Show simple item record

dc.contributor.authorBurke, Tom
dc.contributor.authorAsghar, Sohaib
dc.contributor.authorO’Hara, Jamie
dc.contributor.authorChuang, Margaret
dc.contributor.authorSawyer, Eileen K.
dc.contributor.authorLi, Nanxin; email: n.li@uniqure.com
dc.date.accessioned2021-12-20T16:32:24Z
dc.date.available2021-12-20T16:32:24Z
dc.date.issued2021-12-20
dc.date.submitted2021-04-30
dc.identifierhttps://chesterrep.openrepository.com/bitstream/handle/10034/626586/13023_2021_Article_2152.pdf?sequence=3
dc.identifier.citationOrphanet Journal of Rare Diseases, volume 16, issue 1, page 521
dc.identifier.urihttp://hdl.handle.net/10034/626586
dc.descriptionFrom Springer Nature via Jisc Publications Router
dc.descriptionHistory: received 2021-04-30, collection 2021-12, accepted 2021-12-06, registration 2021-12-07, pub-electronic 2021-12-20, online 2021-12-20
dc.descriptionPublication status: Published
dc.descriptionFunder: uniQure Inc
dc.description.abstractAbstract: Background: Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited. We analysed data from the recent CHESS II study to better understand the clinical, humanistic, and economic burden of severe haemophilia B in Europe. Data from male adults with severe haemophilia B receiving prophylaxis were analysed from the retrospective cross-sectional CHESS II study conducted in Germany, France, Italy, Spain and the United Kingdom. Inhibitors were exclusionary. Patients and physicians completed questionnaires on bleeding, joint status, quality of life, and haemophilia-related direct and indirect costs (2019–2020). All outcomes were summarised using descriptive statistics. Results: A total of 75 CHESS II patients were eligible and included; 40 patients (53%) provided self-reported outcomes. Mean age was 36.2 years. Approximately half the patients were receiving EHL versus standard half-life (SHL) prophylaxis (44% vs 56%). Most patients reported mild or moderate chronic pain (76%) and had ≥ 2 bleeding events per year (70%), with a mean annualised bleed rate of 2.4. Mean annual total haemophilia-related direct medical cost per patient was €235,723, driven by FIX costs (€232,328 overall, n = 40; €186,528 for SHL, €290,620 for EHL). Mean annual indirect costs (€8,973) were driven by early retirement or work stoppage due to haemophilia. Mean quality of life (EQ-5D) score was 0.67. Conclusions: These data document a substantial, persistent real-world burden of severe haemophilia B in Europe. Unmet needs persist for these patients, their caregivers, and society.
dc.languageen
dc.publisherBioMed Central
dc.rightsLicence for this article: http://creativecommons.org/licenses/by/4.0/
dc.sourceeissn: 1750-1172
dc.subjectResearch
dc.subjectHaematology
dc.subjectHaemophilia B
dc.subjectFactor IX
dc.subjectBurden
dc.subjectCost
dc.subjectBleeds
dc.subjectHealth-related quality of life
dc.subjectPatient-reported outcomes
dc.titleClinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe
dc.typearticle
dc.date.updated2021-12-20T16:32:24Z
dc.date.accepted2021-12-06


Files in this item

Thumbnail
Name:
13023_2021_Article_2152.pdf
Size:
896.9Kb
Format:
PDF

This item appears in the following Collection(s)

Show simple item record