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GENE THERAPY REGISTRY

Gene Therapy Registry

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.

PARTICIPATE IN THE GTR

Data will be captured in the registry one of two ways:

  1. Directly via participating hemophilia treatment centres (HTCs)
  • All HTCs that will be administering gene therapy for PWH will be invited to participate. HTCs that will be providing follow up visits for PWH who receive gene therapy will either be invited to participate, or the patient data collected at the follow up site will be sent to the HTC administering gene therapy for inclusion in the registry.

If your HTC is interested in participating in the GTR, please email us at [email protected]. The WFH will guide you through the process of obtaining ethics approval from your local institution.

  1. Through data transfer/linking from existing national hemophilia registries who meet specific criteria and can ensure the following:
  • Collection of the same/similar data fields as the WFH GTR core data set
  • Harmonization and standardization of terminology, particularly for adverse events
  • Patient consent for secondary uses of their data outside of the existing registry
  • Strict data quality management practices and high-quality data
  • Avoidance of duplication of PWH

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.

GTR GOVERNANCE

A comprehensive governance structure—involving several key committees—was established to ensure the success of the GTR. The governance structure includes input from the Scientific Advisory Board (SAB), the National Registries & Hemophilia Treatment Centres Consortium, the steering committee, the industry consortium, and the patient advisory group. Representatives from various patient and healthcare professional organizations—including the International Society of Thrombosis and Hemostasis (ISTH), the European Association for Haemophilia and Allied Disorders (EAHAD), the European Haemophilia Consortium (EHC), and the US National Bleeding Disorders Foundation (NBDF)—are represented on the different committees.

GTR DATA

The WFH GTR is being developed in collaboration with the International Society on Thrombosis and Hemostasis (ISTH), the European Haemophilia Consortium (EHC), the National Bleeding Disorders Foundation (NBDF), 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 Scientific Advisory Board, which provides expert scientific advice on the WFH GTR, particularly focusing on the data charter, research policies, and scientific issues. The Scientific Advisory Board is composed of healthcare professionals and people with hemophilia.

The GTR protocol synopsis is available here.
The GTR core data set was published in the Journal of Thrombosis and Haemostasis and is available here.

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 WFH has appointed GDPR Rep as our representative in the E.U. and the U.K. to act as a point of contact for E.U. and U.K. authorities and any patient enrolled in the GTR in those regions.

Data governance is vital to ensure the accuracy, consistency, and security of the data collected and managed in the GTR. It enables efficient data use and minimizes risks, while also ensuring the GTR provides valuable insights for research, policy-making, and clinical decision-making.

A comprehensive set of standard operating procedures (SOP) have been developed by the GTR Scientific Advisory Board to govern data use in the GTR, covering topics such as:

  • Data access, rights, and requests
  • Communications, reports and publications
  • Collection and processing of safety information
  • Interpretation, evaluation and communication of safety data
  • Data quality

These SOPs ensure the ethical, secure, and efficient management of data, supporting the GTR’’s mission to assess the long-term safety, efficacy, and durability of gene therapy for hemophilia.

For more information on the SOPs, please contact [email protected].

GTR PROGRAMS

A man wearing a stethoscope around his neck talks across a desk to a patient

Data Quality Program

The GTR includes a comprehensive data quality assurance (DQA) program, which is applied to all data entered in the registry. The DQA Program is informed by best practices in the field including the European Medicines Agency’s Data Quality Framework. All data is evaluated on the following quality dimensions:

  • Reliability
  • Extensiveness
  • Coherence
  • Timeliness
  • Relevance

myGTR

myGTR is a patient engagement tool of the WFH Gene Therapy Registry (GTR). myGTR is designed to collect patient-reported outcome data through an automated digital assistant. Patients will receive alerts via their preferred contact method, either by email or text message to access myGTR. myGTR is a web-based application and is not available in any app store.

myGTR will allow patients to continue providing data on their health status; and health-related quality of life (HR-QOL) in a simple manner. At regular intervals (4 times during the first-year post-infusion, and 2 times per year thereafter ), the patient will be prompted to answer a few simple questions regarding their bleeds and treatment; and two of three different HR-QOL questionnaires, on a rotational basis (see Diagram: myGTR).

The HR-QOL questionnaires include:

  1. PROBE (Patient Reported Outcomes Burdens, and Experiences): a questionnaire collecting information about quality of life and other areas important to health.
  2. coreHEM MHO (coreHEM Mental Health Outlook): an instrument to assess mental health outlook of a person with hemophilia who has received gene therapy.
  3. EQ-5D-5L (EuroQol 5 Dimensions 5 Levels): a short tool used to measure quality of life with five questions related to mobility, self-care, usual activities, pain/discomfort, and anxiety/depression.

Data from myGTR will complement clinical data collected by physicians through the WFH GTR. All data entered in myGTR will be stored in the WFH GTR database.

Diagram: myGTR Questionnaire Cadence

A flowchart of the myGTR questionnaire cadence
Frequency: Year 1 = quarterly pushouts (email or text message); Year 2 onwards = every 6 months

SUPPORT FOR THE GTR COMMUNITY

For more information on participating in the GTR, PWH and HTCs are invited to consult the user guides below:

user Guides

User Guide

Basics of hemophilia, gene therapy and long-term data collection

User Guide

Hemophilia treatment centres

User Guide

People with hemophilia

For more information on gene therapy, please visit the WFH Gene Therapy eLearning Centre.

PUBLICATIONS

Publications related to the GTR are available here:

  • Konkle, B. A., Coffin, D., Pierce, G. F., Clark, C., George, L., Iorio, A., Mahlangu, J., Naccache, M., O’Mahony, B., Peyvandi, F., Pipe, S., Quartel, A., Sawyer, E. K., Skinner, M. W., Tortella, B., Watson, C., Winburn, I., & Members of the WFH Gene Therapy Registry Steering Committee. (2020). World Federation of Hemophilia Gene Therapy Registry. Haemophilia, 26(4), 555-741, e151-e222. https://doi.org/10.1111/hae.14015
  • Konkle, B., Pierce, G., Coffin, D., Naccache, M., Clark, R. C., George, L., Iorio, A., O’Mahony, B., Pipe, S., Skinner, M., Watson, C., Peyvandi, F., Mahlangu, J., for the ISTH Subcommittee on Factor VIII, Factor IX, Rare Bleeding Disorders. (2020). Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH. Journal of Thrombosis and Haemostasis, 18(11), 2-3128. https://doi.org/10.1111/jth.15023
  • Konkle, B. A., Peyvandi, F., Coffin, D., Naccache, M., Youttananukorn, T., Pierce, G. F., & the WFH Gene Therapy Registry Scientific Advisory Board. (2024). Landmark endorsement of a global registry: The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), publicly endorses World Federation of Hemophilia Gene Therapy Registry as global standard. Haemophilia, 30(1), 1-252. https://doi.org/10.1111/hae.14912
  • Miesbach, W., Konkle, B., Chowdary, P., Kaczmarek, R., Leebeek, F., Mahlangu, J., Makris, M., Pipe, S. W., Srivastava, A., Voorberg, J., Pierce, G. F., & Peyvandi, F. (2024).
    Recommendations for a minimum data set for monitoring gene therapy in hemophilia: Communication from the ISTH SSC Working Group on Gene Therapy. Journal of Thrombosis and Haemostasis, 22(5), 1510-1515. https://doi.org/10.1016/j.jtha.2023.12.039

CONTACT US

Please contact the GTR team at [email protected].

THANK YOU

THE GTR IS SUPPORTED BY FUNDING FROM:

FOUNDING VISIONARY PARTNERS

ADDITIONAL SUPPORT FOR THE GTR PROVIDED BY:

GDPR Rep logo
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Disclaimer

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.

Fraud Alert

Unauthorized solicitations – Warning

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.

Hemophilia Alliance

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.

India - Mumbai

Year of IHTC designation: 2000

National Institute of Immunohaematology (ICMR) and KEM Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Molecular genetics
  • Dental care
  • Nursing
  • Orthopedic care
  • Physiotherapy

India - Vellore

Year of IHTC designation: 2000

Christian Medical College, Vellore

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Molecular genetics with antenatal diagnosis
  • Nursing
  • Musculoskeletal management – physical and occupational therapy with physiatry
  • Orthopedic care and surgery
  • Blood banking
  • Dental care
  • Psychology/Social services
  • Antenatal diagnosis

Singapore - Singapore

Year of IHTC designation: 2002

Singapore General Hospital Haemophilia Treatment Clinic

Areas of Specialty

  • Adult hematology
  • Laboratory diagnosis
  • Nursing
  • Dental care
  • Orthopedic care (no surgeries)
  • Physiotherapy
  • Psychology

Senegal - Dakar

Year of IHTC designation: 2016

Centre International de Traitement de l’Hémophilie (CITH) de Dakar
Centre National de Transfusion Sanguin (CNTS)

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Blood banking
  • Physiotherapy

Russia - Moscow and St. Petersburg

Year of IHTC designation: 2010

National Medical Research Center of Hematology of the Russian Academy of Medical Sciences

Areas of Specialty

  • Adult hematology
  • Nursing
  • Orthopedic surgery

Netherlands - Utrecht

Year of IHTC designation: 1996

Van Creveldkliniek
University Medical Centre Utrecht

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hematology
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy/Occupational therapy
  • Psychology and social services

Japan - Nara

Year of IHTC designation: 1998

Nara Hemophilia Centre
Nara Medical University

Areas of Specialty

  • Adult and pediatric hematology
  • Dental care
  • Laboratory diagnosis and Molecular genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy

Italy - Milan

Year of IHTC designation: 1972

Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico

Areas of Specialty

  • Adult and pediatric hematology
  • Blood banking
  • Dental care
  • Diagnostic assessment of platelet functional defects
  • Laboratory diagnosis and hemostasis
  • Molecular genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology and social services

Israel - Tel Hashomer

Year of IHTC designation: 1993

National Hemophilia Center, Tel Hashomer
Sheba Medical Center

Areas of Specialty

  • Adult and pediatric hematology
  • Orthopedic surgery
  • Nursing
  • Physiotherapy
  • Dental care
  • Laboratory diagnosis (not genetics)
  • Psychology

France - Paris

Year of IHTC designation: 1972

Paris Haemophilia Centre (Necker (N) and Kremlin Bicêtre (KB) Hospitals)

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Dental care
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology and Social services

Spain - Madrid

Year of IHTC designation: 2018

Hospital Universitario La Paz

Areas of Specialty

  • Adult and pediatric hematology
  • Physiotherapy/occupational therapy
  • Dental care
  • Orthopaedic care (no surgery)
  • Laboratory diagnosis
  • Nursing
  • Psychology/social services

Spain - Valencia

Year of IHTC designation: 1997

Haemostasis and Thrombosis Unit
Haematology Service, University and Polytechnic Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Molecular genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Blood banking
  • Psychology and Social services

Egypt - Cairo

Year of IHTC designation: 2015

Shabrawishi Hospital, Egyptian Society of Hemophilia, and Cairo University

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Molecular genetics
  • Blood banking
  • Physiotherapy

Canada - Toronto

Year of IHTC designation: 2018

University of Toronto Hemophilia Centres
St. Michael’s Hospital

Areas of Specialty

  • Adult hematology services
  • Pediatric hematology services
  • Physiotherapy/occupational therapy
  • Dental care
  • Orthopaedic care (no surgery)
  • Laboratory diagnosis
  • Nursing
  • Psychology/social services

Brazil - Campinas, SP

Year of IHTC designation: 2008

Hemocentro – “Unidade de Hemofilia Cláudio Luiz Pizzigatti Corrêa”
Hemocentro Unicamp, University of Campinas

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and molecular genetics
  • Nursing
  • Dentistry
  • Orthopedic care and surgery
  • Physiotherapy

Belgium - Brussels

Year of IHTC designation: 2013

Haemostasis and Thrombosis Unit
Division of Haematology
Cliniques universitaires Saint-Luc

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis
  • Orthopedic care and surgery
  • Physiotherapy

Austria - Vienna

Year of IHTC designation: 1972

Medical University of Vienna
Department of Medicine I
Clinical Division of Hematology and Hemostaseology

Areas of Specialty

  • Adult and pediatric hematology

Australia - Melbourne

Year of IHTC designation: 2005

Ronald Sawers Haemophilia Treatment Centre

Areas of Specialty

  • Adult hematology
  • Laboratory diagnosis and hemostasis
  • Orthopedic care and surgery
  • Nursing
  • Dental care
  • Blood banking
  • Psychology
  • Social work

Argentina - Buenos Aires

Year of IHTC designation: 1974

Instituto Asistencial “Dr. Alfredo Pavlovsky”
(Fundación de la Hemofilia)

Instituto De Investigaciones Hematológicas “Dr. Mariano R. Castex” (IIHEMA
(Academia Nacional de Medicina)

Areas of Specialty

  • Adult and pediatric hematology
  • Physiotherapy
  • Dental care
  • Orthopaedic care and surgery
  • Laboratory diagnosis and genetic diagnosis
  • Nursing
  • Psychology
  • Social services

South Africa - Johannesburg

Year of IHTC designation: 2016

Haemophilia Comprehensive Care Centre
Charlotte Maxeke Johannesburg Academic Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Dental care
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic surgery
  • Physiotherapy

Sweden - Malmö

Year of IHTC designation: 1976

Malmö Centre for Thrombosis and Haemostasis
Department for Hematology and Coagulation Disorders, Skåne University Hospital
Skåne University Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Blood banking (on request)
  • Dental care and surgery
  • Laboratory diagnosis and genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy

Hemophilia of Georgia

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.

WFH President's Award - Recipients

National Bleeding Disorders Foundation

The National Bleeding Disorders 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.

Legacy Gift

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].

Disclaimer

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.

Website privacy policy

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.

Use of links

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.”

Use of text and images

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].

Accessibility

If you have suggestions on how to make the site more accessible, please contact us at [email protected].

Opt-Out or Change Your Contact Information

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.

Questions about our policies

If you have any questions about this privacy statement, the practices of this site, or your dealings with this website, you can contact us at [email protected].

Fraud alert

Unauthorized solicitations – Warning

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.

International Award for Outstanding Service - Recipients

International Lifetime Achievement Award - Recipients

Previous years

2012Christine Lee
United Kingdom
2006Dr. Bruce Evatt
United States

International Healthcare Volunteer Award - Recipients

Previous years

2012 Piet de Kleijn
The Netherlands
2010 Dr. Mammen Chandy
India
2008 Dr. Man-Chiu Poon
Canada
2006 Dr. Norma de Bosch
Venezuela
2004 Dr. Parttraporn Insarangkura
Thailand

Dr. Carol K. Kasper
United States

Thailand - Bangkok

Year of IHTC designation: 1984

International Hemophilia Training Center – Bangkok
Ramathibodi Hospital
Mahidol University

Areas of Specialty

  • Adult and pediatric hematology
  • Blood banking
  • Dental care and surgery
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology and social services

Twins of the Year Awards

2022 Amman (Jordan) – Dublin (Ireland)
2021 Addis Ababa (Ethiopia) – Minneapolis (U.S.A) and
Tehran (Iran) – Milan (Italy)
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)

Pietrogrande Prize

2023 Nicholas Goddard
United Kingdom
2021 Axel Seuser
Germany
2019 Dr. Adolfo Llinás
Colombia
2017 Pamela Hilliard
Canada
2015 Kathy Mulder
Canada
2013 James Luck
United States
2012 Piet de Kleijn
The Netherlands
2011 Dr. Lily Heijnen
The Netherlands
2010 Dr. Horacio Caviglia
Argentina
2009 Jerome D. Wiedel
United States
2008 Dr. Federico Fernández-Palazzi
Venezuela
2007 Michael Heim
Israel
2006 Brenda Buzzard
United Kingdom
2005 Dr. Marvin Gilbert
United States

Henri Horoszowski Award

2021 Baolai Hua
Yangzhou College, Xuzhou Medical University
The effects of intra-articular injection of TNF-alpha Antagonists in treatment of haemophilic synovitis
2019 Maarten Eerdekens
Belgium
Association between multi-segment foot kinetics and blood induced joint damage in adult patients with hemophilic ankle arthropathy
2017 Fabio Souza
Brazil
Decrease in joint inflammation, swelling and pain after a swimming protocol in animal model of experimental-induced hemarthrosis
2015 SM Javd Mortazavi
Iran
Bilateral total knee arthroplasty in patients with hemophilia: A safe and cost-effective procedure
2013 Lize F.D. van Vulpen
United States
A fusion protein of IL4 and IL10, is equally effective in protecting cartilage from blood-induced damage compared to the individual components
2012 Horacio Caviglia
Argentina
Pseudotumor Surgery in Haemophilia A patients: Comparitive results between inhibitor and non-inhibitor patients
2011 Nick Goddard
United Kingdom and
Monique van Meegeren
The Netherlands
Early onset administration of IL-4 and IL-10 prevents blood-induced cartilage damage in vitro
2010 Lydia Abad-Franch
Spain
Influence of an Aquatic Training Protocol on the Reduced Risk of Cardiovascular Morbidity and Mortality in a Hemophiliac
2009 Sebastien Lobet
Belgium
Impact of Hemophilia Ankle Arthropathy on Gait Disability: Analysis of Energetic and Mechanical Variables
2008 Jose Alberto Tlacuilo-Parra
Mexico
Bone Turnover Markers and Bone Mineral Density in Children with Hemophilia
2007 Natalie Jansen
The Netherlands
Protective Abilities of Interleukin-10 in Blood-induced Cartilage Damage
2006 Axel Seuser and E. Kusch
Germany
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 Netherlands
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
The Netherlands
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
Germany
How to advise young haemophiliacs to find the right sport? A new and safe algorithm
2002 James Luck, Jr. and Mauricio Silva
United States
Long-Term Review of Total Knee Arthroplasty
2001 Federico Fernández-Palazzi
Venezuela
Oxytetracycline Chlorohydrate: A new material for chemical synovial orthoses
2000 Horacio Caviglia
Argentina
Pseudotumours
1999 William J. Ribbans and J.L. Hicks
United Kingdom
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
Colombia
Chromic phosphate synoviorthesis without age restriction should be the first treatment option for chronic active hemophilic synovitis

International Frank Schnabel Volunteer Award - Recipients

Previous years

2012Martha Patricia Monteros Rincon
Mexico
2010Antonia Luque de Garrido
Venezuela
2008Jean Michel Bouchez
France
2006Yuri Zhulyov
Russia
2004Ashok Verma
India

Functional Scales

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:

WBDR hemophilia data sets

WBDR Minimal 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.

WBDR Extended Data Set

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.

United States - Columbus, Ohio

Year of IHTC designation: 2018

Nationwide Children’s Hospital

Areas of Specialty

  • Dental care
  • Laboratory diagnosis
  • Nursing
  • Orthopedic care and surgery
  • Pediatric hematology services
  • Physiotherapy/occupational therapy
  • Psychology/social services

United States - Houston, Texas

Year of IHTC designation: 2000

Gulf States Hemophilia and Thrombophilia Center
University of Texas Health Science Center at Houston

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Blood banking
  • Physiotherapy/ Occupational therapy
  • Psychology/Social services

United States - Los Angeles, California

Year of IHTC designation: 1970

Lifespan Orthopaedic Hemophilia Treatment Center at Luskin Orthopedic Institute for Children

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology/Social services

United States - New York, New York

Year of IHTC designation: 1993

Mount Sinai School of Medicine
Mount Sinai Medical Center

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic care and surgery
  • Molecular genetics
  • Social services

United States - Rochester, Minnesota

Year of IHTC designation: 1974

Mayo (Rochester) Comprehensive Hemophilia Center, and International Hemophilia Training Center (WFH)
Mayo Clinic/Foundation and Mayo Medical School

Areas of Specialty

  • Adult and pediatric hematology
  • Blood banking
  • Laboratory diagnosis and hemostasis
  • Molecular genetics
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy
  • Psychology/ Social services

United States - San Diego, California

Year of IHTC designation: 2018

Hemophilia & Thrombosis Treatment Center
University of California San Diego

Areas of Specialty

  • Adult hematology
  • Physiotherapy/occupational therapy
  • Dental care
  • Orthopaedic care (no surgery)
  • Laboratory diagnosis
  • Nursing
  • Psychology/social services

United Kingdom - London

Year of IHTC designation: 1970

Katharine Dormandy Haemophilia Centre and Thrombosis Unit
Royal Free Hospital

Areas of Specialty

  • Adult hematology
  • Laboratory diagnosis and hemostasis
  • Nursing
  • Orthopedic care and surgery
  • Physiotherapy/Occupational therapy
  • Molecular genetics
  • Psychology/Social services

United Kingdom - Oxford

Year of IHTC designation: 1972

Oxford Haemophilia and Thrombosis Centre
Churchill Hospital

Areas of Specialty

  • Adult and pediatric hematology
  • Laboratory diagnosis and hemostasis
  • Orthopedic care and surgery
  • Nursing
  • Molecular genetics
  • Physiotherapy/Occupational therapy
  • Psychology and social services

United Kingdom - Sheffield

Year of IHTC designation: 1992

Sheffield Haemophilia and Thrombosis Centre
Royal Hallamshire Hospital

Areas of Specialty

  • Adult hematology
  • Laboratory diagnosis and hemostasis
  • Molecular genetics

Our history

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 152 nations.