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)
Featuring the video recorded proceedings of events held in conjunction with important scientific symposia, live webcasts and closed meetings featuring the perspectives of renowned clinical investigators, these videos provide an overview of important advances in the management of a number of solid tumors and hematologic cancers.