Pemphigus vulgaris (PV) is a rare, potentially life threatening, autoimmune blistering skin disease. The International Pemphigus and Pemphigoid Foundation (IPPF) has recently developed a disease registry with the aim to enhance our understanding of autoimmune bullous diseases with the long-term goal of acquiring information to improve patient care. Patients were recruited to the IPPF disease registry through direct mail, e-mail, advertisements, and articles in the IPPF-quarterly, -website, -Facebook webpage, and IPPF Peer Health Coaches to complete a 38-question survey. We present here the initial analysis of detailed clinical information collected on 393 PV patients. We report previously unrecognized gender differences in terms of lesion location, autoimmune comorbidity, and delay in diagnosis. The IPPF disease registry serves as a useful resource and guide for future clinical investigation.
We used the Archimedes Model, a mathematical simulation model (Model) to estimate the clinical- and cost-effectiveness of using LDL particle concentration (LDL-P) as an adjunct or alternative to LDL cholesterol (LDL-C) to guide statin therapy. LDL-P by NMR has been shown to be a better measure of cardiovascular disease (CVD) risk than LDL-C, and may therefore be a better gauge of the need for and response to statin treatment. Using the Model, we conducted a virtual clinical trial comparing the use of LDL-C alone, LDL-P alone, and LDL-C and LDL-P together to guide treatment in the general adult population, and in high-risk, dyslipidemic subpopulations. In the general population, the 5-year major adverse cardiovascular event (MACE) relative risk reduction (RRR) of LDL-P alone compared to the control arm (LDL-C alone) was 5.0% (95% CI, 4.7-5.3; p < .0001); using both LDL-C and LDL-P (dual markers) led to 3.0% RRR compared to the control arm (95% CI, 2.8-3.3; p < .0001). For individuals with diabetes, the RRR was 7.3% (95% CI, 6.4-8.2; p < .0001) for LDL-P alone and 6.9% for dual markers (95% CI, 6.1-7.8; both, p < .0001). In the general population, the costs per quality-adjusted life year (QALY) associated with the use of LDL-P alone were $76,052 at 5 years and $8913 at 20 years and $142,825 at 5 years and $25,505 at 20 years with the use of both markers. In high-risk subpopulations, the use of LDL-P alone was cost-saving at 5 years; whereas the cost per QALY for the use of both markers was $14,250 at 5 years and $859 at 20 years for high-risk dyslipidemics, $19,192 at 5 years and $649 at 20 years for diabetics, and $9030 at 5 years and $7268 at 20 years for patients with prior CHD. In conclusion, the model estimates that using LDL-P to guide statin therapy may reduce the risk of CVD events to a greater extent than does the use of LDL-C alone and maybe cost-effective or cost-saving for high-risk patients.
Understanding the benefit versus risk of glycemic control and hypoglycemia is fundamental to the successful management of patients with T2DM. Our validated hypoglycemia model is an important step in addressing this issue and may be helpful to researchers, clinicians, and payers to determine the patients who are at the highest risk for hypoglycemia, whether a patient is experiencing events at 'higher-than-expected' rates, and the corresponding economic burden.
objeCtives: The aim of this study was to evaluate the long-term cost effectiveness of ticagrelor + aspirin versus clopidogrel + aspirin in patients with acute coronary syndrome (ACS) treated for 12 months in Spain. Methods: The cost effectiveness model consisted of a decision tree (1st year) based on the PLATO study and a long-term Markov model (2nd year onwards). This allowed estimation of cardiovascular events (death, myocardial infarction and non-fatal stroke), survival, health costs, and health related quality of life. A life time horizon was applied. The daily drug cost was € 0.60 and € 2.96 for generic clopidogrel and ticagrelor, respectively. Spanish unit costs and life tables were used; outcomes and costs were discounted at 3%. A sensitivity analysis across subgroups was carried out, and probabilistic sensitivity analysis was used to validate the robustness of the model. Results: Ticagrelor compared to clopidogrel was associated with a gain of 0.1586 life years and 0.1363 years of quality-adjusted life years (QALY), with an incremental cost of € 596. The incremental cost per life year and per QALY gained was € 3,760 and € 4,374, respectively. The probabilistic sensitivity analysis showed that ticagrelor was costeffective versus clopidogrel in > 99 % of the simulations given a willingness-to-pay threshold of € 15,000/QALY. The results were consistent across different subgroups of ACS patients. ConClusions: Ticagrelor + aspirin for 12 months is a cost effective treatment compared to generic clopidogrel + aspirin in patients with ACS treated invasively or conservatively, based on the findings of the PLATO study and Spanish health care costs.objeCtives: This study was conducted to determine the cost-effectiveness of carotid Artery stenting (CAS) versus carotid endarterectomy (CEA) in patients with symptomatic carotid stenosis (more than 50% stenosis) in Korean health care system perspective. Methods: We performed a cost-utility analysis. Costs were estimated from retrospective chart review (CAS= 346, CEA= 331), health insurance claims data, and other national resources and expressed in 2013 KRW. Transition probabilities were estimated from retrospective chart and systematic review. Health utility index was assessed for general population using Time Trade Off (TTO) with health scenario. We used a Markov model to project 15-year costs and quality-adjusted life years (QALYs) for the 2 treatment groups. Results: In the base case analysis, CAS produced 6.49 QALYs, compared with 6.71 QALYs for CEA. The incremental cost of stenting was 1,691,740 KRW. In the base case analysis, CEA for patients with symptomatic stenosis had a greater benefit than CAS, with lower costs. In subgroup for patients with stenosis more than 70% or patient with over 80 years old, CAS was cost-effective. Sensitivity analyses showed that the major stroke or mortality influenced the results. However the results were consistent with the base analysis. ConClusions: Under the current circumstances in Korea, CEA was dominated by CEA in symptomatic ...
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