title: Treatment of infantile hemangiomas with sirolimus in a patient with PHACE syndrome.
abstract: Infantile hemangiomas (IHs) are common benign tumors of childhood. IHs often regress satisfactorily without intervention, but a subset of IHs may lead to functional or cosmetic morbidity necessitating therapy. PHACE syndrome is characterized by a variety of neurocutaneous and vascular anomalies that typically include segmental hemangiomas. We present an infant with PHACE syndrome and segmental IH that failed conventional first-line therapies. Treatment with sirolimus provided benefit with regression of the cutaneous IH. As an inhibitor of the mammalian target of rapamycin (mTOR) pathway, the effective use of sirolimus may shed light on the emerging role of mTOR signaling in the development and pathogenesis of IHs.
authors: Division of Hematology-Oncology, Department of Pediatrics, University of Alabama at Birmingham, Birmingham, Alabama.
authors: Samer
authors: S
authors: Kaylani
authors: Amy J
authors: AJ
authors: Theos
authors: Joseph G
authors: JG
authors: Pressey
mesh_terms: D000903:Antibiotics, Antineoplastic / Q000627:therapeutic use; D001017:Aortic Coarctation / Q000188:drug therapy*; D005124:Eye Abnormalities / Q000188:drug therapy*; D005153:Facial Neoplasms / Q000188:drug therapy*; D005260:Female; D006391:Hemangioma / Q000188:drug therapy*; D006801:Humans; D007231:Infant, Newborn; D020752:Neurocutaneous Syndromes / Q000188:drug therapy*; D020123:Sirolimus / Q000627:therapeutic use*; D012878:Skin Neoplasms / Q000188:drug therapy*
chemical_list: D000903:Antibiotics, Antineoplastic; D020123:Sirolimus