Response to sirolimus in capillary lymphatic venous malformations and associated syndromes: Impact on symptomatology, quality of life, and radiographic response.
Elissa R EngelAdrienne M HammilllDenise AdamsRoderic J PhillipsMichael R JengMegha M TollefsonIonela IacobasDeborah E SchiffShoshana GreenbergerMichael KellyIlona FriedenNibal ZaghloulBeth DroletAmy GeddisDov GoldenbergKiersten W RicciPublished in: Pediatric blood & cancer (2023)
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.