Generalized lymphatic anomaly successfully treated with long-term, low-dose sirolimus.
Generalized lymphatic anomaly is a rare, complex, lymphatic anomaly generally involving soft tissues, spleen, and bones. It can lead to focal skeletal fragility and pathologic effusions. A recent prospective trial of sirolimus for complicated vascular anomalies showed partial response in seven patients with generalized lymphatic anomaly treated with sirolimus with a target trough level of 10-15 ng/mL for 1 year (Adams et al). We describe successful treatment of generalized lymphatic anomaly with a lower-dose, long-term course of sirolimus.
['Dvorakova|Veronika|V|', 'Rea|David|D|', "O'Regan|Grainne M|GM|", 'Irvine|Alan D|AD|http://orcid.org/0000-0002-9048-2044']
D002675:Child, Preschool D006801:Humans D007166:Immunosuppressive Agents D044148:Lymphatic Abnormalities D008279:Magnetic Resonance Imaging D008297:Male D020123:Sirolimus
generalized lymphatic anomaly sirolimus mTOR inhibitor vascular malformation