Efficacy of systemic sirolimus in the treatment of generalized lymphatic anomaly and Gorham-Stout disease.
Kiersten W RicciAdrienne M HammillPaula Mobberley-SchumanStephen C NelsonJulie BlattJulia L Glade BenderCatherine C McCuaigAnna SynakiewiczIlona J FriedenDenise M AdamsPublished in: Pediatric blood & cancer (2019)
Sirolimus appears effective at stabilizing or reducing signs/symptoms of disease in patients with GLA and GSD. Functional impairment and/or QOL improved in the majority of individuals with GLA and GSD with sirolimus treatment.
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