Eghbali A, Melikof L, Taherahmadi H, Bagheri B
Department of Pediatric Hematology and Oncology, Arak University of Medical Sciences, Arak, Iran.
Cancer Research Center and Department of Pharmacology, Semnan University of Medical Sciences, Semnan, Iran.
Haemophilia. 2016 Sep;22(5):e423-6. doi: 10.1111/hae.13051. Epub 2016 Aug 1.
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.
To study the prophylactic effects of tranexamic acid (TXA) for reduction of bleeding episodes in patients with VWD and GT in non-invasive conditions.
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.
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.
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.
在血管性血友病（VWD）和血小板无力症（GT）等血液系统疾病中，减少出血发作非常重要。尽管预防性药物价格可承受，但替代因子非常昂贵。
研究氨甲环酸（TXA）在非侵入性条件下对VWD和GT患者减少出血发作的预防作用。
在伊朗阿拉克的阿米尔卡比尔医院进行了一项单中心对照双盲前后试验。该研究针对17例VWD患者和3例GT患者，最小年龄为2岁。患者接受6个月的安慰剂治疗，以评估出血的频率和严重程度，并记录因子浓缩物和血小板输注的使用频率。之后，患者口服单剂量TXA 25 mg·kg⁻¹·d⁻¹，持续6个月。在研究的两个阶段对上述结果进行研究和比较。对所有患者进行安全性评估。
氨甲环酸使VWD患者1级和2级出血发作次数显著减少（分别为P < 0.001和P < 0.01）。此外，TXA治疗与因子浓缩物的使用显著减少相关（P < 0.05）。使用TXA的GT患者出血发作次数减少；然而，两个研究阶段之间的差异不显著（P = 0.1）。TXA对GT患者血小板输注频率没有影响。TXA治疗与头痛、背痛和肌肉骨骼疼痛有关。治疗后未观察到过敏或血栓栓塞事件病例。
结果表明，TXA在减少VWD患者出血和因子浓缩物使用方面是安全有效的。此外，TXA治疗可减少GT患者的出血。
