title: Intermittent low-dose bevacizumab in hereditary hemorrhagic telangiectasia : A case report.
abstract: BACKGROUND
  Hereditary hemorrhagic telangiectasia is an inherited autosomal dominant disease presenting with recurrent bleeding episodes and iron deficiency anemia due to vascular malformations. Hereditary hemorrhagic telangiectasia is associated with an increased risk of stroke, gastrointestinal bleeding and pulmonary hypertension and life expectancy is significantly reduced. Excess vascular endothelial growth factor (VEGF) plays a key role in the pathophysiology of the disease.
  
  
  METHODS
  Here we report about a male patient with hereditary hemorrhagic telangiectasia presenting with pulmonary and central nervous system involvement experiencing repetitive nosebleeds, necessitating frequent local cauterization and transfusion of more than 100 units of packed red blood cells. After initiation of temporary therapy with the anti-VEGF antibody bevacizumab at a dosage of 1 mg/kg body weight every 2 weeks, the nose bleeding episodes and the epistaxis severity score significantly decreased and long-lasting transfusion independence was achieved. Reinitiation of low-dose bevacizumab after relapse again proved effective without any documented therapy-related adverse events. In comparison to other reported anti-VEGF antibody protocols in hereditary hemorrhagic telangiectasia, our treatment approach proved to be cost-efficient.
  
  
  CONCLUSIONS
  Intermittent low-dose therapy with bevacizumab represents an effective and cost-efficient treatment option for transfusion-dependent patients with hereditary hemorrhagic telangiectasia.
authors.affiliation: IIIrd Medical Department with Haematology, Medical Oncology, Haemostaseology, Infectious Diseases and Rheumathology, Oncologic Center, Paracelsus Medical University, Muellner Hauptstraße 48, 5020, Salzburg, Austria.|Salzburg Cancer Research Institute with Laboratory of Immunological and Molecular Cancer Research and Center for Clinical Cancer and Immunology Trials, Salzburg, Austria.|Cancer Cluster Salzburg, Salzburg, Austria.
authors.forename: Florian
authors.initials: F
authors.lastname: Huemer
authors.affiliation: Department of Otorhinolaryngology, Paracelsus Medical University, Salzburg, Austria.
authors.forename: Martin
authors.initials: M
authors.lastname: Dejaco
authors.affiliation: Blood Group Serology and Transfusion Medicine, Paracelsus Medical University, Salzburg, Austria.
authors.forename: Christoph
authors.initials: C
authors.lastname: Grabmer
authors.affiliation: IIIrd Medical Department with Haematology, Medical Oncology, Haemostaseology, Infectious Diseases and Rheumathology, Oncologic Center, Paracelsus Medical University, Muellner Hauptstraße 48, 5020, Salzburg, Austria.|Salzburg Cancer Research Institute with Laboratory of Immunological and Molecular Cancer Research and Center for Clinical Cancer and Immunology Trials, Salzburg, Austria.|Cancer Cluster Salzburg, Salzburg, Austria.
authors.forename: Thomas
authors.initials: T
authors.lastname: Melchardt
authors.affiliation: Institute of Pathology, Paracelsus Medical University, Salzburg, Austria. d.neureiter@salk.at.
authors.forename: Daniel
authors.initials: D
authors.lastname: Neureiter
authors.affiliation: Blood Group Serology and Transfusion Medicine, Paracelsus Medical University, Salzburg, Austria.
authors.forename: Georg
authors.initials: G
authors.lastname: Mayer
authors.affiliation: IIIrd Medical Department with Haematology, Medical Oncology, Haemostaseology, Infectious Diseases and Rheumathology, Oncologic Center, Paracelsus Medical University, Muellner Hauptstraße 48, 5020, Salzburg, Austria.|Salzburg Cancer Research Institute with Laboratory of Immunological and Molecular Cancer Research and Center for Clinical Cancer and Immunology Trials, Salzburg, Austria.|Cancer Cluster Salzburg, Salzburg, Austria.
authors.forename: Alexander
authors.initials: A
authors.lastname: Egle
authors.affiliation: IIIrd Medical Department with Haematology, Medical Oncology, Haemostaseology, Infectious Diseases and Rheumathology, Oncologic Center, Paracelsus Medical University, Muellner Hauptstraße 48, 5020, Salzburg, Austria.|Salzburg Cancer Research Institute with Laboratory of Immunological and Molecular Cancer Research and Center for Clinical Cancer and Immunology Trials, Salzburg, Austria.|Cancer Cluster Salzburg, Salzburg, Austria.
authors.forename: Richard
authors.initials: R
authors.lastname: Greil
authors.affiliation: IIIrd Medical Department with Haematology, Medical Oncology, Haemostaseology, Infectious Diseases and Rheumathology, Oncologic Center, Paracelsus Medical University, Muellner Hauptstraße 48, 5020, Salzburg, Austria.|Salzburg Cancer Research Institute with Laboratory of Immunological and Molecular Cancer Research and Center for Clinical Cancer and Immunology Trials, Salzburg, Austria.|Cancer Cluster Salzburg, Salzburg, Austria.
authors.forename: Lukas
authors.initials: L
authors.lastname: Weiss
mesh_terms: D020533:Angiogenesis Inhibitors / Q000008:administration & dosage; D000068258:Bevacizumab / Q000008:administration & dosage*; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D004844:Epistaxis / Q000175:diagnosis / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D013683:Telangiectasia, Hereditary Hemorrhagic / Q000150:complications / Q000175:diagnosis* / Q000188:drug therapy*; D016896:Treatment Outcome; D042461:Vascular Endothelial Growth Factor A / Q000037:antagonists & inhibitors
chemical_list: D020533:Angiogenesis Inhibitors; D042461:Vascular Endothelial Growth Factor A; D000068258:Bevacizumab
keywords: Anemia; Bevacizumab; Nosebleeds; Telangiectasia; Transfusion; VEGF