Electronic personal assessment questionnaire for vascular conditions (ePAQ-VAS): development and validity.

BACKGROUND: This paper describes the development and validation of an electronic personal assessment questionnaire for vascular conditions (ePAQ-VAS) that captures the symptomatology, quality of life and clinically relevant data of patients presenting to vascular services. METHODS: A two-stage survey was conducted in patients attending a tertiary vascular department. Patients completed the ePAQ-VAS remotely online, or on site using an electronic tablet. In the first stage of the survey, the responses were used to perform confirmatory factor analysis to assess the construct validity and remove redundant items. The internal reliability of disease-specific scales was investigated. In the second stage of the survey, the acceptability, known-group validity, test-retest reliability, and responsiveness of ePAQ-VAS was assessed. RESULTS: In total, 721 patients completed ePAQ-VAS. Their mean(s.d.) age was 63·5(15·7) years and 468 (64·9 per cent) were men. Some 553 patients (76·7 per cent) completed the questionnaire in clinic and the remainder completed the questionnaire online. The results of the confirmatory factor analysis confirmed the conceptual model for ePAQ-VAS structure and eliminated six items. Internal reliability was acceptable for all the scales (Cronbach's α greater than 0·7). The test-retest reliability measured by the intraclass correlation coefficient ranged from 0·65 to 0·99. The results showed that the instrument was responsive over time with the standardized response mean ranging from 0·69 to 1·60. CONCLUSION: ePAQ-VAS is a holistic data-collection process that is relevant to vascular service users and has potential to contribute to patient-focused care and the collection of aggregate data for service evaluation. A demonstration version of the final version of ePAQ can be viewed at http://demo-questionnaire.epaq.co.uk/home/project?id=VASC_1.7&page=1.

ANTECEDENTES: Este artículo describe el desarrollo y la validación de un cuestionario electrónico de evaluación personal para enfermedades vasculares (ePAQ-VAS) que incluye la sintomatología, la calidad de vida y los datos clínicamente relevantes para los pacientes que son atendidos en los servicios de patología vascular. MÉTODOS: Se llevó a cabo una encuesta en dos fases entre los pacientes atendidos en un servicio de patología vascular de tercer nivel. Los pacientes completaron el cuestionario ePAQ-VAS a distancia en línea o bien en el centro hospitalario mediante una tableta electrónica. En la primera fase de la encuesta, las respuestas se utilizaron para realizar un análisis factorial de confirmación para evaluar la validez del diseño y eliminar los elementos redundantes. Se investigó la fiabilidad interna de las escalas específicas de la enfermedad. En la segunda fase de la encuesta, se evaluó la aceptabilidad, la validez de grupo conocida, la fiabilidad test-retest y la capacidad de respuesta del ePAQ-VAS. RESULTADOS: En total, 721 pacientes completaron el ePAQ-VAS, la edad media fue de 63,5 años (DE 15,7); el 64,9% eran varones (468); el 76% de los pacientes (553) completaron el cuestionario en la clínica y los pacientes restantes lo hicieron electrónicamente a distancia. Los resultados del análisis factorial de confirmación confirmaron el modelo conceptual para la estructura ePAQ-VAS y eliminaron seis ítems. La fiabilidad interna fue aceptable para todas las escalas (alfa de Cronbach > 0,7). La fiabilidad test-retest medida por el coeficiente de correlación intraclase osciló entre 0,65-0,99. Los resultados mostraron que el instrumento responde con el tiempo con una media de respuesta estandarizada que varía de 0,69 a 1,60. CONCLUSIÓN: El ePAQ-VAS es un proceso holístico de recopilación de datos que es relevante para los usuarios de servicios de patología vascular y tiene el potencial de contribuir a la atención centrada en el paciente y a la recopilación de datos agregados para la evaluación del servicio.

Strength of public preferences for endovascular or open aortic aneurysm repair.

BACKGROUND: This study evaluated public preferences for the treatment processes for abdominal aortic aneurysm repair in order to allow them to be incorporated into a cost-effectiveness analysis. METHODS: This was a telephone survey using a trade-off method in UK resident adults (aged at least 18 years) with no previous diagnosis of a vascular condition. RESULTS: Some 167 of 209 participants (79·9 per cent) stated that they would prefer endovascular aneurysm repair (EVAR), 40 (19·1 per cent) preferred open surgery and two (1·0 per cent) stated no preference. Participants preferred EVAR because of the less invasive nature of the intervention and quicker recovery. Participants preferring open surgery cited reasons such as having a single follow-up appointment, and a procedure that felt more permanent. When participants were asked to make a sacrifice in order to have their preferred treatment, 122 (58·4 per cent) favoured EVAR, 18 (8·6 per cent) favoured open surgery and 69 (33·0 per cent) had no preference. Those preferring EVAR were willing to give up a mean of 0·135 expected quality-adjusted life-years (QALYs) to have EVAR, compared with a willingness to give up 0·033 expected QALYs among those preferring open repair. CONCLUSION: These results indicate a clear preference for EVAR over open surgery for aortic aneurysm.

ANTECEDENTES: Este estudio evaluó las preferencias de la opinion pública en relación a las opciones de tratamiento para la reparación del aneurisma de aorta abdominal, con el objetivo de que dichas preferencias se puedan incorporar en un análisis de coste-efectividad. MÉTODOS: Se realizó una encuesta telefónica utilizando el método trade-off (solución de intercambio) en adultos residentes en el Reino Unido (mayores de 18 años) sin diagnóstico previo de enfermedad vascular. RESULTADOS: Un total de 167 (79,9%) de 209 participantes declararon que preferirían la reparación endovascular del aneurisma (endovascular aneurysm repair, EVAR), 40 (19,1%) prefirieron cirugía abierta y dos (1,0%) no tenían preferencia. Los participantes prefirieron el EVAR debido a la naturaleza menos invasiva de la intervención y a tiempos de recuperación más rápidos. Los participantes que preferían la cirugía abierta mencionaron como razones tener una única visita de seguimiento y consideraron que se trataba de un procedimiento más permanente. Cuando se pidió a los participantes que para recibir su tratamiento preferido hicieran un intercambio, 122 (58,4%) se decantaron por la EVAR, 18 (8,6%) por la cirugía abierta y 69 (33%) no tuvieron preferencia. Los que prefirieron EVAR estaban dispuestos a renunciar a una media de 0,135 años de vida ajustados por calidad (QALYs) esperados con tal de recibir una EVAR en comparación con la renuncia de 0,033 QALYs esperada entre quienes preferían la reparación abierta. CONCLUSIÓN: Estos resultados indican una clara preferencia por la EVAR sobre la cirugía abierta, lo que está en desacuerdo con la reciente recomendación de NICE de que la EVAR no debe recomendarse como una opción de tratamiento. Los hallazgos sugieren que se debe prestar mayor atención a las características del proceso de tratamiento. Al no incorporar explícitamente tales preferencias en el proceso de toma de decisiones, NICE corre el riesgo de recomendar opciones de tratamiento que son contrarias a las preferencias de la población del Reino Unido.

Trabecular microstructure is influenced by race and sex in Black and White young adults.

Lower fracture rates in Black men and women compared to their White counterparts are incompletely understood. High-resolution imaging specific to trabecular bone may provide insight. Black participants have enhanced trabecular morphology. These differences may contribute to the lower fracture risk in Black versus White individuals. INTRODUCTION: Lower fracture rates in Black men and women compared to their White counterparts may be explained by favorable bone microstructure in Black individuals. Individual trabecular segmentation (ITS) analysis, which characterizes the alignment and plate- and rod-like nature of trabecular bone using high-resolution peripheral quantitative computed tomography (HR-pQCT), may provide insight into trabecular differences by race/ethnic origin. PURPOSE: We determined differences in trabecular bone microarchitecture, connectivity, and alignment according to race/ethnic origin and sex in young adults. METHODS: We analyzed HR-pQCT scans of 184 adult (24.2 ± 3.4 years) women (n = 51 Black, n = 50 White) and men (n = 34 Black, n = 49 White). We used ANCOVA to compare bone outcomes, and adjusted for age, height, and weight. RESULTS: Overall, the effect of race on bone outcomes did not differ by sex, and the effect of sex on bone outcomes did not differ by race. After adjusting for covariates, Black participants and men of both races had greater trabecular plate volume fraction, plate thickness, plate number density, plate surface area, and greater axial alignment of trabeculae, leading to higher trabecular bone stiffness compared to White participants and women, respectively (p < 0.05 for all). CONCLUSION: These findings demonstrate that more favorable bone microarchitecture in Black individuals compared to White individuals and in men compared to women is not unique to the cortical bone compartment. Enhanced plate-like morphology and greater trabecular axial alignment, established in young adulthood, may contribute to the improved bone strength and lower fracture risk in Black versus White individuals and in men compared to women.

Incorporating indigenous knowledge in health services: a consumer partnership framework.

OBJECTIVES: Healthcare policy and planning should be informed by a partnership between healthcare services and healthcare users. This is critical for people who access care frequently such as indigenous Australians who have a high burden of chronic kidney disease. This study aimed to explore the most appropriate ways of enhancing services by incorporating renal patients' expectations and satisfaction of care in Australia's Northern Territory. STUDY DESIGN: This is a participatory action research. METHODS: Six aboriginal health users with end-stage kidney disease were recruited to form an Indigenous Reference Group. This group met bimonthly between April and November 2017 and meetings took the same structure as a focus group. Findings from these meetings were presented to health policy and planners in a feedback loop implemented by the study. RESULTS: This framework enabled indigenous knowledge to guide the project, indigenous priorities to be identified in this context and timely feedback of information to inform the strengths and priorities of the health service. Changes were recognised and addressed immediately. CONCLUSIONS: This qualitative research framework is a useful mechanism for providing local data to inform patient-centred health system change as expressed by health users. We recommend this consumer partnership framework be embedded into existing operational structures to support the ongoing sustainability of this group.

Examples of bedaquiline introduction for the management of multidrug-resistant tuberculosis in five countries.

BACKGROUND: For the first time in almost 50 years, there are new drugs available for the treatment of tuberculosis (TB), including bedaquiline (BDQ) and delamanid (DLM). The rate of introduction, however, has not kept pace with patient needs. It is estimated that as many as 23% of multidrug-resistant TB (MDR-TB) patients have an indication for receiving BDQ. As this is the first time the MDR-TB community is introducing new medications, it is important to understand how implementation can be developed in a variety of settings. METHODS: A qualitative assessment of country TB programs in which more than 5% of MDR-TB patients were started on BDQ under program conditions. RESULTS: National TB programs in Belarus, France, Georgia, South Africa, and Swaziland all started sizeable cohorts of patients on BDQ in 2015. Common factors observed in these programs included experience with compassionate use/expanded access, support from implementing partners, and adequate national or donor-supported budgets. Barriers to introduction included restriction of BDQ to the in-patient setting, lack of access to companion drugs, and the development of systems for pharmacovigilance. CONCLUSION: The five countries in this paper are examples of the introduction of new therapeutic options for the treatment of TB.

A comparative investigation of methods for longitudinal data with limits of detection through a case study.

The statistical analysis of continuous longitudinal data may be complicated since quantitative levels of bioassay cannot always be determined. Values beyond the limits of detection (LOD) in the assays may not be observed and thus censored, rendering complexity to the analysis of such data. This article examines how both left-censoring and right censoring of HIV-1 plasma RNA measurements, collected for the study on AIDS-related Non-Hodgkin's lymphoma (AR-NHL) in East Africa, affects the quantification of viral load and explores the natural history of viral load measurements over time in AR-NHL patients receiving anticancer chemotherapy. Data analyses using Monte Carlo EM algorithm (MCEM) are compared to analyses where the LOD or LOD/2 (left censoring) value is substituted for the censored observations, and also to other methods such as multiple imputation, and maximum likelihood estimation for censored data (generalized Tobit regression). Simulations are used to explore the sensitivity of the results to changes in the model parameters. In conclusion, the antiretroviral treatment was associated with a significant decrease in viral load after controlling the effects of other covariates. A simulation study with finite sample size shows MCEM is the least biased method and the estimates are least sensitive to the censoring mechanism.

Pragmatic staging of oesophageal cancer using decision theory involving selective endoscopic ultrasonography, PET and laparoscopy.

BACKGROUND: Following CT, guidelines for staging oesophageal and gastro-oesophageal junction (GOJ) cancer recommend endoscopic ultrasonography (EUS), PET-CT and laparoscopy for T3-T4 GOJ tumours. These recommendations are based on generic utilities, but it is unclear whether the test risk outweighs the potential benefit for some patients. This study sought to quantify investigation risks, benefits and utilities, in order to develop pragmatic, personalized staging recommendations. METHODS: All patients with a histological diagnosis of oesophageal or GOJ cancer staged between May 2006 and July 2013 comprised a development set; those staged from July 2013 to July 2014 formed the prospective validation set. Probability thresholds of altering management were calculated and predictive factors identified. Algorithms and models (decision tree analysis, logistic regression, artificial neural networks) were validated internally and independently. RESULTS: Some 953 patients were staged following CT, by [(18) F]fluorodeoxyglucose PET-CT (918), EUS (798) and laparoscopy (458). Of these patients, 829 comprised the development set (800 PET-CT, 698 EUS, 397 laparoscopy) and 124 the validation set (118 PET-CT, 100 EUS, 61 laparoscopy). EUS utility in the 71.8 per cent of patients with T2-T4a disease on CT was minimal (0.4 per cent), its risk exceeding benefit. EUS was moderately accurate for pT1 N0 disease. A number of factors predicted metastases on PET-CT and laparoscopy, although none could inform an algorithm. PET-CT altered management in 23.0 per cent, and laparoscopy in 7.1 per cent, including those with T2 and distal oesophageal tumours. CONCLUSION: Although EUS provided additional information on T and N category, its risk outweighed potential benefit in patients with T2-T4a disease on CT. Laparoscopy seemed justified for distal oesophageal tumours of T2 or greater.

Comparing the decline in coronary heart disease and stroke mortality in neighbouring countries with different healthcare systems.

OBJECTIVE: To examine age and gender specific trends in coronary heart disease (CHD) and stroke mortality in two neighbouring countries, the Republic of Ireland (ROI) and Northern Ireland (NI). DESIGN: Epidemiological study of time trends in CHD and stroke mortality. SETTING/PATIENTS: The populations of the ROI and NI, 1985-2010. INTERVENTIONS: None. MAIN OUTCOME MEASURES: Directly age standardised CHD and stroke mortality rates were calculated and analysed using joinpoint regression to identify years where the slope of the linear trend changed significantly. This was performed separately for specific age groups (25-54, 55-64, 65-74 and 75-84 years) and by gender. Annual percentage change (APC) and 95% CIs are presented. RESULTS: There was a striking similarity between the two countries, with percentage change between 1985 and 1989 and between 2006 and 2010 of 67% and 69% in CHD mortality, and 64% and 62% in stroke mortality for the ROI and NI, respectively. However, joinpoint analysis identified differences in the pace of change between the two countries. There was an accelerated pace of decline (negative APC) in mortality for both CHD and stroke in both countries from the mid-1990s (APC ROI -8% (95% CI -9.5 to 6.5) and NI -6.6% (-6.9 to -6.3)), but the accelerated decrease started later for CHD mortality in the ROI. In recent years, a levelling off in CHD mortality was observed in the 25-54 year age group in NI and in stroke mortality for men and women in the ROI. CONCLUSIONS: While differences in the pace of change in mortality were observed at different time points, similar, substantial decreases in CHD and stroke mortality were achieved between 1985 and 1989 and between 2006 and 2010 in the ROI and NI despite important differences in health service structures. There is evidence of a levelling in mortality rates in some groups in recent years.

The effectiveness and cost-effectiveness of acupressure for the control and management of chemotherapy-related acute and delayed nausea: Assessment of Nausea in Chemotherapy Research (ANCHoR), a randomised controlled trial.

BACKGROUND: Chemotherapy-induced nausea and vomiting remain difficult symptoms to manage in clinical practice. As standard antiemetic drugs do not fully eliminate these symptoms, it is important to explore the adjuvant role of non-pharmacological and complementary therapies in antiemetic management approaches. Acupressure is one such treatment showing highly suggestive evidence so far of a positive effect, meriting further investigation. OBJECTIVES: The primary objective was to assess the effectiveness and cost-effectiveness of self-acupressure using wristbands compared with sham acupressure wristbands and standard care alone in the management of chemotherapy-induced nausea. Secondary objectives included assessment of the effectiveness and cost-effectiveness of the wristbands in relation to vomiting and quality of life and exploration of any age, gender and emetogenic risk effects. DESIGN: Randomised three-arm sham-controlled trial (Assessment of Nausea in Chemotherapy Research or ANCHoR) with an economic evaluation. Arms include the wristband arm, the sham wristband arm and the standard care only arm. Randomisation consisted of minimisation with a random element balancing for gender, age (16-24, > 24-50, >50 years) and three levels of emetogenic chemotherapy (low, moderate and high). Qualitative interviews were incorporated to shed more light on the quantitative findings. SETTING: Outpatient chemotherapy clinics in three regions in the UK involving 14 different cancer units/centres. PARTICIPANTS: Chemotherapy-naive cancer patients receiving chemotherapy of low, moderate and high emetogenic risk. INTERVENTION: The intervention was acupressure wristbands pressing the P6 point (anterior surface of the forearm). MAIN OUTCOME MEASURES: The Rhodes Index for Nausea/Vomiting, the Multinational Association of Supportive Care in Cancer (MASCC) Antiemesis Tool and the Functional Assessment of Cancer Therapy - General (FACT-G). At baseline participants completed measures of anxiety/depression, nausea/vomiting expectation and expectations from using the wristbands. RESULTS: In total, 500 patients were randomised in the study arms (166 standard care, 166 sham acupressure and 168 acupressure) and data were available for 361 participants for the primary outcome. The primary outcome analysis (nausea in cycle 1) revealed no statistically significant differences between the three arms, although the median nausea experience in patients using wristbands (both real and sham ones) was somewhat lower than that in the antiemetics only group (median nausea experience scores for the four cycles: standard care arm 1.43, 1.71, 1.14, 1.14; sham acupressure arm 0.57, 0.71, 0.71, 0.43; acupressure arm 1.00, 0.93, 0.43, 0). A gender effect was evident (p= 0.002), with women responding more favourably to the use of sham acupressure wristbands than men (odds ratio 0.35 for men and 2.02 for women in the sham acupressure group; 1.27 for men and 1.17 for women in the acupressure group). This suggests a placebo effect. No significant differences were detected in relation to vomiting outcomes, anxiety and quality of life. Some transient adverse effects were reported, including tightness in the area of the wristbands, feeling uncomfortable when wearing them and minor swelling in the wristband area (n= 6).There were no statistically significant cost differences associated with the use of real acupressure bands (£70.66 for the acupressure group, £111.13 for the standard care group and £161.92 for the sham acupressure group). In total, 26 subjects took part in qualitative interviews. The qualitative data suggested that participants perceived the wristbands (both real and sham) as effective and helpful in managing their nausea during chemotherapy. CONCLUSIONS: There were no statistically significant differences between the three arms in terms of nausea, vomiting and quality of life, although apparent resource use was less in both the real acupressure arm and the sham acupressure arm compared with standard care only; therefore; no clear conclusions can be drawn about the use of acupressure wristbands in the management of chemotherapy-related nausea and vomiting. However, the study provided encouraging evidence in relation to an improved nausea experience and some indications of possible cost savings to warrant further consideration of acupressure both in practice and in further clinical trials. TRIAL REGISTRATION: ISRCTN87604299. SOURCE OF FUNDING: This project was funded by the NIHR Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 17, No. 26. See the HTA programme website for further project information.

Parent and group leader reflections on a group-based programme for parents and babies.

There is growing interest in supporting families during children's early years to encourage optimal infant development. The data were collected from an evaluation of the Incredible Years (IY) eight-week parenting group for parents and their babies. Feedback obtained from parents (n = 34) and leaders (n = 13) was positive. Retention and attendance rates were high. A detailed costing for the groups indicated that this programme can be delivered for a reasonable cost to meet health visitor objectives with families in the first postpartum months and provides an opportunity to inform parents about resources and other support available for them and their infants.

Principles of early drug discovery.

Developing a new drug from original idea to the launch of a finished product is a complex process which can take 12-15 years and cost in excess of $1 billion. The idea for a target can come from a variety of sources including academic and clinical research and from the commercial sector. It may take many years to build up a body of supporting evidence before selecting a target for a costly drug discovery programme. Once a target has been chosen, the pharmaceutical industry and more recently some academic centres have streamlined a number of early processes to identify molecules which possess suitable characteristics to make acceptable drugs. This review will look at key preclinical stages of the drug discovery process, from initial target identification and validation, through assay development, high throughput screening, hit identification, lead optimization and finally the selection of a candidate molecule for clinical development.

Does trauma risk management reduce psychological distress in deployed troops?

BACKGROUND: Military personnel exposed to potentially traumatic events whilst deployed on operational duties may develop psychological problems. The Royal Marines have made extensive use of Trauma Risk Management (TRiM), a peer-support system that operates through practitioners embedded within operational units. TRiM aims to promote recognition of psychological illness and to facilitate social support. AIMS: To evaluate the effects of TRiM in two units at different stages of implementation. METHODS: Royal Marines and Army personnel were surveyed prior to, during and upon return from an operational deployment to Afghanistan. Participants completed measures of general mental health (GHQ12) and traumatic stress [PCL(C)]. RESULTS: We received responses from 180 pre-deployment, 105 during deployment and 137 post-deployment. Personnel within units with experience of TRiM reported lower levels of psychological distress than personnel in the unit who were using TRiM for the first time. Both groups reported higher psychological distress scores before and during deployment, compared with post-deployment. However, we found personnel who reported having more access to social support during deployment reported less psychological distress. CONCLUSIONS: The use of TRiM may assist in increasing the psychological resilience of military personnel through the facilitation of social support; this may have particular utility during operational deployments.

Impact of organisation and management factors on infection control in hospitals: a scoping review.

This scoping review sought evidence about organisational and management factors affecting infection control in general hospital settings. A literature search yielded a wide range of studies, systematic reviews and reports, but high quality direct evidence was scant. The majority of studies were observational and the standard of reporting was generally inadequate. Positive leadership at ward level and above appears to be a prerequisite for effective action to control infection, although the benefits of good clinical leadership are diffused by supervision of large numbers of staff. Senior clinical leaders need a highly visible presence and clear role boundaries and responsibilities. Team stability and morale are linked to improved patient outcomes. Organisational mechanisms for supporting training, appraisal and clinical governance are important determinants of effective practice and successful change. Rates of infection have been linked to workload, in terms of nurse staffing, bed occupancy and patient turnover. The organisational characteristics identified in the review should be considered risk factors for infection. They cannot always be eliminated or avoided completely, but appropriate assessment will enable targeted action to protect patients.

Reliability of needs assessments in the community care of older people: impact of the single assessment process in England.

BACKGROUND: The single assessment process (SAP) for older people, introduced in England across health and social care agencies from April 2004, aimed at improving assessment processes. We examined the impact of this policy in terms of the reliability of needs identification within statutory social services assessments. METHODS: An observational study compared the accuracy of needs identification in samples of older people before and after SAP introduction. Participants, at risk of entering care homes, were interviewed using standardized measures. Needs elicited from interviews were compared with those from statutory social services assessments to ascertain the reliability of needs identification at both times. Inter-rater reliabilities were calculated using the kappa (k) statistic. A Chi-squared statistic tested the equality of kappa values pre- and post-SAP. RESULTS: Most needs were identified more reliably after SAP introduction (range adjusted k = 0.05-0.58) than before (range adjusted k = -0.09 to 0.28), this being statistically significant for 9 out of 15 needs. Depression, and associated apathy, was an exception. CONCLUSION: Statutory social services assessments better captured need following the introduction of the SAP. However, the extent to which these findings can be attributed to SAP introduction is limited by the introduction of multiple policy initiatives throughout the study period.