{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,8]],"date-time":"2026-06-08T04:13:40Z","timestamp":1780892020413,"version":"3.54.1"},"reference-count":32,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2023,9,25]],"date-time":"2023-09-25T00:00:00Z","timestamp":1695600000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2023,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Haemophilia treatment centres (HTCs) and healthcare providers (HCPs) will need to adapt to a new treatment paradigm with the emergence of adeno\u2010associated virus (AAV)\u2010based gene therapy for the treatment of haemophilia in adults.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>This review examines the upcoming patient and institutional journeys, along with practical aspects of preparedness for clinical delivery of gene therapy by HTCs.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Based on our clinical experience and examination of published literature, we explored the parallel journeys for patients and treatment centres to navigate before, during, and after administration of gene therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The patient journey includes: information gathering; decision making; comprehensive patient assessment; preparation for the infusion itself; short\u2010 and long\u2010term monitoring; lifestyle modifications; and the possible need for immunosuppressive treatment. Informed decision\u2010making may require patient education with extensive discussions and an understanding that not all people with haemophilia will choose or be eligible for gene therapy, although eligibility criteria continue to evolve. The institutional journey includes: consideration of biosafety procedures; planning for product procurement, handling, storage, and administration; development of detailed protocols and guidance documents; contingency planning for immunosuppressive and haemostatic management; consideration of clinical capabilities and staff training needs; coordination of efforts by the full multidisciplinary team; and collaboration between referring, dosing, and follow\u2010up treatment centres. Documented protocols and guidance documents are pivotal for this complex therapy to ensure safe handling, optimal delivery, and post\u2010infusion management and follow\u2010up.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Successful implementation of this new treatment modality will require communication and collaboration among multiple stakeholders.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14867","type":"journal-article","created":{"date-parts":[[2023,9,25]],"date-time":"2023-09-25T14:35:10Z","timestamp":1695652510000},"page":"1430-1441","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["Delivery of gene therapy in haemophilia treatment centres in the United States: Practical aspects of preparedness and implementation"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2558-2089","authenticated-orcid":false,"given":"Steven","family":"Pipe","sequence":"first","affiliation":[{"name":"Departments of Pediatrics and Pathology University of Michigan  Ann Arbor Michigan USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kayla","family":"Douglas","sequence":"additional","affiliation":[{"name":"Director of Pharmacy Mississippi Center for Advanced Medicine  Madison Mississippi USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nina","family":"Hwang","sequence":"additional","affiliation":[{"name":"Pediatric Hematology Center for Inherited Blood Disorders  Orange California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6013-1254","authenticated-orcid":false,"given":"Guy","family":"Young","sequence":"additional","affiliation":[{"name":"Cancer and Blood Disorders Institute Children's Hospital and University of Southern California Keck School of Medicine  Los Angeles California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Priya","family":"Patel","sequence":"additional","affiliation":[{"name":"Pfizer Inc.  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3146-6519","authenticated-orcid":false,"given":"Patrick","family":"Fogarty","sequence":"additional","affiliation":[{"name":"Pfizer Inc.  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