Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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To determine US societal burden of illness, including direct and indirect costs and annual bleed rate (ABR), for persons with hemophilia B (HB), a rare and debilitating genetic disorder, and to examine associations of hemophilia severity and treatment regimens with costs and ABR. ⋯ HB is associated with high economic burden, primarily because of clotting factor costs. Nevertheless, prophylaxis treatment leads to clinical benefits and may reduce other nonfactor costs.
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Multicenter Study Comparative Study
Goal-Directed Fluid Therapy Guided by Cardiac Monitoring During High-Risk Abdominal Surgery in Adult Patients: Cost-Effectiveness Analysis of Esophageal Doppler and Arterial Pulse Pressure Waveform Analysis.
Several minimally invasive techniques for cardiac output monitoring such as the esophageal Doppler (ED) and arterial pulse pressure waveform analysis (APPWA) have been shown to improve surgical outcomes compared with conventional clinical assessment (CCA). ⋯ Cardiac output monitoring during high-risk abdominal surgery is cost-effective and is associated with a reduced rate of hospital mortality and major complications, whatever the device used. The two devices evaluated had negligible costs compared with the observed reduction in hospital costs. Our comparative studies suggest a larger effect with APPWA that needs to be confirmed by further studies.
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Multicenter Study Comparative Study
Modeling Health State Utility Values in Ankylosing Spondylitis: Comparisons of Direct and Indirect Methods.
Cost-effectiveness analyses of technologies for patients with ankylosing spondylitis frequently require estimates of health utilities as a function of the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) and the Bath Ankylosing Spondylitis Functional Index (BASFI). ⋯ These results add to a body of evidence that the statistical model used to estimate health utilities matters. Linear models are not appropriate. The four-class bespoke mixture model approach provides the best performing method to estimate the EuroQol five-dimensional questionnaire values from BASDAI and BASFI.
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Multicenter Study Observational Study
Impact of acute bleeding on daily activities of patients with congenital hemophilia with inhibitors and their caregivers and families: observations from the Dosing Observational Study in Hemophilia (DOSE).
There is limited understanding of the effects of bleeding episodes on the daily lives of patients with congenital hemophilia with inhibitors and their caregivers. This analysis of the Dosing Observational Study in Hemophilia examined the impact of acute bleeding episodes on work, school, and family activities. ⋯ This study demonstrated that in hemophilia with inhibitors, bleeding episodes interfere with the daily activities of patients and their caregivers. Furthermore, documenting only lost days underestimated the impact of bleeding episodes because of the high percentage of days without planned work or school.
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Multicenter Study
Development of a symptom index for patients with primary brain tumors.
This study's primary goals included identifying the highest priority symptoms of patients with advanced brain tumors on treatment, comparing patient priority ratings with those of oncology experts, and constructing a brief symptom index using combined input to assess these symptoms and concerns. ⋯ The NFBrSI-24, an index of the symptoms in advanced brain tumors perceived as most important by both patients and clinicians, improves upon existing measures of brain tumor symptoms through better satisfaction of regulatory requirements for measure development. The findings suggest good reliability and validity, indicating that the NFBrSI-24 is a promising brief assessment of high-priority advanced brain tumor symptoms for research and clinical settings.