Evolving Treatment Paradigms for Patients With Hemoglobinopathies

Release Date: January 14, 2025

Expiration Date: January 14, 2026

Activity Overview

The most common monogenic global disorders worldwide are hemoglobinopathies, which are genetic diseases related to hemoglobin synthesis. The genetic cause of hemoglobinopathies is DNA variants in or near the globin genes. Approximately 7% of the global population carries a DNA variant, with the majority of diagnosed individuals having sickle cell disease (SCD); the minority of patients are affected by β-thalassemia. Although hemoglobinopathies are diverse, their burden is severe and disabling for many individuals and requires specialized multidisciplinary management. This continuing medical education symposium features engaging, case-based discussions among 3 experts, highlighting clinical trial data for gene therapies and strategies for integrating gene therapy into the individualized management of hemoglobinopathies including SCD and transfusion-dependent thalassemia.

This educational activity is an archive of the live/virtual symposium held on December 6, 2024.

Target Audience

This educational activity is directed toward pediatric and adult hematologists, hematologist-oncologists, pediatricians, nurses, nurse practitioners, physician assistants, and other clinicians involved in the management of patients living with hemoglobinopathies.

Learning Objectives

Upon successful completion of this activity, you should be better prepared to:

• Describe the burden of disease of transfusion-dependent thalassemia (TDT)
• Outline the burden of disease of sickle cell disease (SCD)
• Formulate individualized management plans for patients with TDT
• Design personalized treatment plans for patients with SCD
• Evaluate clinical trial data for gene therapy for hemoglobinopathies
• Integrate gene therapy into management plans for patients with hemoglobinopathies

Faculty, Staff, and Planners’ Disclosures

The staff of Physicians’ Education Resource^®, LLC have no relevant financial relationships with ineligible companies.

PER^® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.

Off-Label Disclosure and Disclaimer

This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a health care professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER^® or any company that provided commercial support for this activity.