Pratima Chowdary, MD, MRCP, FRCPath
Royal Free Hospital
London, United Kingdom
David Lillicrap, MD, FRCPC
Johnny Mahlangu, BSc, MBBCh, MMed, FCPath
University of the Witwatersrand and National Health Laboratory Service
Johannesburg, South Africa
Wolfgang Miesbach, MD, PhD
Goethe University Hospital
Margareth Ozelo, MD, PhD
University of Campinas Hemocentro UNICAMP Brazil Campinas
São Paulo, Brazil
Glenn F. Pierce, MD, PhD
World Federation of Hemophilia (WFH)
Steven W. Pipe, MD
University of Michigan
Ann Arbor, Michigan
Flora Peyvandi, MD, PhD
University of Milan
Alok Srivastava, MD, FRACP, FRCPA, FRCP
Christian Medical College
Thierry VandenDriessche, PhD
Vrije University Brussels
This activity was planned by and is intended for physicians (hematologists). It may be relevant to nurse practitioners, physician assistants, nurses managing patients with hemophilia, and scientists with an interest in basic, translational, and clinical research in hemophilia around the world.
STATEMENT OF NEED
An estimated 400,000 people worldwide are living with hemophilia. A variety of increasingly effective treatment options have been available to patients over the past decades. However, only 25% receive adequate treatment globally. After many years of research and clinical trials, gene therapy is moving towards its promise as a revolutionary new treatment option for patients with hemophilia.
Significant progress has occurred over the last decade in gene therapy for patients with hemophilia. It is essential for all members of the hemophilia care team to be knowledgeable about gene therapy for hemophilia and poised for the integration of this new therapeutic approach into clinical practice.
- Describe gene therapy correctly, including basic terms and concepts, for your patients with hemophilia
- Identify patients with hemophilia who are candidates for gene therapy
- Discuss up-to-date information on the current and emerging approaches for treating hemophilia, including various approaches of gene therapy
- Anticipate short- and long-term monitoring requirements for gene therapy
- Identify potential modifications to health care systems needed to enable successful integration of gene therapy and appropriate patients for ongoing and emerging trials
In support of improving patient care, this activity has been planned and implemented by The France Foundation and International Society on Thrombosis and Haemostasis. The France Foundation 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.
The France Foundation designates this enduring material activity for a maximum of 0.25 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
In accordance with the ACCME Standards for Commercial Support, The France Foundation (TFF) and the International Society on Thrombosis and Haemostasis (ISTH) require that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial interest. TFF and ISTH resolve all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all their educational programs. Furthermore, TFF and ISTH seek to verify that all scientific research referred to, reported, or used in a CME/CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. TFF and ISTH are committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest.
Activity Staff Disclosures
The France Foundation staff listed below report that they have no relevant financial relationships to disclose:
- Miriam Giles
- Amanda Noe
- Patrick Harty, PhD
The International Society on Thrombosis and Haemostasis staff listed below report that they have no relevant financial relationships to disclose:
- Cary Clark
Faculty and Plannng Committee Disclosures
The following faculty report that they have relevant financial relationships to disclose:
- Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, serves on the speakers bureau and does contract research for CSL Behring, Catalyst Biosciences, Freeline Therapeutics, Novo Nordisk, Roche, Sanofi, Spark, and Takeda
- Steven W. Pipe, MD, serves as a consultant for Apcintex, ASC Therapeutics, Bayer, BioMarin Catalyst Biosciences, CSL Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sangamo Therapeutics, Sanofi, Takeda, Spark Therapeutics, and uniQure
- Glenn F. Pierce, MD, PhD, receives honoraria for academic consulting from BioMarin, Genentech/Roche, Pfizer, St. Jude, and VarmX. He holds leadership positions with Global Blood Therapeutics, NHF MASAC, and World Federation of Hemophilia
- Pratima Chowdary, MRCP, FRCPath, receives research grant support from Bayer, CSL Behring, Novo Nordisk, Pfizer, and Sobi. She serves as a consultant for Baxter, Biogen, CSL Behring, Freeline, Novo Nordisk, Pfizer, Roche, Shire, and Sobi
- David Lillicrap, MD, serves as a consultant for BioMarin, CSL Behring, Sanofi, and Takeda. He receives research support from Bayer, BioMarin, CSL Behring, Octapharma, and Sanofi
- Wolfgang Miesbach, MD, PhD, receives grant research support from Bayer, Biotest, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, and Takeda/Shire. He serves on the speakers' bureau for Bayer, BioMarin, Biotest, CSL Behring, Chugai, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, and Takeda/Shire. Dr. Miesbach serves on advisory boards for Bayer, BioMarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Takeda/Shire, and uniQure
- Margareth Ozelo, MD, PhD, serves as consultant for BioMarin, Novo Nordisk, Pfizer, Roche, Sanofi, and Takeda. She serves on the speakers' bureau for Bayer, BioMarin, Novo Nordisk, Roche, and Takeda. Dr. Ozelo conducts contract research for BioMarin, Novo Nordisk, Pfizer, Roche, Sanofi, Spark, and Takeda
- Flora Peyvandi, MD, PhD, serves as a consultant for Sanofi and Sobi. She serves on the speakers bureau for Bioverativ, Grifols, Roche, Sanofi, Sobi, Spark, and Takeda
- Thierry VandenDriessche, PhD, holds leadership positions with NHF and ISTH. He receives research funding from Pfizer and Takeda. Dr. VandenDriessche receives honoraria from Baxalta, Shire, Takeda, Bayer, Biotest, and Pfizer
The faculty listed below report that they have no relevant financial relationships to disclose:
- Alok Srivastava, MD, FRACP, FRCPA, FRCP
The France Foundation (TFF) and the International Society on Thrombosis and Haemostasis (ISTH) require CME faculty (speakers) to disclose when products or procedures being discussed are off-label, unlabeled, experimental, and/or investigational, and any limitations on the information that is presented, such as data that are preliminary, or that represent ongoing research, interim analyses, and/or unsupported opinion. Faculty in this activity may discuss information about pharmaceutical agents that is outside of US Food and Drug Administration approved labeling. This information is intended solely for continuing medical education and is not intended to promote off-label use of these medications. TFF and ISTH do not recommend the use of any agent outside of the labeled indications. If you have questions, contact the manufacturer's Medical Affairs department for the most recent prescribing information.
This activity is supported by independent medical educational grants from BioMarin, Sanofi Genzyme, CSL Behring, and Spark Therapeutics.
The France Foundation and the International Society on Thrombosis Haemostasis present this information for educational purposes only. The content is provided solely by faculty who have been selected because of recognized expertise in their field. Participants have the professional responsibility to ensure that products are prescribed and used appropriately on the basis of their own clinical judgment and accepted standards of care. The France Foundation, the International Society on Thrombosis Haemostasis, and the commercial supporter(s) assume no liability for the information herein.
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Topic: Gene Therapy in Hemophilia
Accreditation Type: AMA PRA Category 1 Credit(s)™
Release Date: September 20, 2022
Expiration Date: September 19, 2023
Estimated Time to Complete Activity: 15 minutes
METHOD OF PARTICIPATION/HOW TO RECEIVE CREDIT
- There are no fees for participating in and receiving credit for this activity
- Review the activity objectives and CME/CE information
- Complete the CME/CE activity
- Complete this CME/CE evaluation/attestation form: https://survey.alchemer.com/s3/7020035/9153-ISTH-Gene-Therapy-in-Hemophilia-Self-Assessment-Module
This form provides each participant with the opportunity to comment on how participating in the activity will affect their professional practice; the quality of the instructional process; the perception of enhanced professional effectiveness; the perception of commercial bias; and their views on future educational needs
- If you are requesting AMA PRA Category 1 Credits™ or a certificate of participation—your CME/CE certificate will be available for download
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