title: Tranexamic acid for epistaxis in hereditary hemorrhagic telangiectasia patients: a European cross-over controlled trial in a rare disease.
abstract: BACKGROUND
  Hereditary hemorrhagic telangiectasia (HHT) is a genetic disorder associated with abnormal angiogenesis and disabling epistaxis. Tranexamic acid (TA) has been widely used in the treatment of these severe bleeds but with no properly designed trial.
  
  
  OBJECTIVE
  To demonstrate the efficacy of TA in epistaxis in HHT patients and to explore its safety of use.
  
  
  METHODS
  A randomized, placebo-controlled, double-blind, cross-over trial was conducted. Participants were randomized to receive TA (3 g a day) then placebo or the opposite sequence. The main analysis compared intra-individual mean duration of epistaxis under TA vs. placebo on a log scale. The primary outcome was the mean duration of epistaxis per month, assessed with specific grids to be completed by participants. The number of epistaxis episodes was recorded as a secondary outcome.
  
  
  RESULTS
  A total of 118 randomized patients contributed to the statistical analysis. The mean duration of epistaxis per month was significantly shorter with TA than placebo (0.19 on the log scale; SD = 0.07; P = 0.005), corresponding to a decrease of 17.3% (15.7 min) in the duration of epistaxis per month (CI 95%, 5.5-27.6). The median number of epistaxis episodes per month was 22.1 episodes in the placebo arm vs. 23.3 episodes in the TA arm. No thrombophlebitis was observed.
  
  
  CONCLUSIONS
  In the ATERO study, we demonstrated a significant decrease in the duration of epistaxis in HHT patients taking TA. No safety issues were recorded in our cohort of patients.
authors.affiliation: INSERM, CIC 1407 Lyon, Hospices Civils de Lyon, Service de Pharmacologie Clinique Lyon, Université de Lyon, Lyon, France; Université Lyon 1, Villeurbanne, France; CNRS, UMR5558, Laboratoire de Biométrie et Biologie Evolutive, Equipe Biotatistique-Santé, Villeurbanne, France.
authors.forename: S
authors.initials: S
authors.lastname: Gaillard
authors.forename: S
authors.initials: S
authors.lastname: Dupuis-Girod
authors.forename: F
authors.initials: F
authors.lastname: Boutitie
authors.forename: S
authors.initials: S
authors.lastname: Rivière
authors.forename: S
authors.initials: S
authors.lastname: Morinière
authors.forename: P-Y
authors.initials: PY
authors.lastname: Hatron
authors.forename: G
authors.initials: G
authors.lastname: Manfredi
authors.forename: P
authors.initials: P
authors.lastname: Kaminsky
authors.forename: A-L
authors.initials: AL
authors.lastname: Capitaine
authors.forename: P
authors.initials: P
authors.lastname: Roy
authors.forename: F
authors.initials: F
authors.lastname: Gueyffier
authors.forename: H
authors.initials: H
authors.lastname: Plauchu
mesh_terms: D000328:Adult; D000368:Aged; D000933:Antifibrinolytic Agents / Q000627:therapeutic use*; D018592:Cross-Over Studies; D004311:Double-Blind Method; D004844:Epistaxis / Q000188:drug therapy*; D005060:Europe; D005260:Female; D006470:Hemorrhage / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009389:Neovascularization, Pathologic; D011788:Quality of Life; D035583:Rare Diseases; D013683:Telangiectasia, Hereditary Hemorrhagic / Q000188:drug therapy*; D014148:Tranexamic Acid / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000933:Antifibrinolytic Agents; D014148:Tranexamic Acid
keywords: Osler-Rendu disease; epistaxis; hereditary hemorrhagic telangiectasia; randomized controlled trial; tranexamic acid