Experimental Luspatercept Reduced Need For Transfusions in Beta Thalassemia

MedicalResearch.com Interview with:

Maria Domenica Cappellini, M.D. Principal Investigator, BELIEVE Clinical Trial Fondazione IRCCS Ca’ Granda Policlinico Hospital University of Milan, Milan, Italy

Dr. Cappellini

Maria Domenica Cappellini, M.D.
Principal Investigator, BELIEVE Clinical Trial
Fondazione IRCCS Ca’ Granda Policlinico Hospital
University of Milan, Milan, Italy

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Beta thalassemia is a severe, inherited hemoglobinophathy due to a reduced production of hemoglobin. Standard of care is blood transfusion and iron chelation to remove the accumulated iron by transfusion. This is a very demanding treatment.

Therefore, there is the need to find new approaches for treating these patients. The BELIEVE study showed that the experimental drug luspatercept significantly reduced the need for blood transfusions in patients with beta thalassemia. More than 70 percent of patients who received luspatercept were able to cut blood transfusions by at least one-third over any consecutive 12-week period

MedicalResearch.com: What should readers take away from your report?

Response: The efficacy of  luspatercept in reducing the transfusion burden in beta thalassemia patients. 

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: We are just at the beginning. We need to monitor the patients’ treatment in order to confirm the efficacy of the drug, to prove the duration of response and to identify the different patterns of response in order to identify those patients who could benefit more from this treatment.

Citation:

ASH abstract

The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

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