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
低剂量 PI3K 抑制剂 taselisib 在患有 CLOVES 和 Klippel-Trenaunay 综合征 (KTS) 的成年患者中的安全性和有效性:TOTEM 试验,一项 1/2 期多中心、开放标签、单臂研究
期刊:Genetics in Medicine
影响因子:6.2
doi:10.1038/s41436-021-01290-y
Luu, M; Vabres, P; Devilliers, H; Loffroy, R; Phan, A; Martin, L; Morice-Picard, F; Petit, F; Willems, M; Bessis, D; Jacquemont, M L; Maruani, A; Chiaverini, C; Mirault, T; Clayton-Smith, J; Carpentier, M; Fleck, C; Maurer, A; Yousfi, M; Parker, V E R; Semple, R K; Bardou, M; Faivre, L