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Generalized lymphatic anomaly successfully treated with long‐term,low‐dose sirolimus
Authors:Veronika Dvorakova MUDr  David Rea MCh  Grainne M. O'Regan PhD  Alan D. Irvine MD  DSc
Affiliation:1. Department of Pediatric Dermatology, Our Lady's Children's Hospital, Crumlin, Dublin, Ireland;2. Department of Pediatric Radiology, Our Lady's Children's Hospital, Crumlin, Dublin, Ireland
Abstract: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.
Keywords:generalized lymphatic anomaly  mTOR inhibitor  sirolimus  vascular malformation
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