{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T07:05:56Z","timestamp":1777878356227,"version":"3.51.4"},"reference-count":71,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,2,1]],"date-time":"2026-02-01T00:00:00Z","timestamp":1769904000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"DOI":"10.13039\/501100003196","name":"ministero della salute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003196","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Italian Ministry of Education and Research"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Therapeutic Advances in Hematology"],"published-print":{"date-parts":[[2026,2]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Severe hemophilia traditionally requires lifelong prophylactic treatment, which cannot fully prevent joint damage and significantly impacts patients\u2019 quality of life. In recent years, gene therapy has been extensively researched as a potential cure with a single infusion, but it has not yet gained widespread acceptance.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives:<\/jats:title>\n                    <jats:p>This study examines the knowledge and attitudes of people with severe hemophilia regarding gene therapy to provide guidance to practitioners in the context of shared decision-making.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design:<\/jats:title>\n                    <jats:p>Cross-sectional observational study based on a standardized questionnaire.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>A questionnaire developed by Cutica, Ilaria et al. was evaluated in a self-reported, anonymized survey for a German cohort of 59 people with severe hemophilia. It covered sociodemographic and clinical characteristics, knowledge, attitudes, risk tolerance, and decision-making preferences.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>\n                      Among 59 people with severe hemophilia A and 10 with severe hemophilia B, 59% expressed a predominantly rejective attitude toward gene therapy. Most had only general knowledge of gene therapy. Concerns about long-term side effects (e.g., cancer development) significantly contribute to a negative attitude toward gene therapy (\n                      <jats:italic toggle=\"yes\">p<\/jats:italic>\n                      \u2009=\u20090.005). People with hemophilia and an annual bleeding rate \u2a7e1 show a significantly higher willingness to consider or accept gene therapy (\n                      <jats:italic toggle=\"yes\">p<\/jats:italic>\n                      \u2009=\u20090.001). Rejection of gene therapy is significantly associated with high expectations regarding its duration of efficacy and the minimum clotting factor level.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Further studies are needed to refine educational concepts for people with hemophilia and clear out misconceptions about gene therapy.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/20406207261428774","type":"journal-article","created":{"date-parts":[[2026,3,11]],"date-time":"2026-03-11T18:40:21Z","timestamp":1773254421000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["An analysis of attitudes toward gene therapy in people with severe hemophilia in Germany, a survey-based cross-sectional study"],"prefix":"10.1177","volume":"17","author":[{"given":"Svetlana","family":"Babayeva","sequence":"first","affiliation":[{"name":"Medical Clinic 2, University Hospital Frankfurt, Frankfurt, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Aida","family":"Peyvandi","sequence":"additional","affiliation":[{"name":"Department of Neurosciences and Mental Health, Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico, Milan, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christian","family":"Schepperle","sequence":"additional","affiliation":[{"name":"Interessengemeinschaft H\u00e4mophiler e.V. (IGH), Rottenburg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"G\u00fcnter","family":"Auerswald","sequence":"additional","affiliation":[{"name":"Deutsche H\u00e4mophiliegesellschaft e.V. (DHG), Hamburg, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Steffen","family":"Rauchfuss","sequence":"additional","affiliation":[{"name":"Medical Clinic 2, University Hospital Frankfurt, Frankfurt, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Flora","family":"Peyvandi","sequence":"additional","affiliation":[{"name":"Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ilaria","family":"Cutica","sequence":"additional","affiliation":[{"name":"Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy"},{"name":"Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4506-0061","authenticated-orcid":false,"given":"Wolfgang","family":"Miesbach","sequence":"additional","affiliation":[{"name":"Medical Clinic 2, University Hospital Frankfurt, Theodor-Stern-Kai 7, Frankfurt 60590, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2026,3,11]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-07-820720"},{"key":"e_1_3_5_3_2","unstructured":"Annual Report 2022\/23 German Hemophilia Registry (DHR) https:\/\/www.pei.de\/DE\/newsroom\/pflichtberichte\/dhr\/dhr-berichte-node.html (2024 accessed 9 February 2025)."},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200106073442307"},{"key":"e_1_3_5_5_2","article-title":"Recombinant factor VIIa concentrate versus plasma-derived concentrates for treating acute bleeding episodes in people with haemophilia and inhibitors","volume":"2015","author":"Matino D","year":"2015","unstructured":"Matino D, Makris M, Dwan K, et al. Recombinant factor VIIa concentrate versus plasma-derived concentrates for treating acute bleeding episodes in people with haemophilia and inhibitors. Cochrane Database Syst Rev 2015; 2015: CD004449.","journal-title":"Cochrane Database Syst Rev"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2516.2002.00581.x"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2015-01-528414"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-08-872291"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.47420\/9783769137309"},{"issue":"51","key":"e_1_3_5_10_2","first-page":"887","article-title":"Gene therapy for hemophilia-opportunities and risks","volume":"119","author":"Miesbach W","year":"2022","unstructured":"Miesbach W, Klamroth R, Oldenburg J, et al. Gene therapy for hemophilia-opportunities and risks. 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