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Although the impact on quality of life seems promising, some limitations remain, such as the presence of pre-existing anti-AAV neutralising antibodies and the occurrence of hepatotoxicity. This review follows the CSL Behring-sponsored symposium at the 27th Congress of the European Hematology Association (EHA) 2022 that examined the haemophilia gene therapy process from a 360-degree multidisciplinary perspective. Here, the faculty (haematologist, nurse and haemophilia patient) summarised their own viewpoints from the symposium, with the aim of highlighting the key considerations required to engage with gene therapy effectively, for both patients and providers, as well as the importance of multidisciplinary collaboration, including with industry.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>When considering these new therapies, patients face a complex decision-making process, which includes whether gene therapy is right for them at their current stage of life. The authors agreed that collaboration and tailored education across the multidisciplinary team (including patients and their carers\/families), starting early in the process and continuing throughout the long-term follow-up period, is key for the success of gene therapy. Additionally, patient expectations, which may surround eligibility, follow-up requirements and treatment outcomes, should be continually explored. During these ongoing discussions, transparent communication of the unknown factors, such as anticipated clotting factor levels, long-term factor expression and safety, and psychological changes, is critical. To ensure efficiency and comprehensiveness, clearly\u2011defined protocols should outline the whole process, which should include the recording and management of long-term effects.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusion<\/jats:title>\n                <jats:p>In order to engage effectively, both patients and providers should be familiar with these key considerations prior to their involvement with the haemophilia gene therapy process. The future after the approval of haemophilia gene therapies remains to be seen and real-world evidence is eagerly awaited.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1186\/s13023-024-03181-2","type":"journal-article","created":{"date-parts":[[2024,5,13]],"date-time":"2024-05-13T13:02:38Z","timestamp":1715605358000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient"],"prefix":"10.1186","volume":"19","author":[{"given":"Wolfgang","family":"Miesbach","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Greta","family":"Mulders","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daan","family":"Breederveld","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karen","family":"Pinachyan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra","family":"Le Quellec","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ingrid","family":"Pabinger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,5,13]]},"reference":[{"key":"3181_CR1","doi-asserted-by":"crossref","unstructured":"Srivastava A, Santagostino E, Dougall A, Kitchen S, Sutherland M, Pipe SW et al. 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W. Miesbach has received grant\/research support from Bayer, Biogen Idec, Biotest, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sobi, Takeda, and UniQure, and consultant fees from Biogen Idec, Biotest, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sobi, Takeda, and UniQure. G. Mulders has received grant\/research support from Sobi, consultancy fees from Novo Nordisk and Biomarin, and has received speaker fees from CSL Behring. D. Breederveld has received consultancy fees from CSL Behring and is the owner of an OHS company in Amsterdam. K. Pinachyan is a full-time employee of CSL Behring. S. Le Quellec is a full-time employee of CSL Behring. I. Pabinger has received honoraria for lectures from CSL Behring, Pfizer, Roche, Sobi, Takeda and Technoclone, and consultancy fees from Biomarin, CSL Behring, Sobi and Takeda.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"193"}}