Scientific Literature
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The Danish National Haemoglobinopathy Screening Programme: Report From 16 Years of Screening in a Low-prevalence, Non-endemic Region
The Danish national haemoglobinopathy screening programme seeks to determine parental haemoglobinopathy carrier state antenatally. In this retrospective register based study, we evaluated the 16-year trajectory of this programme, utilising the…
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Analysis of the Pregnancy Status and Outcomes of Pregnant Women with α-Thalassemia: A Retrospective Clinical Study
Background: The prevalence of α-thalassemia is high in Guangxi, and its co-occurrence with pregnancy may impede maternal system functions, with a consequent adverse effect on pregnancy progression and birth outcomes.…
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How I Τreat Non-Transfusion-Dependent β-Thalassemia
The intricate interplay of anemia and iron overload under the pathophysiological umbrella of ineffective erythropoiesis in non-transfusion-dependent β-thalassemia (NTDT) results in a complex variety of clinical phenotypes that are challenging…
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Pregnancy-related Thromboembolism in Women with Sickle Cell Disease: An Analysis of National Medicaid Data
Pregnancy and sickle cell disease (SCD) both individually carry a risk of thromboembolism (TE). Pregnancy in people with SCD may further enhance the prothrombotic effect of the underlying disease. The…
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Speckle Tracking Echocardiography and Β-Thalassemia Major: A Systematic Review
Heart disease is among the primary causes of morbidity and mortality in β-thalassemia major (β-TM). Conventional echocardiography has failed to identify myocardial dysfunction at an early stage among these patients,…
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SLN124, a GalNAc Conjugated 19‐mer siRNA Targeting tmprss6, Reduces Plasma Iron and Increases Hepcidin Levels of Healthy Volunteers
SLN124, an N-acetylgalactosamine conjugated 19-mer short interfering RNA, is being developed to treat iron-loading anemias (including beta-thalassemia and myelodysplastic syndromes) and myeloproliferative neoplasms (polycythemia vera). Through hepatic targeting and silencing…
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Management of the Sickle Cell Trait: An Opinion by Expert Panel Members
The number of individuals with the sickle cell trait exceeds 300 million worldwide, making sickle cell disease one of the most common monogenetic diseases globally. Because of the high frequency…
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Economic and Clinical Burden of Managing Transfusion-dependent β-Thalassemia in the United States
Aims: To describe clinical complications, treatment use, healthcare resource utilization (HCRU), and costs among patients with transfusion-dependent β-thalassemia (TDT) in the United States. Materials and methods: Merative MarketScan Databases were used to…
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Efficacy and Safety of Early-start Deferiprone in Infants and Young Children with Transfusion-dependent Beta Thalassemia: Evidence for Iron Shuttling to Transferrin in a Randomized, Double-blind, Placebo-controlled, Clinical Trial (START)
Children with transfusion‐dependent thalassemia (TDT) require regular blood transfusions that, without iron‐chelation therapy, lead to iron‐overload toxicities. Current practice delays chelation therapy (late‐start) until reaching iron overload (serum ferritin ≥1000 μg/L)…
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Burden of Aging: Health Outcomes Among Adolescents and Young Adults With Sickle Cell Disease
Although ≈95% of children with sickle cell disease (SCD) will reach adulthood in high-income settings, adolescents and young adults (AYA) face difficulties in establishing adult care and experience progression of disease…
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