A study in Milano found that there is a drug that can significantly reduce, even half, the need for blood units transfusion in patients with Mediterranean anemia. Revolutionary therapy reduced 7 out of 10 patients by at least 33% the amount of blood units needed over 3 months. Over time, this reduction in specific patient groups is over 50%.
Scientists’ findings are for adult patients, most of them around the age of 30.
“Our great expectation is that at some point we will relieve as many patients as possible from transfusions. The new data now allows us to be confident that treatment will be possible in the future from childhood, “the study director Ms D. Cappellini, a professor of hematology at the University of Milano, told Reuters.
This is the BELIEVE phase III study, which evaluated the safety and efficacy of luspatercept monoclonal antibody for the treatment of adults with Mediterranean anemia (β-thalassemia), a chronic disease requiring regular red blood cell transfusions in a large number of patients RBC).
The data was presented at the conference by MD Cappellini itself, which featured them exciting, as it has been shown that “this luspatercept helps patients to significantly reduce their dependence on red blood cell transfusions” has ruled out the possibility that in the near future this treatment will definitely relieve some of the patients from blood transfusions.
“Our study lasted 48 weeks. Expanding our research for more time, it is possible to completely relieve some patients of the blood transfusions they need today. It is an extremely important approach because it transforms a very demanding treatment for Mediterranean anemia with frequent transfusions into a treatment that is given by a subcutaneous injection every 3 weeks.
Today, treatment that helps patients with β-thalassemia (Mediterranean anemia) manage their anemia is lifelong transfusions of red blood cells, which over time can lead the body to iron overload and life-threatening co-morbidities. These problems are now limited to this new treatment approach, which greatly reduces the number of transfusions, “says M.Cappellini.
Regarding the safety profile of the biological treatment, the researchers reported that the drug was well tolerated by the patients, while some of them had mild arthralgia in the period following the injection treatment.