A press release from the US Food and Drug Administration (FDA) is announcing the approval of a new treatment for anemia in adult patients with the rare blood disorder beta thalassemia. The treatment is for patients with the most severe form of the disease that require frequent blood transfusions to manage the disease. The ultimate goal of this treatment is to decrease the number of transfusions that a patient needs.
About Beta Thalassemia
Beta thalassemia is a group of genetic disorders which affect the blood. These disorders are characterized by the abnormal production of hemoglobin, the substance in red blood cells which allows them to transport oxygen. The severity of the disorder can vary widely from person to person and depends on the present mutation. These mutations affect the HBB gene which is found on chromosome 11. These mutations can be easily passed down within families. Symptoms of beta thalassemia major, the most severe form, include spleen problems, skeletal abnormalities, poor growth, anemia, liver problems, diabetes, osteoporosis, and heart failure. Treatment for beta thalassemia may include blood transfusions, surgery, and bone marrow transplant, which can be curative for some children. There is a need for improved treatments for patients with severe cases. To learn more about beta thalassemia, click here.
Blood Transfusions Carry Risks
Patients with beta thalassemia major require regular blood transfusions in order to prevent serious symptoms from appearing. However, periodic blood transfusions carry a significant risk: excessive iron buildup, or iron overload, affecting the body. Iron overload can inflict significant issues with the body’s organs, particularly the spleen, and patients are required to undergo iron chelation treatment in order to get rid of the extra iron. Unfortunately, iron chelation can cause unpleasant side effects, leading to diminished quality of life.
A New Treatment to Reduce Transfusions
The treatment that was approved is called luspatercept-aamt (marketed as Reblozyl). In a clinical trial of 336 patients, the drug was able to reduce the need for blood transfusions during the treatment period by a minimum of 33 percent for 21 percent of the patients that were treated with it. Reblozyl comes with an increased risk of hypertension and blood clots, and patients using the drug should be closely monitored for such issues accordingly.