Scientific Literature
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LentiGlobin Administration to Sickle Cell Disease Patients: Effect on Serum Markers and Vaso-Occlusive Crisis
LentiGlobin, an innovative gene therapy, introduces a modified beta-globin gene that yields an anti-sickling hemoglobin variant. It boosts total hemoglobin levels, mitigates hemolysis, curtails inflammation, and addresses iron overload by…
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‘Phenoconversion’ in Adult Patients with β-Thalassemia
Rate and risk factors for phenoconversion from non-transfusion-dependent β-thalassemia (NTDT) to transfusion-dependent β-thalassemia (TDT) during a 10-year follow up of adult patients in Italy.
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Pregnancy Outcomes and Iron Status in β-Thalassemia Major and Intermedia: A Systematic Review and Meta-analysis
Advancements in orally bioavailable iron chelators and MRI methods have improved life expectancy and reproductive potential in Thalassemia Major (TM) and Thalassemia Intermedia(TI). Pregnancy is associated with adverse maternal and…
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A Cross-Sectional, Multicenter, Disease-Specific, Health-Related Quality of Life study in Greek Transfusion Dependent Thalassemia Patients
The assessment of Health-Related Quality of Life (HRQoL) in Thalassemia offers a holistic approach to the disease and facilitates better communication between physicians and patients. This study aimed to evaluate…
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Economic and Clinical Burden of Managing Transfusion-Dependent b-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…
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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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