Words similar to thalassemia
Example sentences for: thalassemia
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In the first study, 144 patients with thalassemia major and relatively low serum ferritin concentrations (1,500 - 3,000 ng/mL) were randomized to either deferiprone (n = 71) or desferrioxamine (n = 73) and followed for one year [ 28 ] . Although, the primary efficacy measure was the reduction of serum ferritin, HIC was assessed in a small subgroup of patients willing to undergo repeat liver biopsy: 21 in the deferiprone and 15 in the desferrioxamine group.
The studies identified were carefully evaluated for eligibility; they were included if they (i) enrolled subjects with thalassemia major - irrespective of age at diagnosis, treatment initiation or study start nor of treatment history in terms of transfusion regimen or iron chelation; (ii) followed patients treated with either desferrioxamine administered subcutaneously or intravenously or oral deferiprone; and (iii) measured hepatic iron concentrations to evaluate treatment efficacy.
Until such a study becomes available, we present this comprehensive and quantitative review of the evidence to aid patients with thalassemia major and their physicians in clinical judgment and treatment decisions.
Measurement of HIC has been established in the field as the reference method for assessing total body iron burden in thalassemia major, and therefore preferred for research purposes.
Improvements in the management of patients with thalassemia major in the past four decades have resulted in "one of the most dramatic alterations in morbidity and mortality associated with a genetic disease" [ 2 ] . Regular red blood cell transfusions extend survival, eliminate complications of anemia, inhibit bone marrow hyperactivity, and support normal growth and development in patients with thalassemia major [ 3 ] . Unfortunately, regular transfusions also lead to the accumulation of tissue iron, loading the body's organs to the point of dysfunction and finally death in the second or third decade of life if left untreated [ 4 ] .
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