A Eghbali1, L Melikof1, H Taherahmadi1, B Bagheri2. 1. Department of Pediatric Hematology and Oncology, Arak University of Medical Sciences, Arak, Iran. 2. Cancer Research Center and Department of Pharmacology, Semnan University of Medical Sciences, Semnan, Iran. bahadordvm@yahoo.com.
Abstract
INTRODUCTION: 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.
RCT Entities:
INTRODUCTION: 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 VWDpatients (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 GTpatients 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 GTpatients. 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 VWDpatients. In addition, TXA therapy can decrease bleeding in GTpatients.
Authors: Nathan T Connell; Veronica H Flood; Romina Brignardello-Petersen; Rezan Abdul-Kadir; Alice Arapshian; Susie Couper; Jean M Grow; Peter Kouides; Michael Laffan; Michelle Lavin; Frank W G Leebeek; Sarah H O'Brien; Margareth C Ozelo; Alberto Tosetto; Angela C Weyand; Paula D James; Mohamad A Kalot; Nedaa Husainat; Reem A Mustafa Journal: Blood Adv Date: 2021-01-12
Authors: Romina Brignardello-Petersen; Abdallah El Alayli; Nedaa Husainat; Mohamad A Kalot; Shaneela Shahid; Yazan Aljabirii; Alec Britt; Hani Alturkmani; Hussein El-Khechen; Shahrzad Motaghi; John Roller; Rezan Abdul-Kadir; Susie Couper; Peter Kouides; Michelle Lavin; Margareth C Ozelo; Angela Weyand; Paula D James; Nathan T Connell; Veronica H Flood; Reem A Mustafa Journal: Blood Adv Date: 2022-01-11