maxime LuuP VabresH DevilliersR LoffroyA PhanL MartinF Morice-PicardF PetitM WillemsD BessisM L JacquemontA MaruaniC ChiaveriniT MiraultJ Clayton-SmithM CarpentierC FleckA MaurerM YousfiV E R ParkerR K SempleM BardouL FaivrePublished in: Genetics in medicine : official journal of the American College of Medical Genetics (2021)
Despite functional improvement, the safety profile of low-dose taselisib precludes its long-term use.