AAV Gene Therapy

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Last Updated: November 18, 2024
First Published: November 18, 2024

Guidelines

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WFH Guidelines in Practice

Frameworks

Guidelines

AAV Gene Therapy Version 2025

Recommended Citation

Pierce, G.F., Ozelo, M.C., Mahlangu, J., Dunn, A.L., Beijlevelt, M., Bender, R., Carcao, M., Coffin, D., Grazzi, E.F., Foster, G.R., et al. (2025). The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025. Haemophilia. https://doi.org/10.1111/hae.70113

Sub-Topic Panel Lead

Margareth C. Ozelo

MD, PhD

Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil

Marlene Beijlevelt

MSc, NP

Amsterdam University Medical Center, Amsterdam, Netherlands

Manuel Carcao

MD, FRCPC

Division of Haematology/Oncology, Department of Paediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada

Shannon Jackson

MD, FRCPC

Division of Hematology, St. Paul’s Hospital and University of British Columbia, Vancouver, British Columbia, Canada

Barbara A. Konkle

Washington Center for Bleeding Disorders, University of Washington, Seattle, Washington, USA

Steven W. Pipe

Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA

Robert Sidonio Jr.

Pediatric Hematology and Medical oncology, Hemophilia of Georgia Center for Bleeding and Clotting Disorders of CHOA, Atlanta, GA, USA

Catherine Lambert

Haemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, Brussels, Belgium

Magdalena Lewandowska

Innovative Hematology/Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA

Richa Mohan

M.Phil.

President, Empowering Minds Society for Research & Development, Delhi, India

Topic Panelists

Johnny Mahlangu

BSc, MBBCh, MMed, FCPath

Department of Molecularm Medicine and Haematology, School of Pathology
Faculty of Health Sciences, University of theWitwatersrand, Johannesburg
South Africa and Clinical Haematology Unit
Charlotte Maxeke Johannesburg Academic Hospital, Parktown, South Africa

Amy L. Dunn

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

Rose Bender

Lived Experience Expert

Yale University School of Medicine, New Haven, Connecticut, USA

Donna Coffin

MSc

World Federation of Hemophilia, Montreal, QC, Canada

Enrico Ferri Grazzi

Lived Experience Expert

Federation of Haemophilia Associations (FedEmo), Milan, Italy

Graham R. Foster

MA, MBBS, PhD, FRCP

Queen Mary University London, London, UK and Barts Health NHS Trust, London, UK

Julie Frantsve-Hawley

PhD

EBQ Consulting, Santa Monica, California, USA

Alfonso Iorio

MD, PhD, FRCPC

Department of Health Research Methods, Evidence, and Impact and Department of Medicine, McMaster University, Ontario, Canada

Radoslaw Kaczmarek

Lived Experience Expert

Indiana University School of Medicine, Indianapolis, Indiana, USA

Gwendolyn E. Kaeser

PhD

GK Medical Writing, LLC, Bend, Oregon, USA

Wolfgang Miesbach

Medical Clinic 2, University Hospital Frankfurt, Frankfurt, Germany

Brian O’Mahony

Lived Experience Expert

Irish Haemophilia Society, Dublin, Ireland

Ulrike M. Reiss

St. Jude Children’s Research Hospital, Memphis, Tennessee, USA

Amy K.Wilson

LICSW

M Health Fairview Center for Bleeding and Clotting Disorders, Minneapolis, Minnesota, USA

Megan Adediran

Lived Experience Expert

Nigeria

Erica Crilly

MN, NP

Division of Paediatric Hematology/Oncology/BMT, Vancouver, British Columbia, Canada

Saliou Diop

Department of Hematology, University Cheikh Anta Diop, Dakar, Senegal.

Miguel Escobar

University of Texas Health Science Center at Houston-McGovern Medical School and the Gulf States Hemophilia and Thrombophilia Center, Houston, Texas, United States.

Marcela Ganzella- Sisdelli

PhD, RN

Fundação Hemocentro de Ribeirão Preto. Ribeirão Preto, São Paulo, Brazil.

Cesar Haddad

Lebanese Association for Hemophilia
Beirut, Lebanon

Ryan Hallock

Lived Experience Expert

USA

Garrett Hayes

Lived Experience Expert

USA

Roland Herzog

PhD

Department of Pediatrics, Herman B Wells Center for Pediatric Research, Indiana University, Indianapolis, USA

M. Joseph John

Department of Clinical Haematology, Haemato-Oncology and Bone Marrow (Stem Cell) Transplantation, Christian Medical College, Punjab, India

Jay Konduros

Lived Experience Expert

Canada

Bishesh Sharma Poudyal

Clinical Hematology And Bone Marrow Transplant1 Unit, Civil Service Hospital,
Nepal

Dawn Rotellini

Lived Experience Expert

USA

Ekawat Suwantaroj

Lived experience expert

Bangkok, Thailand

Jerzy Windyga

MD, PhD

Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland

Guidelines Co-Chair

Glenn F. Pierce

MD, PhD

World Federation of Hemophilia, Montreal, QC, Canada

Emna Gouider

Medical School, University of Tunis El Manar, Hemophilia Centre, Aziza Othmana Hospital, Tunis, Tunisia

AAV Gene Therapy

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