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Article
Open AccessImpact of a simulation-based education approach for health sciences: demo, debrief, and do
Skill-based practice (e.g., communication skills) is important for individuals to incorporate into students' learning and can be challenging in large classes. Simulation-based education (SBE) is a method where...
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Article
Open AccessBurden and Treatment of Achondroplasia: A Systematic Literature Review
Achondroplasia is the most common form of skeletal dysplasia. Recent advances in therapeutic options have highlighted the need for understanding the burden and treatment landscape of the condition. This system...
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Article
Open AccessAssessing the value of bypassing agent therapy used prophylactic versus on-demand, during immune tolerance induction for treatment of inhibitors: a retrospective chart review
Haemophilia A is a bleeding disorder caused by deficiency of coagulation factor VIII (FVIII) which leads to severe and repeated bleedings. There is a need to understand the optimal treatment pathway for FVIII ...
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Article
Open AccessPharmacokinetic parameter driven outcomes model predicts a reduction in bleeding events associated with BAY 81–8973 versus antihemophilic factor (recombinant) plasma/albumin-free method in a Chinese healthcare setting
Long-term prophylactic therapy is considered the standard of care for hemophilia A patients. This study models the long-term clinical and cost outcomes of two factor VIII (FVIII) products using a pharmacokinet...
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Article
Open AccessHealth-related quality of life, direct medical and societal costs among children with moderate or severe haemophilia in Europe: multivariable models of the CHESS-PAEDs study
Haemophilia bears substantial humanistic and economic burden on children and their caregivers. Characterising the differential impact of severe versus moderate paediatric haemophilia is important for clinical ...
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Article
Open AccessDifferential humanistic and economic burden of mild, moderate and severe haemophilia in european adults: a regression analysis of the CHESS II study
The lifelong nature of haemophilia makes patient-centred and societal assessments of its impact important to clinical and policy decisions. Quantifying the humanistic and economic burden by severity is key to ...
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Article
Open AccessClinical, 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
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 CH...
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Article
Open AccessClinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys
Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life. The standard of care for severe hemophilia B in the Uni...
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Article
Open AccessReal-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a nested cohort analysis
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 recom...
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Article
Open AccessInhibitor clinical burden of disease: a comparative analysis of the CHESS data
Patients with hemophilia and inhibitors generally face greater disease burden compared to patients without inhibitors. While raising awareness of relative burden may improve the standard of care for patients w...
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Article
Open AccessThe impact of severe haemophilia and the presence of target joints on health-related quality-of-life
Joint damage remains a major complication associated with haemophilia and is widely accepted as one of the most debilitating symptoms for persons with severe haemophilia. The aim of this study is to describe h...
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Article
Open AccessThe relationship between target joints and direct resource use in severe haemophilia
Target joints are a common complication of severe haemophilia. While factor replacement therapy constitutes the majority of costs in haemophilia, the relationship between target joints and non drug-related dir...
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Article
Open AccessThe cost of severe haemophilia in Europe: the CHESS study
Severe haemophilia is associated with major psychological and economic burden for patients, caregivers, and the wider health care system. This burden has been quantified and documented for a number of European...
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Article
Open AccessImpact of Friedreich’s Ataxia on health-care resource utilization in the United Kingdom and Germany
Friedreich’s Ataxia (FRDA) is a neurodegenerative disorder that causes progressive damage to the central and peripheral nervous systems having a significant impact upon quality of life. With little information...
