Learn About Beta Thalassemia
View Main Condition: Thalassemia
Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body.
Variants (also known as mutations) in the HBB gene cause beta thalassemia. The HBB gene provides instructions for making a protein called beta-globin. Beta-globin is a component (subunit) of hemoglobin. Hemoglobin consists of four protein subunits, typically two subunits of beta-globin and two subunits of another protein called alpha-globin.
Beta thalassemia is a fairly common blood disorder worldwide. Thousands of infants with beta thalassemia are born each year. Beta thalassemia occurs most frequently in people from Mediterranean countries, North Africa, the Middle East, India, Central Asia, and Southeast Asia.
Thalassemia major and thalassemia intermedia are inherited in an autosomal recessive pattern, which means both copies of the HBB gene in each cell have variants. The parents of an individual with an autosomal recessive condition each carry one copy of the altered gene, but they typically do not show signs and symptoms of the condition. Sometimes, however, people with only one HBB gene variant in each cell develop mild anemia. These mildly affected people are said to have thalassemia minor.
Aurora Internal Medicine
Alok Srivastava is a primary care provider, practicing in Internal Medicine in Waterford, Wisconsin. Dr. Srivastava is rated as an Elite provider by MediFind in the treatment of Beta Thalassemia. His top areas of expertise are Blood Clots, Hemophilia A, Beta Thalassemia, Kidney Transplant, and Liver Transplant.
John Porter practices in London, United Kingdom. Mr. Porter is rated as an Elite expert by MediFind in the treatment of Beta Thalassemia. His top areas of expertise are Thalassemia, Anemia, Congenital Hemolytic Anemia, Splenectomy, and Hormone Replacement Therapy (HRT).
Antonella Meloni practices in Pisa, Italy. Ms. Meloni is rated as an Elite expert by MediFind in the treatment of Beta Thalassemia. Her top areas of expertise are Thalassemia, Beta Thalassemia, Hemolytic Anemia, Congenital Hemolytic Anemia, and Splenectomy.
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some donor cells in their body.
Summary: The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). ...
Published Date: May 01, 2023
Published By: National Institutes of Health