The transfusion management of beta thalassemia in the United States

Abstract

The β thalassemia syndromes constitute the most frequent inherited anemia managed with chronic red cell transfusions around the world. 1,2 The prevalence of thalassemia in the United States was underestimated in past surveys that were limited only to the major specialty centers. 3 Recent data show that the aggregate number of patients followed at smaller centers and community practices surpasses those at the major centers. 4 A precise estimate of the total number of individuals with thalassemia in the U.S. is unavailable due to the lack of either a state or national database. Surveys have shown that 10 large thalassemia centers in the country collectively follow about 1100 patients, while additional 1500 patients are estimated to receive care at other hospitals. 4,5 The Thalassemia Western Consortium (TWC) study showed that of the 717 patients, 44% of patients had β thalassemia syndromes (including 15% with HbE β thalassemia) and 55% had various α thalassemia disorders. Transfusion-dependent patients comprised 35% of the population, 9% had received 1 or more life-time transfusions, and 56% had never been transfused. Thus,

Clinical pathology aspect on diagnosis cholelithiasis in β-Thalassemia patient: A case report

“…This continuous transfusion can result in iron overload in many organs, such as the heart, liver, kidneys, endocrine organs and others. The accumulation of free iron (ferrous iron/Fe2+) can cause a Fenton reaction that forms free radicals, causing oxidative stress, which will oxidize cell lipid membranes (lipid peroxidation) and nucleic acid modification, causing cell death, tissue damage and ultimately organ damage [ [9] , [10] , [11] ].…”

Infantile-onset Pompe disease complicated by sickle cell anemia: Case report and management considerations

“…Typically, chronic red blood cell transfusion therapy is utilized in SCA for stroke prevention with a primary goal of maintaining Hb S <30%, which may result in a nadir Hb pre-transfusion of <8 g/dl ( 33 ). In the current case, since this degree of anemia may compromise growth and increase the risk of cardiac stress, a transfusion strategy similar to that utilized in children with beta-thalassemia major was chosen, in which total hemoglobin concentration is maintained at 9–10 g/dl to minimize anemia-related organ dysfunction ( 34 ). Chronic transfusions will cause iron overload, which over many years can increase risk of heart failure but is manageable with iron chelating drugs ( 34 ).…”

“…In the current case, since this degree of anemia may compromise growth and increase the risk of cardiac stress, a transfusion strategy similar to that utilized in children with beta-thalassemia major was chosen, in which total hemoglobin concentration is maintained at 9–10 g/dl to minimize anemia-related organ dysfunction ( 34 ). Chronic transfusions will cause iron overload, which over many years can increase risk of heart failure but is manageable with iron chelating drugs ( 34 ). Hydroxyurea is another option for disease-modifying therapy, but it is not recommended until age 9 months.…”

The Association Between Caregiver Coping and Youth Clinic Attendance and Health-related Quality of Life in Pediatric Sickle Cell Disease

“…monthly3) or receiving chronic blood transfusions (e.g. once every 3 to 4 wk4). Unfortunately, despite its importance for disease management, clinic attendance rates for pediatric SCD are between 46% to 77%,3,5 and youth with SCD report significantly lower HRQOL across domains compared with healthy peers 6–8…”