Highlights From the World Federation of Haemophilia 2022 World Congress
Barbara Konkle, MD
Professor of Medicine
University of Washington School of Medicine
Seattle, Washington, United States
Pratima Chowdary, MD, MRCP, FRCPath
Royal Free Hospital
David Lillicrap, MD, FRCPC
Queen’s University - Kingston, Ontario, Canada
Johnny Mahlangu, BSc, MBBCh, MMed, FCPath
Faculty of the Health Sciences - University of the Witwatersrand and National Health Laboratory Service
Johannesburg, South Africa
Wolfgang Miesbach, MD, PhD
Goethe University Hospital
Glenn F. Pierce, MD, PhD
World Federation of Hemophilia (WFH)
Steven W. Pipe, MD
University of Michigan
Ann Arbor, Michigan, United States
Flora Peyvandi, MD, PhD
University of Milan
Alok Srivastava, MD, FRACP, FRCPA, FRCP
Christian Medical College
Thierry VandenDriessche, PhD
Vrije University Brussels
This activity is intended for physicians (hematologists), 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.
- Discuss up-to-date information on the current and emerging approaches for treating hemophilia, including various approaches of gene therapy
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 France Foundation and the International Society on Thrombosis and Haemostasis. The France Foundation is accredited by the ACCME to provide continuing medical education for physicians.
Physicians: The France Foundation designates this enduring activity for a maximum of 0.25 AMA PRA Category 1 Credit(s)™. 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 planners, reviewers, editors, staff, CME committee, and other members at The France Foundation who control content have no relevant financial relationships to disclose.
The planners, reviewers, editors, staff, CME committee, and other members at the International Society on Thrombosis and Haemostasis who control content have no relevant financial relationships to disclose.
Faculty Disclosures–Activity Faculty
The following faculty report that they have relevant financial relationships to disclose:
- Barbara Konkle, MD, serves as a consultant for Biomarin, Regeneron, Pfizer, Inc., Sanofi, Sigilon, and Takeda. She has received research funding from Octapharma, Pfizer, Inc., Sanofi, Spark Therapeutics, and Takeda.
Faculty Disclosures–Planning Committee
The faculty listed below report that they have no relevant financial relationships to disclose:
- Alok Srivastava, MD, FRACP, FRCPA, FRCP
The following faculty report that they have relevant financial relationships to disclose:
- 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.
- 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.
- 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
- 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
- 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 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 Medical Affairs Department of the manufacturer for the most recent prescribing information.
This activity is supported by independent medical educational grants from BioMarin, Sanofi Genzyme, CSL Behring, and Spark Therapeutics.
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Topic: Gene Therapy in Hemophilia
Accreditation Type: AMA PRA Category 1 Credit(s)™
Release Date: June 21, 2022
Expiration Date: June 20, 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
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- Complete the CME/CE activity
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