Scientific Literature
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Efficacy and Safety οf Mitapivat in Adults with Transfusion-Dependent Α-Thalassaemia or Β-Thalassaemia (ENERGIZE-T): A Double-Blind, Randomised, Multicentre, Placebo-Controlled, Phase 3 Trial
Abstract Background: The absence of disease-modifying therapies for patients with α-thalassaemia and oral disease-modifying therapies for patients with β-thalassaemia has been a substantial unmet need in these patients. We assessed the…
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Delayed Recognition and Significant Morbidity in Adults with Non-Transfusion-Dependent Thalassemia
Abstract: Non-transfusion-dependent thalassemia (NTDT) is characterized by variable degrees of anemia, ineffective erythropoiesis, and iron overload, with a heightened risk of age-related complications. However, the clinical profiles of patients who…
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Global Burden of Thalassemia by Socio-Demographic Index, 1990–2023: Trends, Disparities, and Future Implications
Thalassemia imposes a substantial and unequal global health burden, yet its temporal evolution stratified by sociodemographic development remains incompletely characterized using the most recent Global Burden of Disease (GBD) data. …
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EHA Recommendations for Preconceptual and Antenatal Screening and Prenatal Diagnosis for Hemoglobinopathies
Abstract Thalassemia and sickle cell disease (SCD) are among the most common monogenic disorders worldwide. They cause chronic hemolytic anemia, the consequences and prognosis of which vary considerably depending on…
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In Utero Hematopoietic Cell Transplantation in Fetuses With Α-Thalassemia Major: A Phase 1 Clinical Trial
Abstract In utero hematopoietic cell transplantation (IUHCT) has the potential to treat patients who have hemoglobinopathies by harnessing the unique period of fetal tolerance to maternal cells, thereby enabling semi-allogeneic…
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Transforming The Treatment of Alpha-Thalassemia: A Single-Center Retrospective Study on Hematopoietic Stem Cell Transplantation in Transfusion-Dependent Pediatric Patients
Abstract Hematopoietic stem cell transplantation (HSCT) is the only definitive cure for transfusion-dependent α-thalassemia, though comprehensive studies on its effectiveness are limited. In this retrospective study, we analyzed the clinical…
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Disease-Related Complications and Risk Factors in Hemoglobin H Disease in a Thai Multicentre Registry
Abstract Hemoglobin H (Hb H) disease is a prevalent hereditary hemolytic anemia worldwide. Clinical severity varies by genotype, particularly between deletional and non-deletional forms, as well as the presence of…
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Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease
BACKGROUND Sickle cell disease is characterized by chronic hemolytic anemia and recurrent severe vaso-occlusive crises. Ristoglogene autogetemcel (risto-cel) includes autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited…
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Daily Zinc Supplementation for Infection Prevention in Children With Sickle Cell Anemia: The ZIPS-2 Randomized Clinical Trial
Abstract Importance: Despite existing prevention strategies, infections remain a major cause of morbidity and mortality in children in Africa with sickle cell anemia. Objective: To determine the safety and effectiveness of daily…
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Contemporary Global Burden of Sickle Cell Anaemia Under-5 and Under-20: A Systematic Review and Meta-Analysis
Abstract Sickle cell disease (SCD), a prevalent inherited non-communicable disease, remains a neglected public health priority, especially among children and adolescents in many low- and middle-income countries. The global burden…
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