A History of Hemophilia Treatment: Factor Replacement to Gene Therapy

A History of Hemophilia Treatment: Factor Replacement to Gene Therapy

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A comprehensive educational resource designed by leading experts for the global hemophilia community to help you stay abreast of the evolving science and latest clinical advancements in gene therapy in hemophilia.

 

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Flora Peyvandi, MD, PhD
University of Milan - Milan, Italy

Flora Peyvandi, MD, PhD, is Professor of Internal Medicine at the University of Milan and the Director of the Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy.

Dr. Peyvandi received her medical degree from the University of Milan, Italy, is certified in Haematology and was awarded PhDs from the Maastricht University, The Netherlands and the University of Milan, Italy for her research in the field of rare bleeding disorders. As part of her PhD thesis, she was a research fellow at the Royal Free Hospital, University College London, London, UK in 1997-98 for molecular characterization of rare bleeding disorders and at the Veteran Administration Hospital, Harvard University, Boston, USA in 1998-99 for in vitro expression studies.

Dr. Peyvandi’s basic and medical science research has focused on the investigation of molecular mechanisms of coagulation disorders. Her research in the prevalence and mechanism of coagulation disorders aims at developing cost-effective treatment for the widespread treatment of patients. Dr. Peyvandi has authored and co-authored more than 360 scientific publications published across well-known specialised journals as well as 18 chapters in various books. Since 1999, she has been invited as an expert speaker at more than 128 national and international meetings and congresses. She has been the successful recipient of more than 40 project grants funded by Italian and International organizations and she was the principal investigator of the Establishment of a European Network of Rare Bleeding Disorders program. She participates in clinical, educational and research activities in different parts of the world and she was Chair of the International Society of Thrombosis and Haemostasis (ISTH) Scientific and Standardization Committee on Factor VIII, Factor IX and Rare Coagulation Disorders. She is a member of the ISTH council, Executive Committee of the World Federation of Hemophilia (WFH), Executive Committee of the European Association for Haemophilia and Allied Disorders (EAHAD), and the Medical Advisory Group of the European Hemophilia Consortium (EHC). In 2014 she was awarded the “Great Hippocrates” that is delivered to the Italian medical researcher of the year.

Activity Title: A History of Hemophilia Treatment: Factor Replacement to Gene Therapy

Topic: Gene Therapy in Hemophilia

Accreditation Type: AMA PRA Category 1 Credit(s)™

Release Date: July 1, 2019

Expiration Date: June 30, 2020

Estimated Time to Complete Activity: 30 minutes

CME Information


Activity Title

A History of Hemophilia Treatment: Factor Replacement to Gene Therapy

Topic

Gene Therapy in Hemophilia

Accreditation Type

AMA PRA Category 1 Credit(s)™

Release Date

July 1, 2019

Expiration Date

June 30, 2020

Estimated Time to Complete Activity

30 minutes

LEARNING OBJECTIVE
Upon completion of the activity, participants should be able to:

    • Outline the current and emerging approaches for treating hemophilia, including various approaches of gene therapy

FACULTY
Flora Peyvandi, MD, PhD
University of Milan
Milan, Italy

METHOD OF PARTICIPATION / HOW TO RECEIVE CREDIT

  1. There are no fees for participating in and receiving credit for this activity.
  2. Review the activity objectives and CME/CE information.
  3. Complete the CME/CE activity
  4. Complete the online posttest. A score of 75% is required to successfully complete this activity. The participant may take the test until successfully passed.
  5. Complete the CME/CE evaluation/attestation form, which 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 his/her views on future educational needs.
  6. Credit documentation/reporting:
    • If you are requesting AMA PRA Category 1 Credits™ or a certificate of participation—your CME/CE certificate will be available for download.

CLICK HERE TO VIEW TECHNICAL REQUIREMENTS


ACCREDITED PROVIDER

This activity is provided by The France Foundation.

TARGET AUDIENCE
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
As development of gene therapy for hemophilia continues into phase 3 clinical trials, and approval of this therapeutic approach is anticipated, it is essential for all members of the hemophilia care team to be knowledgeable and poised for the integration of this new therapeutic approach into clinical practice.

ACCREDITATION STATEMENT
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 International Society of Thrombosis and Haemostasis. The France Foundation is accredited by the ACCME to provide continuing medical education for physicians.

CREDIT DESIGNATION
Physicians:
The France Foundation designates this enduring activity 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.

Nurses: Nurses who are certified by the American Nurses Credentialing Center (ANCC) may utilize activities that are certified by ACCME-accredited providers toward their requirement for certification renewal by the ANCC. A certificate of attendance will be provided by The France Foundation, an ACCME accredited provider.

DISCLOSURE POLICY
In accordance with the ACCME Standards for Commercial Support, The France Foundation (TFF) and the International Society of 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 healthcare and not those of a commercial interest.

Activity Staff Disclosures
The reviewers, editors, staff, CME committee, or other members at The France Foundation who control content have no relevant financial relationships to disclose.

The reviewers, editors, staff, or other members at the International Society of Thrombosis and Haemostasis who control content have no relevant financial relationships to disclose.

Faculty Disclosures–Planners

The faculty listed below report that they have no relevant financial relationships to disclose:

  • Claire McLintock, MBChB, FRACP, FRCPA

The faculty listed below report that they have relevant financial relationships to disclose:

  • David Lillicrap, MD, FRCPC, has received honoraria for academic consulting from Bioverativ, CSL Behring, and Octapharma Plasma. He has received research funding from Bayer, BioMarin, Bioverative, CSL-Behring, and Octapharma Plasma.
  • Johnny Mahlangu, BSc, MBBCh, MMed, FCPath has held leadership positions with ISTH and WFH. He has served on the speakers’ bureau for Alnylam, Bayer, Biotest, Biogen, ISTH, Novo Nordisk, Pfizer, Sobi, Shire, Roche, and WFH. Dr. Mahlangu has received research funding from Alnylam, Bayer, Biotest, Biogen, ISTH, Novo Nordisk, Pfizer, Sobi, Shire, Roche, and WFH. He has received honoraria from Amgen, Bayer, Biotest, Biogen, Baxalta, CSL- Behring, Catalyst Biosciences, Chugai, Freeline, LFB, Novo Nordisk, Roche, and Spark.
  • K. John Pasi, MChB, PhD, FRCP, FRCPath, FRCPCH, has received research funding from BioMarin GeneR8 programme, uniQure HOPE-B programme, Sanofi - ATLAS fitusiran program. He has received honoraria from Alnylam, ApcinteX, BioMarin, Catalyst Bio, Chugai, Novo Nordisk, Octapharma Plasma, Pfizer, Roche, Sanofi, Shire, and Sobi.
  • Flora Peyvandi, MD, PhD has held leadership positions with EHC, EMA, and EAHAD. She has served on the speakers’ bureau for Bioverativ, CSL-Behring, Grifols, Novo Nordisk, Roche, Sanofi, Sobi, Spark, Sysmex, and Takeda. Dr. Peyvandi is an advisory board member for Sanofi.
  • Glenn F. Pierce, MD, PhD, has received honoraria for academic consulting from BioMarin, Genentech/Roche, Pfizer, St. Jude, and VarmX. He has held leadership positions with Global Blood Therapeutics, NHF MASAC, and World Federation of Hemophilia.
  • Steven W. Pipe, MD, has received honoraria for academic consulting from CSL-Behring, Novo Nordisk and Pfizer. He has held leadership positions with MASAC-National Hemophilia Foundation. Dr. Pipe has received research funding from Siemens and Shire. He has received honoraria from ApcinteX, Bayer, Biomarin, Bioverativ, Catalyst, CSL-Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sanofi, Shire, Spark, and uniQure.
  • Alok Srivastava, MD, FRACP, FRCPA, FRCP, has received honoraria for academic consulting from Bayer, Novo Nordisk, Roche, and Shire/Takeda. He has held leadership positions as Chair of Gene Therapy Task Force of SSC of the ISTH and Chair of the Steering Committee of the Asia Pacific Hemophilia Working Group. Dr. Srivastava has received research funding from Alnylam, Bayer, and Shire.
  • Thierry VandenDriessche, PhD, has held leadership positions with NHF and ISTH. He has received research funding from Pfizer and Takeda. Dr. VandenDriessche has received honoraria from Baxalta/Shire/Takeda, Bayer, Biotest, and Pfizer.

Faculty Disclosures–Activity Faculty
The following faculty report that they have relevant financial relationships to disclose:

  • Flora Peyvandi, MD, PhD has held leadership positions with EHC, EMA, and EAHAD. She has served on the speakers’ bureau for Bioverativ, CSL-Behring, Grifols, Novo Nordisk, Roche, Sanofi, Sobi, Spark, Sysmex, and Takeda. Dr. Peyvandi is an advisory board member for Sanofi.

DISCLOSURE OF UNLABELED USE
TFF and 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 U.S. 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.

COMMERCIAL SUPPORT ACKNOWLEDGMENT
This activity is supported by an educational grant from BioMarin, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

DISCLAIMER
The France Foundation and International Society of Thrombosis and 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, International Society of Thrombosis and Haemostasis, and the commercial supporter(s) assume no liability for the information herein.

COPYRIGHT INFORMATION
Copyright © 2019 The France Foundation. Any unauthorized use of any materials on the site may violate copyright, trademark, and other laws. You may view, copy, and download information or software ("Materials") found on the Site subject to the following terms, conditions, and exceptions:

  • The materials are to be used solely for personal, noncommercial, informational and educational purposes. The materials are not to be modified. They are to be distributed in the format provided with the source clearly identified. The copyright information or other proprietary notices may not be removed, changed, or altered.
  • Materials may not be published, uploaded, posted, transmitted (other than as set forth herein), without The France Foundation's prior written permission.


PRIVACY POLICY

The France Foundation protects the privacy of personal and other information regarding participants and educational collaborators. The France Foundation will not release personally identifiable information to a third party without the individual's consent, except such information as is required for reporting purposes to the ACCME.

The France Foundation maintains physical, electronic, and procedural safeguards that comply with federal regulations to protect against the loss, misuse or alteration of information that we have collected from you.

Additional information regarding The France Foundation's Privacy Policy can be viewed at http://www.francefoundation.com/privacy-policy.


CONTACT INFORMATION
If you have questions about this CME activity, please contact The France Foundation at 860-434-1650 or This email address is being protected from spambots. You need JavaScript enabled to view it..

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Getting to Know Gene Therapy– Terminology and Concepts

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