ProCE Banner Activity

β-Thalassemia: Expert Insights on New Therapies for an Old Disease

Multimedia

In this CME-certified on-demand Webcast of a CCO symposium at ASH 2019 a panel of experts discusses current management of &beta -thalassemia the evolving role of biologic therapies changing the course of this disease and the promise of gene therapy for a cure including guidance on a series of patient cases and panel question and answer session.

Released: January 15, 2020

Expiration: January 13, 2021

Share

Faculty

Maria Domenica Cappellini

Maria Domenica Cappellini, MD

Professor of Internal Medicine
Chief, Internal Medicine Unit
Internal Medicine Department
University of Milan-Fondazione IRCCS Ca Granda Policlinico Hospital
Milan, Italy

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

ProCE Banner

Supporters

Celgene

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

Maria Domenica Cappellini, MD

Professor of Internal Medicine
Chief, Internal Medicine Unit
Internal Medicine Department
University of Milan-Fondazione IRCCS Ca Granda Policlinico Hospital
Milan, Italy

Maria Domenica Capellini, MD, has disclosed that she is a member of advisory boards for Bluebird Bio, Celgene, CRISPR Therapeutics, Ionis, Novartis, and Sanofi/Genzyme.

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 served on advisory boards for Agios, Bluebird Bio, and Celgene; has received consulting fees from Silence Therapeutics; and has received funds for research support from ApoPharma, Bluebird Bio, Novartis, 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 consulting fees from Acceleron and Celgene and funds for research support from ApoPharma, Celgene, CRISPR/Vertex, La Jolla, Novartis, and Terumo.

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.

Gordon Kelley,

Clinical Editor
Clinical Care Options, LLC

Gordon Kelley 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.

Tina B. Stacy, PharmD, FACEHP, BCOP, CHCP

Executive Vice President, Educational Strategy
General Manager, Oncology

Tina B. Stacy, PharmD, FACEHP, BCOP, CHCP, has no relevant conflicts of interest to report.

June Wasserstrom,

Director, CME Program Development

June Wasserstrom 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 3.0 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 January 15, 2020, through January 13, 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 participants’ knowledge of and competence in using novel curative and noncurative therapeutic options for patients with β-thalassemia.