Rethinking Beta Thalassemia Treatment and Management: Expert Strategies on Novel Therapies

Audience: This activity is intended for healthcare professionals practicing in managed care environments.

This activity is supported by an educational grant from
Agios

Description:
Thalassemia describes a hematologic genetic disorder that includes both deletions and mutations of genes involved in hemoglobin production. Hemoglobin, a protein in red blood cells, is responsible for binding to oxygen in the lungs and delivering it to tissues in the body. The hemoglobin protein comprises various chains, including alpha and beta globin chains, which both play an integral role in the proper oxygenation of the body. Chromosome 11 contains genes involved in beta production, and chromosome 16 contains genes related to alpha production. Severity of thalassemia is dependent upon the extent of genetic changes. It represents a growing global health concern, affecting approximately 23,000 births each year. The burden of transfusion-dependent beta thalassemia is profound, with patients experiencing diminished quality of life and hematology teams managing a range of potential complications. Treatment with regular red blood cell transfusions and iron chelation therapy allows for normal growth and development and improves prognosis. Long-term complications associated with iron overload include stunted growth, dilated cardiomyopathy, liver disease, and endocrinopathies. Advances in treatment have improved outcomes for many patients but predicting response to therapy continues to be a big challenge. Managed care clinicians and providers must be educated surrounding recognition of beta thalassemia and early diagnosis, prompt intervention for both acute treatment needs and long-term treatment goals.

Upon completion of this activity, participants will be able to:

• Analyze recent clinical evidence and guideline recommendations for the therapeutic management of thalassemia
• Integrate novel oral therapies into treatment plans for patients with beta thalassemia by looking at the safety and efficacy of current and emerging treatments
• Formulate and implement a shared-care model to ensure that patient needs are aligned with therapeutic goals
• Assess the effectiveness and challenges of current therapies to address the burden of disease and monitor adverse events
• Evaluate the future direction and managed care considerations for novel therapies in beta thalassemia treatment while minimizing costs and improving outcomes

Faculty:
Sujit Sheth, MD
Chief, Division of Pediatric Hematology/Oncology
Professor of Clinical Pediatrics
Weill Cornell Medicine

Disclosure Information:
As accredited providers, NAMCP and AAMCN must ensure balance, independence, objectivity, and scientific rigor in all of their activities. All faculty participating in this activity are required to disclose to NAMCP/AAMCN any financial relationships with ineligible companies. NAMCP/AAMCN in turn discloses all relevant financial conflicts to the learners. NAMCP/AAMCN disclosure policy adheres to The Standards for Integrity and Independence in Accredited Continuing Education. All relevant conflicts of interest have been mitigated prior to the commencement of the activity. See below for conflicts of interest.

Disclosures (Relevant Financial Relationships with Ineligible Companies in the Last 24 Months):
Dr. Sheth has served as a consultant for Agios, BMS, Novo Nordisk, and Vertex. He has received grant/research support from Agios, BMS, Novo Nordisk, and Regeneron. All relevant financial relationships have been mitigated.

Planning Committee Disclosures (Relevant Financial Relationships with Ineligible Companies in the Last 24 Months):
Bill Williams, MD has no relevant financial relationship with an ineligible company to disclose.
Jeremy Williams has no relevant financial relationship with an ineligible company to disclose.
Jacqueline Cole, RN, MS, CMCN has no relevant financial relationship with an ineligible company to disclose.

NAMCP and/or the presenter has copyright or has received permissions for use of materials provided in this activity.

Accreditation & Designation
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of the National Association of Managed Care Physicians (NAMCP) and American Association of Managed Care Nurses (AAMCN). The National Association of Managed Care Physicians is accredited by the ACCME to provide continuing medical education for physicians.

NAMCP designates this enduring material for a maximum of 1 AMA PRA Category 1 credit(s)^TM. Each physician should claim credit commensurate with the extent of their participation in the activity.

The American Association of Managed Care Nurses is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center's Commission on Accreditation.

Nurses who complete this activity and achieve a passing score will receive 1 hour in nursing continuing professional development.

This activity has been approved by the American Board of Managed Care Nursing for 1.0 contact hours toward CMCN recertification requirements.

NAMCP and/or this website do not provide medical advice, diagnosis, or treatment. NAMCP does not endorse or imply endorsement of the content on any linked website. This website is to be used as an informational resource. With any health-related concern, consult with your physician or healthcare professional.

This activity is supported by an educational grant from
Agios

NAMCP and/or this website does not provide medical advice, diagnosis or treatment. NAMCP does not endorse or imply endorsement of the content on any linked website. This website is to be used as an informational resource. With any health related concern, consult with your physician or healthcare professional.