Alpha-thalassemia: Current Paradigms and the Evolving Therapeutic Landscape – A GameOn! Challenge

Overview

Provider Statement

This continuing education activity is provided by

Support Statement

This activity is supported by an educational grant from Agios Pharmaceuticals, Inc.

There is no non-commercial support for this activity.

Education activities are distinguished as separate from endorsement of commercial products. When commercial products are displayed, participants will be advised that accredited status as a provider refers only to its continuing education activities and does not imply ANCC Commission on Accreditation endorsement of any commercial products.

Activity Description

Alpha-thalassemia is an autosomal recessive disorder characterized by the reduction or absence of α-globin chain production due to mutations in the HBA1 and HBA2 genes. The loss of α-globin chains leads to an excess of unpaired β-like globin chains; these form insoluble homotetramers resulting in intracellular precipitation, ineffective erythropoiesis, and acute hemolytic anemia seen in the severe forms of α-thalassemia. It is estimated that the more severe forms of α-thalassemia (eg, hemoglobin H [HbH] disease and Hb Bart’s hydrops fetalis) occur in approximately 1 in 1,000,000 individuals in Northern Europe and North America. However, the prevalence of α-thalassemia is growing in these regions, but studies indicate that α-thalassemia is underrecognized and underdiagnosed. This educational gaming activity has been designed to review the pathophysiology of α-thalassemia, the risk for acute and chronic complications, and the burden of disease; describe the diagnostic pathway and differential diagnosis of α-thalassemia, including the clinical presentation and heterogeneity in symptomology; and outline aspects of the management of α-thalassemia, including the evolving therapeutic landscape and investigational compounds in clinical trials.

Target Audience

The intended audience for this activity is hematologist-oncologists, pediatricians, hematology nurses, and other health care professionals involved in the management of patients with α-thalassemia.

Learning Objectives

Upon successful completion of this activity, participants should be better able to:

• Outline the pathophysiology of α-thalassemia, the risk for acute and chronic complications, and the burden of disease.
• Review the diagnostic pathway and differential diagnosis of α-thalassemia, including the clinical presentation and heterogeneity in symptomology.
• Summarize aspects of the management of α-thalassemia, including the evolving therapeutic landscape and investigational compounds in clinical trials.

Faculty

Kevin H. M. Kuo, MD, MSc, FRCPC
Clinician-Investigator and Staff Hematologist, Red Blood Cell Disorders Program
Division of Medical Oncology and Hematology, Department of Medicine, University Health Network
Associate Professor, Division of Hematology, Department of Medicine, University of Toronto
Assistant Professor (status), Institute of Health Policy, Management and Evaluation, Dalla Lana School of Public Health, University of Toronto
Toronto, ON

Sujit Sheth, MD
Harold Weill Professor of Pediatric Hematology
Department of Pediatrics
Weill Cornell Medicine
New York, NY

Planners/Reviewers

Ronald A. Codario, MD, EMBA, FACP, FNLA, RPVI, CHCP
Barbara A. Niedz, PhD, RN, CPHQ

Accreditation

Vindico Medical Education is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Vindico Medical Education is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s Commission on Accreditation.

Credit Designation

Vindico Medical Education designates this enduring material for a maximum of 0.50 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Successful completion of this CME activity, which includes participation in the evaluation component, enables the learner to earn up to 0.50 MOC points in the American Board of Pediatrics’ (ABP) Maintenance of Certification (MOC) program. It is the CME activity provider’s responsibility to submit learner completion information to ACCME for the purpose of granting ABP MOC credit.

Upon successful completion, learners who opt-in by providing their ABP ID and DOB (MM/DD) will earn MOC points equivalent to the amount of CME credits claimed for the activity.

Vindico Medical Education will provide a maximum of 0.50 contact hours for nurses. Nurses should claim only the credit commensurate with the extent of their participation in the activity.

This enduring material is approved for 1 year from the date of original release, December 15, 2022, to December 14, 2023.

How to Participate in This Activity and Obtain CE Credit

To participate in this CE activity, you must read the objectives, answer the polling and pretest questions, view the content, and complete the posttest and evaluation. Provide only one (1) correct answer for each question. A satisfactory score is defined as answering 3 of the 4 posttest questions correctly. Upon receipt of the completed materials, if a satisfactory score on the posttest is achieved, Vindico Medical Education will issue an AMA PRA Category 1 Credit(s)™ or Contact Hour(s) certificate.

Disclosures

Vindico Medical Education 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 continuing education 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.

Faculty report the following relevant financial relationship(s)

Kevin H. M. Kuo, MD, MSc, FRCPC
Advisor: Bioverativ, Sangamo, Sanofi
Consultant: Agios, Alexion, Apellis, Aruvant, bluebird bio, Celgene/Bristol Myers Squibb, Forma Therapeutics, Novartis, Pfizer
Independent Research Contractor: Agios, Alexion, Celgene/Bristol Myers Squibb, Forma Therapeutics, Global Blood Therapeutics, Novartis, Novo Nordisk, Pfizer

Sujit Sheth, MD
Consultant: Agios, AustinPx, Bristol Myers Squibb/Celgene, bluebird bio, Chiesi, Forma Therapeutics, FulcrumTx
Independent Research Contractor: Agios, Bristol Myers Squibb/Celgene, Forma Therapeutics
Clinical Trial Steering Committee: Vertex Pharmaceuticals

Planners/Reviewers report the following relevant financial relationship(s)

Ronald A. Codario, MD, EMBA, FACP, FNLA, RPVI, CHCP
No relevant financial relationships to disclose.

Barbara A. Niedz, PhD, RN, CPHQ
No relevant financial relationships to disclose.

Vindico Medical Education staff report the following relevant financial relationship(s)

No relevant financial relationships to disclose.

Signed disclosures are on file at Vindico Medical Education, Office of Medical Affairs and Compliance.

Unlabeled and Investigational Usage

The audience is advised that this continuing medical education activity may contain references to unlabeled uses of US Food and Drug Administration (FDA)–approved products or to products not approved by the FDA for use in the United States. The faculty members have been made aware of their obligation to disclose such usage. All activity participants will be informed if any speakers/authors intend to discuss either non–FDA-approved or investigational use of products/devices.

Copyright Statement

Created and published by Vindico Medical Education, 6900 Grove Road, Building 100, Thorofare, NJ 08086-9447. Telephone: 856-994-9400; Fax: 856-384-6680. Printed in the USA. © 2022 Vindico Medical Education. All rights reserved. No part of this publication may be reproduced without written permission from the publisher. The material presented at or in any of Vindico Medical Education continuing medical education activities does not necessarily reflect the views and opinions of Vindico Medical Education. Neither Vindico Medical Education nor the faculty endorse or recommend any techniques, commercial products, or manufacturers. The faculty/authors may discuss the use of materials and/or products that have not yet been approved by the US Food and Drug Administration. All readers and continuing education participants should verify all information before treating patients or utilizing any product.

CE Questions?

Contact us at cme@vindicoCME.com