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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
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  Data: 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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  Data: <searchLink fieldCode="AU" term="%22Mussert+CMA%22">Mussert CMA</searchLink>; Department of Pediatric Hematology and Oncology, Erasmus Medical Center Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Miesbach+W%22">Miesbach W</searchLink>; Department of Haemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany. Electronic address: https://twitter.com/Miesbach.<br /><searchLink fieldCode="AU" term="%22Chowdary+P%22">Chowdary P</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Lillicrap+D%22">Lillicrap D</searchLink>; Department of Pathology and Molecular Medicine, Queen's University, Kingston, Canada. Electronic address: https://twitter.com/DavidLillicrap.<br /><searchLink fieldCode="AU" term="%22Mahlangu+J%22">Mahlangu J</searchLink>; Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.<br /><searchLink fieldCode="AU" term="%22Peyvandi+F%22">Peyvandi F</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Pipe+SW%22">Pipe SW</searchLink>; Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Srivastava+A%22">Srivastava A</searchLink>; Haematology Research Unit, St. John's Research Institute and St. John's Medical College Hospital, Bengaluru, India.<br /><searchLink fieldCode="AU" term="%22Voorberg+J%22">Voorberg J</searchLink>; Department of Molecular Hematology, Sanquin Research, Amsterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Pierce+GF%22">Pierce GF</searchLink>; World Federation of Hemophilia, Montreal. Electronic address: https://twitter.com/gfp55.<br /><searchLink fieldCode="AU" term="%22Kaczmarek+R%22">Kaczmarek R</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Batty+P%22">Batty P</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Cutica+I%22">Cutica I</searchLink>; Department of Oncology and Hemato-oncology, University of Milan, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Nathwani+A%22">Nathwani A</searchLink>; Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK; Department of Haematology, Cancer Institute, University College London, UK.<br /><searchLink fieldCode="AU" term="%22Leebeek+FWG%22">Leebeek FWG</searchLink>; Department of Hematology, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands. Electronic address: f.leebeek@erasmusmc.nl.
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  Data: <searchLink fieldCode="JN" term="%22101170508%22">Journal of thrombosis and haemostasis : JTH</searchLink> [J Thromb Haemost] 2026 Feb; Vol. 24 (2), pp. 747-765. <i>Date of Electronic Publication: </i>2025 Oct 27.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier%22">Elsevier </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101170508 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1538-7836 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215387836%22">15387836 </searchLink><i>NLM ISO Abbreviation: </i>J Thromb Haemost <i>Subsets: </i>MEDLINE
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        Value: 10.1016/j.jtha.2025.09.041
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      – Code: eng
        Text: English
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      – TitleFull: 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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              Text: 2026 Feb
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