Scientific Literature
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Erythropoiesis in Lower-risk Myelodysplastic Syndromes and Beta Thalassemia
The hematologic disorders myelodysplastic syndromes and beta-thalassemia are characterized by ineffective erythropoiesis and anemia, often managed with regular blood transfusions. Erythropoiesis, the process by which sufficient numbers of functional erythrocytes are produced from hematopoietic…
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Association of Osteoporosis and Sarcopenia with Fracture Risk in Transfusion-Dependent Thalassemia
Patients with transfusion-dependent thalassaemia (TDT) have an increased risk of osteoporosis and fractures. They also have several potential factors associated with sarcopenia. There has been currently no study on sarcopenia…
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Lung Function Decline in Children with Sickle Cell Disease Treated with Hydroxyurea
Sickle cell disease (SCD), the most common form of an inherited hematological disorder, is caused by a genetic variant that leads to the synthesis of an abnormal haemoglobin, HbS. Deoxygenated…
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Splenectomy Significantly Associated with Thrombosis but Not with Pulmonary Hypertension in Patients with Transfusion-Dependent Thalassemia: A Meta-analysis of Observational Studies
Introduction: Thromboembolism (TE) and pulmonary hypertension (PH) constitute frequently occurring complications in patients with transfusion-dependent thalassemia and have been associated with splenectomy in different studies. Nevertheless, the size of the…
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Deferiprone vs Deferoxamine for Transfusional Iron Overload in Sickle Cell Disease and Other Anemias: Pediatric Subgroup Analysis of the Randomized, Open-label FIRST Study
Background: Children with sickle cell disease (SCD) who are chronically transfused often, require iron chelation therapy. There are limited data that allow for comparison of the efficacy and safety of…
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Pneumococcal Infections in Children with Sickle Cell Disease Before and After Pneumococcal Conjugate Vaccines
Children with sickle cell disease (SCD) are at increased risk of invasive pneumococcal infections (IPD). Over 25 years the Georgia Emerging Infections Program/CDC Active Bacterial Core Surveillance network identified 104…
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Impact of Imatinib on Reducing the Painful Crisis in Patients with Sickle Cell Disease
Introduction: Sickle cell disease (SCD) is a common hemoglobinopathy worldwide that causes painful crises and hospitalization of patients. These attacks decrease survival and cause chronic end-organ damage in these patients. Hypothesis: For this reason,…
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Health-Related Quality-of-Life Profile of Pediatric Patients with β-Thalassemia after Hematopoietic Stem Cell Transplantation
Matched hematopoietic stem cell transplantation (HSCT) is a feasible and curative treatment in pediatric patients with beta thalassemia major (β-TM). However, little data are available regarding patients and their parents’…
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Modelling the Public Health Impact of Voxelotor in the Management of Sickle Cell Disease in France
Sickle cell disease (SCD) is an inherited blood disorder in which sickle hemoglobin (HbS) polymerizes, leading to red blood cell sickling and chronic hemolytic anemia, vaso-occlusive crises, and end-organ damage…
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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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