Recently, Canada made strides towards better, more accessible treatments for patients with beta thalassemia when they approved REBLOZYL (luspatercept). Biopharmaceutical companies Bristol Myers Squibb Canada (“BMS Canada”) and Acceleron Pharma (“Acceleron”) announced that REBLOZYL was an effective option for patients whose blood disorder resulted in red blood cell transfusion-dependent anemia. According to an article published in Leukemia Research:
The term RBC-transfusion-dependence is widely used by hematologists to describe a condition of severe anemia typically arising when [the production of red blood cells] is reduced such that a person continuously requires ≥1 RBC-transfusions over a specified interval. Defining a person as RBC-transfusion-dependent has important implications in diverse hematological disorders especially because it strongly correlated with decreased survival.
REBLOZYL
In many cases, patients with beta thalassemia require RBC transfusions. These reduce symptoms and improve quality of life (QOL). Whether or not these transfusions are correlated with decreased or increased survival rate is highly controversial. Regardless, RBC transfusions can often be disruptive and time-consuming.
REBLOZYL offers patients another therapeutic option: the first Canadian approved erythroid maturation agent. This type of therapy works by regulating and improving red blood cell maturation. As a result, hemoglobin levels rise. Additionally, REBLOZYL avoids some of the health difficulties linked to RBC transfusions, such as abnormally high iron levels and associated damage.
Health Canada approved REBLOZYL based on data from the Phase 3 BELIEVE clinical trial. The study showed that REBLOZYL decreased the need for RBC transfusions.
Beta Thalassemia
HBB gene mutations cause beta thalassemia. Considered to be a rare genetic blood disorder, beta thalassemia is characterized by low hemoglobin levels. As described by the Mayo Clinic, hemoglobin is:
a protein in your red blood cells that carries oxygen to your body’s organs and tissues and transports carbon dioxide from your organs and tissues back to your lungs.
Beta thalassemia can be minor, intermediate, or major. Symptom severity varies based on subtype. However, symptoms may include:
- Anemia
- Excess iron levels
- Fatigue
- Muscle weakness
- Abnormal spleen enlargement
- Blood clots
- Headaches
- Pale skin
- Dizziness
- Difficulty breathing
Learn more about beta thalassemia.
