{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,14]],"date-time":"2026-02-14T15:11:18Z","timestamp":1771081878370,"version":"3.50.1"},"reference-count":38,"publisher":"Georg Thieme Verlag KG","issue":"03","license":[{"start":{"date-parts":[[2022,12,14]],"date-time":"2022-12-14T00:00:00Z","timestamp":1670976000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Hamostaseologie"],"published-print":{"date-parts":[[2023,6]]},"abstract":"<jats:title>Zusammenfassung<\/jats:title><jats:p>Die Gentherapie hat sich in den vergangenen Jahren zu einer realistischen Behandlungsoption f\u00fcr Patienten mit H\u00e4mophilie entwickelt. Unter Ber\u00fccksichtigung der verf\u00fcgbaren Literatur und der pers\u00f6nlichen Erfahrungen aus klinischen Studien diskutieren wir hier wesentliche Aspekte der Gentherapie der H\u00e4mophilie A und B mit Vektoren auf Basis des Adeno-assoziierten Virus (AAV) einschlie\u00dflich zu erwartender Ergebnisse, Risiken, Nebenwirkungen und Auswirkungen auf patientenrelevante Endpunkte. \u00dcberlegungen zur Auswahl von Patienten, zum Aufkl\u00e4rungs- und Einwilligungsprozess, zur ambulanten Durchf\u00fchrung und \u00dcberwachung der Gentherapie sowie zur Erfassung von Daten werden dargestellt. Die Notwendigkeit interdisziplin\u00e4rer Zusammenarbeit mit der Hepatologie und anderen Fachgruppen wird dargelegt. Besondere Ber\u00fccksichtigung finden strukturelle und organisatorische Voraussetzungen der Zentren, die Gentherapie durchf\u00fchren (Dosierungszentren oder \u201eHub\u201c), und der heimatnahen Zentren, die Patienten der Gentherapie zuf\u00fchren und die Nachsorge \u00fcbernehmen (\u201eSpoke\u201c). Wir geben eine Empfehlung zur Nutzung einer elektronischen Plattform zum sicheren und verz\u00f6gerungsfreien Datenaustausch zwischen diesen Zentren und den Patienten. Diese elektronische Plattform wird auch die Prim\u00e4rdatenquelle sein f\u00fcr Pharmakovigilanz, Studien, nationale und internationale Register sowie f\u00fcr die fr\u00fche Nutzenbewertung dieser neuen Therapieform. \u00dcberlegungen zur Finanzierung der Gentherapie sowie zur m\u00f6glichen zuk\u00fcnftigen Nutzung bei Jugendlichen und Kindern werden diskutiert. In einem sich rasch entwickelnden wissenschaftlichen Umfeld sollen diese Empfehlungen den betreuenden Einrichtungen und Kostentr\u00e4gern erste Orientierung in Vorbereitung auf die Anwendung der Gentherapie nach ihrer Zulassung geben.<\/jats:p>","DOI":"10.1055\/a-1957-4477","type":"journal-article","created":{"date-parts":[[2022,12,15]],"date-time":"2022-12-15T00:23:26Z","timestamp":1671063806000},"page":"196-207","source":"Crossref","is-referenced-by-count":9,"title":["Gentherapie der H\u00e4mophilie: Empfehlung der Gesellschaft f\u00fcr Thrombose- und H\u00e4mostaseforschung (GTH)"],"prefix":"10.1055","volume":"43","author":[{"given":"Wolfgang","family":"Miesbach","sequence":"additional","affiliation":[{"name":"Medizinische Klinik 2, Institut f\u00fcr Transfusionsmedizin, Universit\u00e4tsklinikum Frankfurt, Frankfurt, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Johannes","family":"Oldenburg","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Robert","family":"Klamroth","sequence":"additional","affiliation":[{"name":"Klinik f\u00fcr Innere Medizin \u2013 Angiologie und H\u00e4mostaseologie, Zentrum f\u00fcr Gef\u00e4\u00dfmedizin, Vivantes Klinikum im Friedrichshain, Berlin, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hermann","family":"Eichler","sequence":"additional","affiliation":[{"name":"Institut f\u00fcr Klinische H\u00e4mostaseologie und Transfusionsmedizin, Universit\u00e4t und Universit\u00e4tsklinikum des Saarlandes, Homburg\/Saar, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J\u00fcrgen","family":"Koscielny","sequence":"additional","affiliation":[{"name":"Gerinnungsambulanz mit H\u00e4mophiliezentrum, Charit\u00e9, Berlin, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Susanne","family":"Holzhauer","sequence":"additional","affiliation":[{"name":"Klinik f\u00fcr P\u00e4diatrie m. S. Onkologie und H\u00e4matologie, Charit\u00e9, Universit\u00e4tsmedizin, Berlin, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katharina","family":"Holstein","sequence":"additional","affiliation":[{"name":"II. Medizinische Klinik und Poliklinik, Universit\u00e4tsklinikum Hamburg-Eppendorf, Hamburg, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Johanna A. Kremer","family":"Hovinga","sequence":"additional","affiliation":[{"name":"Universit\u00e4tsklinik f\u00fcr H\u00e4matologie und H\u00e4matologische Zentrallabor, Universit\u00e4tsspital Bern, Universit\u00e4t Bern, Bern, Schweiz"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lorenzo","family":"Alberio","sequence":"additional","affiliation":[{"name":"Division of Haematology and Haematology Central Laboratory, University Hospital of Lausanne (CHUV) and University of Lausanne (UNIL), Lausanne, Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Martin","family":"Olivieri","sequence":"additional","affiliation":[{"name":"H\u00e4mophiliezentrum LMU Klinikum - Bereich P\u00e4diatrie, Dr. von Haunerschen Kinderspital, LMU M\u00fcnchen, M\u00fcnchen, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ralf","family":"Kn\u00f6fler","sequence":"additional","affiliation":[{"name":"Universit\u00e4tsklinikum Dresden Klinik\/Poliklinik f\u00fcr Kinder- und Jugendmedizin Bereich H\u00e4matologie, Dresden, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christoph","family":"Male","sequence":"additional","affiliation":[{"name":"Abteilung f\u00fcr Kinder- und Jugendheilkunde, Medizinische Universit\u00e4t Wien, \u00d6sterreich"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andreas","family":"Tiede","sequence":"additional","affiliation":[{"name":"Klinik f\u00fcr H\u00e4matologie, H\u00e4mostaseologie, Onkologie und Stammzelltransplantation, Medizinische Hochschule Hannover, Hannover, Deutschland"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2022,12,14]]},"reference":[{"issue":"11","key":"ref1","doi-asserted-by":"crossref","first-page":"E3408","DOI":"10.3390\/jcm9113408","article-title":"The German Hemophilia Registry: Growing with Its Tasks","volume":"9","author":"H Duda","year":"2020","journal-title":"J Clin Med"},{"issue":"08","key":"ref2","doi-asserted-by":"crossref","first-page":"540","DOI":"10.7326\/M19-1208","article-title":"Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries","volume":"171","author":"A Iorio","year":"2019","journal-title":"Ann Intern Med"},{"issue":"04","key":"ref3","doi-asserted-by":"crossref","first-page":"591","DOI":"10.1111\/hae.14012","article-title":"The World Federation of Hemophilia Annual Global Survey 1999-2018","volume":"26","author":"J S Stonebraker","year":"2020","journal-title":"Haemophilia"},{"key":"ref4","doi-asserted-by":"crossref","DOI":"10.1111\/hae.14046","volume-title":"WFH Guidelines for the Management of Hemophilia, 3rd edition","author":"A Srivastava","year":"2020"},{"key":"ref5","volume-title":"Arzneimittel zur Therapie der angeborenen und erworbenen H\u00e4mophilie und der von-Willebrand-Erkrankung, in Querschnitts-Leitlinien zur Therapie mit Blutkomponenten und Plasmaderivaten 2020","author":"J Oldenburg"},{"issue":"06","key":"ref6","doi-asserted-by":"crossref","first-page":"946","DOI":"10.1111\/hae.13834","article-title":"Real-life experience in switching to new extended half-life products at European haemophilia centres","volume":"25","author":"F Peyvandi","year":"2019","journal-title":"Haemophilia"},{"issue":"16","key":"ref7","doi-asserted-by":"crossref","first-page":"2231","DOI":"10.1182\/blood.2020009217","article-title":"Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies","volume":"137","author":"M U Callaghan","year":"2021","journal-title":"Blood"},{"issue":"01","key":"ref8","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1002\/ajh.1146","article-title":"Changes in the occurrence of and risk factors for hemophilia-associated intracranial hemorrhage","volume":"68","author":"R Nuss","year":"2001","journal-title":"Am J Hematol"},{"issue":"13","key":"ref9","doi-asserted-by":"crossref","first-page":"2038","DOI":"10.1182\/blood-2015-01-528414","article-title":"Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens","volume":"125","author":"J Oldenburg","year":"2015","journal-title":"Blood"},{"issue":"09","key":"ref10","doi-asserted-by":"crossref","first-page":"2112","DOI":"10.1111\/jth.15444","article-title":"Hemophilic arthropathy: Current knowledge and future perspectives","volume":"19","author":"R Gualtierotti","year":"2021","journal-title":"J Thromb Haemost"},{"issue":"01","key":"ref11","doi-asserted-by":"crossref","first-page":"448","DOI":"10.1186\/s12891-021-04319-0","article-title":"Quality of life and its predictors among adult patients with haemophilic arthropathy. 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