scholarly journals Projecting the impact of triple CFTR modulator therapy on intravenous antibiotic requirements in cystic fibrosis using patient registry data combined with treatment effects from randomised trials

Thorax ◽  
2021 ◽  
pp. thoraxjnl-2020-216265
Author(s):  
Ruth H Keogh ◽  
Rebecca Cosgriff ◽  
Eleni-Rosalina Andrinopoulou ◽  
Keith G Brownlee ◽  
Siobhán B Carr ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Prediction Models ◽  
Population Level ◽  
Registry Data ◽  
Standards Of Care ◽  
Exacerbation Rate ◽  
Population Total ◽  
Potential Health ◽  
Healthcare Needs ◽  
The Impact

BackgroundCystic fibrosis (CF) is a life-threatening genetic disease, affecting around 10 500 people in the UK. Precision medicines have been developed to treat specific CF-gene mutations. The newest, elexacaftor/tezacaftor/ivacaftor (ELEX/TEZ/IVA), has been found to be highly effective in randomised controlled trials (RCTs) and became available to a large proportion of UK CF patients in 2020. Understanding the potential health economic impacts of ELEX/TEZ/IVA is vital to planning service provision.MethodsWe combined observational UK CF Registry data with RCT results to project the impact of ELEX/TEZ/IVA on total days of intravenous (IV) antibiotic treatment at a population level. Registry data from 2015 to 2017 were used to develop prediction models for IV days over a 1-year period using several predictors, and to estimate 1-year population total IV days based on standards of care pre-ELEX/TEZ/IVA. We considered two approaches to imposing the impact of ELEX/TEZ/IVA on projected outcomes using effect estimates from RCTs: approach 1 based on effect estimates on FEV1% and approach 2 based on effect estimates on exacerbation rate.ResultsELEX/TEZ/IVA is expected to result in significant reductions in population-level requirements for IV antibiotics of 16.1% (~17 800 days) using approach 1 and 43.6% (~39 500 days) using approach 2. The two approaches require different assumptions. Increased understanding of the mechanisms through which ELEX/TEZ/IVA acts on these outcomes would enable further refinements to our projections.ConclusionsThis work contributes to increased understanding of the changing healthcare needs of people with CF and illustrates how Registry data can be used in combination with RCT evidence to estimate population-level treatment impacts.

scholarly journals Development and external validation of 1- and 2-year mortality prediction models in cystic fibrosis

2019 ◽  
Vol 54 (3) ◽  
pp. 1900224 ◽  
Author(s):  
Sanja Stanojevic ◽  
Jenna Sykes ◽  
Anne L. Stephenson ◽  
Shawn D. Aaron ◽  
George A. Whitmore
Keyword(s):  
Cystic Fibrosis ◽  
Health Status ◽  
Prediction Models ◽  
Risk Model ◽  
External Validation ◽  
Registry Data ◽  
Receiver Operating Curve ◽  
Clinical Tool ◽  
Risk Of Death ◽  
The Uk

IntroductionWe aimed to develop a clinical tool for predicting 1- and 2-year risk of death for patients with cystic fibrosis (CF). The model considers patients' overall health status as well as risk of intermittent shock events in calculating the risk of death.MethodsCanadian CF Registry data from 1982 to 2015 were used to develop a predictive risk model using threshold regression. A 2-year risk of death estimated conditional probability of surviving the second year given survival for the first year. UK CF Registry data from 2007 to 2013 were used to externally validate the model.ResultsThe combined effect of CF chronic health status and CF intermittent shock risk provided a simple clinical scoring tool for assessing 1-year and 2-year risk of death for an individual CF patient. At a threshold risk of death of ≥20%, the 1-year model had a sensitivity of 74% and specificity of 96%. The area under the receiver operating curve (AUC) for the 2-year mortality model was significantly greater than the AUC for a model that predicted survival based on forced expiratory volume in 1 s <30% predicted (AUC 0.95 versus 0.68 respectively, p<0.001). The Canadian-derived model validated well with the UK data and correctly identified 79% of deaths and 95% of survivors in a single year in the UK.ConclusionsThe prediction models provide an accurate risk of death over a 1- and 2-year time horizon. The models performed equally well when validated in an independent UK CF population.

Use of Nutrition Information and Understanding of “Percent Daily Value” on Nutrition Facts Tables: Evaluating the Impact of a National Public Education Campaign among Youth and Young Adults in Canada

2019 ◽  
Vol 80 (4) ◽  
pp. 200-204 ◽  
Author(s):  
Brittany Cormier ◽  
Lana Vanderlee ◽  
David Hammond
Keyword(s):  
Population Level ◽  
Correct Recall ◽  
Nutrition Information ◽  
Education Campaign ◽  
Knowledge Based ◽  
Public Education Campaign ◽  
Youth And Young Adults ◽  
Campaign Message ◽  
Health Canada ◽  
The Impact

Purpose: In 2010, Health Canada implemented a national campaign to improve understanding of “percent daily value” (%DV) in Nutrition Facts Tables (NFTs). This study examined sources of nutrition information and knowledge of %DV information communicated in the campaign. Methods: Respondents aged 16–30 years completed the Canada Food Study in 2016 (n = 2665). Measures included sources of nutrition information, NFT use, and %DV knowledge based on the campaign message (“5% DV or less is a little; 15% DV or more is a lot”). A logistic regression examined correlates of providing “correct” responses to %DV questions related to the campaign messaging. Results: Overall, 7.2% (n = 191) respondents correctly indicated that 5% is “a little”, and 4.3% (n = 115) correctly indicated 15% DV was “a lot”. Only 4.0% (n = 107) correctly answered both. Correct recall of %DV amounts was not associated with number of information sources reported, but was greater among those who were female, were younger, and reported greater NFT understanding and serving size information use (P < 0.05 for all). Conclusions: Results show low awareness of messaging from the Nutrition Facts Education Campaign among young Canadians. Such a mass media campaign may be insufficient on its own to enhance population-level understanding of %DV.

scholarly journals Understanding and responding to COVID-19 in Wales: protocol for a privacy-protecting data platform for enhanced epidemiology and evaluation of interventions

BMJ Open ◽  
2020 ◽  
Vol 10 (10) ◽  
pp. e043010
Author(s):  
Jane Lyons ◽  
Ashley Akbari ◽  
Fatemeh Torabi ◽  
Gareth I Davies ◽  
Laura North ◽  
...  
Keyword(s):  
Policy Development ◽  
Healthcare Utilisation ◽  
Population Data ◽  
Population Level ◽  
Risk Groups ◽  
Service Utilisation ◽  
Natural Experiments ◽  
Spread Of Infection ◽  
The Impact

IntroductionThe emergence of the novel respiratory SARS-CoV-2 and subsequent COVID-19 pandemic have required rapid assimilation of population-level data to understand and control the spread of infection in the general and vulnerable populations. Rapid analyses are needed to inform policy development and target interventions to at-risk groups to prevent serious health outcomes. We aim to provide an accessible research platform to determine demographic, socioeconomic and clinical risk factors for infection, morbidity and mortality of COVID-19, to measure the impact of COVID-19 on healthcare utilisation and long-term health, and to enable the evaluation of natural experiments of policy interventions.Methods and analysisTwo privacy-protecting population-level cohorts have been created and derived from multisourced demographic and healthcare data. The C20 cohort consists of 3.2 million people in Wales on the 1 January 2020 with follow-up until 31 May 2020. The complete cohort dataset will be updated monthly with some individual datasets available daily. The C16 cohort consists of 3 million people in Wales on the 1 January 2016 with follow-up to 31 December 2019. C16 is designed as a counterfactual cohort to provide contextual comparative population data on disease, health service utilisation and mortality. Study outcomes will: (a) characterise the epidemiology of COVID-19, (b) assess socioeconomic and demographic influences on infection and outcomes, (c) measure the impact of COVID-19 on short -term and longer-term population outcomes and (d) undertake studies on the transmission and spatial spread of infection.Ethics and disseminationThe Secure Anonymised Information Linkage-independent Information Governance Review Panel has approved this study. The study findings will be presented to policy groups, public meetings, national and international conferences, and published in peer-reviewed journals.

scholarly journals Impact of COVID-19 on multiple sclerosis care and management: Results from the European Committee for Treatment and Research in Multiple Sclerosis survey

2021 ◽  
pp. 135245852110053
Author(s):  
Emilio Portaccio ◽  
Mattia Fonderico ◽  
Bernhard Hemmer ◽  
Tobias Derfuss ◽  
Bruno Stankoff ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Access To Care ◽  
Online Survey ◽  
Dimethyl Fumarate ◽  
Standards Of Care ◽  
Treatment Practices ◽  
European Committee ◽  
Disease Modifying Therapy ◽  
Anti Cd20 ◽  
The Impact

Background: The spread of Coronavirus disease-19 (COVID-19) poses unique challenges in the management of people with multiple sclerosis (PwMS). Objectives: To collect data about the impact of COVID-19 emergency on access to care for PwMS and on MS treatment practices. Methods: Between March and July 2020, the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) promoted an online survey covering patient access to care, management of relapses and visits, disease-modifying therapy (DMT) and experience with COVID-19. Results: Three-hundred and sixty neurologists from 52 countries (68% from Europe) completed the survey. 98% reported COVID-19-related restrictions. Telemedicine was adopted to overcome the limited access to care and was newly activated (73%) or widely implemented (17%). 70% reported changes in DMT management. Interferons and glatiramer were considered safe. Dimethyl fumarate, teriflunomide and fingolimod were considered safe except for patients developing lymphopenia. No modifications were considered for natalizumab in 64%, cladribine in 24%, anti-CD20 in 22% and alemtuzumab in 17%; 18% (for alemtuzumab and cladribine) and 43% (for anti-CD20) considered postponing treatment. Conclusion: The ECTRIMS survey highlighted the challenges in keeping standards of care in clinical practice. Telemedicine clearly needs to be implemented. Gathering data on DMT safety will remain crucial to inform treatment decisions.

scholarly journals The Impact of an Efflux Pump Inhibitor on the Activity of Free and Liposomal Antibiotics against Pseudomonas aeruginosa

Pharmaceutics ◽  
2021 ◽  
Vol 13 (4) ◽  
pp. 577
Author(s):  
Douweh Leyla Gbian ◽  
Abdelwahab Omri
Keyword(s):  
Cystic Fibrosis ◽  
Pseudomonas Aeruginosa ◽  
Efflux Pump ◽  
Antimicrobial Activities ◽  
Dilution Method ◽  
Efflux Pump Inhibitor ◽  
Signal Production ◽  
Lung Infections ◽  
The Impact ◽  
Microbroth Dilution

The eradication of Pseudomonas aeruginosa in cystic fibrosis patients has become continuously difficult due to its increased resistance to treatments. This study assessed the efficacy of free and liposomal gentamicin and erythromycin, combined with Phenylalanine arginine beta-naphthylamide (PABN), a broad-spectrum efflux pump inhibitor, against P. aeruginosa isolates. Liposomes were prepared and characterized for their sizes and encapsulation efficiencies. The antimicrobial activities of formulations were determined by the microbroth dilution method. Their activity on P. aeruginosa biofilms was assessed, and the effect of sub-inhibitory concentrations on bacterial virulence factors, quorum sensing (QS) signals and bacterial motility was also evaluated. The average diameters of liposomes were 562.67 ± 33.74 nm for gentamicin and 3086.35 ± 553.95 nm for erythromycin, with encapsulation efficiencies of 13.89 ± 1.54% and 51.58 ± 2.84%, respectively. Liposomes and PABN combinations potentiated antibiotics by reducing minimum inhibitory and bactericidal concentrations by 4–32 fold overall. The formulations significantly inhibited biofilm formation and differentially attenuated virulence factor production as well as motility. Unexpectedly, QS signal production was not affected by treatments. Taken together, the results indicate that PABN shows potential as an adjuvant of liposomal macrolides and aminoglycosides in the management of lung infections in cystic fibrosis patients.

Household and Population-Level Behavioural Changes Due to COVID-19 Pandemic: A Smart Thermostat Based Comparative Data Analysis

2021 ◽  
Vol 10 (1) ◽  
pp. 1-6
Author(s):  
Kirti Sundar Sahu ◽  
Arlene Oetomo ◽  
Niloofar Jalali ◽  
Plinio P. Morita
Keyword(s):  
Data Analysis ◽  
Population Level ◽  
World Health ◽  
Motion Sensors ◽  
Household Level ◽  
Work From Home ◽  
Significant Difference ◽  
Health Organization ◽  
Smart Thermostat ◽  
The Impact

The World Health Organization declared the coronavirus outbreak as a pandemic on March 11, 2020. To inhibit the spread of COVID-19, governments around the globe, including Canada, have implemented physical distancing and lockdown measures, including a work-from-home policy. Canada in 2020 has developed a 24-Hour Movement Guideline for all ages laying guidance on the ideal amount of physical activity, sedentary behaviour, and sleep (PASS) for an individual in a day. The purpose of this study was to investigate changes on the household and population-level in lifestyle behaviours (PASS) and time spent indoors at the household level, following the implementation of physical distancing protocols and stay-at-home guidelines. For this study, we used 2019 and 2020 data from ecobee, a Canadian smart Wi-Fi thermostat company, through the Donate Your Data (DYD) program. Using motion sensors data, we quantified the amount of sleep by using the absence of movement, and similarly, increased sensor activation to show a longer duration of household occupancy. The key findings of this study were; during the COVID-19 pandemic, overall household-level activity increased significantly compared to pre-pandemic times, there was no significant difference between household-level behaviours between weekdays and weekends during the pandemic, average sleep duration has not changed, but the pattern of sleep behaviour significantly changed, specifically, bedtime and wake up time delayed, indoor time spent has been increased and outdoor time significantly reduced. Our data analysis shows the feasibility of using big data to monitor the impact of the COVID-19 pandemic on the household and population-level behaviours and patterns of change.

scholarly journals GPS Coordinates for Modelling Correlated Herd Effects in Genomic Prediction Models Applied to Hanwoo Beef Cattle

Animals ◽  
2021 ◽  
Vol 11 (7) ◽  
pp. 2050
Author(s):  
Beatriz Castro Dias Cuyabano ◽  
Gabriel Rovere ◽  
Dajeong Lim ◽  
Tae Hun Kim ◽  
Hak Kyo Lee ◽  
...  
Keyword(s):  
Environmental Factors ◽  
Genomic Prediction ◽  
Prediction Models ◽  
Phenotypic Expression ◽  
Genetic Evaluation ◽  
Genomic Breeding ◽  
Breeding Values ◽  
Korean Cattle ◽  
Evaluation Programs ◽  
The Impact

It is widely known that the environment influences phenotypic expression and that its effects must be accounted for in genetic evaluation programs. The most used method to account for environmental effects is to add herd and contemporary group to the model. Although generally informative, the herd effect treats different farms as independent units. However, if two farms are located physically close to each other, they potentially share correlated environmental factors. We introduce a method to model herd effects that uses the physical distances between farms based on the Global Positioning System (GPS) coordinates as a proxy for the correlation matrix of these effects that aims to account for similarities and differences between farms due to environmental factors. A population of Hanwoo Korean cattle was used to evaluate the impact of modelling herd effects as correlated, in comparison to assuming the farms as completely independent units, on the variance components and genomic prediction. The main result was an increase in the reliabilities of the predicted genomic breeding values compared to reliabilities obtained with traditional models (across four traits evaluated, reliabilities of prediction presented increases that ranged from 0.05 ± 0.01 to 0.33 ± 0.03), suggesting that these models may overestimate heritabilities. Although little to no significant gain was obtained in phenotypic prediction, the increased reliability of the predicted genomic breeding values is of practical relevance for genetic evaluation programs.

scholarly journals The effects of nano-curcumin as a nutritional strategy on clinical and inflammatory factors in children with cystic fibrosis: the study protocol for a randomized controlled trial

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Saeedeh Talebi ◽  
Mahammad Safarian ◽  
Mahmood Reza Jaafari ◽  
Seyed Javad Sayedi ◽  
Zahra Abbasi ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Controlled Trial ◽  
Genetic Disorder ◽  
The Body ◽  
Inflammatory Factors ◽  
Secondary Outcome ◽  
Nasopharyngeal Swab ◽  
Nutritional Strategy ◽  
Cftr Protein ◽  
The Impact

Abstract Background Cystic fibrosis (CF) is a genetic disorder, which is caused by the CFTR protein defects. Along with CFTR dysfunction, inflammation plays a key role in the disease outcomes. Inflammation may develop due to the internal dysfunction of the CFTR protein or external factors. Curcumin affects the CFTR protein function primarily as a corrector and potentiator and secondary as an anti-inflammatory and antimicrobial agent. The present study aims to assess the impact of nano-curcumin on clinical and inflammatory markers in children with CF. Methods This prospective, double blind control trial will be conducted at the Akbar Children’s Hospital in Mashhad, Iran. Children with CF will be enrolled based on the eligibility criteria. Placebo and curcumin with the maximum dose of 80 mg considering the body surface of the patients will be administrated for 3 months. The primary outcome is to evaluate inflammation based on serum interleukin-6, interleukin-10, and hs-CRP, stool calprotectin, and neutrophil count of nasopharyngeal swab. The secondary outcome involved clinical assessment via spirometry, anthropometrics, and quality of life. They will be assessed before and after 3 months. Discussion Due to the multifarious effects of curcumin on CF disease, it could be proposed as a nutritional strategy in the treatment of cystic fibrosis. Trial registration Iranian Registry of Clinical Trials IRCT20200705048018N1. Registered on July 10, 2020.

scholarly journals Potential health impact of increasing adoption of sustainable dietary practices in Sweden

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Emma Patterson ◽  
Patricia Eustachio Colombo ◽  
James Milner ◽  
Rosemary Green ◽  
Liselotte Schäfer Elinder
Keyword(s):  
Health Impact ◽  
Milk Intake ◽  
Processed Meat ◽  
Years Of Life Lost ◽  
Equal Weight ◽  
Dietary Practices ◽  
Swedish Population ◽  
Potential Health ◽  
The Impact ◽  
Red And Processed Meat

Abstract Background An urgent transition to more sustainable diets is necessary for the improvement of human and planetary health. One way to achieve this is for sustainable practices to become mainstream. We estimated the potential health impact of wider adoption of dietary practices deemed by consumers, researchers and stakeholders in Sweden to be niche, sustainable and with the potential to be scaled up. Methods A life table method was used to estimate the impact - changes in years of life lost (YLL) - over periods of 20 and 30 years in the Swedish population had the practices been adopted in 2010–11, when the last national adult dietary survey was conducted. The practices modelled were reducing red and processed meat (by 25, 50 and 100%), and assuming, for each stage, replacement by an equal weight of poultry/fish and vegetables +/− legumes; reducing milk intake (by 25, 50 and 100%); and reducing sugar-sweetened beverage intake (by 25, 50 and 100%). Using population data together with data on cause-specific mortality and relative risks for diet-disease outcomes, impacts were estimated for each scenario separately and in combination, for the outcomes ischaemic heart disease (IHD), ischaemic stroke, diabetes type 2 and colorectal cancer. Results For a “moderate” combination of scenarios (changes at the 50% level), reductions of 513,200 YLL (lower-upper uncertainty estimate 59,400-797,900) could have been achieved over 20 years and 1,148,500 YLL (135,900-1,786,600) over 30 years. The majority (over 90%) of YLLs prevented were related to IHD, and the majority were in men. The singular practice that had the most impact was reducing the intake of red and processed meat and replacing it with a mixture of vegetables and legumes. Reducing milk intake resulted in an increase in YLL, but this was compensated for by other scenarios. Conclusion If these practices were more widely adopted, they would be expected to lead to improvements in public health in Sweden. Over the long term, this would translate to many premature deaths postponed or prevented from a number of chronic diseases, to the benefit of individuals, society, the climate and the economy.

scholarly journals Quantifying the consequence of applying conservative assumptions in the assessment of oil spill effects on polar cod (Boreogadus saida) populations

Polar Biology ◽  
2021 ◽  
Vol 44 (3) ◽  
pp. 575-586
Author(s):  
Pepijn De Vries ◽  
Jacqueline Tamis ◽  
Jasmine Nahrgang ◽  
Marianne Frantzen ◽  
Robbert Jak ◽  
...  
Keyword(s):  
Oil Spill ◽  
Oil Spills ◽  
Population Level ◽  
Threshold Concentration ◽  
The Arctic ◽  
Polar Cod ◽  
Boreogadus Saida ◽  
Precautionary Approach ◽  
Recovery Duration ◽  
The Impact

AbstractIn order to assess the potential impact from oil spills and decide the optimal response actions, prediction of population level effects of key resources is crucial. These assessments are usually based on acute toxicity data combined with precautionary assumptions because chronic data are often lacking. To better understand the consequences of applying precautionary approaches, two approaches for assessing population level effects on the Arctic keystone species polar cod (Boreogadus saida) were compared: a precautionary approach, where all exposed individuals die when exposed above a defined threshold concentration, and a refined (full-dose-response) approach. A matrix model was used to assess the population recovery duration of scenarios with various but constant exposure concentrations, durations and temperatures. The difference between the two approaches was largest for exposures with relatively low concentrations and short durations. Here, the recovery duration for the refined approach was less than eight times that found for the precautionary approach. Quantifying these differences helps to understand the consequences of precautionary assumptions applied to environmental risk assessment used in oil spill response decision making and it can feed into the discussion about the need for more chronic toxicity testing. An elasticity analysis of our model identified embryo and larval survival as crucial processes in the life cycle of polar cod and the impact assessment of oil spills on its population.

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