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Jul 17, 2019

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)

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