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dc.contributor.authorO’Hara, Jamie
dc.contributor.authorHirst, Ceri; orcid: 0000-0001-9094-2437; email: ceri.hirst@bayer.com
dc.contributor.authorCabre Marquez, Jose Francisco
dc.contributor.authorBurke, Tom
dc.date.accessioned2021-01-15T16:14:22Z
dc.date.available2021-01-15T16:14:22Z
dc.date.issued2021-01-15
dc.date.submitted2020-09-11
dc.identifierhttps://chesterrep.openrepository.com/bitstream/handle/10034/624186/13023_2021_Article_1676_nlm.xml?sequence=2
dc.identifierhttps://chesterrep.openrepository.com/bitstream/handle/10034/624186/13023_2021_Article_1676.pdf?sequence=3
dc.identifier.citationOrphanet Journal of Rare Diseases, volume 16, issue 1, page 33
dc.identifier.urihttp://hdl.handle.net/10034/624186
dc.descriptionFrom Springer Nature via Jisc Publications Router
dc.descriptionHistory: received 2020-09-11, accepted 2021-01-05, registration 2021-01-05, online 2021-01-15, pub-electronic 2021-01-15, collection 2021-12
dc.descriptionPublication status: Published
dc.descriptionFunder: Bayer; doi: http://dx.doi.org/10.13039/100004326
dc.description.abstractAbstract: Background: Untreated hemophilia A patients may experience recurrent bleeding events leading to debilitating joint damages. While RCT and pharmacokinetic data support the value of Kovaltry [an unmodified full-length recombinant factor VIII (FVIII) product], real world evidence in children is lacking. This report describes a descriptive and multivariate analysis of the effectiveness of Kovaltry in children with hemophilia A in the real-world setting, using data from medical chart abstraction and cross-sectional surveys of physicians, patients, and caregivers. Results: Male patients aged < 18 years with moderate or severe hemophilia A, residing in five European countries and treated with FVIII were studied. The co-primary endpoints were the annualized bleeding rate (ABR) and the annual FVIII utilization rate. Twenty nine patients treated with Kovaltry were included, of whom 93% had severe disease and 75% were on continuous prophylactic treatment. The mean ABR was 2.66 ± 2.06, with rates decreasing with age. The children received on average 2.45 infusions per week, consistent across age groups (median 3; range 1–3). There were no reports of inhibitor development or adverse events in the study (AEs), and all patients were satisfied or very satisfied with the treatment. An exploratory multivariate analysis suggests no significant difference in ABR or units utilized between Kovaltry and some extended half life products in children with severe hemophilia A, though characteristics of these patient cohorts were markedly different. Conclusion: This analysis demonstrates the effectiveness and safety of Kovaltry in a pan-European pediatric population with severe hemophilia A.
dc.languageen
dc.publisherBioMed Central
dc.rightsLicence for this article: http://creativecommons.org/licenses/by/4.0/
dc.sourceeissn: 1750-1172
dc.subjectLetter to the Editor
dc.subjectHaematology
dc.subjectHemophilia A
dc.subjectKovaltry
dc.subjectReal world evidence
dc.subjectFactor utilization
dc.subjectClinical burden
dc.titleReal-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis
dc.typeother
dc.date.updated2021-01-15T16:14:21Z
dc.date.accepted2021-01-05


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