scholarly journals The Rapid and Accurate Categorisation of Critically Ill Patients (RACE) to Identify Outcomes of Interest for Longitudinal Studies: A Feasibility Study

2017 ◽  
Vol 45 (4) ◽  
pp. 476-484 ◽  
Author(s):  
A. M. Deane ◽  
C. L. Hodgson ◽  
P. Young ◽  
L. Little ◽  
V. Singh ◽  
...  
Keyword(s):  
Gold Standard ◽  
Functional Outcomes ◽  
Clinical Approach ◽  
Baseline Assessment ◽  
Inception Cohort ◽  
Eligibility Criteria ◽  
Standard Assessment ◽  
The Impact ◽  
Blinded Assessor

The capacity to measure the impact of an intervention on long-term functional outcomes might be improved if research methodology reflected our clinical approach, which is to individualise goals of care to what is achievable for each patient. The objective of this multicentre inception cohort study was to evaluate the feasibility of rapidly and accurately categorising patients, who were eligible for simulated enrolment into a clinical trial, into unique categories based on premorbid function. Once a patient met eligibility criteria a rapid ‘baseline assessment’ was conducted to categorise patients into one of eight specified groups. A subsequent ‘gold standard’ assessment was made by an independent blinded assessor once patients had recovered sufficiently to allow such an assessment to occur. Accuracy was predefined as agreement in >80% of assessments. One hundred and twenty-two patients received a baseline assessment and 104 (85%) were categorised to a unique category. One hundred and six patients survived to have a gold standard assessment performed, with 100 (94%) assigned to a unique category. Ninety-two patients had both a baseline and gold standard assessment, and these agreed in 65 (71%) patients. It was not feasible to rapidly and accurately categorise patients according to premorbid function.

scholarly journals Indirect costs associated with ulcerative colitis: a systematic literature review of real-world data

2019 ◽  
Vol 19 (1) ◽  
Author(s):  
Joelle Constantin ◽  
Petar Atanasov ◽  
Daniel Wirth ◽  
Andras Borsi
Keyword(s):  
Ulcerative Colitis ◽  
Sick Leave ◽  
Indirect Costs ◽  
Cochrane Library ◽  
Real World Data ◽  
Eligibility Criteria ◽  
Productivity Costs ◽  
Productivity Losses ◽  
The Impact

Abstract Background The economic burden of ulcerative colitis (UC), specifically related to indirect costs, is not extensively documented. Understanding and quantifying it is required by health care decision makers. Aim To assess the impact of indirect costs of UC in observation studies. Method A systematic literature search was conducted in MEDLINE®, Embase® and Cochrane Library to capture all relevant publications reporting outcomes on absenteeism, presenteeism and productivity losses in moderate to severe UC. Eligibility criteria for inclusion into the review were established using a predefined PICOS scheme. All costs were adjusted to 2017 currency values (USD dollars, $). Results In total, 18 studies reporting data on indirect costs were included in the analysis. Absenteeism costs were classified into three categories: sick leave, short-term and long-term disability. Most of the studies captured absenteeism costs related specifically to sick leave, which was experienced on average by 10 to 24% patients with UC. Only three studies captured presenteeism costs, as these are difficult to measure, however costs ranged from 1602 $ to 2947 $ per patient year. The proportion of indirect costs accounted for 35% of total UC costs (Total UC costs were defined as the sum of healthcare costs, productivity costs and out-of-pocket costs). Discussion A limited number of studies were identified describing the indirect costs in patients with moderate to severe UC. Insufficient data on different components of costs allowed a limited analysis on the impact of indirect costs in patients with UC. Further studies are needed to gain an understanding of the influence of UC on patients’ functional abilities.

scholarly journals Long-Term Growth in Phenylketonuria: A Systematic Review and Meta-Analysis

Nutrients ◽  
2019 ◽  
Vol 11 (9) ◽  
pp. 2070 ◽  
Author(s):  
Fatma Ilgaz ◽  
Alex Pinto ◽  
Hülya Gökmen-Özel ◽  
Julio César Rocha ◽  
Esther van Dam ◽  
...  
Keyword(s):  
Systematic Review ◽  
Meta Analysis ◽  
Normal Growth ◽  
Optimal Growth ◽  
Current Evidence ◽  
Eligibility Criteria ◽  
Dietary Practices ◽  
Dietary Treatments ◽  
The Impact

There is an ongoing debate regarding the impact of phenylketonuria (PKU) and its treatment on growth. To date, evidence from studies is inconsistent, and data on the whole developmental period is limited. The primary aim of this systematic review was to investigate the effects of a phenylalanine (Phe)-restricted diet on long-term growth in patients with PKU. Four electronic databases were searched for articles published until September 2018. A total of 887 results were found, but only 13 articles met eligibility criteria. Only three studies had an adequate methodology for meta-analysis. Although the results indicate normal growth at birth and during infancy, children with PKU were significantly shorter and had lower weight for age than reference populations during the first four years of life. Impaired linear growth was observed until the end of adolescence in PKU. In contrast, growth impairment was not reported in patients with mild hyperphenylalaninemia, not requiring dietary restriction. Current evidence indicates that even with advances in dietary treatments, “optimal” growth outcomes are not attained in PKU. The majority of studies include children born before 1990s, so further research is needed to show the effects of recent dietary practices on growth in PKU.

scholarly journals Does preoperative bladder compliance affect long-term functional outcomes after laser prostatectomy?

2021 ◽  
Author(s):  
Sangjun Yoo ◽  
Min Chul Cho ◽  
Min Soo Choo ◽  
Sung Yong Cho ◽  
Hyeon Jeong ◽  
...  
Keyword(s):  
Functional Outcomes ◽  
Multivariable Analysis ◽  
Laser Prostatectomy ◽  
Bladder Compliance ◽  
Laser Enucleation ◽  
Symptom Changes ◽  
The Impact ◽  
And Storage ◽  
Holmium Laser Enucleation

Introduction: We assessed the effects of preoperative bladder compliance on the long-term functional outcomes, especially focused on postoperative storage symptom changes, after laser prostatectomy. Materials and Methods: From January 2008 to March 2014, 1608 men who underwent laser prostatectomy, including holmium laser enucleation or photo-vaporization of the prostate, were included in the analysis. We divided patients into 3 groups according to bladder compliance on a baseline urodynamic study: < 12.5; 12.5–25.0; ≥25 mL/cm H2O. A multivariable analysis was performed to determine the impact of bladder compliance on long-term functional outcomes after laser prostatectomy. Results: Bladder compliance was less than 12.5 ml/cm H2O in 50 (3.1%), 12.5-25 ml/cm H2O in 232 (14.4%) patients. As bladder compliance decreased, the baseline International Prostate Symptom (I-PSS) total score and storage sub-score were increased; the voiding sub-score remain unchanged. At postoperative 36 months, improvements in the I-PSS total score and storage sub-score were significantly higher in < 12.5 mL/cm H2O group compared to other groups, although those were equivalent at postoperative 1 and 12 months. On the multivariable analysis, decreased bladder compliance < 12.5 mL/cm H2O was significantly associated with superior improvement in storage sub-score at postoperative 36 months, although it was not associated with voiding sub-score. Conclusion: In patients with preoperative bladder compliance < 12.5 mL/cm H2O, storage symptoms could be further improved at 36 months after laser prostatectomy compared to others. Thus, laser prostatectomy could be a considerable treatment option for patients with severely decreased bladder compliance

Impact of functional outcomes on long-term quality of life after open retropubic radical prostectomy.

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 9580-9580
Author(s):  
Bjoern Loeppenberg ◽  
Christian von Bodman ◽  
Marko Brock ◽  
Joachim Noldus ◽  
Jueri Palisaar
Keyword(s):  
Quality Of Life ◽  
Erectile Dysfunction ◽  
Erectile Function ◽  
Functional Outcomes ◽  
Term Survival ◽  
Eortc Qlq C30 ◽  
Eortc Qlq ◽  
The Impact

9580 Background: Patients who underwent open retropubic radical prostatectomy (ORRP) for prostate-cancer (PCA) have excellent long-term survival. Besides oncologic safety, recovery of continence and erectile function are highly important, as adverse functional outcomes may have a detrimental effect on health-related quality of life (HRQOL). We report the long-term HRQOL of PCA survivors after ORRP using standardized tools. Methods: Men treated between August 2003 and December 2007 with ORRP for localized PCA at a single academic hospital received validated questionnaires (International consultation on incontinence questionnaire (ICIQ), International index of erectile function (IIEF-5), Erection hardness score (EHS), EORTC QLQ-C30) to assess functional outcomes and HRQOL. Results were correlated with the global-health score (GHS) of the EORTC QLQ-C30 to assess the impact of ORRP on HRQOL. Results: In the study period 1936 men underwent ORRP of whom 1156 (59.7%) received a nerve-sparing (NS) procedure. Questionnaire return-rate was 59% (n=1141) comprising the final study cohort. Median follow-up (FU) was 62 months. Mean age at surgery and FU was 63.7±6.2 and 69.2±6.2 years, respectively. Biochemical recurrence (BCR) occurred in 17.5% (n=200/1141) and 2% (n=40/1936) deceased. Mean GHS in the study population was 71.5±20.8. In the ICIQ 28% (n=320) scored 0 indicating complete continence and 9.9% (n=113) scored ≥11 indicating severe incontinence. The corresponding GHS was 78.1±19.5 and 55.4±21.8, respectively. 68.5% (n=782) of patients used no pads and 17.9% (n=204) ≥2 pads. Corresponding GHS scores were 74.9±19.8 and 58.9±20.7. Using the IIEF-5 in men who received NS, 24.1% (n=154) had no erectile dysfunction versus 50% (n=318) using the EHS. Corresponding GHS scores were 82.2±16.3 and 74.7±19.8, respectively. Patients with BCR had a GHS of 66.8±21.8 versus 72.5±20.5 for patients without. Men who achieved the Trifecta and Pentafecta criteria had a GHS of 83.1±15.1 and 83.3±15, respectively. Conclusions: Incontinence severely impacts the HRQOL of long-term survivors after ORRP while erectile dysfunction and BCR have a lesser effect. Every effort should be undertaken to maintain functional integrity.

Methodological issues in the design and analysis of cluster randomized trials

2021 ◽  
pp. 113-128
Author(s):  
Kathy J. Baisley ◽  
Richard J. Hayes ◽  
Lawrence H. Moulton
Keyword(s):  
Gold Standard ◽  
Randomized Trials ◽  
Controlled Trials ◽  
Cluster Randomized Trials ◽  
Methodological Issues ◽  
Eligibility Criteria ◽  
Experimental Conditions ◽  
Cluster Randomized ◽  
And Control ◽  
The Impact

Randomized controlled trials are the accepted gold standard for evaluating the effects of interventions to improve health. In the majority of such trials, individuals are randomly allocated to the experimental conditions under study, for example, to treatment and control arms. However, in some situations it is more appropriate to randomly allocate groups of individuals to the treatment arms. These groups are referred to as clusters, and trials of this kind are known as cluster randomized trials (CRTs). Examples of clusters include schools, villages, workplaces, or health facilities, but there are many other possible choices. In some CRTs, all individuals within the selected clusters are automatically included. In others, there may be additional eligibility criteria. Similarly, the impact of the intervention may be measured in all individuals in the cluster, or in a random subsample. This chapter aims to discuss methodological issues that arise in the design and analysis of CRTs

scholarly journals Accounting for heterogeneity in wild adult samples to measure insecticide resistance in Anopheles malaria vectors

2021 ◽  
Author(s):  
Inga E. Holmdahl ◽  
Caroline O. Buckee ◽  
Lauren M. Childs
Keyword(s):  
Spatial Structure ◽  
Insecticide Resistance ◽  
Gold Standard ◽  
Time Frame ◽  
Malaria Vectors ◽  
Bed Nets ◽  
Insecticide Exposure ◽  
Mosquito Collection ◽  
The Impact

Background Systematic, long-term, and spatially representative monitoring of insecticide resistance in mosquito populations is urgently needed to quantify its impact on malaria transmission, and to combat failing interventions when resistance emerges. Resistance assays on wild-caught adult mosquitoes (known as adult-capture) offer an alternative to the current protocols, and can be done cheaply, in a shorter time frame, and in the absence of an insectary. However, quantitative assessments of the performance of these assays relative to the gold standard, which involves rearing larvae in an insectary, are lacking. Methodology/Principal findings We developed a discrete-time deterministic mosquito lifecycle model to simulate insecticide resistance assays from adult-captured mosquito collection in a heterogeneous environment compared to the gold standard larval capture methods, and to quantify possible biases in the results. We incorporated non-lethal effects of insecticide exposure that have been demonstrated in laboratory experiments, spatial structure, and the impact of multiple exposure to insecticides and natural ageing on mosquito death rates during the assay. Using output from this model, we compared the results of these assays to true resistance as measured by the presence of the resistance allele. In simulated samples of 100 test mosquitoes, reflecting WHO-recommended sample sizes, we found that compared to adult-captured assays (MSE = 0.0059), larval-captured assays were a better measure of true resistance (MSE = 0.0018). Using a correction model, we were able to improve the accuracy of the adult-captured assay results (MSE = 0.0038). Bias in the adult-capture assays was dependent on the level of insecticide resistance rather than coverage of bed nets or spatial structure. Conclusions/Significance Using adult-captured mosquitoes for resistance assays has logistical advantages over the standard larval-capture collection, and may be a more accurate sample of the mosquito population. These results show that adult-captured assays can be improved using a simple mathematical approach and used to inform resistance monitoring programs.

scholarly journals Direct reprogramming of astrocytes to neurons leads to functional recovery after stroke

2020 ◽  
Author(s):  
Jessica Livingston ◽  
Tina Lee ◽  
Emerson Daniele ◽  
Clara Phillips ◽  
Alexandra Krassikova ◽  
...  
Keyword(s):  
Functional Outcome ◽  
Functional Outcomes ◽  
Treatment Options ◽  
Brain Repair ◽  
Novel Approach ◽  
Regenerative Approach ◽  
Cortical Stroke ◽  
The Impact

AbstractStroke is the leading cause of adult disability with few treatment options for stroke survivors. Astrocyte reprogramming to neurons enables the targeted in vivo generation of new cells at the site of injury and represents a novel approach for brain repair. A number of studies have demonstrated successful conversion of astrocytes to neurons in various models of brain injury and disease; however, the impact of this strategy on tissue and functional outcome following stroke is not well established. Using AAV delivery of the transcription factor NeuroD1, we reprogrammed astrocytes 7 days after endothelin-1 induced cortical stroke, and studied the long-term cellular and functional outcomes. We found that by 63 days post-stroke, 20% of neurons in the perilesional cortex were reprogrammed. Furthermore, reprogrammed neurons had matured into regionally appropriate neuronal subtypes. Importantly, this treatment was associated with improved functional outcome using the foot fault test and gait analysis. Together, our findings indicate that in vivo reprogramming is a promising regenerative approach for stroke repair.

scholarly journals The Influence of Hearing Impairment on Mental Age in Down Syndrome: Preliminary Results

2021 ◽  
Vol 9 ◽  
Author(s):  
Amanda Saksida ◽  
Davide Brotto ◽  
Giulia Pizzamiglio ◽  
Elena Bianco ◽  
Sara Bressan ◽  
...  
Keyword(s):  
Down Syndrome ◽  
Cognitive Decline ◽  
Hearing Impairment ◽  
Functional Outcomes ◽  
Young Patients ◽  
Receptive Language ◽  
Language Abilities ◽  
Mental Age ◽  
The Impact

The increased life expectancy for patients with Down Syndrome (DS) has elicited the need to improve their quality of life by enhancing functional outcomes and identifying the factors that contribute to their long-term cognitive decline. Although the majority of individuals with DS have issues with hearing impairment (HI) since early childhood, to our knowledge no study has investigated whether HI represents a potential modulator of cognitive decline over time. The present explorative cohort study, albeit very preliminary due to the limited cohort (17 children), highlights the significant relation of a significant HI not only with receptive language abilities, but also with mental age in young patients with DS. Additional studies are required to confirm the link between HI and mental age and to assess the impact of audiological treatment on the enhancement of functional outcomes and of cognitive decline in individuals with DS.

scholarly journals Efficient Ways to Combat Doping in a Sports Education Context!? A Systematic Review on Doping Prevention Measures Focusing on Young Age Groups

2021 ◽  
Vol 3 ◽  
Author(s):  
Katharina Pöppel
Keyword(s):  
School Context ◽  
Age Groups ◽  
Young Age ◽  
Added Value ◽  
Prevention Measures ◽  
Target Groups ◽  
Eligibility Criteria ◽  
Screening Process ◽  
The Impact

Youth is characterized by testing and crossing natural boundaries, sometimes with the help of performance-enhancing substances. In this context, doping prevention measures play a crucial role to protect individuals both within and outside the context of elite sport. Based on the PRISMA guidelines, a systematic literature search was conducted in the databases ProQuest (ERIC), Scopus, PSYNDEX/PsychInfo, PubMed, and Web of Science Core Collection to provide an overview of the impact of doping prevention measures, with particular attention to the underlying understanding of learning. As a result of the screening process, 30 of the initial 5,591 articles met the previously defined and recorded eligibility criteria. The analysis led to heterogeneous results regarding content, implementation, target group, or outcome variables considered relevant. Two-thirds of the studies related to the competitive sports context. Nevertheless, there has been a growing interest in studying doping prevention and its effects on non-elite athlete target groups in recent years. In terms of effectiveness, many measures did not achieve long-term changes or did not collect any follow-up data. This contrasts with understanding learning as sustained change and reduces the intended long-term protection of prevention measures, especially for adolescent target groups. Even young age groups from 10 years upwards benefited from doping prevention measures, and almost all doping prevention measures enabled their participants to increase their physical and health literacy. No conclusion can be drawn as to whether doping prevention measures based on constructivist ideas are superior to cognitivist approaches or a combination of both. Nevertheless, programs that actively engage their participants appear superior to lecture-based knowledge transfer. Most of the prevention measures offered a benefit-orientation so that participants can achieve added value, besides trying to initiate health-promoting change through rejection. Because of the lack of sustained changes, a further modification in doping prevention seems necessary. The review results support the value of primary prevention. Doping prevention measures should enable tailored learning and development options in the sense of more meaningful differentiation to individual needs. The implementation in a school context or an online setting is promising and sees doping as a problem for society. The review highlights the importance of accompanying evaluation measures to identify efficient prevention components that promote health and protect young people.

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