Scientific Literature
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Newborn Screening for Sickle Cell Disease in Catalonia between 2015 and 2022: Epidemiology and Impact on Clinical Events
In 2015, Catalonia introduced sickle cell disease (SCD) screening in its newborn screening (NBS) program along with standard-of-care treatments like penicillin, hydroxyurea, and anti-pneumococcal vaccination. Few studies have assessed the…
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Management of Transfusion-Dependent β-Thalassemia (TDT): Expert Insights and Practical Overview from the Middle East
β-Thalassaemia is one of the most common monogenetic diseases worldwide, with a particularly high prevalence in the Middle East region. As such, we have developed long-standing experience with disease management…
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Challenges of Iron Chelation in Thalassemic Children
Thalassaemia treatment still relies on supportive care, mainly including blood transfusion and iron chelation therapy. Iron chelation is considered the main factor responsible for the marked improvement in survival rates…
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TIF Literature
Assessing Disability in Thalassaemia: A Position Statement by the Thalassaemia International Federation Farmakis, D., Angastiniotis, M., Papingiotis, G., Cannon, L., & Eleftheriou, A. European Journal of Haematology.(2024). Exciting Science from…
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Iron Chelation Therapy for Children with Transfusion‐Dependent β‐Thalassemia: How Young Is Too Young?
In this review, we provide a summary of evidence on iron overload in young children with transfusion-dependent β-thalassemia (TDT) and explore the ideal timing for intervention. Key data from clinical…
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Bone Health Impairment in Patients with Hemoglobinopathies: From Biological Bases to New Possible Therapeutic Strategies
Hemoglobinopathies are monogenic disorders affecting hemoglobin synthesis. Thalassemia and sickle cell disease (SCD) are considered the two major hemoglobinopathies. Thalassemia is a genetic disorder and one of the major hemoglobinopathies…
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Early Detection of Renal Complication in Children With Sickle Cell Disease: A Single Center Prospective Study
Introduction: This observational cross-sectional study aimed to identify predictors of renal complications in pediatric patients with sickle cell disease (SCD) at King Salman Armed Forces Hospital, Tabuk, Saudi Arabia, over…
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Hoping for a Normal Life: Decision‐Making on Hematopoietic Stem Cell Transplantation by Patients With a Hemoglobinopathy and Their Caregivers
Background: To provide insight into the perspectives of children and young adults with transfusion-dependent thalassemia and sickle cell disease and their caregivers regarding the decision for hematopoietic stem cell transplantation…
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Liver Disease in Patients With Transfusion-Dependent β-Thalassemia: The Emerging Role of Metabolism Dysfunction-Associated Steatotic Liver Disease
In this Editorial, we highlight the possible role that metabolism dysfunction-associated steatotic liver disease (MASLD) may play in the future, regarding liver disease in patients with transfusion-dependent β-thalassemia (TDBT). MASLD…
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Global Longitudinal Strain by Cardiac Magnetic Resonance Associated With Cardiac Iron and Complications in β-Thalassemia Major Patients
Background: The aim of this cross-sectional study was to investigate the association of left ventricular (LV) strain parameters with demographics, clinical data, cardiovascular magnetic resonance (CMR) findings, and cardiac complications…
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