Denosumab Promising for Transfusion Dependent Thalassemia-Induced...
HealthDay News — Denosumab (DNM), a fully human monoclonal antibody against the receptor activator of nuclear factor kappa-B ligand (RANKL), may be beneficial for managing transfusion-dependent...
View ArticleManaging Atrial Fibrillation in Patients With Beta Thalassemia Major
The use of transfusion and iron chelation therapy has extended the lifespan of patients with beta thalassemia major (BTM), allowing patients who may previously have died in childhood to survive to over...
View ArticleA Novel Health-Related Quality of Life Assessment Tool for...
A new tool for measuring patient-reported outcomes (PROs) has been developed for patients with nontransfusion-dependent beta thalassemia (NTDT) and was presented in the American Journal of Hematology....
View ArticleNew Iron Chelator May Improve Cardiac Function in Patients With Beta Thalassemia
A longitudinal study, published in the American Journal of Hematology, demonstrated that a new oral chelator, deferasirox, can improve cardiac iron overload and effectively maintain cardiac iron levels...
View ArticleLuspatercept May Improve Hemoglobin Levels in Patients With Beta Thalassemia
Luspatercept, a novel recombinant fusion protein that targets specific transforming growth factor (TGF)-beta proteins involved in late-stage red blood cell maturation, may lead to improvements in...
View ArticleThe Efficacy of Intrabone Gene Therapy for Transfusion-Dependent Beta...
Intrabone allogeneic hematopoietic stem cell (HSC) gene therapy after a reduced-toxicity myeloablative conditioning regimen may be safe and efficacious in both adult and pediatric patients with...
View ArticleWhite Matter Changes and Headache in Thalassemia
Thalassemia and white matter changes (WMCs) in the brain may be associated with greater likelihood of experiencing headache, according to a study published in the British Journal of Haematology....
View ArticleDoes Thalassemia Increase the Risk of Malignancies?
Screening for leukemia and lymphoma should be offered to patients with thalassemia when they have overlapping symptoms such as anemia, fatigue, and splenomegaly, according to an article published in...
View ArticleReducing Transfusion Burden and Addressing Unmet Needs in Beta Thalassemia
The beta thalassemias are common inherited blood disorders characterized by ineffective erythropoiesis, often leading to severe anemia. If left untreated, serious complications, such as heart failure,...
View ArticleUtility of Flow Cytometry to Detect Red Blood Cell Alloimmunization in...
The use of flow cytometry may allow more accurate detection of red blood cell (RBC) autoantibody formation in patients with thalassemia compared with direct Coombs test, according to results reported...
View ArticleAs Survival Increases in Beta Thalassemia, Renal Complications Emerge
Beta thalassemias are a group of common monogenic disorders, with the highest prevalence in the Mediterranean region, Africa, and Southeast Asia. However, beta thalassemias are increasingly being...
View ArticleImproving Bone Mass With Iron Chelation Therapy in Transfusion-Dependent Beta...
Iron chelation therapy (ICT) with a combination of deferasirox (DFX) and deferoxamine (DFO) may improve bone mass in patients with transfusion-dependent beta thalassemia (TDT) while lowering serum...
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Mineral and Hormonal Dysfunction Associated With Lower BMD in Thalassemia Major
Hypothyroidism, hyperglycemia, and low adrenocorticotrophic hormone (ACTH) levels are associated with lower bone mass in patients with thalassemia major, according to study results published in The...
View ArticleNovel Conditioning Regimen for Haploidentical HSCT in Severe Thalassemia
A novel conditioning regimen for hematopoietic stem cell transplantation (SCT) from haploidentical donors in patients with severe thalassemia appears to be safe and efficacious, yielding results...
View ArticleManaging Complications Associated With Increased Longevity in Beta Thalassemia
Beta thalassemia syndromes are among the most common monogenic disorders that occur globally and are generally inherited as autosomal recessive diseases. The beta globin gene HBB is found on the short...
View ArticleLuspatercept Improves Transfusion Burden in Patients with...
According to the results of a study published in The New England Journal of Medicine, luspatercept, a first-in-class erythroid maturation agent, increased the percentage of patients with...
View ArticleAltered Bone Marrow Microenvironment Induces Defect of Hematopoietic Stem...
Impaired hematopoietic stem cell (HSC) self-renewal in β-thalassemia is induced by an altered bone marrow microenvironment and correction of HSC-stromal niche crosstalk rescues thalassemic HSC...
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Ferriprox Twice-a-Day Tablet Dosing Approved for Transfusional Iron Overload...
The Food and Drug Administration (FDA) has approved a twice-a-day formulation of Ferriprox® (deferiprone; Chiesi) for the treatment of transfusional iron overload due to thalassemia syndromes when...
View ArticlePromising New Noninvasive Test Developed for β-Thalassemia in At-Risk...
A novel noninvasive prenatal diagnosis (NIPD) assay for β-thalassemia appears to be highly reliable and accurate and, with optimization, could serve as an alternative to traditional invasive prenatal...
View ArticleFDA Grants Orphan Drug Status to Mitapivat for Thalassemia
The Food and Drug Administration (FDA) has granted Orphan Drug designation to mitapivat (AG-348; Agios Pharmaceuticals) for the treatment of thalassemia. Mitapivat is a first-in-class, oral, selective...
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