Hematopoietic Stem Cells Primed for Erythroid Lineage in β-Thalassemia
How might β-thalassemia, an erythropoietic disorder, affect “erythroid branching” in hematopoietic differentiation?
How might β-thalassemia, an erythropoietic disorder, affect “erythroid branching” in hematopoietic differentiation?
The FDA has granted Orphan Drug designation to mitapivat (AG-348; Agios Pharmaceuticals) for the treatment of thalassemia.
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.
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.
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.
Splenectomy may be a safe therapeutic options for children with nonmalignant hematologic diseases who have proper vaccinations and follow-up care.
Neither transcriptional nor proteomic profiling revealed significant differences between F- and A-cells at baseline or after HbF-inducing treatment.
When compared with placebo, luspatercept increased the percentage of patients with transfusion-dependent beta-thalassemia who had a reduction in transfusion burden.
As the life expectancy for patients with beta thalassemia continues to increase, more and more patients are experiencing age-related complications.
Both event-free and overall survival at 3 years were 96% for patients with transfusion-dependent thalassemia who underwent transplant.