The COVID-19 pandemic forced (or quickened the advent of) online short course statistics training for non-statistical professionals. Participants in traditional statistics programs generally form a cohort who progress together over some protracted timescale, forging support networks and friendship groups, some of which will be maintained throughout professional careers. By contrast, short courses for professional purposes are often standalone and half a day to one week in duration. Participant focus lies with obtaining skills for usage within the foreseeable future, such that creating social networks with other participants is of little or no importance. We present our experiences with two years of online, examination-free, short course statistics training for the period from March 2020 to 2022.

Several factors may affect the quality of education (QoE). The challenges have intensified over the past 20 months of COVID-19. Our goal is to understand the effect of transitioning to online-learning on QoE amongst those undertaking statistical courses. A total of 714 evaluation forms were collected from students over a period of 12 years. Students were asked to rate the overall QoE of the research methods course. More than 90% of the students agreed that the overall QoE was very good/excellent over the past 12 years. Despite the online delivery of the course since 2020, more than 90% reported a very good/excellent QoE. A synergy of factors may contribute to the retention of an overall good QoE. Further studies could assess the effectiveness of online learning.

Background: With the widespread introduction of newborn screening for cystic fibrosis (CF), there has been considerable emphasis on the need to develop objective markers of lung health that can be used during infancy. We hypothesised that in a newborn screened (NBS) UK cohort, evidence of airway inflammation and infection at one year would be associated with adverse structural and functional outcomes at the same age. Methods: Infants underwent lung function testing, chest CT scan and bronchoscopy with bronchoalveolar lavage (BAL) at 1 year of age when clinically well. Microbiology cultures were also available from routine cough swabs. Results: 65 infants had lung function, CT and BAL. Mean (SD) lung clearance index and forced expiratory volume in 0.5 s z-scores were 0.9(1.2) and -0.6(1.1) respectively; median Brody II CF-CT air trapping score on chest CT=0 (interquartile range 0-1, maximum possible score 27). Infants isolating any significant pathogen by 1 yr of age had higher LCI z-score (mean difference 0.9; 95%CI:0.4-1.4; p = 0.001) and a trend towards higher air trapping scores on CT (p = 0.06). BAL neutrophil elastase was detectable in 23% (10/43) infants in whom BAL supernatant was available. This did not relate to air trapping score on CT. Conclusions: In this UK NBS cohort at one year of age, lung and airway damage is much milder and associations between inflammation, abnormal physiology and structural changes were at best weak, contrary to our hypothesis and previously published reports. Continued follow-up will clarify longer term implications of these very mild structural, functional and inflammatory changes. (C) 2020 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

Lung clearance index (LCI) in the early years was associated with LCI during adolescence in children with cystic fibrosis. Pre-school LCI may help to identify children in whom treatment could be intensified.https://bit.ly/2yKyMbM

Background Less invasive perinatal and paediatric autopsy methods, such as imaging alongside targeted endoscopy and organ biopsy, may address declining consent rates for traditional autopsy, but their acceptability and accuracy are not known. Objectives The aims of this study were to provide empirical data on the acceptability and likely uptake for different types of autopsy among key stakeholders (study 1); and to analyse existing autopsy data sources to provide estimates of the potential efficacy of less invasive autopsy (LIA) and its projected utility in clinical practice (study 2). Review methods Study 1: this was a mixed-methods study. Parents were involved in research design and interpretation of findings. Substudy 1: a cross-sectional survey of 859 parents who had experienced miscarriage, termination of pregnancy for fetal anomaly, stillbirth, infant or child death, and interviews with 20 responders. Substudy 2: interviews with 25 health professionals and four coroners. Substudy 3: interviews with 16 religious leaders and eight focus groups, with 76 members of the Muslim and Jewish community. Study 2: a retrospective analysis of national data in addition to detailed information from an existing in-house autopsy database of > 5000 clinical cases that had undergone standard autopsy to determine the proportion of cases by clinical indication group for which tissue sampling of specific internal organs significantly contributed to the diagnosis. Results Substudy 1: 91% of participants indicated that they would consent to some form of LIA, 54% would consent to standard autopsy, 74% to minimally invasive autopsy (MIA) and 77% to non-invasive autopsy (NIA). Substudy 2: participants viewed LIA as a positive development, but had concerns around the limitations of the technology and de-skilling the workforce. Cost implications, skills and training requirements were identified as implementation challenges. Substudy 3: religious leaders agreed that NIA was religiously permissible, but MIA was considered less acceptable. Community members indicated that they might consent to NIA if the body could be returned for burial within 24 hours. Study 2: in 5–10% of cases of sudden unexplained death in childhood and sudden unexplained death in infants, the final cause of death is determined by routine histological sampling of macroscopically normal organs, predominantly the heart and lungs, and in this group routine histological sampling therefore remains an important aspect of investigation. In contrast, routine histological examination of macroscopically normal organs rarely (< 0.5%) provides the cause of death in fetal cases, making LIA and NIA approaches potentially highly applicable. Limitations A key limitation of the empirical research is that it is hypothetical. Further research is required to determine actual uptake. Furthermore, because of the retrospective nature of the autopsy data set, findings regarding the likely contribution of organ sampling to final diagnosis are based on extrapolation of findings from historical autopsies, and prospective data collection is required to validate the conclusions. Conclusions LIA is viable and acceptable (except for unexplained deaths), and likely to increase uptake. Further health economic, performance and implementation studies are required to determine the optimal service configuration required to offer this as routine clinical care. Funding The National Institute for Health Research Health Technology Assessment programme.

We analyse paediatric ophthalmic data from a large sample of children aged between 3 and 8 years. We use a Bayesian additive conditional bivariate copula regression model with sinh‐arcsinh marginal densities with location, scale, and shape parameters that depend smoothly on a covariate. We perform Bayesian inference about the unknown quantities of our model using a specially tailored Markov chain Monte Carlo algorithm. We gain new insights about the processes, which determine transformations in visual acuity with respect to age, including the nature of joint changes in both eyes as modelled with the age‐related copula dependence parameter. We analyse posterior predictive distributions to identify children with unusual sight characteristics, distinguishing those who are bivariate, but not univariate outliers. In this way, we provide an innovative tool that enables clinicians to identify children with unusual sight who may otherwise be missed. We compare our simultaneous Bayesian method with a two‐step frequentist generalised additive modelling approach.

To compare results from a third (1995–2010) cohort of children with medulloblastoma with two previous series (J Neurosurg 86:13–21, 1997; Arch Dis Child 54:200–203, 1979) to analyse the effects of management changes aimed at improving both overall and event-free survivals (OS and EFS) and functional outcomes. Review of neuro-oncology and imaging databases and previously published results. There was no statistically significant improvement in the 5-year OS for 104 children diagnosed 1995–2010, 61.5% (95% CI, 52.9, 71.6), compared with 50% of the 80 children presenting 1980–1990 (J Neurosurg 86:13–21, 1997) (difference 11.5%; 95% CI, 2.8, 25.4). Five-year OS for 96 children suitable for risk-stratification was overall 66% (95% CI, 57.9, 75.8); standard risk 77.8% (95% CI, 67.4, 89.7); high risk < 3 years 50.0% (95% CI, 32.3, 77.5); high risk ≥ 3 years 54.5% (95% CI, 37.2, 79.9); 5-year EFS were standard risk 68.5% (95% CI, 57.2, 82.1); high risk < 3 years 40.0% (95% CI, 23.4, 68.4); and high risk ≥ 3 years 36.4% (95% CI, 20.9, 63.2); overall 55.2% (95% CI, 46.1, 66.1). Of 62/63 ≥ 5-year survivor, 9 died later from tumour relapse and 4 from second malignancy. Functional outcomes of 62 of the 63 ≥ 5-year survivors: 67.7% had educational issues requiring remedial input; 18% restricted mobility indoors and outdoors; 59.7% hearing impairment (42% prescribed aids).

Background Community-based women's groups practising participatory learning and action (PLA) can reduce maternal and neonatal mortality in low-income countries. However, it is not clear whether these reductions are associated with subsequent increased or decreased rates of childhood death and disability. We assessed the impact on child deaths and disability beyond the perinatal period among participants in the earliest trial in Nepal 2001-2003. Methods Household interviews were conducted with mothers or household heads. At cluster and individual levels, we analysed disability using pairwise log relative risks and survival using multilevel logistic models. Findings From 6075 children and 6117 mothers alive at 4 weeks post partum, 44 419 children (73%) were available for interview a mean 11.5 years later. Rates of child deaths beyond the perinatal period were 36.6 and 52.0 per 1000 children in the intervention and control arms respectively. Rates of disability were 62.7 and 85.5 per 1000 children in the intervention and control arms respectively. Individual-level analysis, including random effects for cluster pairing and adjusted for baseline maternal literacy, socioeconomic status and maternal age, showed lower, statistically non significant, odds of child deaths (OR 0.70 (95% CI 0.43 to 1.18) and disability (0.64 (0.39 to 1.06)) in the intervention arm. Conclusion Community-level exposure to women's groups practising PLA did not significantly impact childhood death or disability or death beyond the perinatal period. Follow-up of other trials with larger sample sizes is warranted in order to explore the possibility of potential long-term survival and disability benefits with greater precision.

Are adolescents with constipation more likely to suffer psychological maladjustment? 1 What percentage of Chiari I-type headaches show improvement after foramen magnum decompression (FMD)? 2 Does Bacillus Calmette-Guerin (BCG) vaccination reduce early childhood hospitalisation in Denmark? 3 Is diagnosis of coeliac disease associated with differences in adolescent anthropometry? 4 Does visual feedback affect the rate of chest compressions? 5 These are all questions asked in recent issues of this journal. In each case, the authors collated information from a sample of individuals to yield an answer to their question. Differing study types were used ranging from observational audits and surveys through to randomised parallel and crossover trials. The study designs, participants, settings, sample sizes and key statistics are summarised in table 1. View this table: Table 1 Description of the five studies Despite these differences, the same basic principle is followed for each. A sample of the relevant group of individuals is identified and from observing what happens to this sample, inferences are made about the wider population. The inferences may be beneficial to similar individuals and those involved in their care. For example, clinicians trying to determine whether to perform FMD2 or parents considering the pros and cons of BCG vaccination.3 How well a question is answered by the study depends on how large a sample was studied in conjunction with other factors such as the variability of the measurements and/or event rates. Both researchers and patients intuitively understand that findings based on a larger sample are likely to be more accurate and will have more confidence in results based on a randomised trial of 1000 individuals than if only 10 patients had been recruited. What is less intuitive is that if a treatment is not shown to be effective in a small sample, it may still have benefits. Similarly, we are …

With the advent of novel designer molecules for cystic fibrosis (CF) treatment, there is huge need for early-life clinical trial outcomes, such as infant lung function (ILF). We investigated the degree and tracking of ILF abnormality during the first 2 years of life in CF newborn screened infants.Forced expiratory volume in 0.5 s (FEV0.5), lung clearance index (LCI) and plethysmographic functional residual capacity were measured at ∼3 months, 1 year and 2 years in 62 infants with CF and 34 controls.By 2 years there was no significant difference in FEV0.5z-score between CF and controls, whereas mean LCI z-score was 0.81 (95% CI 0.45–1.17) higher in CF. However, there was no significant association between LCI z-score at 2 years with either 3-month or 1-year results. Despite minimal average group changes in any ILF outcome during the second year of life, marked within-subject changes occurred. No child had abnormal LCI or FEV0.5on all test occasions, precluding the ability to identify “high-risk” infants in early life.In conclusion, changes in lung function are mild and transient during the first 2 years of life in newborn screened infants with CF when managed according to a standardised UK treatment protocol. Their potential role in tracking disease to later childhood will be ascertained by ongoing follow-up.

The ideology of Emancipatory Disability Research (EDR) reflected in the phrase ‘Nothing about us without us’, was first put forward in the 1990s. Although it aimed to place research control in the hands of the ‘researched’, i.e., people with disability, this rarely happens even today, 25 years later.The Community Action Research on Disability (CARD) programme in Uganda embraced and modified the EDR approach, recognising the need for including people with disability in the research process from concept to outcome, and nurturing participation and collaboration between all the stakeholders in achieving action-based research. The research teams always included people with disability and staff from Disability People’s Organisations (DPOs) as well as academics and service providers. It endeavoured to generate and carry out research around issues that mattered to people with disability and their families. Leadership roles were assigned by team members. The objectives of the CARD programme were: (1) to fund teams to carry out action-based research on disability in Uganda; (2) to develop research and administrative capacity to manage the initiative within the academic registrar’s office at Kyambogo University; (3) to incorporate new knowledge generated from the studies into the ongoing local community-based rehabilitation and special education courses; and, (4) to ensure wide dissemination of research findings to all stakeholder groups.CARD ran for 5 years, commissioning 21 action research studies in the field of disability and community-based services. This paper describes the process, presents the 12 completed studies, examines the extent to which the objectives were achieved and evaluates the experiences of the participating research teams, particularly in relation to the inclusion of its members with disability. It concludes with recommendations for future initiatives designed to promote validity, good value and inclusive approaches in disability research.

Psychological research and clinical practice rely heavily on psychometric testing for measuring psychological constructs that represent symptoms of psychopathology, individual difference characteristics, or cognitive profiles. Test-retest reliability assessment is crucial in the development of psychometric tools, helping to ensure that measurement variation is due to replicable differences between people regardless of time, target behavior, or user profile. While psychological studies testing the reliability of measurement tools are pervasive in the literature, many still discuss and assess this form of reliability inappropriately with regard to the specified aims of the study or the intended use of the tool. The current paper outlines important factors to consider in test-retest reliability analyses, common errors, and some initial methods for conducting and reporting reliability analyses to avoid such errors. The paper aims to highlight a persistently problematic area in psychological assessment, to illustrate the real-world impact that these problems can have on measurement validity, and to offer relatively simple methods for improving the validity and practical use of reliability statistics.

The purpose of this study was to investigate the effects of dietary nitrate supplementation, in the form of beetroot juice, on acute mountain sickness (AMS) symptoms and physiological responses, in a group of young males trekking to Mount Everest Base Camp (EBC). Forty healthy male students (mean age (SD): 16 (1) yrs) trekked to EBC over 11 days. Following an overnight fast, each morning participants completed the Lake Louise AMS questionnaire and underwent a series of physiological tests: resting blood pressure as well as resting and exercising heart rate, respiratory rate, and peripheral oxygen saturation. The exercise test consisted of a standardised 2-min stepping protocol and measurements were taken in the last 10 s. Participants in the intervention arm of the study consumed 140 ml of concentrated beetroot juice daily, containing approximately 10 mmol of nitrate, while those in the control arm consumed 140 ml of concentrated blackcurrant cordial with negligible nitrate content. Drinks were taken for the first seven days at high altitude (days 2-8), in two equal doses; one with breakfast, and one with the evening meal. Mixed modelling revealed no significant between-groups difference in the incidence of AMS (Odds Ratio - nitrate vs. control: 1.16 (95% CI: 0.59; 2.29)). Physiological changes occurring during ascent to high altitude generally were not significantly different between the two groups (Model Coef (95% CI) - average difference nitrate vs. control: systolic blood pressure, 0.16 (-4.47; 4.79); peripheral oxygen saturation, 0.28 (-0.85; 1.41); heart rate, 0.48 (-8.47; 7.50) (Model Coef (95% CI) - relative difference nitrate vs. control: ventilatory rate, 0.95 (0.82; 1.08)). Modelling revealed that diastolic blood pressure was 3.37 mmHg (0.24; 6.49) higher for participants in the beetroot juice, however this difference was no larger than that found at baseline and no interaction effect was observed. Supplementation with dietary nitrate did not significantly change symptoms of AMS or alter key physiological variables, in a group of adolescent males during a high altitude trekking expedition. There was no evidence of harm from dietary nitrate supplementation in this context. Given the wide confidence intervals in all models, a larger sample size would be required to exclude a false negative result. Our data suggest that prolonged oral nitrate supplementation is safe and feasible at altitude but has little physiological or clinical effect. (C) 2016 Elsevier Inc. All rights reserved.

Objectives: Long term effects of exercise on FEV1, LCI, VO2Peak, 10m-MSWT and CFQ-R. Methods: Children aged 10±3 (mean±SD, range 6-15) yrs with average FEV1 of 86.6±15.3%pred were randomised by minimisation into control (n=34) or intervention groups (n=37). Both received specialist CF care. The intervention group also received weekly supervised exercise for 24-months. Results: Data for 68/71 were available. There was an average annual FEV1 decline of 1.7%pred in both groups and a 15% increase in VO2Peak, but no between-group differences. Significant between-group differences in 10m-MSWT favoured the intervention group. Perception of treatment burden reduced significantly in the intervention group. Increased physical and total-CFQ domains approached significance, with no between-groups differences in other CFQ-R domains. Conclusion: Despite modest deterioration in lung function over 2yrs, weekly supervised exercise significantly improved functional exercise capacity in the intervention group with quality of life benefits.

To compare the diagnostic yield of whole-body post-mortem computed tomography (PMCT) imaging to post-mortem magnetic resonance (PMMR) imaging in a prospective study of fetuses and children.

Knowledge about long-term variability of lung function in healthy children is essential when monitoring and treating those with respiratory disease over time. The aim of this study was to define the natural variability in spirometry in young children after an interval of 12 months.The Size and Lung function In Children study was a prospective study designed to assess spirometry and body size, shape and composition in a multi-ethnic population of London school children. 14 schools with a wide range of socioeconomic circumstances were recruited. Spirometric and anthropometric assessments and parental questionnaires pertaining to respiratory symptoms, previous medical history, pubertal status and socioeconomic circumstances were completed at baseline and ∼1 year later.Technically acceptable spirometry data on two occasions ∼1 year apart (range 9–16 months) were available in 758 children (39% boys, mean±sdage 8.1±1.6 years), 593 of whom were classified as “healthy”. Mean±sdwithin-subject between-test variability was 0.05±0.6 z-scores, with 95% of all the children achieving a between-test variability within ±1.2 z-scores (equating to ∼13% predicted).Natural variations of up to 1.2 z-scores occur in healthy children over ∼1 year. These must be considered when interpreting results from annual reviews in those with lung disease who are otherwise stable, if unnecessary further investigations or changes in treatment are to be avoided.