Researchers sought to determine whether vitamin D deficiency before hematopoietic stem cell transplantation would impact risk of GVHD in thalassemia major.
Researchers sought to determine whether longitudinal strain with conventional TTE parameters could identify subclinical myocardial dysfunction associated with iron overload in thalassemia.
Researchers sought to determine the prevalence of fractures and osteoporosis among adolescents and young adults with transfusion-dependent thalassemia.
Zynteglo is a one-time gene therapy that works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells.
Beti-cel is an investigational one-time gene therapy that works by adding functional copies of a modified form of the beta-globin gene into a patient’s own hematopoietic stem cells.
Researchers sought to identify lipid profiles of red blood cell membranes in patients with beta-thalassemia, in the context of IVS mutations and hydroxyurea treatment.
Researchers sought to determine mortality rates among patients with beta-thalassemia and pulmonary arterial hypertension who had right heart catheterization.
Researchers sought to determine whether silymarin would be safe and effective in decreasing serum ferritin levels in patients with beta-thalassemia intermedia.
Researchers sought to assess the outcomes for patients with transfusion-dependent thalassemia based on the use of a fresh blood product vs a stored blood product.
Researchers sought to determine whether amlodipine with chelation therapy would be more effective in reducing cardiac iron overload in transfusion-dependent thalassemia.
Betibeglogene autotemcel (beti-cel) is a one-time gene therapy that works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells.
Researchers sought to determine whether there is a need for regular kidney surveillance to detect dysfunction among pediatric patients with transfusion-dependent thalassemia.
Researchers sought to determine a correlation between endocrine levels and iron overload in patients with transfusion-dependent beta thalassemia and growth retardation.
Researchers sought to determine whether MRI T2 is an effective way to detect iron concentrations in the heart, liver, and pancreas during chelation therapy for patients with thalassemia major.
Researchers sought to determine whether carvedilol would improve left ventricular diastolic function in patients with transfusion‑dependent thalassemia who had iron overload cardiomyopathy.
Researchers sought to validate the usefulness of cardiac T2* magnetic resonance imaging to evaluate cardiac siderosis in patients with thalassemia major.
Researchers sought to determine whether TBI could help improve treatment with allogeneic blood or marrow transplantation in patients with severe hemoglobinopathies.
Researchers sought to determine whether deferasirox would be a good option for long-term management of iron overload in patients who receive iron chelation therapy.
Researchers sought to determine whether CRISPR-Cas9 gene therapy would be an effective treatment for patients with sickle cell disease or -thalassemia.
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.
Antoine N. Saliba, MD, and colleagues summarized current literature surrounding the frequent complications that bring patients with thalassemia into the ED.