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New Agents and Therapeutic Strategies in β-Thalassemia

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Watch this on-demand Webcast capturing a live CCO Webinar to get up to date on the latest clinical data informing optimal management of β-thalassemia including discussion of new and investigational therapeutic strategies.

Released: December 08, 2020

Expiration: December 07, 2021

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Faculty

Jeanne Boudreaux

Jeanne Boudreaux, MD

Associate Professor of Pediatrics
Director of the Comprehensive Thalassemia Program
Clinical Director of Hematology

Aflac Cancer & Blood Disorders Center
Childrens Healthcare of Atlanta
Emory University
Atlanta, Georgia

Janet L. Kwiatkowski

Janet L. Kwiatkowski, MD, MSCE

Professor of Pediatrics
Perelman School of Medicine of the University of Pennsylvania
Director, Thalassemia Program
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania

Sujit Sheth

Sujit Sheth, MD

Professor
Department of Pediatrics
Weill Cornell Medicine
New York, New York

Provided by

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Supporters

Bristol Myers Squibb

Target Audience

This program is intended for hematologists and other clinicians involved in the management of patients with β-thalassemia.

Learning Objectives


  • Evaluate the medical need for novel therapeutic strategies for the management of β-thalassemia
  • Describe the safety and efficacy evidence for novel approaches to reducing transfusion dependence through the use of erythroid maturation agents
  • Explain the mechanism of action of gene therapy as a curative approach to β-thalassemia
  • Examine key efficacy and safety data on emerging gene therapy strategies and their potential role in the management of patients with β-thalassemia
  • Identify patients with β-thalassemia who are candidates for ongoing clinical trials

Disclosure

Clinical Care Options, LLC (CCO) requires instructors, planners, managers, and other individuals who are in a position to control the content of this activity to disclose any relevant conflict of interest (COI) they may have as related to the content of this activity. All identified COI are thoroughly vetted and resolved according to CCO policy. CCO is committed to providing its learners with high-quality CME/CE activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of a commercial interest.

The faculty reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CME/CE activity:

Faculty Disclosure

Primary Author

Jeanne Boudreaux, MD

Associate Professor of Pediatrics
Director of the Comprehensive Thalassemia Program
Clinical Director of Hematology

Aflac Cancer & Blood Disorders Center
Childrens Healthcare of Atlanta
Emory University
Atlanta, Georgia

Jeanne Boudreaux, MD, has disclosed that she has received consulting fees from Bluebird Bio, Chiesi, and Celgene.

Janet L. Kwiatkowski, MD, MSCE

Professor of Pediatrics
Perelman School of Medicine of the University of Pennsylvania
Director, Thalassemia Program
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania

Janet L. Kwiatkowski, MD, MSCE, has disclosed that she has been a consultant for Agios, Bluebird Bio, Celgene (now Bristol-Myers Squibb), Imara, and Silence Therapeutics and has received funds for research support from ApoPharma, Bluebird Bio, Novartis, Sangamo, and Terumo BCT.

Sujit Sheth, MD

Professor
Department of Pediatrics
Weill Cornell Medicine
New York, New York

Sujit Sheth, MD, has disclosed that he has received funds for research support from Bristol-Myers Squibb/Celgene, DisperSol/DST, and La Jolla and consulting fees from Agios, Bluebird Bio, Bristol-Myers Squibb/Celgene, Chiesi, and CRISPR/Vertex.

Staff Disclosure

Staff

Rachael M. Andrie, PhD

Clinical Editor

Rachael M. Andrie, PhD, has no relevant conflicts of interest to report.

Megan Cartwright, PhD

Senior Clinical Editor

Megan Cartwright, PhD, has no relevant conflicts of interest to report.

Jerfiz Constanzo, PhD, MBA

Scientific Director

Jerfiz Constanzo, PhD, MBA, has no relevant conflicts of interest to report.

Kevin Obholz, PhD

Editorial Director, Hematology/Oncology

Kevin Obholz, PhD, has no relevant conflicts of interest to report.

Timothy A. Quill, PhD

Senior Managing Editor

Timothy A. Quill, PhD, has no relevant conflicts of interest to report.

June Wasserstrom,

Director, CME Program Development

June Wasserstrom has no relevant conflicts of interest to report.

Bing-e Xu, PhD

Scientific Director

Bing-e Xu, PhD, has no relevant conflicts of interest to report.

Instructions for Credit

Accreditation

Joint Accreditation Statement

In support of improving patient care, Clinical Care Options, LLC (CCO) is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Physician Continuing Medical Education

Credit Designation

CCO designates this enduring material for a maximum of 2.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Disclosure of Unlabeled Use

Disclosure of Unlabeled Use

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications.

The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

Disclaimer

Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Additional Information

Participation in this self-study activity should be completed in approximately hours. To successfully complete this activity and receive credit, participants must follow these steps during the period from December 08, 2020, through December 07, 2021:

1. Register online at http://www.clinicaloptions.com.
2. Read the target audience, learning objectives, and faculty disclosures.
3. Study the educational activity online or printed out.
4. Submit answers to the posttest questions and evaluation questions online.

You must receive a test score of at least % and respond to all evaluation questions to receive a certificate. After submitting the evaluation, you may access your online certificate by selecting the certificate link on the posttest confirmation page. Records of all CME/CE activities completed can be found on the "CME/CE Manager" page. There are no costs/fees for this activity.

Program Medium

This program has been made available online.


Goal


The goal of this activity is to improve the knowledge and competence of learners in using novel curative and noncurative therapeutic options for patients with β-thalassemia.