title: Successful Everolimus Treatment of Kaposiform Hemangioendothelioma With Kasabach-Merritt Phenomenon: Clinical Efficacy and Adverse Effects of mTOR Inhibitor Therapy.
abstract: Kasabach-Merritt phenomenon (KMP) is a life-threatening consumptive coagulopathy associated with underlying kaposiform hemangioendothelioma (KHE) in infancy. We describe the case of a 3-month-old girl with KHE complicated by KMP who responded dramatically to treatment with everolimus, a mechanistic target of rapamycin (mTOR) inhibitor. Immunohistochemical expression of mTOR was found in the KHE biopsy specimens, which may explain the improvement of KMP and reduction in KHE tumor size with mTOR inhibitor treatment. This effective use of everolimus may shed light on the emerging role of mTOR signaling in the development and pathogenesis of KHE and KMP.
authors.affiliation: *Department of Pediatrics, Gifu Prefectural General Medical Center Departments of †Pediatrics ‡Orthopedic Surgery ∥Pathology, Gifu University Graduate School of Medicine, Gifu University, Gifu §Department of Pediatrics and Cell Transplantation, Mie University Graduate School of Medicine, Tsu, Japan.
authors.forename: Hideki
authors.initials: H
authors.lastname: Matsumoto
authors.forename: Michio
authors.initials: M
authors.lastname: Ozeki
authors.forename: Tomohiro
authors.initials: T
authors.lastname: Hori
authors.forename: Kaori
authors.initials: K
authors.lastname: Kanda
authors.forename: Norio
authors.initials: N
authors.lastname: Kawamoto
authors.forename: Akihito
authors.initials: A
authors.lastname: Nagano
authors.forename: Eiichi
authors.initials: E
authors.lastname: Azuma
authors.forename: Tatsuhiko
authors.initials: T
authors.lastname: Miyazaki
authors.forename: Toshiyuki
authors.initials: T
authors.lastname: Fukao
mesh_terms: D000068338:Everolimus / Q000627:therapeutic use*; D005260:Female; D006390:Hemangioendothelioma / Q000737:chemistry / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D007150:Immunohistochemistry; D007223:Infant; D059885:Kasabach-Merritt Syndrome / Q000737:chemistry / Q000150:complications / Q000188:drug therapy*; D012514:Sarcoma, Kaposi / Q000737:chemistry / Q000150:complications / Q000188:drug therapy*; D058570:TOR Serine-Threonine Kinases / Q000032:analysis / Q000037:antagonists & inhibitors; D016896:Treatment Outcome
chemical_list: D000068338:Everolimus; C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases