CO29 Real-World Benefit of Ataluren Treatment in Milestones Not Related to Ambulatory Function in Nonsense Duchenne Muscular Dystrophy Versus Standard of Care
To present the most recent data on real-world ataluren benefits on upper limb and pulmonary function, compared to standard of care (SoC). (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: D. Machado, E. Paula, I.D.L. Bretas, A.L.M. Freitas, A.P.C. dos Santos Source Type: research
CO30 Disease Burden and Efficacy of Current Treatment for Non-Neuronopathic MPS II Patients: A Systematic Literature Review
Mucopolysaccharidosis II (MPSII, Hunter syndrome) is an ultra-rare genetic disease (incidence rate: 0.38 to 1.09/100,000 live male births) with manifestations caused by mutations in the iduronate-2-sulfatase gene, leading to impaired enzymatic function and accumulation of glycosaminoglycans in multiple systems and organs. The non-neuronopathic phenotype (nnMPSII) is characterized by delayed recognition and absent or milder CNS manifestations. Although enzyme replacement therapy (ERT) for MPSII was approved in 2006, ERT is unable to access the CNS, and characterization of unmet need has predominantly focused on the severe p...
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: E. Schmidt, A.D. Ocampo, G.A. DeOcampo, N.J. Engmann Source Type: research
CO31 Progressive Pulmonary Fibrosis: A Modelling Analysis of Long-Term Progression Based on Inbuild
The objective of our analysis was to model disease progression or death over the lifetime of PPF patients. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: C. Raspin, A. Ramon, M. Baldwin, A. Diamantopoulos, C. Valenzuela, V. Cottin Source Type: research
CO32 Validation of a Scoring Algorithm for the Clinician-Reported Outcome ”Prurigo Activity and Severity (PAS)’’ Tool: Results Based on Clinical Studies of Dupilumab in Adults with Prurigo Nodularis
This study validated the psychometric properties of a PAS score (range: 0 to 11) derived as the sum of the items assessing the estimated number of pruriginous lesions, percentage of pruriginous lesions with excoriations/crusts, and percentage of healed pruriginous lesions and estimated the within-patient improvement threshold of PAS score in PN patients participating in the dupilumab clinical trials. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: C. Zeidler, S. Stander, S. Rhoten, S. Wratten, D. Zhang, J. Msihid, E. Brookes, J. O ’Malley, A. Bansal, S. Wiggins, J. Zahn, R.B. Thomas, D. Bahloul Source Type: research
CO34 Mortality and Clinical Complications Among Patients with Transfusion-Dependent Beta-Thalassemia in France
This study describes the mortality and clinical complications in patients with TDT in France. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: J. Baldwin, C. Udeze, N. Li, L. Boulmerka, L. Dahal, G. Pesce, N. Quignot, H. Jiang, F. Galacteros Source Type: research
CO35 Clinical Outcomes Associated with Sodium Zirconium Cyclosilicate for Treating Hyperkalaemia in Patients Receiving Haemodialysis
This study assessed the projected impact of SZC versus placebo on clinical outcomes including kidney transplants in individuals on haemodialysis in Italy. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: P.M. Ferraro, D. Booth, J. Silvanto, P. McEwan, N. Rao Source Type: research
CO36 Comparative Analysis of Coding Schemas for Assessing Major Bleeding Risk Between Non-Vitamin K Antagonist Oral Anticoagulants and Warfarin
This study aimed to investigate the impact of utilizing different diagnosis coding schemas on evaluating the major bleeding risk associated with non-vitamin K antagonist oral anticoagulants (NOACs) and warfarin. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: H.T. Chien, T.F. Chao, R. Wang, S.Y. Lin, F.J. Lin Source Type: research
CO37 Inventory-Facilitated Goal Setting Interview Conducted by Disease Experts Yields a Broader Range of Goal Areas
Goal attainment scaling (GAS) quantifies the impact of an intervention on personal goals. GAS interviewers work with patients/caregivers to set goals that are meaningful, specific, attainable, and relevant. Although interviewer expertise in the condition and use of a goal inventory are thought to improve goal setting, their impact has not been systematically investigated. Here we assessed the impacts of interviewer expertise and the use of a goal inventory on the number of goals, goal type, time to set goals, and goal quality in individuals with dyslexia. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: K. Cheema, C. St. Jean, J. Stanley, J. Cummine, W. Hodgetts, C. Chapman, K. Rockwood, S.E. Howlett, G. Sevinc Source Type: research
CO38 Real-World Comparative COVID-19 Vaccine Effectiveness of a Third Dose of mRNA-1273 Versus BNT162b2 Among Immunocompromised Adults in the US
The objective of this study was to compare the real-world effectiveness of the third dose of mRNA-1273 versus BNT162b2 in the US. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: E. Beck, T. Sun, L. Li, M. Georgieva, N. Van de Velde Source Type: research
CO40 Non-Communicable Diseases Prevention in India: A Review of Suitable Instruments for Surveillance and a General Overview of Cultural, Behavioral Practices and Risk Factors for the Adult Population
ICMR estimates that the proportion of deaths due to Non-Communicable Diseases (NCDs) in India has increased from 37.9% in 1990 to 61.8% in 2016. This paper will try to find outcome assessments to assess the severity of non-communicable NCD ’s in India and specific cultural practices. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: K. Jarodia, K. Rudell Source Type: research
CO41 Long-Term Clinical Outcomes in People with Heart Failure with Preserved Ejection Fraction (HFpEF) Treated with Standard Care
To model clinical outcomes in a cohort of patients with symptomatic HFpEF over a lifetime horizon. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: L. Sun, V. Dayer, R.N. Hansen, Y. Du, T. Williamson, S.X. Kong, P. Mernagh, R. Singh, K. Folkerts, S. Allen, S. Sullivan Source Type: research
CO42 4P Care and Adjusting Therapy for Hypothyroidism – A Potential to Reduce Costs of Healthcare and Loss of Productivity
Hypothyroidism affects 25 million people in the US, causing health complications and increased healthcare utilization for many patients despite well-established hormone replacement therapy. A significant effort goes into understanding how personalized care for chronic patients can improve health outcomes, and reduce associated healthcare costs. We focused on two complications frequently affecting hypothyroid patients: (I) fatigue – known to increase workplace absenteeism and the economic burden on healthcare payers; (II) excessive body weight – known to increase the risk of health complications and total cost of care. ...
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: V. H ögqvist Tabor, M. Tarasova, G. Cohen-Skalli, F. Dubois, B. Curran, B. Candea, M. Murphy Source Type: research
CO43 Projecting Cardiovascular Benefits Associated With Tirzepatide's Effects on Cardiovascular Risk Factors in Patients With Type 2 Diabetes in France: A Simulation Study
Tirzepatide is a GIP/GLP-1 receptor agonist approved for the treatment of type 2 diabetes (T2D) that is associated with superior reductions in glycated hemoglobin and body weight versus a range of existing therapies. The present analysis aimed to model the benefits on cardiovascular (CV) outcomes associated with tirzepatide from demonstrated improvements in CV risk factors (RF) versus no change in RF, with a focus on major adverse cardiovascular events (MACE) as the primary outcome. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: B. Osumili, N. Debackere, L. Ludwig, H. Sharland Source Type: research
CO45 A Systematic Review of Quality of Life and Gastroesophageal Reflux Disease
Gastroesophageal reflux disease (GERD) is a common condition associated with functional impairment and loss of productivity. Management and the patient perspective may be better understood when considering the quality of life patients experience. The purpose of this study was to perform a systematic review involving GERD and quality of life parameters. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: M. Goldsmith, S. Das, J. Hoffsten Source Type: research
CO48 A Systematic Review of Effectiveness and Safety of Acupuncture on Suicidal Behavior
In situations where death by suicide is a major global issue and effective prevention and management approaches are lacking, acupuncture improves some risk factors for suicide, including depression, substance abuse, and post-traumatic stress disorder and has been used for a long time in clinical settings. This review aimed to investigate the effectiveness and safety of acupuncture for suicidal behaviors. (Source: Value in Health)
Source: Value in Health - December 1, 2023 Category: International Medicine & Public Health Authors: C.Y. Kwon Source Type: research
