A new drug can significantly reduce, even at half, the need for blood transfusions to patients with thalassemia.
In seven out of 10 patients, the revolutionary therapy reduced the number of blood units needed over three months by at least 33 percent. Over time, this decline in specific patient groups is over 50 percent.
The findings of the scientists are for adult patients, most of them around the age of 30. “Our great hope is that, at some point, we will free as many patients as possible from [the need for] transfusions. The new data now allows us to be confident that in the future treatment will be possible from childhood,” the head of the study, Ms. D. Cappellini, professor of hematology at the University of Milan, told the Athens-Macedonian News Agency (ANA).
This is a dream that may become reality in a few years and help many patients improve their quality of life. The results of the international study with Greek participation from hospitals in Athens, Thessaloniki and Patras were announced at the 60th Annual Congress of the American Society of Hematology, held in San Diego, USA.
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).