Video-based assessments of activities of daily living: generating real-world evidence in pediatric rare diseases.

INTRODUCTION: Preserving function and independence to perform activities of daily living (ADL) is critical for patients and carers to manage the burden of care and improve quality of life. In children living with rare diseases, video recording ADLs offer the opportunity to collect the patients' experience in a real-life setting and accurately reflect treatment effectiveness on outcomes that matter to patients and families. AREAS COVERED: We reviewed the measurement of ADL in pediatric rare diseases and the use of video to develop at-home electronic clinical outcome assessments (eCOA) by leveraging smartphone apps and artificial intelligence-based analysis. We broadly searched PubMed using Boolean combinations of the following MeSH terms 'Rare Diseases,' 'Quality of Life,' 'Activities of Daily Living,' 'Child,' 'Video Recording,' 'Outcome Assessment, Healthcare,' 'Intellectual disability,' and 'Genetic Diseases, Inborn.' Non-controlled vocabulary was used to include human pose estimation in movement analysis. EXPERT OPINION: Broad uptake of video eCOA in drug development is linked to the generation of technical and clinical validation evidence to confidently assess a patient's functional abilities. Software platforms handling video data must align with quality regulations to ensure data integrity, security, and privacy. Regulatory flexibility and optimized validation processes should facilitate video eCOA to support benefit/risk drug assessment.

Erratum: Author Correction: Using a smartphone-based self-management platform to support medication adherence and clinical consultation in Parkinson's disease.

[This corrects the article DOI: 10.1038/s41531-016-0003-z.].

Recruitment and Ongoing Engagement in a UK Smartphone Study Examining the Association Between Weather and Pain: Cohort Study.

BACKGROUND: The huge increase in smartphone use heralds an enormous opportunity for epidemiology research, but there is limited evidence regarding long-term engagement and attrition in mobile health (mHealth) studies. OBJECTIVE: The objective of this study was to examine how representative the Cloudy with a Chance of Pain study population is of wider chronic-pain populations and to explore patterns of engagement among participants during the first 6 months of the study. METHODS: Participants in the United Kingdom who had chronic pain (≥3 months) and enrolled between January 20, 2016 and January 29, 2016 were eligible if they were aged ≥17 years and used the study app to report any of 10 pain-related symptoms during the study period. Participant characteristics were compared with data from the Health Survey for England (HSE) 2011. Distinct clusters of engagement over time were determined using first-order hidden Markov models, and participant characteristics were compared between the clusters. RESULTS: Compared with the data from the HSE, our sample comprised a higher proportion of women (80.51%, 5129/6370 vs 55.61%, 4782/8599) and fewer persons at the extremes of age (16-34 and 75+). Four clusters of engagement were identified: high (13.60%, 865/6370), moderate (21.76%, 1384/6370), low (39.35%, 2503/6370), and tourists (25.44%, 1618/6370), between which median days of data entry ranged from 1 (interquartile range; IQR: 1-1; tourist) to 149 (124-163; high). Those in the high-engagement cluster were typically older, whereas those in the tourist cluster were mostly male. Few other differences distinguished the clusters. CONCLUSIONS: Cloudy with a Chance of Pain demonstrates a rapid and successful recruitment of a large, representative, and engaged sample of people with chronic pain and provides strong evidence to suggest that smartphones could provide a viable alternative to traditional data collection methods.

Community health promotion and medical provision for neonatal health-CHAMPION cluster randomised trial in Nagarkurnool district, Telangana (formerly Andhra Pradesh), India.

BACKGROUND: In the mid-2000s, neonatal mortality accounted for almost 40% of deaths of children under 5 years worldwide, and constituted 65% of infant deaths in India. The neonatal mortality rate in Andhra Pradesh was 44 per 1,000 live births, and was higher in the rural areas and tribal regions, such as the Nagarkurnool division of Mahabubnagar district (which became Nagarkurnool district in Telangana in 2014). The aim of the CHAMPION trial was to investigate whether a package of interventions comprising community health promotion and provision of health services (including outreach and facility-based care) could lead to a reduction of the order of 25% in neonatal mortality. METHODS AND FINDINGS: The design was a trial in which villages (clusters) in Nagarkurnool with a population < 2,500 were randomised to the CHAMPION package of health interventions or to the control arm (in which children aged 6-9 years were provided with educational interventions-the STRIPES trial). A woman was eligible for the CHAMPION package if she was married and <50 years old, neither she nor her husband had had a family planning operation, and she resided in a trial village at the time of a baseline survey before randomisation or married into the village after randomisation. The CHAMPION intervention package comprised community health promotion (including health education via village health worker-led participatory discussion groups) and provision of health services (including outreach, with mobile teams providing antenatal check-ups, and facility-based care, with subsidised access to non-public health centres [NPHCs]). Villages were stratified by travel time to the nearest NPHC and tribal status, and randomised (1:1) within strata. The primary outcome was neonatal mortality. Secondary outcomes included maternal mortality, causes of death, health knowledge, health practices including health service usage, satisfaction with care, and costs. The baseline survey (enumeration) was carried out between August and November 2007. After randomisation on 18 February 2008, participants, data collectors, and data analysts were not masked to allocation. The intervention was initiated on 1 August 2008. After an inception period, the assessment start date was 1 December 2008. The intervention ended on 31 May 2011, and data collection was completed on 30 November 2011. Primary analyses followed the intention to treat principle. In all, 14,137 women were enrolled in 232 control villages, and 15,532 in 232 intervention villages. Of these, 4,885 control women had 5,474 eligible pregnancies and gave birth to 4,998 eligible children. The corresponding numbers in intervention villages were 5,664 women, 6,351 pregnancies, and 5,798 children. Of the live-born babies, 343 (6.9%) in the control arm and 303 (5.2%) in the intervention arm died in their first 28 days of life (risk ratio 0.76, 95% CI 0.64 to 0.90, p = 0.0018; risk difference -1.59%, 95% CI -2.63% to -0.54%), suggesting that there were 92 fewer deaths (95% CI 31 to 152) as a result of the intervention. There were 9 (0.16%) maternal deaths in the control arm compared to 13 (0.20%) in the intervention arm (risk ratio 1.24, 95% CI 0.53 to 2.90, p = 0.6176; 1 death was reported as a serious adverse event). There was evidence of improved health knowledge and health practices including health service usage in the intervention arm compared to the control arm. Women in the intervention arm were more likely to rate their delivery and postnatal care as good or very good. The total cost of the CHAMPION interventions was US$1,084,955 ($11,769 per life saved, 95% CI $7,115 to $34,653). The main limitations of the study included that it could not be masked post-randomisation and that fetal losses were not divided into stillbirths and miscarriages because gestational age was not reliably reported. CONCLUSIONS: The CHAMPION trial showed that a package of interventions addressing health knowledge and health seeking behaviour, buttressing existing health services, and contracting out important areas of maternal and child healthcare led to a reduction in neonatal mortality of almost the hypothesized 25% in small villages in an Indian state with high mortality rates. The intervention can be strongly justified in much of rural India, and is of potential use in other similar settings. Ongoing changes in maternal and child health programmes make it imperative that a similar intervention that establishes ties between the community and health facilities is tested in different settings. TRIAL REGISTRATION: ISRCTN registry ISRCTN24104646.

Using a smartphone-based self-management platform to support medication adherence and clinical consultation in Parkinson's disease.

The progressive nature of Parkinson's disease, its complex treatment regimens and the high rates of comorbid conditions make self-management and treatment adherence a challenge. Clinicians have limited face-to-face consultation time with Parkinson's disease patients, making it difficult to comprehensively address non-adherence. Here we share the results from a multi-centre (seven centres) randomised controlled trial conducted in England and Scotland to assess the impact of using a smartphone-based Parkinson's tracker app to promote patient self-management, enhance treatment adherence and quality of clinical consultation. Eligible Parkinson's disease patients were randomised using a 1:1 ratio according to a computer-generated random sequence, stratified by centre and using blocks of variable size, to intervention Parkinson's Tracker App or control (Treatment as Usual). Primary outcome was the self-reported score of adherence to treatment (Morisky medication adherence scale -8) at 16 weeks. Secondary outcomes were Quality of Life (Parkinson's disease questionnaire -39), quality of consultation for Parkinson's disease patients (Patient-centred questionnaire for Parkinson's disease), impact on non-motor symptoms (Non-motor symptoms questionnaire), depression and anxiety (Hospital anxiety and depression scale) and beliefs about medication (Beliefs about Medication Questionnaire) at 16 weeks. Primary and secondary endpoints were analysed using a generalised linear model with treatment as the fixed effect and baseline measurement as the covariate. 158 patients completed the study (Parkinson's tracker app = 68 and TAU = 90). At 16 weeks Parkinson's tracker app significantly improved adherence, compared to treatment as usual (mean difference: 0.39, 95%CI 0.04-0.74; p = 0.0304) with no confounding effects of gender, number of comorbidities and age. Among secondary outcomes, Parkinson's tracker app significantly improved patients' perception of quality of consultation (0.15, 95% CI 0.03 to 0.27; p = 0.0110). The change in non-motor symptoms was -0.82 (95% CI -1.75 to 0.10; p = 0.0822). 72% of participants in the Parkinson's tracker app group continued to use and engage with the application throughout the 16-week trial period. The Parkinson's tracker app can be an effective and novel way of enhancing self-reported medication adherence and quality of clinical consultation by supporting self-management in Parkinson's disease in patients owning smartphones. Further work is recommended to determine whether the benefits of the intervention are maintained beyond the 16 week study period.

Cloudy with a Chance of Pain: Engagement and Subsequent Attrition of Daily Data Entry in a Smartphone Pilot Study Tracking Weather, Disease Severity, and Physical Activity in Patients With Rheumatoid Arthritis.

BACKGROUND: The increasing ownership of smartphones provides major opportunities for epidemiological research through self-reported and passively collected data. OBJECTIVE: This pilot study aimed to codesign a smartphone app to assess associations between weather and joint pain in patients with rheumatoid arthritis (RA) and to study the success of daily self-reported data entry over a 60-day period and the enablers of and barriers to data collection. METHODS: A patient and public involvement group (n=5) and 2 focus groups of patients with RA (n=9) supported the codesign of the app collecting self-reported symptoms. A separate "capture app" was designed to collect global positioning system (GPS) and continuous raw accelerometer data, with the GPS-linking providing local weather data. A total of 20 patients with RA were then recruited to collect daily data for 60 days, with entry and exit interviews. Of these, 17 were loaned an Android smartphone, whereas 3 used their own Android smartphones. RESULTS: Of the 20 patients, 6 (30%) withdrew from the study: 4 because of technical challenges and 2 for health reasons. The mean completion of daily entries was 68% over 2 months. Patients entered data at least five times per week 65% of the time. Reasons for successful engagement included a simple graphical user interface, automated reminders, visualization of data, and eagerness to contribute to this easily understood research question. The main barrier to continuing engagement was impaired battery life due to the accelerometer data capture app. For some, successful engagement required ongoing support in using the smartphones. CONCLUSIONS: This successful pilot study has demonstrated that daily data collection using smartphones for health research is feasible and achievable with high levels of ongoing engagement over 2 months. This result opens important opportunities for large-scale longitudinal epidemiological research.

Smartphone- and internet-assisted self-management and adherence tools to manage Parkinson's disease (SMART-PD): study protocol for a randomised controlled trial (v7; 15 August 2014).

BACKGROUND: Nonadherence to treatment leads to suboptimal treatment outcomes and enormous costs to the economy. This is especially important in Parkinson's disease (PD). The progressive nature of the degenerative process, the complex treatment regimens and the high rates of comorbid conditions make treatment adherence in PD a challenge. Clinicians have limited face-to-face consultation time with PD patients, making it difficult to comprehensively address non-adherence. The rapid growth of digital technologies provides an opportunity to improve adherence and the quality of decision-making during consultation. The aim of this randomised controlled trial (RCT) is to evaluate the impact of using a smartphone and web applications to promote patient self-management as a tool to increase treatment adherence and working with the data collected to enhance the quality of clinical consultation. METHODS/DESIGN: A 4-month multicentre RCT with 222 patients will be conducted to compare use of a smartphone- and internet-enabled Parkinson's tracker smartphone app with treatment as usual for patients with PD and/or their carers. The study investigators will compare the two groups immediately after intervention. Seven centres across England (6) and Scotland (1) will be involved. The primary objective of this trial is to assess whether patients with PD who use the app show improved medication adherence compared to those receiving treatment as usual alone. The secondary objectives are to investigate whether patients who receive the app and those who receive treatment as usual differ in terms of quality of life, quality of clinical consultation, overall disease state and activities of daily living. We also aim to investigate the experience of those receiving the intervention by conducting qualitative interviews with a sample of participants and clinicians, which will be administered by independent researchers. TRIAL REGISTRATION: ISRCTN45824264 (registered 5 November 2013).

The Support to Rural India's Public Education System (STRIPES) trial: a cluster randomised controlled trial of supplementary teaching, learning material and material support.

BACKGROUND: The aim of the STRIPES trial was to assess the effectiveness of providing supplementary, remedial teaching and learning materials (and an additional 'kit' of materials for girls) on a composite of language and mathematics test scores for children in classes two, three and four in public primary schools in villages in the Nagarkurnool division of Andhra Pradesh, India. METHODS: STRIPES was a cluster randomised trial in which 214 villages were allocated either to the supplementary teaching intervention (n = 107) or to serve as controls (n = 107). 54 of the intervention villages were further randomly allocated to receive additional kit for girls. The study was not blinded. Analysis was conducted on the intention to treat principle, allowing for clustering. RESULTS: Composite test scores were significantly higher in the intervention group (107 villages; 2364 children) than in the control group (106 villages; 2014 children) at the end of the trial (mean difference on a percentage scale 15.8; 95% CI 13.1 to 18.6; p<0.001; 0.75 Standard Deviation (SD) difference). Composite test scores were not significantly different in the 54 villages (614 girls) with the additional kits for girls compared to the 53 villages (636 girls) without these kits at the end of the trial (mean difference on a percentage scale 0.5; 95% CI -4.34 to 5.4; p = 0.84). The cost per 0.1 SD increase in composite test score for intervention without kits is Rs. 382.97 (£4.45, $7.13), and Rs.480.59 (£5.58, $8.94) for the intervention with kits. CONCLUSIONS: A 18 month programme of supplementary remedial teaching and learning materials had a substantial impact on language and mathematics scores of primary school students in rural Andhra Pradesh, yet providing a 'kit' of materials to girls in these villages did not lead to any measured additional benefit. TRIAL REGISTRATION: Controlled-Trials.com ISRCTN69951502.

Predictors of maternal psychological distress in rural India: a cross-sectional community-based study.

BACKGROUND: Maternal common mental disorders are prevalent in low-resource settings and have far-reaching consequences for maternal and child health. We assessed the prevalence and predictors of psychological distress as a proxy for common mental disorders among mothers in rural Jharkhand and Orissa, eastern India, where over 40% of the population live below the poverty line and access to reproductive and mental health services is low. METHOD: We screened 5801 mothers around 6 weeks after delivery using the Kessler-10 item scale, and identified predictors of distress using multiple hierarchical logistic regression. RESULTS: 11.5% (95% CI: 10.7-12.3) of mothers had symptoms of distress (K10 score >15). High maternal age, low asset ownership, health problems in the antepartum, delivery or postpartum periods, caesarean section, an unwanted pregnancy for the mother, small perceived infant size and a stillbirth or neonatal death were all independently associated with an increased risk of distress. The loss of an infant or an unwanted pregnancy increased the risk of distress considerably (AORs: 7.06 95% CI: 5.51-9.04 and 1.49, 95% CI: 1.12-1.97, respectively). LIMITATIONS: We did not collect data on antepartum depression, domestic violence or a mother's past birth history, and were therefore unable to examine the importance of these factors as predictors of psychological distress. CONCLUSIONS: Mothers living in underserved areas of India who experience infant loss, an unwanted pregnancy, health problems in the perinatal and postpartum periods and socio-economic disadvantage are at increased risk of distress and require access to reproductive healthcare with integrated mental health interventions.

Effect of a participatory intervention with women's groups on birth outcomes and maternal depression in Jharkhand and Orissa, India: a cluster-randomised controlled trial.

BACKGROUND: Community mobilisation through participatory women's groups might improve birth outcomes in poor rural communities. We therefore assessed this approach in a largely tribal and rural population in three districts in eastern India. METHODS: From 36 clusters in Jharkhand and Orissa, with an estimated population of 228 186, we assigned 18 clusters to intervention or control using stratified randomisation. Women were eligible to participate if they were aged 15-49 years, residing in the project area, and had given birth during the study. In intervention clusters, a facilitator convened 13 groups every month to support participatory action and learning for women, and facilitated the development and implementation of strategies to address maternal and newborn health problems. The primary outcomes were reductions in neonatal mortality rate (NMR) and maternal depression scores. Analysis was by intention to treat. This trial is registered as an International Standard Randomised Controlled Trial, number ISRCTN21817853. FINDINGS: After baseline surveillance of 4692 births, we monitored outcomes for 19 030 births during 3 years (2005-08). NMRs per 1000 were 55.6, 37.1, and 36.3 during the first, second, and third years, respectively, in intervention clusters, and 53.4, 59.6, and 64.3, respectively, in control clusters. NMR was 32% lower in intervention clusters adjusted for clustering, stratification, and baseline differences (odds ratio 0.68, 95% CI 0.59-0.78) during the 3 years, and 45% lower in years 2 and 3 (0.55, 0.46-0.66). Although we did not note a significant effect on maternal depression overall, reduction in moderate depression was 57% in year 3 (0.43, 0.23-0.80). INTERPRETATION: This intervention could be used with or as a potential alternative to health-worker-led interventions, and presents new opportunities for policy makers to improve maternal and newborn health outcomes in poor populations. FUNDING: Health Foundation, UK Department for International Development, Wellcome Trust, and the Big Lottery Fund (UK).

The STRIPES trial--support to rural India's public education system.

BACKGROUND: Performance of primary school students in India lags far below government expectations, and major disparity exists between rural and urban areas. The Naandi Foundation has designed and implemented a programme using community members to deliver after-school academic support for children in over 1,100 schools in five Indian states. Assessments to date suggest that it might have a substantial effect. This trial aims to evaluate the impact of this programme in villages of rural Andhra Pradesh and will compare test scores for children in three arms: a control and two intervention arms. In both intervention arms additional after-school instruction and learning materials will be offered to all eligible children and in one arm girls will also receive an additional 'kit' with a uniform and clothes. METHODS/DESIGN: The trial is a cluster-randomised controlled trial conducted in conjunction with the CHAMPION trial. In the CHAMPION trial 464 villages were randomised so that half receive health interventions aiming to reduce neonatal mortality. STRIPES will be introduced in those CHAMPION villages which have a public primary school attended by at least 15 students at the time of a baseline test in 2008. 214 villages of the 464 were found to fulfil above criteria, 107 belonging to the control and 107 to the intervention arm of the CHAMPION trial. These latter 107 villages will serve as control villages in the STRIPES trial. A further randomisation will be carried out within the 107 STRIPES intervention villages allocating half to receive an additional kit for girls on the top of the instruction and learning materials. The primary outcome of the trial is a composite maths and language test score. DISCUSSION: The study is designed to measure (i) whether the educational intervention affects the exam score of children compared to the control arm, (ii) if the exam scores of girls who receive the additional kit are different from those of girls living in the other STRIPES intervention arm. One of the goals of the STRIPES trial is to provide benefit to the controls of the CHAMPION trial. We will also conduct a cost-benefit analysis in which we calculate the programme cost for 0.1 standard deviation improvement for both intervention arms. TRIAL REGISTRATION: Current controlled trials ISRCTN69951502.

Mental health consequences of war: a brief review of research findings.

Among the consequences of war, the impact on the mental health of the civilian population is one of the most significant. Studies of the general population show a definite increase in the incidence and prevalence of mental disorders. Women are more affected than men. Other vulnerable groups are children, the elderly and the disabled. Prevalence rates are associated with the degree of trauma, and the availability of physical and emotional support. The use of cultural and religious coping strategies is frequent in developing countries.

Is it possible to carry out high-quality epidemiological research in psychiatry with limited resources?

PURPOSE OF REVIEW: This review covers the epidemiological research from developing countries to identify the scope of research in the various aspects of epidemiology and the strengths of research. The period covered is June 2004 to June 2005. The literature survey used searches of Medline, key psychiatric journals and personal correspondence with leading psychiatric researchers from developing countries. RECENT FINDINGS: There is a new interest in epidemiological studies in developing countries. These studies have been driven by three factors, namely international collaboration; specific situations like disasters, terrorism and severe acute respiratory syndrome; and international attention to specific topics like maternal depression. There is limited long-term research on specific conditions. There are a number of leads suggesting local social-cultural factors contribute to the distribution of psychiatric problems. Child psychiatry continues to be low in priority. There is also lack of research in areas such as personality disorders and organic psychiatric disorders. SUMMARY: It is possible to carry out high quality epidemiological research in developing countries. Such research has provided new insights into the distribution, causation, course and outcome of mental disorders. There is need for greater attention to the development of epidemiological assessment tools to suit local conditions. Specific centers/institutions developing long-term research interests on specific subjects would be valuable for future efforts. There is also need to widen the conditions to be studied.