Response to sirolimus in capillary lymphatic venous malformations and associated syndromes: Impact on symptomatology, quality of life, and radiographic response.

Elissa R Engel,Adrienne Hammill,Denise Adams,Roderic J Phillips,Michael Jeng,Megha M Tollefson,Ionela Iacobas,Deborah Schiff,Shoshana Greenberger,Michael Kelly,Ilona Frieden,Nibal Zaghloul,Beth Drolet,Amy Geddis,Dov Goldenberg,Kiersten Ricci

Pediatric blood & cancer（2023）

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摘要

Sirolimus appears to be effective at reducing complications and improving QOL in patients with CLVM and associated syndromes. In this patient cohort, sirolimus was well tolerated and resulted in few treatment-related toxicities.

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关键词

CLOVES,Klippel-Trenaunay syndrome (KTS),PIK3CA-related overgrowth spectrum (PROS),capillary lymphatic venous malformation (CLVM),mammalian target of rapamycin (mTOR),sirolimus (rapamycin)

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