ClinicalTrials.gov. A database of privately and publicly funded clinical studies conducted around the world. https://clinicaltrials.gov/. Accessed March 12, 2018.
Dunbar CE, High KA, Joung JK, et al. Gene therapy comes of age. Science. 2018;359(6372).
VandenDriessche T, Chuah MK. Hemophilia Gene Therapy: Ready for Prime Time? Hum Gene Ther. 2017;28:1013-1023.
Kay MA, Manno CS, Ragni MV, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet. 2000;24:257-261.
Roth DA, Tawa N, Jr, O'Brien J, et al. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med. 2001;344:1735-1742.
Chauah M, Collen D, VandenDriessche T. Clinical gene transfer studies for hemophilia A. Semin Thromb Hemost. 2004;30:249-256.
Powell JS, Ragni MV, White GC, 2nd, et al. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood. 2003;102:2038-2045.
Grieger JC, Samulski RJ. Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps. J Virol. 2005;79:9933-9944.
Sharma R, Anguela XM, Doyon Y, et al. In vivo genome editing of the albumin locus as a platform for protein replacement therapy. Blood. 2015;126:1777-1784.
Bowen D. Haemophilia A and haemophilia B: Molecular insights. Mol Pathol. 2002;55:127-144.
Simioni P, Tormene D, Tognin G, et al. X-linked thrombophilia with a mutant factor IX (factor IX Padua). N Engl J Med. 2009;361:1671-1675.
Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371:1994-2004.
Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011;365:2357-2365.
George L, Sullivan S, Giermasz A, et al. Hemophilia B gene therapy with a high-specific-activity factor IX variant. N Engl J Med. 2017;377:2215-2227.
Miesbach W, Meijer K, Coppens M, et al. Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B. Blood. 2018;131:1022-1031.
Rangarajan S, Walsh L, Lester W, et al. AAV5-factor VIII gene transfer in severe hemophilia A. N Engl J Med. 2017;377:2519-2530.
George L, Ragni M, Samelson-Jones BJ, et al. Spk-8011: Preliminary results from a phase 1/2 dose escalation trial of an investigational AAV-mediated gene. Presented at: 59th ASH Annual Meeting & Exposition; December 9-12, 2017; Atlanta, Georgia. Abstract 604.

CME / ABIM MOC

Test Your Knowledge of Gene Therapy for Hemophilia

Authors: John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH
CME / ABIM MOC Released: 5/3/2018
THIS ACTIVITY HAS EXPIRED
Valid for credit through: 5/3/2019

Target Audience and Goal Statement

This activity is intended for hematologists, pediatricians, and other healthcare professionals who manage patients with hemophilia.

The goal of this activity is to review the latest developments in gene therapy for patients with hemophilia.

Upon completion of this activity, participants will have increased knowledge regarding the:

Research on gene therapy in hemophilia
New findings from clinical trials on gene therapy in hemophilia

Disclosures

As an organization accredited by the ACCME, Medscape, LLC, requires everyone who is in a position to control the content of an education activity to disclose all relevant financial relationships with any commercial interest. The ACCME defines "relevant financial relationships" as financial relationships in any amount, occurring within the past 12 months, including financial relationships of a spouse or life partner, that could create a conflict of interest.

Medscape, LLC, encourages Authors to identify investigational products or off-label uses of products regulated by the US Food and Drug Administration, at first mention and where appropriate in the content.

Faculty

John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH

Professor of Haemostasis and Thrombosis, Barts and the London School of Medicine and Dentistry, London, United Kingdom

Disclosures

Disclosure: John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Alnylam Pharmaceuticals, Inc; Biogen; BioMarin Pharmaceutical Inc.; Octapharma; Roche; Shire; Swedish Orphan Biovitrum
Served as a speaker or a member of a speakers bureau for: Bayer HealthCare Pharmaceuticals; Biotest; Novo Nordisk; Pfizer Inc

Dr Pasi does not intend to discuss off-label uses of drugs, mechanical devices, biologics, or diagnostics approved by the FDA for use in the United States.

Dr Pasi does intend to discuss investigational drugs, mechanical devices, biologics, or diagnostics not approved by the FDA for use in the United States.

Editors

Charlotte Warren

Senior Scientific Director, Medscape, LLC

Disclosures

Disclosure: Charlotte Warren has disclosed no relevant financial relationships.

Melinda Tanzola, PhD

Scientific Director, Medscape, LLC

Disclosures

Disclosure: Melinda Tanzola has disclosed no relevant financial relationships.

Steering Committee

John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH

Disclosures

As listed above.

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

Professor of Haematology, Faculty of Health Sciences, Department of Molecular Medicine and Haematology, University of the Witwatersrand; Head of Clinical Haematology Ward and Service, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa

Disclosures

Disclosure: Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Amgen Inc.; Bayer HealthCare; Biotest; Biogen; Baxalta; CSL Behring; Catalyst Pharmaceutical Partners, Inc.; Novo Nordisk; Roche; Spark
Served as a speaker or a member of a speakers bureau for: Alnylam Pharmaceuticals, Inc; Bayer HealthCare; Biotest; Biogen; Novo Nordisk; Pfizer Inc.; Swedish Orphan Biovitrum; Shire; Roche
Received grants for clinical research from: Bayer HealthCare; Biogen; BioMarin Pharmaceutical Inc.; CSL Behring; Novo Nordisk; Swedish Orphan Biovitrum; Roche; Unique Pharmaceuticals

Margareth C. Ozelo, MD, PhD

Assistant Professor; Director, IHTC Hemocentro UNICAMP, University of Campinas; Director, WFH International Hemophilia Training Center, Sao Paulo, Brazil

Disclosures

Disclosure: Margareth C. Ozelo, MD, PhD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: BioMarin Pharmaceutical Inc.; CSL Behring; Novo Nordisk; Pfizer Inc.; Roche; Shire
Served as a speaker or a member of a speakers bureau for: BioMarin Pharmaceutical Inc.; Grifols; Novo Nordisk; Pfizer Inc.; Roche; Shire
Received grants for clinical research from: Bioverativ; Pfizer Inc.; Shire

Flora Peyvandi, MD, PhD

Professor of Internal Medicine, University of Milan; Director, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy

Disclosures

Professor of Internal Medicine, University of Milan; Director, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy Disclosure: Flora Peyvandi, MD, PhD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Kedrion Biopharma Inc.; LFB SA
Served as a speaker or a member of a speakers bureau for: Ablynx; Alnylam Pharmaceuticals, Inc; F. Hoffmann-La Roche; Grifols; Shire; Swedish Orphan Biovitrum
Other: Advisory board member for Ablynx; F. Hoffmann-La Roche; Shire

Steven W. Pipe, MD

Professor of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, United States

Disclosures

Disclosure: Steven W. Pipe, MD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Alnylam Pharmaceuticals, Inc; Bayer HealthCare; BioMarin Pharmaceutical Inc.; Bioverativ; CSL Behring; Novo Nordisk; Pfizer Inc.; Roche; Spark; UniQure
Received grants for clinical research from: Pfizer Inc.; Siemens Healthcare Diagnostics Inc.; Shire

Ulrike M. Reiss, MD

Associate Member; Director of Clinical Hematology; Director, Pediatric Hemophilia Treatment Center, Department of Hematology, St. Jude Children’s Research Hospital, Memphis, Tennessee, United States

Disclosures

Disclosure: Ulrike M. Reiss, MD, has disclosed no relevant financial relationships.

Additional Planners

Amanda Bok

Course Director, Chief Executive Officer, European Haemophilia Consortium (EHC), Brussels, Belgium

Disclosures

Disclosure: Amanda Bok has disclosed no relevant financial relationships.

Neil Frick, MS

Course Director, Vice President for Medical Programs and Information, National Hemophilia Foundation, New York, New York.

Disclosures

Disclosure: Neil Frick, MS, has disclosed no relevant financial relationships.

Fiona Robinson, PhD

Course Director, Educational Materials Manager, World Federation of Hemophilia (WFH), Montreal, Quebec

Disclosures

Disclosure: Fiona Robinson, PhD, has disclosed no relevant financial relationships.

Georghia Michael, PhD

Medical Writing Consultant, World Federation of Hemophilia (WFH), Montreal, Quebec

Disclosures

Disclosure: Georghia Michael, PhD, has disclosed the following relevant financial relationships:
Owns stock, stock options, or bonds from: GlaxoSmithKline plc.

CME Reviewer

Amy H. Seung, PharmD, BCOP

Disclosures

Disclosure: Amy H. Seung, PharmD, BCOP, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: BeyondSpring Pharmaceuticals

Peer Reviewer

This activity has been peer reviewed and the reviewer has disclosed no relevant financial relationships.

Accreditation Statements

In support of improving patient care, Medscape, LLC 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.

For Physicians

Medscape, LLC designates this enduring material 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.

Medscape, LLC staff have disclosed that they have no relevant financial relationships.

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 0.25 MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit. Aggregate participant data will be shared with commercial supporters of this activity.

Contact This Provider

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This activity is designed to be completed within the time designated on the title page; physicians should claim only those credits that reflect the time actually spent in the activity. To successfully earn credit, participants must complete the activity online during the valid credit period that is noted on the title page. To receive AMA PRA Category 1 Credit™, you must receive a minimum score of 70% on the post-test.

Follow these steps to earn CME/CE credit*:

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From Medscape Education Oncology

CME / ABIM MOC

Test Your Knowledge of Gene Therapy for Hemophilia

Authors: John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCHFaculty and Disclosures

THIS ACTIVITY HAS EXPIRED

CME / ABIM MOC Released: 5/3/2018

Valid for credit through: 5/3/2019

processing....

Abbreviations

A1AT = alpha-1-anti-trypsin
AAV = adeno-associated virus
ABR = annual bleeding rate
ALT = alanine aminotransferase
APOE = apolipoprotein E
ELISPOT = enzyme-linked immunospot
hAAT = human α1-antitrypsin
HCR = hepatic control region
mRNA = messenger RNA
PCR = polymerase chain reaction
QoL = quality of life
RNAi = RNA interference
tRNA = transfer RNA

« Return to: Test Your Knowledge of Gene Therapy for Hemophilia

CME / ABIM MOC Information

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References

Faculty and Disclosures

Faculty

John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH

Editors

Charlotte Warren

Melinda Tanzola, PhD

Steering Committee

John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

Margareth C. Ozelo, MD, PhD

Flora Peyvandi, MD, PhD

Steven W. Pipe, MD

Ulrike M. Reiss, MD

Additional Planners

Amanda Bok

Neil Frick, MS

Fiona Robinson, PhD

Georghia Michael, PhD

CME Reviewer

Amy H. Seung, PharmD, BCOP

Peer Reviewer

CME / ABIM MOC

Test Your Knowledge of Gene Therapy for Hemophilia

Target Audience and Goal Statement

Disclosures

Faculty

John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH

Editors

Charlotte Warren

Melinda Tanzola, PhD

Steering Committee

John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

Margareth C. Ozelo, MD, PhD

Flora Peyvandi, MD, PhD

Steven W. Pipe, MD

Ulrike M. Reiss, MD

Additional Planners

Amanda Bok

Neil Frick, MS

Fiona Robinson, PhD

Georghia Michael, PhD

CME Reviewer

Amy H. Seung, PharmD, BCOP

Peer Reviewer

Accreditation Statements

For Physicians

Instructions for Participation and Credit

Test Your Knowledge of Gene Therapy for Hemophilia

Abbreviations