Anemia | Maria-Domenica Cappellini, MD

Novel and Emerging Therapeutic Strategies in the Management of Hematologic Disorder-Related Anemia — Part 2: Our interview with Dr Cappellini highlights the following topics and cases from her practice. Pathophysiology of thalassemia (00:00) Evolution of therapies for thalassemia (4:20) Prevalence and incidence of thalassemia (9:23) Classification and genetic inheritance of thalassemia (11:52) Novel treatments under investigation for thalassemia; role of gene therapy and EMAs (19:27) Emerging data with luspatercept and sotatercept for patients with thalassemia (23:15) Results of the Phase III BELIEVE trial evaluating luspatercept versus placebo for adult patients with beta thalassemia who require regular RBC transfusions (26:28) Tolerability and side effects associated with luspatercept (28:35) Ongoing Phase II BEYOND study evaluating the efficacy and safety of luspatercept in adults with beta thalassemia who are not transfusion dependent (32:37) Activity of luspatercept in patients with anemia of chronic disease and MDS (33:48) Effect of luspatercept on quality of life for patients with thalassemia (35:43) Optimal selection of patients with thalassemia who would benefit from luspatercept (39:11) Gene therapy for transfusion-dependent beta thalassemia (42:06) Gene therapy process for patients with thalassemia (45:26) Perspective on novel approaches to the treatment of thalassemia (51:16) Select publications