title: Efficacy of tranexamic acid for the prevention of bleeding in patients with von Willebrand disease and Glanzmann thrombasthenia: a controlled, before and after trial.
abstract: BACKGROUND
  Reducing bleeding episodes is very important in haematology disorders like von Willebrand disease (VWD) and Glanzmann thrombasthenia (GT). Replacement factors are very expensive although prophylactic drugs are affordable.
  
  
  OBJECTIVE
  To study the prophylactic effects of tranexamic acid (TXA) for reduction of bleeding episodes in patients with VWD and GT in non-invasive conditions.
  
  
  METHODS
  A controlled, double-blind before and after single-centre trial was performed in Amir-Kabir Hospital (Arak, Iran). The study was done on 17 patients with VWD and three patients with GT with minimum age of 2 years. Patients were received placebo for 6 months to evaluate the frequency and severity of bleeding and also to record the frequency of use of factor concentrates and platelet transfusion. After that, patients were given oral single dose of TXA 25 mg kg(-1) day(-1) for 6 months. The mentioned outcomes were studied and compared between two phases of study. Safety assessment was done in all patients.
  
  
  RESULTS
  Tranexamic acid caused a significant reduction in number of Grade 1 and Grade 2 bleeding episodes in VWD patients (P < 0.001 and P < 0.01 respectively). In addition, TXA therapy was associated with significant decrease in the use of factor concentrates (P < 0.05). Number of bleeding episodes decreased in GT patients who used TXA; however, difference between two phases of studies was not significant (P = 0.1). TXA had no effect in the frequency of platelet transfusions in GT patients. TXA therapy was associated with headache, back pain and musculoskeletal pain. No case of allergy or thromboembolic events was seen following treatment.
  
  
  CONCLUSIONS
  The results suggest that TXA is safe and effective to reduce bleeding and use of factor concentrates in VWD patients. In addition, TXA therapy can decrease bleeding in GT patients.
authors.affiliation: Department of Pediatric Hematology and Oncology, Arak University of Medical Sciences, Arak, Iran.
authors.forename: A
authors.initials: A
authors.lastname: Eghbali
authors.affiliation: Department of Pediatric Hematology and Oncology, Arak University of Medical Sciences, Arak, Iran.
authors.forename: L
authors.initials: L
authors.lastname: Melikof
authors.affiliation: Department of Pediatric Hematology and Oncology, Arak University of Medical Sciences, Arak, Iran.
authors.forename: H
authors.initials: H
authors.lastname: Taherahmadi
authors.affiliation: Cancer Research Center and Department of Pharmacology, Semnan University of Medical Sciences, Semnan, Iran. bahadordvm@yahoo.com.
authors.forename: B
authors.initials: B
authors.lastname: Bagheri
mesh_terms: D000933:Antifibrinolytic Agents / Q000627:therapeutic use*; D002675:Child, Preschool; D065187:Controlled Before-After Studies; D004311:Double-Blind Method; D005260:Female; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D007223:Infant; D007492:Iran; D008297:Male; D017713:Platelet Transfusion; D013915:Thrombasthenia / Q000150:complications / Q000188:drug therapy*; D014148:Tranexamic Acid / Q000627:therapeutic use*; D016896:Treatment Outcome; D014842:von Willebrand Diseases / Q000150:complications / Q000188:drug therapy*
chemical_list: D000933:Antifibrinolytic Agents; D014148:Tranexamic Acid
keywords: Glanzmann thrombasthenia; VWD; bleeding; tranexamic acid