A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and summary of product characteristics: communication from the ISTH SSC working group on gene therapy.

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Title: A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and summary of product characteristics: communication from the ISTH SSC working group on gene therapy.
Authors: Mussert CMA; Department of Pediatric Hematology and Oncology, Erasmus Medical Center Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, the Netherlands., Miesbach W; Department of Haemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany. Electronic address: https://twitter.com/Miesbach., Chowdary P; Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK; Department of Haematology, Cancer Institute, University College London, UK. Electronic address: https://twitter.com/chowdarypm., Lillicrap D; Department of Pathology and Molecular Medicine, Queen's University, Kingston, Canada. Electronic address: https://twitter.com/DavidLillicrap., Mahlangu J; Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa., Peyvandi F; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy; Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Ca' Granda Ospedale Maggiore Policlinico and Fondazione Luigi Villa, Milan, Italy. Electronic address: https://twitter.com/flora_peyvandi., Pipe SW; Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA., Srivastava A; Haematology Research Unit, St. John's Research Institute and St. John's Medical College Hospital, Bengaluru, India., Voorberg J; Department of Molecular Hematology, Sanquin Research, Amsterdam, the Netherlands., Pierce GF; World Federation of Hemophilia, Montreal. Electronic address: https://twitter.com/gfp55., Kaczmarek R; Herman B Wells Center for Pediatric Research Indiana University School of Medicine, Indianapolis, Indiana, USA; Ludwik Hirszfeld, Polish Academy of Sciences, Institute of Immunology and Experimental Therapy, Wroclaw, Poland. Electronic address: https://twitter.com/RKaczmarekDr., Batty P; Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK; Department of Haematology, Cancer Institute, University College London, UK. Electronic address: https://twitter.com/paul_batty1., Cutica I; Department of Oncology and Hemato-oncology, University of Milan, Milan, Italy., Nathwani A; Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK; Department of Haematology, Cancer Institute, University College London, UK., Leebeek FWG; Department of Hematology, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands. Electronic address: f.leebeek@erasmusmc.nl.
Source: Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2026 Feb; Vol. 24 (2), pp. 747-765. Date of Electronic Publication: 2025 Oct 27.
Publication Type: Journal Article; Review; Practice Guideline
Journal Info: Publisher: Elsevier Country of Publication: England NLM ID: 101170508 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1538-7836 (Electronic) Linking ISSN: 15387836 NLM ISO Abbreviation: J Thromb Haemost Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1538-7836
DOI:10.1016/j.jtha.2025.09.041