Efficacy of tranexamic acid for the prevention of bleeding in patients with von Willebrand disease and Glanzmann thrombasthenia: a controlled, before and after trial.

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.

Association between serum ferritin level and diastolic cardiac function in patients with major ß-thalassemia.

BACKGROUND: Prevention of myocardial siderosis is a key step to reduce rate of mortality in thalassemic patients. Our objective was to study association between echocardiography parameters and serum ferritin level in patients with major thalassemia. MATERIALS AND METHODS: Sixty-six patients with major thalassemia were studied in Amir Kabir hospital, Arak, Iran. Serum ferritin levels were measured during 3 months in patients with no symptoms of infection. It was measured by enzyme-linked immunosorbent assay (ELISA). Ejection Fraction (EF), Fractional Shortening (FS) and Early/Late ratio (E/A) were studied by echocardiography. RESULTS: Fifty two percent were female and 48% were male. Mean age was 16 ± 9 years and the age range was3-26years. Mean serum ferritin level was1912 ± 1748 ng/dl and its range was from 303 to 8333 ng/dl. There were significant correlations between serum ferritin level and EF(r=0.3 and P=0.05) and also between serum ferritin level and FS. CONCLUSION: Due to significant association between serum ferritin level and echo parameters, it is beneficial that all patients with major thalassemia undergo echocardiography to gain better understating about cardiac function.

Association between serum ferritin level, cardiac and hepatic T2-star MRI in patients with major ß-thalassemia.

BACKGROUND: Frequent blood transfusion is often associated with iron overload. Proper use of iron chelators to treat iron overload requires an accurate measurement of iron levels. Magnetic resonance T2-star (T2* MRI) can measure iron level in the heart and liver. Our goal was to see whether an association exists between serum ferritin level and T2* MRI in patients with major beta thalassemia. MATERIALS AND METHODS: Sixty patients with a diagnosis of major beta thalassemia were enrolled in the study. They were older than five years old and needed regular transfusion. Cardiac and hepatic T2*MRI and mean serum ferritin levels were measured within 3 months. RESULTS: No significant correlation was observed between serum ferritin level and cardiac T2*MRI (p=0.361, r=-0.120).However, a significant correlation was observed between serum ferritin and liver T2*MRI (p=0.021, r=-0.297). CONCLUSION: Our results showed an association between hepatic T2*MRI and serum ferritin level.