Hematopoietic Stem Cells Primed for Erythroid Lineage in β-Thalassemia
How might β-thalassemia, an erythropoietic disorder, affect “erythroid branching” in hematopoietic differentiation?
Thalassemias
FDA Grants Orphan Drug Status to Mitapivat for Thalassemia
The FDA has granted Orphan Drug designation to mitapivat (AG-348; Agios Pharmaceuticals) for the treatment of thalassemia.
Promising New Noninvasive Test Developed for β-Thalassemia in At-Risk Pregnancies
Compared to invasive prenatal diagnosis, a noninvansive assay may be a viable alternative for genotyping a fetus in pregnancy at high-risk for β-thalassemia.
Ferriprox Twice-a-Day Tablet Dosing Approved for Transfusional Iron Overload Due to Thalassemia Syndromes
The FDA has approved a twice-a-day formulation of Ferriprox® (deferiprone; Chiesi) for the treatment of transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate.
Altered Bone Marrow Microenvironment Induces Defect of Hematopoietic Stem Cells in β-Thalassemia
For patients with β-thalassemia who undergo HSCT, exposure to a normal microenvironment and the impairment of HSCs may be reversible in the presence of a normal microenvironment and in vivo protein restoration.
Assessing Infectious, Thrombotic Complications of Splenectomy in Children With Nonmalignant Hematologic Disorders
Splenectomy may be a safe therapeutic options for children with nonmalignant hematologic diseases who have proper vaccinations and follow-up care.
F-Erythroblasts Not Significantly Different From Non-HbF-Expressing Cells
Neither transcriptional nor proteomic profiling revealed significant differences between F- and A-cells at baseline or after HbF-inducing treatment.
Luspatercept Improves Transfusion Burden in Patients with Transfusion-Dependent Beta-Thalassemia
When compared with placebo, luspatercept increased the percentage of patients with transfusion-dependent beta-thalassemia who had a reduction in transfusion burden.
Managing Complications Associated With Increased Longevity in Beta Thalassemia
As the life expectancy for patients with beta thalassemia continues to increase, more and more patients are experiencing age-related complications.
Novel Conditioning Regimen for Haploidentical HSCT in Severe Thalassemia
Both event-free and overall survival at 3 years were 96% for patients with transfusion-dependent thalassemia who underwent transplant.
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