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Research Letter
January 6, 2021

Mucocutaneous Adverse Events Associated With Oral Sirolimus for the Treatment of Vascular Anomalies

Author Affiliations
  • 1Department of Dermatology, Boston Children’s Hospital, Harvard Medical School, Boston, Massachusetts
  • 2Vascular Anomalies Center, Boston Children’s Hospital, Boston, Massachusetts
  • 3University of Massachusetts Medical School, Boston
  • 4Boston Children’s Hospital, Division of Hematology/Oncology, Harvard Medical School, Boston, Massachusetts
  • 5Complex Vascular Anomalies Program, Division of Oncology, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania
JAMA Dermatol. 2021;157(2):233-235. doi:10.1001/jamadermatol.2020.5180

Sirolimus is a mammalian target of rapamycin (mTOR) inhibitor that blocks signals from the phosphatidylinositol 3-kinase (PI3K)/protein kinase B (AKT) pathway, which coordinates cell proliferation and survival central to the development of vascular anomalies.1,2 Oral sirolimus is widely used to treat varied vascular anomalies and is associated with diverse adverse events (AEs), including leukopenia, gastrointestinal symptoms, infectious complications, and dyslipidemia.1,2 Limited data exist regarding dermatologic AEs in populations with vascular anomalies.

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