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Evolution of haemophilia integrated care in the era of gene therapy: Treatment centre’s readiness in United States and EU
dc.contributor.authorMiesbach, Wolfgang
dc.contributor.authorPasi, K. John
dc.contributor.authorPipe, Steven W.
dc.contributor.authorHermans, Cedric
dc.contributor.authorO’Mahony, Brian
dc.contributor.authorGuelcher, Christine
dc.contributor.authorSteiner, Bruno
dc.contributor.authorSkinner, Mark W.
dc.date.accessioned2021-08-03T18:14:21Z
dc.date.available2022-08-03 14:14:20en
dc.date.available2021-08-03T18:14:21Z
dc.date.issued2021-07
dc.identifier.citationMiesbach, Wolfgang; Pasi, K. John; Pipe, Steven W.; Hermans, Cedric; O’Mahony, Brian; Guelcher, Christine; Steiner, Bruno; Skinner, Mark W. (2021). "Evolution of haemophilia integrated care in the era of gene therapy: Treatment centre’s readiness in United States and EU." Haemophilia (4): 511-514.
dc.identifier.issn1351-8216
dc.identifier.issn1365-2516
dc.identifier.urihttps://hdl.handle.net/2027.42/168442
dc.publisherWiley Periodicals, Inc.
dc.subject.othergene therapy
dc.subject.otherhaemophilia care
dc.subject.otherhaemophilia treatment
dc.subject.othertreatment centres
dc.titleEvolution of haemophilia integrated care in the era of gene therapy: Treatment centre’s readiness in United States and EU
dc.typeArticle
dc.rights.robotsIndexNoFollow
dc.subject.hlbsecondlevelOncology and Hematology
dc.subject.hlbtoplevelHealth Sciences
dc.description.peerreviewedPeer Reviewed
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/168442/1/hae14309.pdf
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/168442/2/hae14309_am.pdf
dc.identifier.doi10.1111/hae.14309
dc.identifier.sourceHaemophilia
dc.identifier.citedreferenceIorio A, Skinner MW, Clearfield E, et al. Core outcome set for gene therapy in haemophilia: results of the coreHEM multistakeholder project. Haemophilia. 2018; 24 ( 4 ): e167 ‐ e172.
dc.identifier.citedreferenceMalouin RA, McKernan L, Forsberg A, et al. Impact of the 340B pharmacy program on services and supports for persons served by haemophilia treatment centers in the United States. Matern Child Health J. 2018; 22 ( 9 ): 1240 ‐ 1246.
dc.identifier.citedreferenceSrivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of haemophilia. Haemophilia. 2020; 26 ( Suppl 6 ): 1 ‐ 158.
dc.identifier.citedreferencePai M, Key NS, Skinner M, et al. NHF‐McMaster guideline on care models for haemophilia management. Haemophilia. 2016; 22 ( Suppl 3 ): 6 ‐ 16.
dc.identifier.citedreferenceColvin BT, Astermark J, Fischer K, et al. European principles of haemophilia care. Haemophilia. 2008; 14 ( 2 ): 361 ‐ 374.
dc.identifier.citedreferenceNoone D, O’Mahony B, Peyvandi F, et al. Evolution of haemophilia care in Europe: 10 years of the principles of care. Orphanet J Rare Dis. 2020; 15 ( 1 ): 184.
dc.identifier.citedreferenceYeung CH, Santesso N, Pai M, et al. Care models in the management of haemophilia: a systematic review. Haemophilia. 2016; 22 ( Suppl 3 ): 31 ‐ 40.
dc.identifier.citedreferencePierce GF, Pasi KJ, Coffin D, et al. Towards a global multidisciplinary consensus framework on haemophilia gene therapy: report of the 2nd World Federation of Haemophilia Gene Therapy Round Table. Haemophilia. 2020; 26 ( 3 ): 443 ‐ 449.
dc.identifier.citedreferenceMiesbach W, O’Mahony B, Key NS, et al. How to discuss gene therapy for haemophilia? A patient and physician perspective. Haemophilia. 2019; 25 ( 4 ): 545 ‐ 557.
dc.identifier.citedreferenceKonkle BA, Coffin D, Pierce GF, et al. World Federation of Haemophilia Gene Therapy Registry. Haemophilia. 2020; 26 ( 4 ): 563 ‐ 564.
dc.identifier.citedreferenceEAHAD‐EHC Joint Statement on Promoting hub‐and‐spoke model for the treatment of haemophilia and rare bleeding disorders using gene therapies. May 2020. Available at https://eahad.org/wp‐content/uploads/2020/05/Hub‐and‐Spoke.pdf. Accessed November 2020.
dc.identifier.citedreferenceKonkle BA, Pierce G, Coffin D, et al. Core data set on safety, efficacy, and durability of haemophilia gene therapy for a global registry: Communication from the SSC of the ISTH. J Thromb Haemost. 2020; 18: 3074 ‐ 3077.
dc.identifier.citedreferenceKonkle BA, Recht M, Hilger A, et al. The critical need for postmarketing surveillance in gene therapy for haemophilia. Haemophilia. 2020; 00: 1 ‐ 6.
dc.working.doiNOen
dc.owningcollnameInterdisciplinary and Peer-Reviewed


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