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Öğe Assessment of diastolic function in children and adolescents with beta-thalassemia major by tissue Doppler imaging(Aves, 2011) Yavuz, Taner; Nisli, Kemal; Oner, Naci; Ertugrul, Turkan; Salcioglu, Zafer; Aydogan, Gonul; Karakas, ZeynepAim: The purpose of this study was to analyze myocardial diastolic function in patients with beta-thalassemia major before development of overt cardiomyopathy using pulsed wave tissue Doppler imaging, and compare data with conventional Doppler echocardiography. Material and Method: The study included 61 beta-thalassemia major subjects (age 4 to 20 years; mean age, 10.7 +/- 4.1 years; 32 females and 29 males) with normal left ventricular function and 52 healthy control subjects, matched for age and sex. All participants underwent M-mode echocardiography and left ventricular systolic function was measured; diastolic functions of the right and left ventricul were analysed using tissue Doppler imaging and conventional Doppler echocardiography. SPSS for Windows 13.0 software programme was used for statistical analysis, and the student's t-test was used to compare data. This study was approved by the ethics committee of the Istanbul Medical Faculty. Results: Body surface area was significantly smaller in the patients than in the controls (1.0 +/- 0.2 vs. 1.2 +/- 0.3 m(2), p < 0.01). Transmitral early peak velocity and ratio were found to be significantly increased in patients compared with the controls (p < 0.01 and p=0.039, respectively). Late peak velocity of mitral valve and all Doppler velocities across the tricuspid valve did not differ between groups (p > 0.05). In patients with beta-thalassemia major, the early diastolic velocities of the myocardium at the base of the left and right ventricle, at the middle segment of the left and right ventricle, and the interventricular septum were found to be higher than controls (p < 0.05). We also found significantly higher late diastolic velocities at the base and middle segments of right ventricle, early and late diastolic velocities ratio at the base of the right ventricle and at the middle segment of the left and right ventricle, and the interventricular septum compared with controls (p < 0.05). Conclusions: These findings acquired from the young aged beta-thalassemia major patients with normal ventricular systolic function were believed to be results from high preload and hyperdynamic response to chronic anemia rather than true ventricular restriction. We suggest that long-term follow-up studies should be carried out in patients with beta-thalassemia major using tissue Doppler in order to evaluate the diagnostic accuracy of this imaging technique in diagnosis of early stages of cardiac involvement. (Turk Arch Ped 2011; 46: 26-32)Öğe Deferasirox in children with transfusion-dependent thalassemia or sickle cell anemia: A large cohort real-life experience from Turkey (REACH-THEM)(Wiley, 2019) Antmen, Bulent; Karakas, Zeynep; Yesilipek, Mehmet Akif; Kupesiz, Osman Alphan; Sasmaz, Ilgen; Uygun, Vedat; Kurtoglu, ErdalObjectives To evaluate the long-term efficacy and safety of deferasirox therapy in a large observational cohort of children with transfusion-dependent thalassemia (TDT) and sickle cell anemia (SCA) in Turkey. Methods This was a multicenter, prospective cohort study including TDT and SCA patients aged 2-18 years with iron overload (>= 100 mL/kg of pRBC or a serum ferritin [SF] level >1000 mu g/L) receiving deferasirox. Patients were followed for up to 3 years according to standard practice. Results A total of 439 patients were evaluated (415 [94.5%] TDT, 143 [32.6%] between 2 and 6 years). Serum ferritin levels consistently and significantly decreased across 3 years of deferasirox therapy from a median of 1775.5 to 1250.5 mu g/L (P < 0.001). Serum ferritin decreases were noted in TDT (1804.9 to 1241 mu g/L), SCA (1655.5 to 1260 mu g/L), and across age groups of 2-6 years (1971.5 to 1499 mu g/L), 7-12 years (1688.5 to 1159.8 mu g/L), and 13-18 years (1496.5 to 1107 mu g/L). Serum ferritin decreases were also noted for all deferasirox dose groups but only significant in patients with doses >= 30 mg/kg/d (n = 120, -579.6 median reduction, P < 0.001). Only 9 (2%) patients had adverse events suspected to be related to deferasirox. Serum creatinine slightly increased but remained within the normal range. Conclusions Deferasirox has long-term efficacy and safety in children with TDT and SCA, although higher doses (>= 30 mg/kg/d) may be required to achieve iron balance.