Catherine Skari

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…

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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…

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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…

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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,…

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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…

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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…

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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…

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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…

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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…

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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…

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