Introduction, faculty disclosures

Overview of BT Epidemiology and burden of BT for patients and caregivers Pathophysiology of BT, including new and emerging therapeutic targets BT classification o  Subtype (major, intermedia, minor) o  Severity o  Transfusion dependence Techniques to accurately diagnose BT and exclude other potential diagnoses Monitoring hepatic and myocardial iron overload using magnetic resonance imaging techniques

Management of BT  Non-transfusion-dependent BT Standard-of-care options to prevent iron overload and accumulation of iron overload-related complications over time Iron overload therapy with iron chelators, including sequencing, combination, and increased dosing frequency Indications for transfusion Novel investigational treatment options Pyruvate kinase activation Transfusion-dependent BT Standard-of-care options to safely reduce transfusion burden Preventing iron overload using iron chelation therapy Gene therapy and gene editing techniques combined with hematopoietic stem cell transplantation Implementation Considerations Emerging efficacy and safety data Emerging small-molecule inhibitors Management of patients with BT over their lifetimes Supportive care: preventing complications Best practices: putting it all together Patient case(s)

Summary, conclusions, and best practice recap

By the end of the session the participant will be able to:

• Review the genetics and pathophysiology of BT, with an emphasis on current and novel therapeutic targets.
• Evaluate guideline-recommended procedures useful in the differential diagnose of BT and magnetic resonance imaging techniques used to measure hepatic and myocardial iron overload.
• Review standard-of-care treatment options for BT, and describe the unmet needs in this patient population, including gaps between real-life care in the clinical setting compared with guideline recommendations.
• Describe the latest safety and efficacy data supporting the use of novel therapeutic treatment strategies, and select treatment options on the basis of disease subtype and severity and patient characteristics.

ACCME Activity #202436906

ACCREDITATION FOR THIS COURSE HAS EXPIRED. YOU MAY VIEW THE PROGRAM, BUT CME / CE IS NO LONGER AVAILABLE AND NO CERTIFICATE WILL BE ISSUED.

As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved through content review by ScientiaCME.

Disclosure of Faculty: Sherif M. Badawy, MD, MBBCh, MS, Associate Professor of Pediatrics, Northwestern University Feinberg School of Medicine, has no relevant financial disclosures.

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.

Faculty WILL NOT discuss off-label uses.

All relevant financial relationships have been mitigated.

ScientiaCME adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CE activity, including faculty, planners, reviewers or others are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity.

Commercial Support Disclosure: This program is supported by an educational grant from Agios Pharmaceuticals and Vertex Pharmaceuticals.

• Read the learning objectives above
• Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowledge gained by participating in this CME activity.
• View the online activity. You may view this is in more than one session, and may pause or repeat any portion of the presentation if you need to.
• Minimum participation threshold: Take the post-test. A score of 70% or higher is required to pass and proceed to the activity evaluation.
• Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

Cultural/Linguistic Competence & Health Disparities

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