- Research Article
- 10.1016/j.jval.2025.09.3393
MSR108 Feasibility Assessment of an Indirect Treatment Comparison of Seladelpar vs. Elafibranor in Patients With Primary Biliary Cholangitis
- Dec 01, 2025
- Value in Health
- David E Jones + 13 more +13
Publications from 2021 to 2026
Showing 10 of 13 papers
MSR108 Feasibility Assessment of an Indirect Treatment Comparison of Seladelpar vs. Elafibranor in Patients With Primary Biliary Cholangitis
CO170 Exploring the Relationship Between Surrogate Endpoints and Clinical Outcomes in Primary Biliary Cholangitis: A Systematic Literature Review and Meta-Analysis
Effects of switching from a metered dose inhaler to a dry powder inhaler on climate emissions and asthma control: post-hoc analysis
ObjectiveTo compare the effects of switching from a pressurised metered dose inhaler (pMDI)-based to a dry powder inhaler (DPI)-based maintenance therapy versus continued usual care on greenhouse gas emissions (carbon dioxide equivalents, CO2e) and asthma control.MethodsThis post-hoc analysis was based on a subset of 2236 (53%) patients from the Salford Lung Study in Asthma who at baseline were using a pMDI-based controller therapy. During the study patients were randomised to fluticasone furoate/vilanterol (FF/VI) via the ELLIPTA DPI (switched from pMDI to DPI) (n=1081) or continued their usual care treatment (n=1155), and were managed in conditions close to everyday clinical practice. Annual CO2e (kg) was calculated for the total number of maintenance and rescue inhalers prescribed. Asthma control was assessed by the proportion of ACT responders (composite of ACT total score ≥20 and/or increase from baseline ≥3).ResultsThe groups were well matched for demographic characteristics and baseline Asthma Control Test (ACT) total score (mean age: 49 years; mean ACT score: usual care, 16.6; FF/VI, 16.5). Annual CO2e kg per patient (maintenance plus rescue therapy) was significantly lower with FF/VI DPI treatment (‘switch’ group) than usual care (least squares geometric mean 108 kg (95% CI 102 to 114) vs 240 kg (95% CI 229 to 252), p<0.001). Asthma control was consistently superior over the 12 months in the FF/VI DPI group compared with usual care.ConclusionsPatients switching from a pMDI-based to a DPI-based maintenance therapy more than halved their inhaler carbon footprint without loss of asthma control. The remaining inhaler carbon footprint could be reduced through switches from pMDI to DPI rescue medications or alternative lower-carbon footprint rescue inhalers if available. Asthma control improved in both groups, with greater control demonstrated in those initiated on FF/VI DPI.Trial registration numberNCT01706198.
Read moreRevue systématique des études en vie réelle d’efficacité et de tolérance de mépolizumab dans le traitement de l’asthme sévère éosinophilique
Teaching Horse Riding: Is the Role of the Horse Recognized?
Abstract This study investigated what methods professional trainers use in their teaching. Interviews with nine professional trainers in sports dressage were analyzed using a qualitative approach. The results showed that trainers had developed a “good eye” using their prior experiences. The trainers helped the riders solve their problems but did not make clear their observations or reasoning for specific instructions. Thus, trainers could further develop the riders’ own practical knowledge. The role of the horse was not made explicit in most cases, showing that recognition of the horse as a sentient and thinking individual is not built into the current teaching practice. Teaching methods would be improved if trainers encouraged riders to pay more attention to the perspective of the horse, to examine how to interpret and attend to the horse’s reactions, and to reflect on these issues in dialog with them.
Read moreIntrepid: Clinical Effectiveness of Once-Daily Single-Inhaler Fluticasone Furoate/Umeclidinium/Vilanterol Versus Multiple-Inhaler Triple Therapy in Usual Clinical Practice
Results from the Survey of Antibiotic Resistance (SOAR) 2011-14 in the Democratic Republic of Congo, Ivory Coast, Republic of Senegal and Kenya.
To assess antibiotic susceptibility of community-acquired respiratory tract isolates from Ivory Coast, Kenya, Democratic Republic of Congo (DRC) and Senegal in 2011-14. Bacterial isolates were collected and MICs determined using Etest(®) for all antibiotics except erythromycin, for which testing was by disc diffusion. Susceptibility was assessed using CLSI, EUCAST and pharmacokinetic/pharmacodynamic (PK/PD) breakpoints. For macrolide interpretation, CLSI breakpoints were adjusted for incubation in CO2. Susceptibility to penicillin (using CLSI oral or EUCAST breakpoints) was low among isolates of Streptococcus pneumoniae from the DRC and Kenya (17.4% and 19%, respectively) but higher among isolates from the Ivory Coast (70%) and Senegal (85.7%). Penicillin susceptibility using CLSI iv breakpoints was higher in all countries, but still only 69.6% in the DRC. Macrolide susceptibility (based on CLSI erythromycin disc diffusion breakpoints) was also low in Kenya (∼65%) but 87%-100% elsewhere. Haemophilus influenzae were only collected in the DRC and Senegal, with β-lactamase prevalence of 39% and 4%, respectively. Furthermore, β-lactamase-negative ampicillin-resistant (BLNAR) isolates were found in DRC (four isolates, 17%), but only two isolates were found in Senegal (by EUCAST definition). Amoxicillin/clavulanic acid in vitro susceptibility was 73.9% in the DRC and 100% in Senegal based on CLSI breakpoints, but this reduced to 65.2% in the DRC when BLNAR rates were considered. Clarithromycin susceptibility was >95% in both countries. There was considerable variability in antibiotic susceptibility among the African countries participating in the surveillance programme. Thus, continued surveillance is necessary to track future changes in antibiotic resistance. Use of EUCAST versus CLSI breakpoints showed profound differences for cefaclor and ofloxacin against S. pneumoniae, with EUCAST showing lower susceptibility.
Read moreThe burden of Duchenne muscular dystrophy
Objective:The objective of this study was to estimate the total cost of illness and economic burden of Duchenne muscular dystrophy (DMD).Methods:Patients with DMD from Germany, Italy, United Kingdom, and United States were identified through Translational Research in Europe–Assessment & Treatment of Neuromuscular Diseases registries and invited to complete a questionnaire online together with a caregiver. Data on health care use, quality of life, work status, informal care, and household expenses were collected to estimate costs of DMD from the perspective of society and caregiver households.Results:A total of 770 patients (173 German, 122 Italian, 191 from the United Kingdom, and 284 from the United States) completed the questionnaire. Mean per-patient annual direct cost of illness was estimated at between $23,920 and $54,270 (2012 international dollars), 7 to 16 times higher than the mean per-capita health expenditure in these countries. Indirect and informal care costs were substantial, each constituting between 18% and 43% of total costs. The total societal burden was estimated at between $80,120 and $120,910 per patient and annum, and increased markedly with disease progression. The corresponding household burden was estimated at between $58,440 and $71,900.Conclusions:We show that DMD is associated with a substantial economic burden. Our results underscore the many different costs accompanying a rare condition such as DMD and the considerable economic burden carried by affected families. Our description of the previously unknown economic context of a rare disease serves as important intelligence input to health policy evaluations of intervention programs and novel therapies, financial support schemes for patients and their families, and the design of future cost studies.
Read moreThe Treatment of Pediatric Supracondylar Humerus Fractures
Based on the best current evidence and a systematic review of published studies, 14 recommendations have been created to guide clinical practice and management of supracondylar fractures of the humerus in children. Two each of these recommendations are graded Weak and Consensus; eight are graded Inconclusive. The two Moderate recommendations include nonsurgical immobilization for acute or nondisplaced fractures of the humerus or posterior fat pad sign, and closed reduction with pin fixation for displaced type II and III and displaced flexion fractures.
Read moreAcute kidney injury in patients with acute lung injury: Impact of fluid accumulation on classification of acute kidney injury and associated outcomes*
It has been suggested that fluid accumulation may delay recognition of acute kidney injury. We sought to determine the impact of fluid balance on the incidence of nondialysis requiring acute kidney injury in patients with acute lung injury and to describe associated outcomes, including mortality. Analysis of the Fluid and Catheter Treatment Trial, a factorial randomized clinical trial of conservative vs. liberal fluid management and of management guided by a central venous vs. pulmonary artery catheter. Acute Respiratory Distress Syndrome Network hospitals. One thousand patients. None. The incidence of acute kidney injury, defined as an absolute rise in creatinine of ≥0.3 mg/dL or a relative change of >50% over 48 hrs, was examined before and after adjustment of serum creatinine for fluid balance. The incidence of acute kidney injury before adjustment for fluid balance was greater in those managed with the conservative fluid protocol (57% vs. 51%, p = .04). After adjustment for fluid balance, the incidence of acute kidney injury was greater in those managed with the liberal fluid protocol (66% vs. 58%, p = .007). Patients who met acute kidney injury criteria after adjustment of creatinine for fluid balance (but not before) had a mortality rate that was significantly greater than those who did not meet acute kidney injury criteria both before and after adjustment for fluid balance (31% vs. 12%, p < .001) and those who had acute kidney injury before but not after adjustment for fluid balance (31% vs. 11%, p = .005). The mortality of those patients meeting acute kidney injury criteria after but not before adjustment for fluid balance was similar to patients with acute kidney injury both before and after adjustment for fluid balance (31% vs. 38%, p = .18). Fluid management influences serum creatinine and therefore the diagnosis of acute kidney injury using creatinine-based definitions. Patients with "unrecognized" acute kidney injury that is identified after adjusting for positive fluid balance have higher mortality rates, and patients who have acute kidney injury before but not after adjusting for fluid balance have lower mortality rates. Future studies of acute kidney injury should consider potential differences in serum creatinine caused by changes in fluid balance and the impact of these differences on diagnosis and prognosis.
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