The Gene Therapy Registry (GTR) is a prospective, observational, and longitudinal registry designed to collect long-term data on people with hemophilia (PWH) who receive gene therapy.
The primary objective of the GTR is to determine the long-term safety of factor VIII and factor IX gene therapies for PWH. Secondary objectives are to determine the long-term efficacy and the durability of factor VIII and factor IX gene therapies in PWH, and to assess long-term quality of life and burden of disease post gene-therapy infusion.
Data will be captured in the registry one of two ways:
If you are a patient with hemophilia who has received gene therapy and is interested in participating in the GTR, please contact your HTC.
Further information can be found in the user guide on the basics of hemophilia, gene therapy, and long-term data collection, and the user guide for people with hemophilia.
The WFH GTR is being developed in collaboration with the International Society on Thrombosis and Hemostasis (ISTH), the European Haemophilia Consortium (EHC), the National Hemophilia Foundation (NHF), the American Thrombosis and Hemostasis Network (ATHN), the Patient-Reported Outcomes Burdens and Experiences (PROBE) study group, and experts in the field of gene therapy. The WFH GTR is governed by the GTR Steering Committee, and the data is governed by the WFH GTR Scientific Advisory Board, composed of health care professionals and people with hemophilia.
Our database is housed in a secure data centre with appropriate physical, administrative and technical safeguards in place. These procedures are designed to protect information from misuse, unauthorized access, interference, alteration, loss and/or disclosure, which meets or exceeds privacy and security regulation requirements in Canada, the U.S.A, and Europe. Data policy guidelines adhere to U.S.A, Canadian and European data privacy regulations.
The GTR includes a comprehensive data quality program, which will be applied to all data entered in the gene therapy registry. All data will be evaluated on the following data quality dimensions:
For more information on participating in the GTR, PWH and HTCs are invited to consult the user guides below:
Basics of hemophilia, gene therapy and long-term data collection
Hemophilia treatment centres
People with hemophilia
Publications prepared by the GTR Steering Committee are available here:
The information on the WFH website is provided for general information purposes only. The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH recommends that you contact your physician or local treatment centre. Before administering any products, the WFH urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.
While every effort has been made to ensure the accuracy of the information on this site, the WFH does not guarantee the information is accurate, and is not responsible in any way whatsoever for damages arising out of the use of this website or any of the information contained herein.
Messages posted to WFH discussion forums, Facebook, Twitter, and other social media platforms do not represent the opinions of the World Federation of Hemophilia, its staff, or Board of Directors. The author of a message is solely responsibility for its content. Information posted on WFH social networks and platforms should never be a substitute for individualized professional medical advice, even when the author has medical qualifications or is considered an authority. Information posted to a discussion group should not be used to diagnose or treat a specific health problem without consulting a qualified healthcare professional. The WFH recommends that you contact your physician or local treatment centre if you have any individual questions or concerns.
References and links to other websites or references to other organizations, products, services, or publications do not constitute endorsement or approval by the WFH. The WFH is not responsible and assumes no liability for the content of any linked websites.
The WFH has been made aware of various correspondences—circulated via e-mail and telephone—coming from individuals impersonating WFH staff or falsely stating that they are associated with the WFH. These correspondences, which may seek to obtain money using the name of someone affiliated with the WFH, are fraudulent and come from outside of our organization.
If you receive a suspicious solicitation, exercise extreme caution. In the case of an email, look at the email address to see if it looks suspicious (for example, all WFH emails come from @wfh.org).
We are asking you to remain vigilant, and if you have any doubts about the correspondence, please forward the email to the WFH at [email protected] or call +1 514-875-7944.
National Hemophilia Center, Tel Hashomer
Sheba Medical Center
Hospital Universitario La Paz
Haemostasis and Thrombosis Unit
Haematology Service, University and Polytechnic Hospital
Haemophilia Comprehensive Care Centre
Charlotte Maxeke Johannesburg Academic Hospital
Singapore General Hospital Haemophilia Treatment Clinic
Centre International de Traitement de l’Hémophilie (CITH) de Dakar
Centre National de Transfusion Sanguin (CNTS)
National Medical Research Center of Hematology of the Russian Academy of Medical Sciences
University Medical Centre Utrecht
Nara Hemophilia Centre
Nara Medical University
Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico
National Institute of Immunohaematology (ICMR) and KEM Hospital
Christian Medical College, Vellore
International Hemophilia Training Center – Bangkok
Paris Haemophilia Centre (Necker (N) and Kremlin Bicetre (KB) Hospitals)
Shabrawishi Hospital, Egyptian Society of Hemophilia, and Cairo University
University of Toronto Hemophilia Centres
St. Michael’s Hospital
Hemocentro – “Unidade de Hemofilia Cláudio Luiz Pizzigatti Corrêa”
Hemocentro Unicamp, University of Campinas
Haemostasis and Thrombosis Unit
Division of Haematology
Cliniques universitaires Saint-Luc
Hemofiliecentrum Leuven, University of Leuven
Medical University of Vienna
Department of Medicine I
Clinical Division of Hematology and Hemostaseology
Ronald Sawers Haemophilia Centre
Instituto Asistencial “Dr. Alfredo Pavlovsky”
(Fundación de la Hemofilia)
Instituto De Investigaciones Hematológicas “Dr. Mariano R. Castex” (IIHEMA
(Academia Nacional de Medicina)
Malmö Centre for Thrombosis and Haemostasis
Department for Hematology and Coagulation Disorders, Skåne University Hospital
Skåne University Hospital
Katharine Dormandy Haemophilia Centre and Thrombosis Unit
Royal Free Hospital
Manchester Haemophilia Comprehensive Care Centre (Children’s and Adult’s)
Manchester Royal Infirmary and Manchester Children’s Hospital
Oxford Haemophilia Centre and Thrombosis Centre
Sheffield Haemophilia and Thrombosis Centre
Royal Hallamshire Hospital
|2006||Dr. Bruce Evatt|
Recognizing that training women leaders ensures the diversity our community need to thrive; the Hemophilia Alliance has been a longtime supporter of the Susan Skinner Memorial Fund. They deepened their engagement with the global community through the establishment of the Travel Grant Program, which allows US-based multidisciplinary healthcare professionals the opportunity to attend WFH global training workshops. The wealth of experience and perspective attendees bring back home to their communities helps patients across the country. Their patience and support as we grappled with changing norms in these challenging times gave us the flexibility to meet our community’s needs with revamped programming.
Hemophilia of Georgia has stood with us for over three decades, playing an early role in distributing donated factor via the WFH Humanitarian Aid program. Our relationship has deepened over the years as they continued to support this program and came on board as a founding Chapter Challenge champion in 2016. Their belief in educating future leaders is clear in their investment in the Youth Leadership Workshops, which has led to the training of over 50 young adults. They also support workshops devoted to educating providers and patient leaders on how to collect and use data to advocate for better patient care. Their staff generously volunteer their time as trainers and as well as serving on various committees.
The National Hemophilia Foundation helps advance our mission on several fronts. They inspire others to follow their lead as a founding champion of the NHF Chapter Challenge. Their commitment to equality of access is demonstrated in their support of the Cornerstone Initiative, which lays the foundation for care in the world's most underserved regions, as well as programs that aim to raise awareness and improve care of women with bleeding disorders and those with von Willebrand disease. We also benefit from their steadfast engagement with various programs including the WFH Twinning Program and their individual leaders’ contribution to our leadership and committees.
Do you sometimes wish you could do more? A bequest is a simple way for you to make a significant gift without changing anything about your financial security and lifestyle today. Your legacy will sustain care for the generations to come.
Download these sample clauses to give to your legal advisor.
We understand if you prefer to remain anonymous, but we would be very honoured if you would let us know of your intentions so that we may thank you personally. We would also be happy to answer any questions you may have or give you additional information. Please contact Jennifer Laliberté at 514‑875‑7944 ext.2822 or [email protected].
The information on the WFH USA website is provided for general information purposes only. The WFH USA does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH USA recommends that you contact your physician or local treatment centre. Before administering any products, the WFH USA urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.
While every effort has been made to ensure the accuracy of the information on this site, the WFH USA does not guarantee the information is accurate, and is not responsible in any way whatsoever for damages arising out of the use of this website or any of the information contained herein.
Messages posted to WFH USA discussion forums, Facebook, Twitter, and other social media platforms do not represent the opinions of the World Federation of Hemophilia, its staff, or Board of Directors. The author of a message is solely responsibility for its content. Information posted on WFH USA social networks and platforms should never be a substitute for individualized professional medical advice, even when the author has medical qualifications or is considered an authority. Information posted to a discussion group should not be used to diagnose or treat a specific health problem without consulting a qualified healthcare professional. The WFH USA recommends that you contact your physician or local treatment centre if you have any individual questions or concerns.
References and links to other websites or references to other organizations, products, services, or publications do not constitute endorsement or approval by the WFH USA. The WFH USA is not responsible and assumes no liability for the content of any linked websites.
The WFH USA does not collect personally identifying information about you when you visit our site, unless you choose to provide such information to us. Providing such information is strictly voluntary. This policy is your guide to how we will handle information we learn about you from your visit to our website.
Throughout our website, we provide links to other servers which may contain information of interest to our readers. We take no responsibility for, and exercise no control over, the organizations, views, or accuracy of the information contained on other servers. When linking to the WFH USA, we request that you ensure that there are no associated connections for commercial purposes. Any official use of the name WFH USA or the use of its logo needs to be approved by the WFH USA. If you have a link you’d like us to consider adding to our website, please send an email to [email protected] with the subject “Link request.”
All information, photographs, graphics, and other material on this website are protected by copyright. While users of this website may use the content for personal, non-commercial use, none of the content of this website can be modified, translated, transferred or sold, copied, reproduced, published, or distributed, in whole or in part, without the prior written consent of the WFH USA. To view a copy of the WFH USA’s permissions guidelines, click here.
The WFH USA encourages the dissemination of its information and welcomes proposals on the reprinting, redistribution, and translation of its materials. If you would like to publish information that you find on our website, please send your request to [email protected].
If you have suggestions on how to make the site more accessible, please contact us at [email protected].
Our website provides users the opportunity to opt-out of receiving communications from us through a special online form or via the communication they have received. You may, at all times, choose to receive which communications within the organization you can receive or none at all. You may also update your contact information previously provided to us via another online form.
The WFH USA has been made aware of various correspondences—circulated via e-mail and telephone—coming from individuals impersonating WFH USA staff or falsely stating that they are associated with the WFH USA. These correspondences, which may seek to obtain money using the name of someone affiliated with the WFH USA, are fraudulent and come from outside of our organization.
If you receive a suspicious solicitation, exercise extreme caution. In the case of an email, look at the email address to see if it looks suspicious (for example, all WFH USA emails come from @wfhusa.org or @wfh.org).
We are asking you to remain vigilant, and if you have any doubts about the correspondence, please forward the email to the WFH USA at [email protected] or call 1-877-417-7944.
University of North Carolina Hemophilia & Thrombosis Center
Nationwide Children’s Hospital
Gulf States Hemophilia and Thrombophilia Center
University of Texas Health Science Center at Houston
Orthopaedic Hemophilia Treatment Center
Mount Sinai School of Medicine
Mount Sinai Medical Center
Mayo (Rochester) Comprehensive Hemophilia Center, and International Hemophilia Training Center (WFH)
Mayo Clinic/Foundation and Mayo Medical School
Hemophilia & Thrombosis Treatment Center
University of California San Diego
|2012||Piet de Kleijn
|2010||Dr. Mammen Chandy
|2008||Dr. Man-Chiu Poon
|2006||Dr. Norma de Bosch
|2004||Dr. Parttraporn Insarangkura
Dr. Carol K. Kasper
|2020||Aluva (India) – Newcastle (UK)|
|2019||Yogyakarta / Solo (Indonesia) – Utrecht (Netherlands)|
|2018||Abidjan (Ivory Coast) – Brussels (Belgium)|
|2017||Santa Cruz de la Sierra (Bolivia) – Buenos Aires (Argentina)|
|2016||Kampala (Uganda) – London (UK)|
|2015||Varna (Bulgaria) – Bonn (Germany)|
|2014||Arequipa (Peru) – Los Angeles (USA)|
|2013||Davangere (India) – Liverpool (UK)
Ludhiana (India) – Detroit (USA)
|2012||Hanoi (Vietnam) – Melbourne (Australia)|
|2011||Manado (Indonesia) – Utrecht (Netherlands)|
|2010||Delhi (India) – Winnipeg (Canada)
Yaoundé (Cameroon) – Geneva (Switzerland)
|2009||Chisinau (Moldova) – Warsaw (Poland)
Colombo (Sri Lanka) – Vellore (India)
|2008||Gaborone (Botswana) – Philadelphia (USA)|
|2007||Vientiane (Laos) – Brest (France)
Damascus (Syria) – Montpellier (France)
|2006||Lima (Peru) – Fort Worth (USA)|
|2005||Yerevan (Armenia) – Minneapolis (USA)
Casablanca (Morocco) – Caen (France)
|2004||Beirut (Lebanon) – Geneva (Switzerland)
Santo Domingo (Dominican Republic) – Caracas (Venezuela)
|2003||Cairo (Egypt) – Knoxville (USA)|
|2002||Moscow (Russia) – Liverpool (UK)
Panama – Valencia (Spain)
|2001||Bangalore (India) – St. Louis (USA)|
|2000||Pune (India) – Bradford (UK)|
|1999||Montevideo (Uruguay) – Buenos Aires (Argentina)
Tianjin (China) – Calgary (Canada)
|1998||Plovdiv (Bulgaria) – Bonn (Germany)|
|1997||Bogota (Colombia) – Los Angeles (USA)
Tartu (Estonia) – Stockholm (Sweden)
|1996||Timisoara (Romania) – Munich (Germany)
Riga (Latvia) – Munster (Germany)
|1995||Klaipeda (Lithuania) – Malmo (Sweden)|
|1994||Bratislava (Slovakia) – Tel Hashomer (Israel)|
|2019||Dr. Adolfo Llinás
|2012||Piet de Kleijn
|2011||Dr. Lily Heijnen
|2010||Dr. Horacio Caviglia
|2009||Jerome D. Wiedel
|2008||Dr. Federico Fernández-Palazzi
|2005||Dr. Marvin Gilbert
Yangzhou College, Xuzhou Medical University
|The effects of intra-articular injection of TNF-alpha Antagonists in treatment of haemophilic synovitis|
|Association between multi-segment foot kinetics and blood induced joint damage in adult patients with hemophilic ankle arthropathy|
|Decrease in joint inflammation, swelling and pain after a swimming protocol in animal model of experimental-induced hemarthrosis|
|2015||SM Javd Mortazavi
|Bilateral total knee arthroplasty in patients with hemophilia: A safe and cost-effective procedure|
|2013||Lize F.D. van Vulpen
|A fusion protein of IL4 and IL10, is equally effective in protecting cartilage from blood-induced damage compared to the individual components|
|Pseudotumor Surgery in Haemophilia A patients: Comparitive results between inhibitor and non-inhibitor patients|
United Kingdom and
Monique van Meegeren
|Early onset administration of IL-4 and IL-10 prevents blood-induced cartilage damage in vitro|
|Influence of an Aquatic Training Protocol on the Reduced Risk of Cardiovascular Morbidity and Mortality in a Hemophiliac|
|Impact of Hemophilia Ankle Arthropathy on Gait Disability: Analysis of Energetic and Mechanical Variables|
|2008||Jose Alberto Tlacuilo-Parra
|Bone Turnover Markers and Bone Mineral Density in Children with Hemophilia|
|Protective Abilities of Interleukin-10 in Blood-induced Cartilage Damage|
|2006||Axel Seuser and E. Kusch
|Multi-Center Study of Orthopedic Outcome of the Lower Extremities in 249 Children with Hemophilia|
|2005||Frank van Genderen, Nico van Meeteren, Lily Heijnen, Piet de Kleijn, Marijke van den Berg, Paul Helders
|The Hemophilia Activities List: Final development and validation of a hemophilic-specific self-assessment questionnaire on functional abilities|
|2004||Goris Roosendaal, Zalima N Jahangier, Kim MG Jacobs, Johannes WJ Bijlsma, Floris PJG Lafeber
|Radiosynoviorthesis using YTTRIUM-90 results in adverse direct effects on cartilage: a human in vitro study|
|2003||Axel Seuser, Thomas Wallny, Günter Schumpe, Elmar Trunz, Hans-Hermann Brackman
|How to advise young haemophiliacs to find the right sport? A new and safe algorithm|
|2002||James Luck, Jr. and Mauricio Silva
|Long-Term Review of Total Knee Arthroplasty|
|Oxytetracycline Chlorohydrate: A new material for chemical synovial orthoses|
|1999||William J. Ribbans and J.L. Hicks
|Communication and Co-operation for the New Millennium – The Future of the Musculoskeletal Committee in the 21st Century|
|1998||Adolfo Llinas, Mauricio Silva, Monica Duarte, Gonzalo Ucros, Graciela Perini, Angela Maria Cerquera, Andres Forero
|Chromic phosphate synoviorthesis without age restriction should be the first treatment option for chronic active hemophilic synovitis|
|2012||Martha Patricia Monteros Rincon|
|2010||Antonia Luque de Garrido|
|2008||Jean Michel Bouchez|
The outcomes of interest collected in the WBDR include demographic characteristics, clinical and treatment-related outcomes, burden of disease outcomes, and quality of life. The quality of life scale, EQ-5D-5L, is part of the WBDR extended data set. It is available in more than 130 languages and in different modes of administration.
Arabic (Algeria, Morocco, UAE)
English (Ghana, India, Jamaica, Kenya, Nigeria, and the Philippines)
French (Algeria, Belgium, and Morocco)
Spanish (Argentina, Panama, Spain, and the U.S.A)
The outcomes of interest collected by the WBDR include demographic characteristics, clinical and treatment-related outcomes, burden of disease outcomes, and patient-reported outcomes.
The WBDR includes five functional scales as part of its extended data set:
The WBDR Minimal Data Set (MDS) is a set of data on demographics, diagnosis, symptoms and treatment which are collected on patients participating in the WBDR.
The WBDR Extended Data Set (EDS) is a larger set of data, with more details than the MDS, which includes questions on patient demographics, diagnosis, symptoms, treatment and burden of disease. The EDS can be used by hemophilia treatment centers to collect data on patients participating in the WBDR. The WBDR Extended Data Set (EDS), was added to the database in March 2019.
The WFH was established in 1963 by Frank Schnabel, a Canadian businessman born with severe hemophilia A. He believed that patient organizations could be much more effective—and do more to represent the interests of people living with bleeding disorders—if they worked together. The first WFH Congress was held in Copenhagen, Denmark, on June 25, 1963, and was attended by representatives from 12 countries. Now, the WFH and our global network of national member organizations (NMOs) represent the interests of people with hemophilia and other inherited bleeding disorders in 147 nations.