title: Treatment of epistaxis in hereditary hemorrhagic telangiectasia with tranexamic acid - a double-blind placebo-controlled cross-over phase IIIB study.
abstract: BACKGROUND
  Epistaxis is the most frequent manifestation in hereditary hemorrhagic telangiectasia, in which no optimal treatment exists. It can lead to severe anemia and reduced quality of life. Positive effects of tranexamic acid, an antifibrinolytic drug, have been reported on epistaxis related to this disorder. We sought to evaluate the efficacy of treating nosebleeds in hereditary hemorrhagic telangiectasia with tranexamic acid.
  
  
  METHODS
  In a randomized, double-blind, placebo controlled, cross-over phase IIIB study, 1 gram of tranexamic acid or placebo was given orally 3 times daily for 3 months for a total of 6 months.
  
  
  RESULTS
  22 patients were included in the intention-to-treat analysis. Hemoglobin levels, the primary outcome measure, did not change significantly (p=0.33). The secondary outcome measure was epistaxis score and patients reported a statistically significant reduction in nosebleeds, equaling a clinically relevant 54% diminution (p=0.0031), as compared to the placebo period. No severe side effects were observed.
  
  
  CONCLUSIONS
  Tranexamic acid reduces epistaxis in patients with hereditary hemorrhagic telangiectasia. (Clinical trial registration numbers: BfArM 141 CHC 9008-001 and ClinicalTrials.gov NCT01031992).
authors.affiliation: Department of Otorhinolaryngology, Essen University Hospital, Germany; Morbus Osler-Selbsthilfe e.V. German HHT self-help group, Niederkrüchten, Germany; Formerly of: Department of Otorhinolaryngology, Hospitals of the University of the Saarland, Homburg/Saar, Germany. Electronic address: http://geisthoff.de.
authors.forename: Urban W
authors.initials: UW
authors.lastname: Geisthoff
authors.affiliation: Medical Practice for Haemostaseology and Transfusion Medicine, Saarbrücken, Germany; Formerly of: Department of Haemostaseology and Transfusion Medicine, Hospitals of the University of the Saarland, Homburg/Saar, Germany.
authors.forename: Ulrich T
authors.initials: UT
authors.lastname: Seyfert
authors.affiliation: Formerly of: Department of Otorhinolaryngology, Hospitals of the University of the Saarland, Homburg/Saar, Germany; B.A.D. Gesundheitsvorsorge und Sicherheitstechnik GmbH, Team Prevent, Munich, Germany; Medical Practice Dr. Rolf Kübler, Stuttgart, Germany.
authors.forename: Marcus
authors.initials: M
authors.lastname: Kübler
authors.affiliation: Formerly of: Department of Haemostaseology and Transfusion Medicine, Hospitals of the University of the Saarland, Homburg/Saar, Germany; Department of Anaesthesiology, Klinikum Braunschweig, Braunschweig, Germany.
authors.forename: Birgitt
authors.initials: B
authors.lastname: Bieg
authors.affiliation: Formerly of: Department of Otorhinolaryngology, Hospitals of the University of the Saarland, Homburg/Saar, Germany; Department of Otorhinolaryngology, Ruprecht-Karls-University, Heidelberg, Germany.
authors.forename: Peter K
authors.initials: PK
authors.lastname: Plinkert
authors.affiliation: Institute of Medical Biostatistics, Epidemiology and Informatics, Division Medical Biometry, University Medical Center, Johannes Gutenberg University, Mainz, Germany; Formerly of: Institute of Medical Biostatistics, Epidemiology and Informatics, University of the Saarland, Homburg/Saar, Germany.
authors.forename: Jochem
authors.initials: J
authors.lastname: König
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D000933:Antifibrinolytic Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D018592:Cross-Over Studies; D004311:Double-Blind Method; D004334:Drug Administration Schedule; D004844:Epistaxis / Q000175:diagnosis / Q000209:etiology / Q000517:prevention & control*; D005260:Female; D005858:Germany; D006801:Humans; D008297:Male; D008875:Middle Aged; D013683:Telangiectasia, Hereditary Hemorrhagic / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D013997:Time Factors; D014148:Tranexamic Acid / Q000008:administration & dosage / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D000933:Antifibrinolytic Agents; D014148:Tranexamic Acid
keywords: Antifibrinolytics; Epistaxis; Hereditary hemorrhagic telangiectasia; Osler-Rendu-Weber syndrome; Tranexamic acid