Scientific Literature
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Renal Function in β-Thalassemia Major Patients Treated with Two Different Iron-Chelation Regimes
Background Renal injury in transfusion dependent β thalassemia patients (TDT) has been attributed to iron overload, chronic anemia and iron-chelation therapy (ICT) toxicity. We studied renal function in TDT patients…
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Prenatal Diagnosis Leads to Early Diagnosis of Transfusion-Dependent Thalassemia and Better Growth Outcomes
Thalassemia is the most common hematological transfusion-dependent disease in Thailand. Even though prenatal diagnosis (PND) can detect the condition, many new cases are diagnosed in pediatric practice. This study assessed…
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Glucose Metabolism and Insulin Response to Oral Glucose Tolerance Test (OGTT) in Prepubertal Patients with Transfusion-dependent β-Thalassemia (TDT): A Long-term Retrospective Analysis
Glucose dysregulation (GD), including prediabetes and diabetes mellitus (DM), is a common complication of transfusion-dependent β-thalassemia (TDT) patients. The prevalence increases with age and magnitude of iron overload, affecting a…
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Effect of HBB genotype on survival in a cohort of transfusion-dependent thalassemia patients in Cyprus
Initiation of regular transfusion in transfusion-dependent thalassemia (TDT) is based on the assessment of clinical phenotype. Pathogenic HBB variants causing β-thalassemia are important determinants of phenotype and could be used to aid…
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The Transfusion Management of Beta Thalassemia in the United States
These are the first recommendations directed toward hematologists and transfusion medicine specialists that are designed to specifically address concerns that are pertinent to the thalassemia population in the U.S. Here,…
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Pregnancy Outcomes in Women Affected by Fetal Alpha-Thalassemia: A Case Control Study
To evaluate the possible associations between fetal α-thalassemia and risk of adverse pregnancy outcomes using a provincial woman-child health service information database in China. This was a case control study…
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How to Facilitate Decision-Making for Hematopoietic Stem Cell Transplantation in Patients With Hemoglobinopathies. The Perspectives of Healthcare Professionals
Hematopoietic stem cell transplantation decision-making for hemoglobinopathy patients is a complex process, and it remains difficult for health care professionals to decide whether and when a hematopoietic stem cell transplantation…
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The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease
Crizanlizumab is a monoclonal antibody that binds to P-selectin. On October 28, 2020, a conditional marketing authorization valid through the European Union (EU) was issued for crizanlizumab for the prevention…
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Economic Evaluation of Betibeglogene Autotemcel (Beti-cel) Gene Addition Therapy in Transfusion-Dependent β-Thalassemia
Background: Standard of care (SoC) for transfusion-dependent β-thalassemia (TDT) requires lifelong, regular blood transfusions as well as chelation to reduce iron accumulation. Objective: This study investigates the cost-effectiveness of betibeglogene…
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Research in Sickle Cell Disease: From Bedside to Bench to Bedside
Sickle cell disease (SCD) is an exemplar of bidirectional translational research, starting with a remarkable astute observation of the abnormally shaped red blood cells that motivated decades of bench research…
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