title: Gorham-Stout Disease Successfully Treated With Sirolimus and Zoledronic Acid Therapy.
abstract: Gorham-Stout disease is a life-threatening disorder often manifested by lymphatic malformation and osteolysis. Unfortunately, available therapies are not uniformly effective and often carry substantial morbidity. We report an 18-year-old male with Gorham-Stout disease manifested by lytic rib lesions and an intractable pleural effusion that responded dramatically to the combination of the mammalian target of rapamycin (mTOR) inhibitor sirolimus and the aminobisphosphonate zoledronic acid after failing interferon therapy. This tolerable therapeutic combination has demonstrated synergism in preclinical cancer models and merits further study in vascular anomalies.
authors.affiliation: Departments of *Pediatrics, Division of Hematology and Oncology, Children's of Alabama †Pathology, University of Alabama at Birmingham, Birmingham, AL ‡Department of Radiology §Cancer and Blood Disorders Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
authors.forename: Stuart L
authors.initials: SL
authors.lastname: Cramer
authors.forename: Shi
authors.initials: S
authors.lastname: Wei
authors.forename: Arnold C
authors.initials: AC
authors.lastname: Merrow
authors.forename: Joseph G
authors.initials: JG
authors.lastname: Pressey
mesh_terms: D000293:Adolescent; D050071:Bone Density Conservation Agents / Q000008:administration & dosage; D004164:Diphosphonates / Q000008:administration & dosage; D004359:Drug Therapy, Combination / Q000379:methods*; D006801:Humans; D007093:Imidazoles / Q000008:administration & dosage; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D008297:Male; D010015:Osteolysis, Essential / Q000188:drug therapy*; D020123:Sirolimus / Q000008:administration & dosage; D000077211:Zoledronic Acid
chemical_list: D050071:Bone Density Conservation Agents; D004164:Diphosphonates; D007093:Imidazoles; D007166:Immunosuppressive Agents; D000077211:Zoledronic Acid; D020123:Sirolimus