Implications of Emerging Treatments for Beta-Thalassemia

Introduction

Thalassemia Continues to Be an Evolving Global Burden due to Migration

The Pathophysiology of Disease

Hallmark of Disease in Thalassemia

The Role of Transfusion and the Associated Challenges

Evidence for Iron Chelation Therapy in TDT Is Established

Treatment Options for Thalassemia

Gene Therapy in Thalassemia: Patients Who Stopped Transfusions

Gene Therapy in Thalassemia: Changes in the Number of Transfusions

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia

Treatment Options for Thalassemia (cont)

Luspatercept

BELIEVE Trial: Study Design

BELIEVE Trial: Rate of Erythroid Response

BELIEVE Trial: Secondary Endpoints

BELIEVE Trial: Reduction in Transfusion Burden During Any 12- and 24-Week Interval

Potential Impact of the BELIEVE Trial Efficacy Results

BELIEVE Trial: Safety

Potential Implications of the Safety Findings from the BELIEVE Trial

Novel Agents and Their Mechanisms of Action

Novel Therapy in Thalassemia

Hydroxyurea and Bitopertin

Novel Therapy in Thalassemia (cont)

Novel Therapy in Thalassemia (cont)

Novel Therapies Targeting Iron Dysregulation Are Under Development

Considerations for the Future

Abbreviations

Abbreviations (cont)