The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025

Glenn F Pierce¹, Margareth C Ozelo², Johnny Mahlangu³, Amy L Dunn⁴, Marlene Beijlevelt⁵, Rose Bender⁶, Manuel Carcao⁷, Donna Coffin¹, Enrico Ferri Grazzi⁸, Graham R Foster⁹, Julie Frantsve-Hawley¹⁰, Alfonso Iorio¹¹, Shannon Jackson¹², Radoslaw Kaczmarek¹³, Gwendolyn E Kaeser¹⁴, Barbara A Konkle¹⁵, Magdalena Lewandowska¹⁶, Wolfgang Miesbach¹⁷, Brian O'Mahony¹⁸, Steven Pipe¹⁹, Ulrike M Reiss²⁰, Amy K Wilson²¹, Emna Gouider²²; WFH AAV Gene Therapy Guidelines Expert Panel

Collaborators, Affiliations

Collaborators

WFH AAV Gene Therapy Guidelines Expert Panel:
Megan Adediran, Marlene Beijlevelt, Rose Bender, Manuel Carcao, Erica Crilly, Saliou Diop, Amy Dunn, Miguel Escobar, Enrico Ferri Grazzi, Graham R Foster, Marcela Ganzella-Sisdelli, Emna Gouider, Cesar Haddad, Ryan Hallock, Garrett Hayes, Roland Herzog, Alfonso Iorio, Shannon Jackson, M Joseph John, Radoslaw Kaczmarek, Jay Konduros, Barbara A Konkle, Catherine Lambert, Magdalena Lewandowska, Johnny Mahlangu, Wolfgang Miesbach, Richa Mohan, Brian O'Mahony

Affiliations

¹ World Federation of Hemophilia, Montréal, Québec, Canada.
² Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil.
³ Department of Molecular Medicine and Haematology, School of Pathology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa and Clinical Haematology Unit, Charlotte Maxeke Johannesburg Academic Hospital, Parktown, South Africa.
⁴ Division of Hematology/Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio, USA and The Ohio State University College of Medicine, Columbus, Ohio, USA.
⁵ Amsterdam University Medical Center, Amsterdam, Netherlands.
⁶ Yale University School of Medicine, New Haven, Connecticut, USA.
⁷ Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
⁸ Federation of Haemophilia Associations (FedEmo), Milan, Italy.
⁹ Queen Mary University London, London, UK and Barts Health NHS Trust, London, UK.
¹⁰ EBQ Consulting, Santa Monica, California, USA.
¹¹ Department of Health Research Methods, Evidence, and Impact and Department of Medicine, McMaster University, Ontario, Canada.
¹² Division of Hematology, St. Paul's Hospital and University of British Columbia, Vancouver, British Columbia, Canada.
¹³ Indiana University School of Medicine, Indianapolis, Indiana, USA.
¹⁴ GK Medical Writing, LLC, Bend, Oregon, USA.
¹⁵ Washington Center for Bleeding Disorders, University of Washington, Seattle, Washington, USA.
¹⁶ Innovative Hematology/Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA.
¹⁷ Medical Clinic 2, University Hospital Frankfurt, Frankfurt, Germany.
¹⁸ Irish Haemophilia Society, Dublin, Ireland.
¹⁹ Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA.
²⁰ St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
²¹ M Health Fairview Center for Bleeding and Clotting Disorders, Minneapolis, Minnesota, USA.
²² Service D'hématologie Biologique Hemophilia Center Aziza Othmana, University Tunis El Manar, Tunis, Tunisia.

PMID: 41137692
DOI: 10.1111/hae.70113

The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025

Glenn F Pierce et al. Haemophilia. 2025.

. 2025 Oct 25.

doi: 10.1111/hae.70113. Online ahead of print.

Authors

Collaborators

WFH AAV Gene Therapy Guidelines Expert Panel:
Megan Adediran, Marlene Beijlevelt, Rose Bender, Manuel Carcao, Erica Crilly, Saliou Diop, Amy Dunn, Miguel Escobar, Enrico Ferri Grazzi, Graham R Foster, Marcela Ganzella-Sisdelli, Emna Gouider, Cesar Haddad, Ryan Hallock, Garrett Hayes, Roland Herzog, Alfonso Iorio, Shannon Jackson, M Joseph John, Radoslaw Kaczmarek, Jay Konduros, Barbara A Konkle, Catherine Lambert, Magdalena Lewandowska, Johnny Mahlangu, Wolfgang Miesbach, Richa Mohan, Brian O'Mahony

Affiliations

¹ World Federation of Hemophilia, Montréal, Québec, Canada.
² Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil.
³ Department of Molecular Medicine and Haematology, School of Pathology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa and Clinical Haematology Unit, Charlotte Maxeke Johannesburg Academic Hospital, Parktown, South Africa.
⁴ Division of Hematology/Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio, USA and The Ohio State University College of Medicine, Columbus, Ohio, USA.
⁵ Amsterdam University Medical Center, Amsterdam, Netherlands.
⁶ Yale University School of Medicine, New Haven, Connecticut, USA.
⁷ Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
⁸ Federation of Haemophilia Associations (FedEmo), Milan, Italy.
⁹ Queen Mary University London, London, UK and Barts Health NHS Trust, London, UK.
¹⁰ EBQ Consulting, Santa Monica, California, USA.
¹¹ Department of Health Research Methods, Evidence, and Impact and Department of Medicine, McMaster University, Ontario, Canada.
¹² Division of Hematology, St. Paul's Hospital and University of British Columbia, Vancouver, British Columbia, Canada.
¹³ Indiana University School of Medicine, Indianapolis, Indiana, USA.
¹⁴ GK Medical Writing, LLC, Bend, Oregon, USA.
¹⁵ Washington Center for Bleeding Disorders, University of Washington, Seattle, Washington, USA.
¹⁶ Innovative Hematology/Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA.
¹⁷ Medical Clinic 2, University Hospital Frankfurt, Frankfurt, Germany.
¹⁸ Irish Haemophilia Society, Dublin, Ireland.
¹⁹ Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA.
²⁰ St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
²¹ M Health Fairview Center for Bleeding and Clotting Disorders, Minneapolis, Minnesota, USA.
²² Service D'hématologie Biologique Hemophilia Center Aziza Othmana, University Tunis El Manar, Tunis, Tunisia.

PMID: 41137692
DOI: 10.1111/hae.70113

No abstract available

Keywords: AAV gene therapy; bleeding disorders; haemophilia; management guidelines; monitoring; outcomes; shared decision‐making.

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References

10 | References

1. A. Srivastava, E. Santagostino, A. Dougall, et al., “WFH Guidelines for the Management of Hemophilia,” Haemophilia 26, no. S6 (2020): 1–158.
1. J. Windyga, S. Apte, M. Frei‐Jones, et al., “Disease and Treatment Burden of Patients With Haemophilia Entering the explorer6 Non‐Interventional Study,” European Journal of Haematology 113, no. 5 (2024): 631–640.
1. W. Miesbach, P. Chowdary, M. Coppens, et al., “Delivery of AAV‐Based Gene Therapy Through Haemophilia Centres‐A Need for Re‐Evaluation of Infrastructure and Comprehensive Care: A Joint Publication of EAHAD and EHC,” Haemophilia 27, no. 6 (2021): 967–973.
1. W. Miesbach, K. J. Pasi, S. W. Pipe, et al., “Evolution of Haemophilia Integrated Care in the Era of Gene Therapy: Treatment Centre's Readiness in United States and EU,” Haemophilia 27, no. 4 (2021): 511–514.
1. W. Miesbach, P. Batty, P. Chowdary, et al., “Adeno‐Associated Virus‐Based Gene Therapy for Hemophilia‐Addressing the Gaps,” Research and Practice in Thrombosis and Haemostasis 9, no. 1 (2025): 102673.

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The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025

Collaborators

Affiliations

The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025

Authors

Collaborators

Affiliations

References

10 | References

LinkOut - more resources

Full Text Sources