According to a story from Hematology Advisor, the drug luspatercept (marketed as Reblozyl) recently demonstrated its ability to reduce transfusion burden in patients with beta thalassemia that depend on regular blood transfusions. First approved late last year for the treatment of anemia associated with the disorder, the latest study indicates that the drug could have benefits in other ways as well.
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.
The phase 3 clinical trial included a total of 336 patients with transfusion-dependent beta thalassemia. These patients received either luspatercept plus best supportive care (BSC) or a placebo plus BSC. Patients were treated for at least 48 weeks. In the trial, 21.4 percent of patients that received the drug saw their transfusion burden reduced; meanwhile, only 4.5 of patients in the placebo group experienced a reduction. The drug also performed better than placebo in secondary endpoints as well.
Adverse events were common in the study, with most patients in both groups experiencing them. The luspatercept group showed a slightly higher rate of them. Regardless, the sustained beneficial response from the drug was promising and a five year long extension study has begun that will monitor the long-term safety and efficacy impacts of the drug.
Overall, it seems like luspatercept could have a greater benefit than initially expected for beta thalassemia patients that require regular transfusions. The original study can be found here in The New England Journal of Medicine.