ABSTRACT
The aim of the study is to assess the relationship between T2* magnetic resonance imaging (MRI) values and age, serum ferritin level, left ventricular ejection fraction (LVEF), splenectomy status, and to identify appropriate modifications to chelation therapy based on T2* MRI results of children with thalassaemia major. Sixty-four patients with thalassaemia major (37 girls/27 boys) older than 8 years of age were enrolled in the study. Based on the first T2* MRI, the patients' myocardial iron depositions were classified into three groups: T2* MRI <10 ms (high risk group), T2* MRI 10-20 ms (medium-risk group) and T2* MRI >20 ms (low-risk group). There was no significant relationship between T2* MRI value and ages, serum ferritin levels and splenectomy status of thalassaemia major patients. The mean LVEFs were 60, 75, and 72.5 % in the high-, medium-, and low-risk groups, respectively (P = 0.006). The mean cardiac iron concentrations calculated from the T2* MRI values were 4.96 ± 1.93, 1.65 ± 0.37, and 0.81 ± 0.27 mg/g in the high-, medium-, and low-risk groups, respectively. Chelation therapies were re-designed in 24 (37.5 %) patients according to cardiac risk as assessed by cardiac T2* MRI. In conclusion, until recently, T2* MRI has been employed to demonstrate cardiac siderosis without a direct relationship with the markers used in follow-up of patients with thalassaemia. However, modifications of chelation therapies could reliably be planned according to severity of iron load displayed by T2* MRI.
Subject(s)
Iron Chelating Agents/therapeutic use , Iron Overload/diagnosis , Iron Overload/drug therapy , Magnetic Resonance Imaging , Myocardium/pathology , beta-Thalassemia/complications , Adolescent , Adult , Child , Echocardiography , Female , Ferritins/blood , Heart/physiopathology , Humans , Iron/metabolism , Iron Overload/etiology , Male , Myocardium/metabolism , Splenectomy , Stroke Volume , Transfusion Reaction , Treatment Outcome , Young Adult , beta-Thalassemia/diagnosis , beta-Thalassemia/therapyABSTRACT
Congenital factor VII deficiency is the most common form of rare coagulation factor deficiencies. This article presents a retrospective evaluation of 73 factor VII deficiency cases that had been followed at our center. The study consisted of 48 males and 25 females (2 months-19 years). Thirty-one (42.5%) of them were asymptomatic. Out of symptomatic patients, 17 had severe clinical symptoms, whereas 8 presented with moderate and 17 with mild symptoms. The symptoms listed in order of frequency were as follows: epistaxis, petechia or ecchymose, easy bruising, and oral cavity bleeding. The genotype was determined in 8 patients. Recombinant activated factor VII (rFVIIa) was used to treat 49 bleeding episodes in 8 patients after 2002. In 2 patients with repeated central nervous system bleeding prophylaxis with rFVIIa was administered. No allergic and thrombotic events were observed during both treatment and prophylaxis courses. Antibody occurrence was not detected in the patients during treatment.
