Antoine N. Saliba, MD, and colleagues summarized current literature surrounding the frequent complications that bring patients with thalassemia into the ED.
In communities where consanguineous marriages are commonplace, nonsibling matched donor transplants may represent a viable option in patients with beta-thalassemia.
Preliminary results suggest that CRISPR-Cas9 editing of BCL11A may benefit patients with sickle cell disease and β-thalassemia by potentially eliminating vaso-occlusive episodes or the need for transfusion.
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.
When compared with placebo, luspatercept increased the percentage of patients with transfusion-dependent beta-thalassemia who had a reduction in transfusion burden.
Mortality in beta-thalassemia has decreased due to medical advances, allowing physicians to observe and manage disease-related complications in adult patients.