Suitable Outcome Measures Research Articles

The potential of eye-tracking as a sensitive measure of behavioural change in response to intervention

One challenge to the development of effective interventions to support learning and behavioural change in neurodevelopmental disorders is a lack of suitable outcome measures. Eye-tracking has been used widely to chart cognitive development and clinically-relevant group differences in many populations. This proof-of-concept study investigates whether it also has the potential to act as a marker of treatment effects, by testing its sensitivity to differential change over a short period of exposure to an iPad app in typically developing children. The app targets a key skill in early social communication development, by rewarding attention to people, operationalised via a finger-tap on screen. We measured attention to images taken from the app, and a selection of matched stimuli to test generalisation of effects, at baseline and two weeks later. Children were assigned to either an app-exposure or no-app condition in the intervening period. The app exposure group showed increases in fixation on people for images from the app, and for distant-generalisation photographs, at high levels of complexity. We conclude that, with careful selection of stimuli, eye-tracking has the potential to make a valuable contribution to the range of outcome measures available for psycho-behavioural interventions in neurodevelopmental disorders.

Left Ventricular Diastolic Dysfunction is Associated with Renal Dysfunction, Poor Survival and Low Health Related Quality of Life in Cirrhosis

The presence of left ventricular diastolic dysfunction (LVDD) in patients with cirrhosis leads to a restriction of activities and a poor health related quality of life (HRQoL), which should be taken into consideration when treating them for liver and cardiac complications. The prevalence, complications, predictors of HRQoL and survival in cirrhotic patients with LVDD were studied. We report a prospective cohort study of 145 consecutive cirrhotic patients with LVDD who were evaluated for cardiac functional status at enrollment and followed up for hepatic complications, cardiac events, outcome and HRQoL using the Minnesota Living With Heart Failure Questionnaire (MLHFQ) over a period of 2 years. In total, 145 (mean age 61 years, 59% male) patients were included. Seventeen patients died with 10.5%, 22.5% and 40% mortality rates in patients with Grades 1, 2 and 3 LVDD respectively over 24 months. The parameters that were significant for predicting mortality on bivariate analysis were MELD, MELDNa, hepatic venous pressure gradient, MLHFQ, and left ventricular (LV) diastolic function (e' and E/e' ratio), but only MELD, MELDNa and E/e' remained significant on multivariate analysis. The E/e' ratio (8.7±3.3 in survivors vs. 9.1±2.3 in non-survivors) predicted outcome. On univariate analysis, the predictors of poor HRQoL were the Child-Pugh score ≥9.8 (OR 2.6; 95% confidence intervals (CI) 2.3-9.1, P=0.041), MELD score≥15.7 (OR 2.48; 95% CI 1.4-3.9, P=0.029), refractory ascites (OR 1.9; 95% CI 1.1-6.1, P=0.050), and E/e' ratio ≥7.6 (OR 1.9; 95% CI 1.8-7.1, P=0.036) The presence of Class II/III (P=0.046) symptoms of heart failure and MLHFQ≥ 45 (P=0.042) were predictors of mortality at 24 months. The grade of LVDD correlates with liver function, clinical events, risk of renal dysfunction and HRQoL. Evaluation of novel therapies which target symptomatic improvement in LVDD, should be done with suitable outcome measures, including HRQoL assessment.

Double Anterior Stereotactic Cingulotomy for Intractable Oncological Pain

Background: Stereotactic anterior cingulotomy has been used in the treatment of patients suffering from refractory oncological pain due to its effects on pain perception. However, the optimal targets as well as suitable candidates and outcome measures have not been well defined. We report our initial experience in the ablation of 2 cingulotomy targets on each side and the use of the Brief Pain Inventory (BPI) as a perioperative assessment tool. Methods: A retrospective review of all patients who underwent stereotactic anterior cingulotomy in our Department between November 2015 and February 2017 was performed. All patients had advanced metastatic cancer with limited prognosis and suffered from intractable oncological pain. Results: Thirteen patients (10 women and 3 men) underwent 14 cingulotomy procedures. Their mean age was 54 ± 14 years. All patients reported substantial pain relief immediately after the operation. Out of the 6 preoperatively bedridden patients, 3 started ambulating shortly after. At the 1-month follow-up, the mean preoperative Visual Analogue Scale score decreased from 9 ± 0.9 to 4 ± 2.7 (p = 0.003). Mean BPI pain severity and interference scores decreased from levels of 29 ± 4 and 55 ± 12 to 16 ± 12 (p = 0.028) and 37 ± 15 (p = 0.043), respectively. During the 1- and 3-month follow-up visits, 9/11 patients (82%) and 5/7 patients (71%) available for follow-up reported substantial pain relief. No patient reported worsening of pain during the study period. Neuropsychological analyses of 6 patients showed stable cognitive functions with a mild nonsignificant decline in focused attention and executive functions. Adverse events included transient confusion or mild apathy in 5 patients (38%) lasting 1-4 weeks. Conclusions: Our initial experience indicates that double stereotactic cingulotomy is safe and effective in alleviating refractory oncological pain.

Stem Cells for Cartilage Repair: Preclinical Studies and Insights in Translational Animal Models and Outcome Measures.

Due to the restricted intrinsic capacity of resident chondrocytes to regenerate the lost cartilage postinjury, stem cell-based therapies have been proposed as a novel therapeutic approach for cartilage repair. Moreover, stem cell-based therapies using mesenchymal stem cells (MSCs) or induced pluripotent stem cells (iPSCs) have been used successfully in preclinical and clinical settings. Despite these promising reports, the exact mechanisms underlying stem cell-mediated cartilage repair remain uncertain. Stem cells can contribute to cartilage repair via chondrogenic differentiation, via immunomodulation, or by the production of paracrine factors and extracellular vesicles. But before novel cell-based therapies for cartilage repair can be introduced into the clinic, rigorous testing in preclinical animal models is required. Preclinical models used in regenerative cartilage studies include murine, lapine, caprine, ovine, porcine, canine, and equine models, each associated with its specific advantages and limitations. This review presents a summary of recent in vitro data and from in vivo preclinical studies justifying the use of MSCs and iPSCs in cartilage tissue engineering. Moreover, the advantages and disadvantages of utilizing small and large animals will be discussed, while also describing suitable outcome measures for evaluating cartilage repair.

P.357 - The EUROMAC registry for rare glycogen storage diseases: preliminary report

EUROMAC was established to collect comprehensive data on GSDV and related glycogen storage diseases, with the goal to facilitate the characterization of the natural history of rare muscle glycogenoses, report on estimated prevalence and regional differences in phenotype/genotype, assist in the selection of suitable outcome measures, create a comprehensive cohort of patients with rare muscle glycogenoses, from which potentially participants in clinical trials can be recruited, and assist in developing standards of care for muscle glycogenoses. 256 patients were recruited (Denmark n = 10, France n = 23, Germany n = 21, Greece n = 3, Italy n = 34, the Netherlands n = 24, Spain n = 65, the UK n = 70; Turkey n = 6). Diagnosed diseases included GSDV (n = 244), GSD IIIa (n = 2), GSDVII (n = 5), GSDIX (n = 2), GSDX (n = 1), GSDXIV (n = 1), and GSDXV (n = 1). Average age was 66 years (range; 12–87); average age at diagnosis was 31 years. Most diagnoses were based on a molecular genetic confirmatory test (n = 228). Average BMI was 26.1. GSDV population (n = 244): R50X is a hotspot mutation in GSDV and 144 of 244 carried this mutation either as homozygous or compound heterozygous, with a clearly higher incidence in northern European countries in the registry. The average CK level was 2,643 IU/L, but 18 patients had normal CK levels. 29 were reported to have wasting, and interestingly, in several the wasting was preferentially in muscles around the hip. New information has emerged for BMI, CK levels and muscle weakness. Increased BMI may reflect low physical activity levels in the reported sample. CK is not always increased in this population. Muscle weakness in GSDV was reported at a lower frequency than investigations in smaller cohorts that were aimed specifically at studying this. This is probably not surprising as fixed weakness easily can go unnoticed unless specifically tested for. Unfortunately, considerable diagnostic delay was evident, which highlights the need for better awareness of these diseases. The authors would like to thank the study coordinators, patients, and the European Union for funding.

Responsiveness of gait analysis parameters in a cohort of 71 CMT subjects

Detection of worsening in the slowly progressive Charcot–Marie–Tooth disease (CMT) is difficult. As previous clinical scales showed low responsiveness, novel outcome measures are under study, including innovative approaches such as quantitative muscle MRI and instrumented movement analysis. Since gait analysis proved able to reliably quantify CMT locomotor deficits, we aimed to explore whether it can be a sensitive-to-change outcome measure in CMT studies. Clinical and biomechanical evaluations were performed in 71 CMT subjects at baseline and after a mean (±sd) of 28.9 ± 9.5 months. Locomotor tasks included natural walking, ascending and descending steps. Instrumented analysis of such tasks provided indexes related to muscle strength (kinetic parameters) and joint movement (kinematic parameters). Parameter responsiveness was expressed as Standardized Response Mean (SRM). Considering the whole CMT group, several parameters showed moderate responsiveness; subgrouping subjects according to disease severity allowed reaching high responsiveness (SRM >0.80). CMT Examination Score showed moderate responsiveness (SRM 0.53) in the minimally affected group; kinematic parameters were more responsive in this group, whereas kinetic parameters in the most severely affected one. Biomechanical parameters can represent suitable outcome measures for CMT by showing moderate-to-high responsiveness. These data suggest that appropriate selection of patient population and outcome measures is crucial for clinical trials' design.

VISUOMOTOR INTEGRATION IN PRESYMPTOMATIC FAMILIAL ALZHEIMER’S DISEASE

As we enter an era of prevention trials for Alzheimer's disease (AD), it is vital to have sensitive cognitive tests that can identify and track subtle cognitive decline in preclinical AD. A circle-tracing task has identified visuomotor integration deficits in presymptomatic mutation carriers for Huntington's disease. As there is some evidence that visuomotor integration is impaired in early AD, we tested for similar deficits in presymptomatic individuals carrying mutations for autosomal dominant familial AD (FAD). Thirty-one participants at risk of FAD were recruited: 19 presymptomatic mutation carriers (MC) and 12 non-carriers (NC). MC were on average 7.0 years from expected symptom onset and were well matched with NC for mean age and performance on standard cognitive tests (Table). Participants completed 12 45-second circle-tracing trials, half with serial-subtraction, using a stylus to trace as quickly and accurately as possible round an annulus on a tablet screen. Six trials were ‘direct’: participants could see their arm and their tracing path on the tablet. Six trials were ‘indirect’: the participant's arm was covered by a box, but they could view a copy of the annulus and their tracing path on a monitor positioned behind the tablet (Figure 1). Outcome variables were tracing speed (number of rotations) and error rate (number of deviations outside the annulus per rotation). GEE models were used to compare outcomes between groups after adjustment for age, gender and education. As expected, the indirect condition was more difficult than the direct condition, with slower tracing (p<0.001) and higher error rates (p<0.01). There was no evidence of an interaction between mutation status and task condition. Across both conditions, MC made more errors than NC (difference=0.297 (95% CI 0.062–0.532), p=0.013) (Figure 2); there was no significant difference in tracing speed. Within MC, error rate did not correlate with years to expected onset. Subtle deficits in visuomotor integration are an early feature of cognitive decline that can be detected in presymptomatic FAD. These deficits may appear at a similarly early stage to subtle memory decline. Computerised tasks measuring visuomotor integration may be suitable outcome measures in AD prevention trials. Set-up of the circle-tracing task (reprinted from Figure 1A in Say et al., Visuomotor integration deficits precede clinical onset in Huntington's disease. Neuropsychologia 2011, Vol. 49, No. 2, 264–270, Elsevier). In the indirect condition the participant's arm was covered by a box, not shown here. Comparison of mutation carriers and non-carriers for speed and accuracy on the Direct and Indirect conditions of the circle-tracing task. Error bars show 95% confidence intervals for the mean. Asterisk shows significant difference between the groups.

A Randomized, Double-Blind, Placebo-Controlled Pilot Clinical Study on ColdZyme&amp;lt;sup&amp;gt;&amp;amp;reg;&amp;lt;/sup&amp;gt; Mouth Spray against Rhinovirus-Induced Common Cold

Common colds incur significant costs in terms of sick leave and personal discomfort for affected individuals. This study investigated the performance of ColdZyme® Mouth Spray (ColdZyme), a protective barrier against common cold, in rhinovirus-inoculated healthy volunteers. This randomized, doubleblind, placebo-controlled pilot study was conducted on 46 healthy volunteers inoculated with rhinovirus 16 via the nose. Subjects self-administered ColdZyme or placebo 6 times daily for 11 days. Symptoms were recorded daily in a diary. Rhinovirus 16 in nasal and oropharyngeal samples at days 0, 3, 4, 6, 7 and 10 were quantified by RT-qPCR. The primary outcome measure was the reduction in viral load in oropharyngeal samples. Rhinovirus 16 was only detected in 35 out of 46 inoculated subjects. Exploratory analysis measuring the total viral load (i.e., area under the curve (AUC)) for days 3 - 10 in successfully inoculated subjects found that ColdZyme treatment resulted in a lower total viral load in the oropharynx (p = 0.023). In subjects who experienced symptomatic common cold, irrespectively, if virus were detected, treatment with ColdZyme resulted in a reduction in the number of days with common cold symptoms from 6.5 to 3.0 days (p = 0.014) in comparison to placebo. ColdZyme reduced virus infection in the oropharynx and reduced the number of days with common cold symptoms and highlights the possible importance of the oropharynx in common cold infections. Suitable outcome measures for a feasible study on ColdZyme are total viral load in the oropharynx in subjects having detectable virus present in nasal or oropharyngeal samples.

Progression characteristics of the European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS): a 2 year cohort study

The European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) is a prospective international registry investigating the natural history of Friedreich's ataxia. We used data from EFACTS to assess disease progression and the predictive value of disease-related factors on progression, and estimated sample sizes for interventional randomised clinical trials. We enrolled patients with genetically confirmed Friedreich's ataxia from 11 European study sites in Austria, Belgium, France, Germany, Italy, Spain, and the UK. Patients were seen at three visits-baseline, 1 year, and 2 years. Our primary endpoint was the Scale for the Assessment and Rating of Ataxia (SARA). Secondary outcomes were the Inventory of Non-Ataxia Signs (INAS), the Spinocerebellar Ataxia Functional Index (SCAFI), phonemic verbal fluency (PVF), and the quality of life measures activities of daily living (ADL) and EQ-5D-3L index. We estimated the yearly progression for each outcome with linear mixed-effect modelling. This study is registered with ClinicalTrials.gov, number NCT02069509, and follow-up assessments and recruitment of new patients are ongoing. Between Sept 15, 2010, and Nov 21, 2013, we enrolled 605 patients with Friedreich's ataxia. 546 patients (90%) contributed data with at least one follow-up visit. The progression rate on SARA was 0·77 points per year (SE 0·06) in the overall cohort. Deterioration in SARA was associated with younger age of onset (-0·02 points per year [0·01] per year of age) and lower SARA baseline scores (-0·07 points per year [0·01] per baseline point). Patients with more than 353 GAA repeats on the shorter allele of the FXN locus had a higher SARA progression rate (0·09 points per year [0·02] per additional 100 repeats) than did patients with fewer than 353 repeats. Annual worsening was 0·10 points per year (0·03) for INAS, -0·04 points per year (0·01) for SCAFI, 0·93 points per year (0·06) for ADL, and -0·02 points per year (0·004) for EQ-5D-3L. PVF performance improved by 0·99 words per year (0·14). To detect a 50% reduction in SARA progression at 80% power, 548 patients would be needed in a 1 year clinical trial and 184 would be needed for a 2 year trial. Our results show that SARA is a suitable clinical rating scale to detect deterioration of ataxia symptoms over time; ADL is an appropriate measure to monitor changes in daily self-care activities; and younger age at disease onset is a major predictor for faster disease progression. The results of the EFACTS longitudinal analysis provide suitable outcome measures and sample size calculations for the design of upcoming clinical trials of Friedreich's ataxia. European Commission.

An audit of in-utero transfers in a district general hospital

Objective: To assess the quantity and nature of transfers from a district general hospital with the aim of identifying suitable outcome measures for the assessment of future service improvements. Background: In-utero transfer is an essential part of daily obstetric practice. A need for an in-utero transfer will arise if the referring unit is unable to cope with the particular clinical condition at the time of transfer for a variety of reasons including: neonatal gestational thresholds, specialist maternal care, antenatal diagnosis requiring specialist postnatal care, bed/cot capacity or staffing issues. It has been reported that most major perinatal centres in the United Kingdom are regularly unable to meet in-house demand. Approximately 25% of women will remain undelivered 96 h after in-utero transfer. To avoid in-utero transfers, which is clearly a source of significant stress and anxiety for parents, the rationale for transfer has to be clear and the final decision made at consultant obstetric level. Method: Retrospective analysis of case notes of women who had in-utero transfers from 01/01/15 to 30/08/15. Results: 39 in-utero transfers identified over the 6 months period. Gestations ranged from 24 + 4 to 36 weeks. 34 of the transfers involved singletons, the other 5 were twin pregnancies. 36 out of the 39 patients (92%) had consent for transfer documented in their notes/IUT proforma. 72% of the transfers were due to cot availability (>28 weeks) in our unit, 10% of these being more that 34 weeks gestation. 14 patients (36%) required tocolytics i.e. Nifedipine or Atosiban prior to or during transfer. Only 33% of the in-utero transfers delivered at the receiving hospital. All 12 patients (31%) with a positive actim partus on transfer were discharged home. Conclusion and recommendations: Our results highlight an apparent lack of capacity in the neonatal service, resulting in considerable numbers in-utero transfers. We need to document maternal consent with every patient being transferred in-utero and to review and develop guidance on the use of Actim Partus in the department.

The Dash and Quick-Dash: Based on Consensus Findings for Internal Consistency and Factor Structure – Are They Suitable and Valid Outcome Measures for the Upper Limb?

The Dash and Quick-Dash: Based on Consensus Findings for Internal Consistency and Factor Structure – Are They Suitable and Valid Outcome Measures for the Upper Limb?

Identifying and Promoting Best Practices in Residency Application and Selection in a Complex Academic Health Network.

Medical education institutions have a social mandate to produce a diverse physician workforce that meets the public's needs. Recent reports have framed the admission process outcome of undergraduate and postgraduate medical education (UGME and PGME) programs as a key determinant of the collective contributions graduating cohorts will make to society, creating a sense of urgency around the issue of who gets accepted. The need for evidence-informed residency application and selection processes is growing because of the increasing size and diversity of the applicant pool and the need for equity, fairness, social accountability, and health human resource planning. The selection literature, however, is dominated by a UGME focus and emphasizes determination of desirable qualities of future physicians and selection instrument reliability and validity. Gaps remain regarding PGME selection, particularly the creation of specialty-specific selection criteria, suitable outcome measures, and reliable selection systems.In this Perspective, the authors describe the University of Toronto's centralized approach to defining system-level best practices for residency application and selection. Over the 2012-2013 academic year, the Best Practices in Application and Selection working group reviewed relevant literature and reports, consulted content experts, surveyed local practices, and conducted iterative stakeholder consultations on draft recommendations. Strong agreement arose around the resulting 13 principles and 24 best practices, which had either empirical support or face validity. These recommendations, which are shared in this article, have been adopted by the university's PGME advisory committee and will inform a national initiative to improve trainees' transition from UGME to PGME in Canada.

Preventing alcohol misuse in young people: an exploratory cluster randomised controlled trial of the Kids, Adults Together (KAT) programme

BackgroundInvolvement of parents/carers may increase the effectiveness of primary school-based alcohol-misuse prevention projects. However, few interventions have been designed for pre-adolescent children, or specifically involve parents/carers. The Kids, Adults Together (KAT) programme in primary schools aimed to reduce alcohol misuse through such an approach.ObjectiveTo determine the value and feasibility of conducting an effectiveness trial of KAT.DesignParallel-group cluster randomised exploratory trial with an embedded process evaluation. Schools were the unit of randomisation.SettingPrimary schools (n = 9) in south Wales, UK.ParticipantsPupils in Year 5/6 (aged 9–11 years) and their parents/carers; school staff.InterventionThe Kids, Adults Together programme consisted of (1) classwork addressing the effects of alcohol; (2) a family event for children and parents/carers; and (3) a ‘goody bag’ containing fun items, including a digital versatile disc (DVD) for families to watch together. The intervention comprised KAT plus existing alcohol-related activities and lessons. Control-group schools continued with existing alcohol-related lessons and activities.Main outcome measuresKey outcomes related to the progression criteria for a potential future effectiveness trial. These included the acceptability, participation equity, feasibility and implementation of KAT; the recruitment and retention of research participants; and the acceptability and feasibility of research processes, including data collection methods and outcome measures.ResultsNine schools (free school meal entitlement ranging from 1% to 37.2%) participated. Two of five intervention schools withdrew but all four control schools were retained, and these seven schools facilitated all research data collections. Programme acceptability and participation rates were high in all three intervention schools (parent/carer participation rates ranged from 45.1% to 65.7%), although implementation quality varied. At baseline, approximately 75% of eligible children (n = 418) provided data, of whom 257 also provided data at follow-up. Only 27 parents/carers (estimated response rate 6.5%) completed interviews. Most children were willing to complete questionnaires but measures were not appropriate for this age group. Measures of alcohol consumption produced inconsistent responses. Intermediate outcomes on family communication showed no evidence of intervention effectiveness.ConclusionsIn the three schools that received the KAT intervention, it was found to be acceptable to schools and pupils and there were good levels of participation from parents/carers from across a range of socioeconomic groups. However, two intervention schools withdrew from the trial. Findings from intermediate outcomes on family communication did not support programme theory. In addition, the study highlighted challenges in identifying suitable outcome measures for children aged 9–11 years and the feasibility of long-term follow-up via secondary schools.Future workIt would not be appropriate to proceed to an effectiveness trial of KAT. There are doubts/uncertainties about the potential effects of KAT; suitability of measures; the large number of schools which would be required for an effectiveness trial of KAT, and the cost of this; feasibility of follow-up in secondary schools; and programme implementation and theory. There is a need to develop and validate measures for children aged 9–11 years; to test the feasibility of follow-up data collection methods in secondary schools; and to further consider sample size requirements and feasibility.Trial registrationCurrent Controlled Trials ISRCTN80672127.FundingThe exploratory trial of this project was funded by the National Institute for Health Research Public Health Research programme and the process evaluation was funded by the Economic and Social Research Council. The work was undertaken with the support of The Centre for the Development and Evaluation of Complex Interventions for Public Health Improvement (DECIPHer), a UK Clinical Research Collaboration Public Health Research Centre of Excellence. Joint funding (MR/KO232331/1) from the British Heart Foundation, Cancer Research UK, Economic and Social Research Council, the Welsh Government and the Wellcome Trust, under the auspices of the UK Clinical Research Collaboration, is gratefully acknowledged. This project will be published in full inPublic Health Research; Vol. 3, No. 15. See the NIHR Journals Library website for further project information.

Group facilitators' perceptions of the attributes that contribute to the effectiveness of group‐based chronic disease self‐management education programs

AbstractAimThis qualitative study aimed to explore group facilitators' perceptions of the attributes contributing to the effectiveness of group‐based chronic disease self‐management education programs.MethodsFourteen group facilitators across a range of disciplines (dietitians, nurses, exercise physiologists, social workers and physiotherapists) individually participated in semistructured interviews, which explored facilitators' awareness of the theoretical basis of the programs they implement, their experiences of implementation and their opinions on the attributes contributing to program effectiveness. The interviews were audio‐recorded, transcribed verbatim and analysed using thematic content analysis and seeking patterning of responses.ResultsGroup facilitators were uncertain about the evidence base and theoretical development of their programs, and very few were offered any formal training prior to facilitating group education programs. Additionally, the outcome measures used by the group facilitators were limited. Group interactions, a non‐didactic delivery style, a multidisciplinary team, and using practical activities, were highlighted as attributes contributing to group program effectiveness.ConclusionsThe present study suggests that group facilitators had limited training and were unaware of the rationale for their programs, which may limit quality control. Health professionals should have an understanding of the basis of their program and be adequately trained prior to facilitating group education programs for chronic disease management. Group facilitators should additionally ensure a focus on the collection of suitable outcome measures to allow the quality improvement of programs in order to improve patient outcomes.

Asynchronous telemedicine applications in rehabilitation of acquired speech-language disorders in neurologic patients

Asynchronous telemedicine applications in rehabilitation of acquired speech-language disorders in neurologic patients Lilian Beijer,1 Toni Rietveld2 1Sint Maartenskliniek, 2Centre of Language Studies, Radboud University of Nijmegen, Nijmegen, The Netherlands Abstract: In this time of an aging population in the Western world and a concomitant need for cost reduction, there is an obvious need for innovative health care delivery. One of the consequences is that a growing number of telemedicine applications are emerging in different health care domains. Also in the area of speech-language (SL) disorders, particularly neurogenic disorders, telemedicine is rapidly gaining interest. In this paper, we place applications for neurogenic SL disorders in a telemedicine taxonomy in order to establish common features along the dimensions of functionality, application, and technology, and their components. Thus, we aim at identifying common features in a wide variety of telemedicine applications and to establish common interests of stakeholders in health care for classified groups of telemedicine applications. This may facilitate decision-making with regard to expansion of innovative products, and give directions to measures needed for upscaling and structural embedding of feasible and effective SL telemedicine applications in health care. Common interests of stakeholders in health care, established using telemedicine taxonomy, is a key factor in decision-making with regard to which telemedicine applications should be given priority for genuine utilization. Priorities of health care institutions, patients, and reimbursement companies are also leading for researchers aiming at solid scientific evidence for the beneficial effects of target applications. That is, although research results tend to indicate the potential of telemedicine in the area of SL pathology, the alleged benefits of most applications have not been confirmed according to the accepted standards for clinical outcome testing as yet. Methodologic obstacles and the lack of adequate speech materials and suitable outcome measures for efficacy and effectiveness testing partially account for this. From the perspective of scientific evidence, the benefits of asynchronous SL telemedicine applications concern data storage and data analyses. To facilitate implementation of telemedicine, there is a call for development of information and communication technology infrastructures that allow feasible applications which meet requirements with regard to licensure and medical privacy laws. For applications with evidence for beneficial effects, we are challenged to develop new business models that apply to a new health care environment. Keywords: telemedicine, speech, language, disorders, rehabilitation&nbsp

Reflecting on the methodological challenge of recruiting older care home residents to podiatry research

Successful randomisd controlled trials (RCTs) require successful participant recruitment; poor recruitment leads to poor, under-powered studies, and may waste grant funds. Recruitment of older care home residents to RCTs is challenging. This is problematic for podiatry, because older people within care home settings are high users of podiatry services; therefore it is essential that strategies are employed to maximise recruitment to RCTs. We describe the experience of recruiting to a feasibility study of a podiatry intervention to reduce falls in care home residents in the East of Scotland. This was the first phase of a two phase project consisting of the feasibility study to acquire data (recruitment strategy, selection of suitable outcome measures) to inform the conduct of the second phase, an exploratory RCT. Recruitment difficulties became apparent early in the study. Difficulties arose when it came to assessing whether or not potential participants fulfilled certain inclusion criteria: (1) Presence of foot pain (defined as foot pain lasting for at least a day in the last month and a positive response of “some days” or “most/every days” to at least one item on the Manchester Foot Pain and Disability Index (MFPDI)) (2) Ability to provide informed consent. The reasons for these difficulties are that (1) we discovered that in the area in which our study was conducted, the majority of care home residents receive basic NHS podiatry care to treat any superficial lesions (i.e. pathological nails and skin callus) thus the prevalence of foot pain resulting from these lesions was lower than we had originally anticipated, and (2) the care homes that we engaged for this phase of the study had residents who were far more dependent and with much higher levels cognitive impairment than we anticipated, making obtaining informed consent difficult. Based on the existing inclusion criteria, it was deemed unlikely that we would meet our recruitment target for the subsequent exploratory randomised controlled trial (n=40).

Treatment of Unawareness of Deficits in Patients With Acquired Brain Injury

To review and evaluate the effectiveness and methodological quality of available treatment methods for unawareness of deficits after acquired brain injury (ABI). Systematic literature search for treatment studies for unawareness of deficits after ABI. Information concerning study content and reported effectiveness was extracted. Quality of the study reports and methods were evaluated. A total of 471 articles were identified; 25 met inclusion criteria. 16 were uncontrolled or single-case studies. Nine were of higher quality: 2 randomized controlled trials, 5 single case experimental designs, 1 single-case design with pre- and posttreatment measurement, and 1 quasi-experimental controlled design. Overall, interventions consisted of multiple components including education and multimodal feedback on performance. Five of the 9 high-quality studies reported a positive effect of the intervention on unawareness in patients with some knowledge of their impairments. Effect sizes ranged from questionable to large. Patients with ABI may improve their awareness of their disabilities and possibly attain a level at which they personally experience problems when they occur. At present, because of lack of evidence, no recommendation can be made for treatment approaches for persons with severe impairment of self-awareness in the chronic phase of ABI. We recommended developing and evaluating theory-driven interventions specifically focused on disentangling the components of treatment that are successful in improving awareness. High-quality intervention studies are urgently needed using controlled designs (eg, single-case experimental designs, randomized controlled trials) based on a theoretic perspective with a detailed description of the content of the intervention and suitable outcome measures.

Preparing an E-learning-based Speech Therapy (EST) efficacy study: Identifying suitable outcome measures to detect within-subject changes of speech intelligibility in dysarthric speakers

We explored the suitability of perceptual and acoustic outcome measures to prepare E-learning based Speech Therapy (EST) efficacy tests regarding speech intelligibility in dysarthric speakers. Eight speakers with stroke (n = 3), Parkinson’s disease (n = 4) and traumatic brain injury (n = 1) participated in a 4 weeks EST trial. A repeated measures design was employed. Perceptual measures were (a) scale ratings for “ease of intelligibility” and “pleasantness” in continuous speech and (b) orthographic transcription scores of semantically unpredictable sentences. Acoustic measures were (c) “intensity during closure” (ΔIDC) in the occlusion phase of voiceless plosives, (d) changes in the vowel space of /a/, /e/ and /o/ and (e) the F0 variability in semantically unpredictable sentences. The only consistent finding concerned an increased (instead of the expected decreased) ΔIDC after EST, possibly caused by increased speech intensity without articulatory adjustments. The importance of suitable perceptual and acoustic measures for efficacy research is discussed.

Informing the NHS Outcomes Framework: evaluating meaningful health outcomes for children with neurodisability using multiple methods including systematic review, qualitative research, Delphi survey and consensus meeting

BackgroundThe identification of suitable outcome measures will improve the evaluation of integrated NHS care for the large number of children affected by neurodisability, and has the potential to encourage the provision of more appropriate and effective health care. This research sought to appraise the potential of patient-reported outcome measures (PROMs) for children and young people with neurodisability.AimThis research aimed (i) to identify key outcomes of health care for children with neurodisability, beyond morbidity and mortality, from the perspectives of children, parents and professionals; (ii) to critically appraise existing generic multidimensional PROMs; and (iii) to examine whether or not the key outcomes might be measured by existing PROMs. We also sought agreement on a definition of neurodisability.MethodsData were gathered in three main ways, (i) a systematic review identified eligible generic multidimensional PROMs and peer-reviewed studies evaluating psychometric performance using English-language questionnaires. Studies were appraised for methodological quality and psychometric performance was appraised using standard criteria. (ii) Focus groups and interviews with children and young people with neurodisability, and separately with parents, sought to identify important outcomes of NHS care, and their feedback on example PROM questionnaires. (iii) An online Delphi survey was conducted with a multidisciplinary sample of health professionals to seek agreement on appropriate NHS outcomes. In addition, we convened a consensus meeting with a small nominal group of young people, parents and professionals; the group sought agreement on a core set of important health outcomes.ResultsFrom the systematic review, we identified 126 papers that reported eligible evidence regarding the psychometric performance of 25 PROMs. Evidence of psychometric robustness was more favourable for a small number of PROMs: KIDSCREEN (generic), DISABKIDS (chronic-generic) and Child Health Utility 9D (preference-based measure). The Pediatric Quality of Life Inventory and KINDL offer both self-report and a proxy report version for a range of age bands, but evidence of their psychometric performance was weaker. Evidence was lacking in one or more respects for all candidate PROMs, in both general populations and those with neurodisability. Proxy reporting was found generally to be poorly correlated with self-report. Focus groups and interviews included 54 children and young people, and 53 parents. The more important health outcomes were felt to be communication, emotional well-being, pain, mobility, independence/self-care, worry/mental health, social activities and sleep. In addition, parents of children with intellectual impairment identified behaviour, toileting and safety as important outcomes. Participants suggested problems with the face validity of example PROM questionnaires for measuring NHS care. In the Delphi survey, 276 clinicians from a wide range of professions contributed to at least one of four rounds. Professionals rated pain, hearing, seeing, sleep, toileting, mobility and communication as key goals for the NHS but also identified treating neurological symptoms as important. Professionals in the Delphi survey and parents working with the research team agreed a proposed definition for neurodisability. The consensus meeting confirmed overlap between the outcomes identified as important by young people, parents and professionals, but not complete agreement.ConclusionsThere was agreement between young people, parents and professionals regarding a core suite of more important health outcomes: communication, emotional well-being, pain, mobility, independence/self-care, worry/mental health, social activities and sleep. In addition, behaviour, toileting and safety were identified as important by parents. This research suggests that it would be appropriate to measure these constructs using PROMs to assess health care. None of the candidate PROMs in the review adequately captures all of the identified constructs, and there is inadequate evidence that candidate PROMs are psychometrically robust for use across children with neurodisability. Further consultation with young people, families and professionals is warranted to support the use of PROMs to measure NHS outcomes. Research to test potential PROMs with different age groups and conditions would be valuable.FundingThe National Institute for Health Research Health Services and Delivery Research programme.

Developing a complex intervention to improve prescribing safety in primary care: mixed methods feasibility and optimisation pilot study

Objectives(A) To measure the extent to which different candidate outcome measures identified high-risk prescribing that is potentially changeable by the data-driven quality improvement in primary care (DQIP) intervention.(B) To explore...