Suitable Outcome Measures Research Articles

Health State Utilities Associated With Treatment Burden in Cystic Fibrosis: A Patient Valuation Study

BACKGROUNDWhile recent advancements in the treatment of cystic fibrosis (CF) have improved survival, reducing high levels of treatment burden remains a priority issue for many people with CF (pwCF). However, economic evaluations of novel interventions may fail to capture their impact on treatment burden due to a lack of suitable outcome measures. This study aimed to estimate health state utilities (HSUs), for changes in treatment burden associated with different CF treatments. RESEARCH QUESTIONWhat value do pwCF place on changes in treatment burden associated with intravenous antibiotic treatment of pulmonary exacerbations, inhaled medicines, and physiotherapy? STUDY DESIGNAND METHODS: Adults attending a specialist CF center were invited to participate in a web-based time trade-off interview. Participants valued their own health, and five health state vignettes describing varying levels of intensity of physiotherapy, inhaled, and IV antibiotic treatment. HSUs for additional instances of each treatment type were estimated using mixed effect linear regression models. RESULTSFifty one people with CF completed the interview (median age, 30 years; range, 19-66); 53% were female; mean FEV(1) % predicted, 65% [SD, 20%]).Mean utility scores for own health were very similar between the EQ-5D index value (0.81, SD 0.20) and the TTO value (0.82, SD 0.20), however limited concordance was observed at the individual level. Adjusted utility decrements associated with treatment burden were -0.037 (SE 0.008) for an additional annual IV antibiotic treatment, -0.029 (SE 0.014) for an additional daily physiotherapy session, and -0.019 (SE 0.013) for an additional daily inhaled medicine. INTERPRETATIONIncreasing treatment burden was associated with decreasing HSU values. The utility decrements associated with treatment burden changes suggest meaningful differences in health-related quality of life (HRQoL) for pwCF. These findings align with existing literature on the impact of treatment burden on HRQoL, and highlight the importance of considering treatment burden in economic evaluations of interventions in CF.

Patient-reported measures of tinnitus for individuals with neurofibromatosis type 2-related schwannomatosis: Recommendations for clinical trials.

Neurofibromatosis type 2-related schwannomatosis is a genetic disease characterized by the development of bilateral vestibular schwannomas, ependymomas, meningiomas, and cataracts. Mild to profound hearing loss and tinnitus are common symptoms reported by individuals with neurofibromatosis type 2. While tinnitus is known to have a significant and negative impact on the quality of life of individuals from the general population, the impact on individuals with neurofibromatosis type 2 is unknown. Consensus regarding the selection of suitable patient-reported outcome measures for assessment could advance further research into tinnitus in neurofibromatosis type 2 patients. The purpose of this work is to achieve a consensus recommendation by the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration for patient-reported outcome measures used to evaluate quality of life in the domain of tinnitus for neurofibromatosis type 2 clinical trials. The Response Evaluation in Neurofibromatosis and Schwannomatosis Patient-Reported Outcomes Communication Subgroup systematically evaluated patient-reported outcome measures of quality of life in the domain of tinnitus for individuals with neurofibromatosis type 2 using previously published Response Evaluation in Neurofibromatosis and Schwannomatosis rating procedures. Of the 19 identified patient-reported outcome measures, 3 measures were excluded because they were not validated as an outcome measure or could not have been used as a single outcome measure for a clinical trial. Sixteen published patient-reported outcome measures for the domain of tinnitus were scored and compared on their participant characteristics, item content, psychometric properties, and feasibility for use in clinical trials. The Tinnitus Functional Index was identified as the most highly rated measure for the assessment of tinnitus in populations with neurofibromatosis type 2, due to strengths in the areas of item content, psychometric properties, feasibility, and available scores. Response Evaluation in Neurofibromatosis and Schwannomatosis currently recommends the Tinnitus Functional Index for the assessment of tinnitus in neurofibromatosis type 2 clinical trials.

An Internet Mantram Repetition Program to Promote Well-being in Breast Cancer Survivors: A Feasibility Randomized Controlled Trial.

Introduction: The primary objective of this study was to assess the feasibility of a 6-week internet-delivered Mantram Repetition Program (MRP) for women recently treated for breast cancer. A secondary objective explored changes in perceived stress, psycho-spiritual measures, and cytokines in the treatment group compared to a waitlist. Methods: A feasibility study (ORBIT model Phase IIa) with a randomized controlled trial pilot was conducted. Eligible women recently treated for breast cancer were randomized to the treatment group (n = 14) or a waitlist group (n = 12) and participated for 12 weeks. During weeks 1-6, the treatment group received the MRP intervention while the waitlisted group was inactive. During weeks 7-12, the treatment group was inactive while the waitlisted group received the MRP intervention. The primary outcomes were feasibility and acceptability of the internet-delivered MRP intervention protocol. Participants completed pre and post-intervention psycho-spiritual health assessments. A subset of participants provided serum for cytokine analyses at enrollment and week 6, coinciding with the period in which the treatment group receiving the MRP intervention. Results: Overall study attrition was 19.2%. MRP adherence for both groups was 86% at post-intervention and 90% in the treatment group at 6-week follow-up. Pre-to-post-intervention analyses pooling both groups' data demonstrated decreased perceived stress (p = .045) and increased spiritual well-being (p =.004). IFN-γ and IL-17A were increased in the waitlisted group and decreased in the treatment group (p = 0.048). Conclusion: Feasibility of a 6-week, internet-delivered MRP intervention for breast cancer survivors was established. Psycho-spiritual variables and serum cytokines are suitable clinical outcome measures for future MRP studies with breast cancer survivors. Data suggest MRP may reduce perceived stress and support spiritual well-being in women with breast cancer; however, additional studies are needed.

Outcome Measures for Shoulder Joint Assessment in Patients with Stroke: A Narrative Review

Objective: Understanding the influence of evidence-based practice on rehabilitating patients with stroke can be achieved through the use of suitable outcome measures. Aim: The purpose of this study is to consolidate the existing outcome measures related to the Shoulder Joint assessment in patients with stroke. This review aims to provide education to neurologists, physiotherapists, academics, and researchers regarding available resources for diagnosing, prognosing, and rehabilitating shoulder joint in stroke patients. Materials and Methods: Electronic searches were performed on databases including Medline, PubMed, Cochrane, Physiotherapy Evidence Database (PEDro) and Google Scholar using terms related to outcome measures utilised in the diagnosis, prognosis, and rehabilitation of shoulder joint in stroke patients. Results: Total seven outcome measures {Stroke Specific Shoulder Disability Index (SSSDI), Fugl Meyer Assessment of Upper Extremity, Motor Assessment Scale, Chedoke-McMaster Stroke Assessment, Orpington Prognostic Scale, Motricity Index, and Stroke Rehabilitation Assessment of Movement} were included in this study used in the diagnosis, prognosis, and rehabilitation of upper extremity in shoulder joints having shoulder domains were identified. SSSDI and Fugl Meyer Assessment of Upper Extremity (FMA-UE) have excellent reliability and good validity among them. But, FMA-UE does not measure the functional assessment of shoulder joint. Currently, among the scales used for shoulder assessment in stroke patients, only the SSSDI covers all domains comprehensively, setting it apart from other scales. Conclusion: The SSSDI is a single tool available for assessing the shoulder domains in stroke patients having excellent reliability

1. Validity and reliability of CAMCOG‐DS2 and other cognitive outcomes measures for clinical trials of AD in DS

AbstractBackgroundDown syndrome (DS) is an important population to include in clinical trials aiming to prevent or delay Alzheimer’s disease (AD). This talk will summarise previous work in trajectory modelling to identify the earliest ages of change in cognition and the optimal sample sizes needed for clinical trials in DS. It will present validate data on the new version of the Cambridge Cognitive Examination–Down syndrome version II (CAMCOG‐DS‐II) alongside established cognitive tests. The CAMCOG‐DS‐II has been developed to assess cognitive impairments associated with dementia in people with DS.MethodThis was an observational, multicentre, longitudinal study (n = 174) across seven existing cohorts in six different European languages to validate cognitive tests in adults with DS aged 25 years and older. It assessed the relationships between tests of cognition, behaviour, function, and health. Cognitive assessments included the CAMCOG‐DS‐II, the CANTAB Paired Associates Learning (PAL) Task, the modified Cued Recall Test (mCRT) and the Purdue Pegboard Test. We examined correlations between tests, group differences between those with and without dementia and explored test‐retest reliability of the CAMCOG‐DS‐II.ResultOur results showed that completion rates for the CAMCOG‐DS‐II were high, and floor and ceiling effects were low. The CAMCOG‐DS‐II memory sub‐score correlated significantly with the mCRT total recall score (Spearman’s ∼0.5), and the praxis sub‐score significantly correlated with the Purdue Pegboard total score (Spearman’s >0.7).ConclusionThere is currently a critical lack of suitable diagnostic and outcome measures that can be used in clinical trials. These results show that the CAMCOG‐DS‐II is a feasible test of cognition and will help to provide sensitive cognitive endpoints for clinical trials which are seeking to find suitable treatments for AD in adults with DS. Future analysis will include trajectories of change from baseline to one year of follow up for each primary outcome measure.

Measuring the success of programmes of care for people living with dementia: a protocol for consensus building with consumers to develop a set of Core Outcome Measures for Improving Care (COM-IC)

IntroductionThe Core Outcome Measures for Improving Care (COM-IC) project aims to deliver practical recommendations on the selection and implementation of a suite of core outcomes to measure the effectiveness of...

Longitudinal structure-function analysis of molecularly-confirmed CYP4V2 Bietti Crystalline Dystrophy

ObjectivesBietti Crystalline Dystrophy (BCD) is an autosomal recessive progressive retinal disease caused by mutations in CYP4V2. We have characterised the natural history including structural and functional measures to identify potential outcome metrics for future clinical trials.MethodsMolecularly-confirmed BCD patients with biallelic variants in CYP4V2 were retrospectively identified from Moorfields Eye Hospital (UK). Clinical details including results of molecular genetic testing, best-corrected visual acuity (BCVA) and spectral-domain optical coherence tomography (OCT) scans were extracted. From OCT scans, ellipsoid zone (EZ) measures, foveal thickness of the whole retina, outer retina and choroid were measured. Age-related changes of clinical parameters were assessed with linear mixed models.ResultsTwenty-eight BCD patients were identified, with median age at baseline of 37 years (interquartile range [IQR]: 30–49.5). Median follow-up was 7.7 years (IQR: 3.4–14.5). Most patients (41.7%) showed chorioretinal atrophy at baseline. All OCT parameters showed significant age-related loss (p < 0.05), with EZ measures and choroidal thickness displaying the most rapid degeneration (2.3–3.3% per year vs 0.6–1.5% per year). Median BCVA was 0.2 LogMAR (IQR: 0–0.5) at baseline and showed small age-related loss ( + 0.016 LogMAR per year, p = 0.0019). Patients exhibited substantial phenotypic variability.ConclusionsBCD presents between age 25 and 40, and slowly progresses to an advanced chorioretinal atrophy and vision loss by age 60. BCVA may be preserved until late, and is seemingly poorly representative of disease progression. OCT parameters capturing EZ and choroid changes may afford more suitable trial outcome measures.

A Virtual Reality-Assisted Cognitive Behavioral Therapy for and With Inuit in Québec: Protocol for a Proof-of-Concept Randomized Controlled Trial.

Emotion regulation is an ability related to psychological well-being; when dysregulated, individuals may have psychiatric symptoms and maladapted physiological responses. Virtual reality-assisted cognitive behavioral therapy (VR-CBT) is an effective psychotherapy to target and strengthen emotion regulation; however, it currently lacks cultural sensitivity and can be improved by adapting it to the cultural context of service users. During previous participatory research, we co-designed a culturally adapted cognitive behavioral therapy (CBT) manual and 2 virtual reality (VR) environments to function as a complement to therapy (VR-CBT) for Inuit who would like to access psychotherapy. Emotion regulation skill building will occur in virtual environments that have interactive components such as heart rate biofeedback. We describe a protocol for a proof-of-concept 2-arm randomized controlled trial (RCT) with Inuit (n=40) in Québec. The primary aims of this research are to investigate the feasibility, benefits, and challenges of the culturally adapted VR-CBT intervention versus an established VR self-management that is available commercially. We will also investigate self-rated mental well-being and objective psychophysiological measures. Finally, we will use proof-of-concept data to identify suitable primary outcome measures, conduct power calculations in a larger trial for efficacy, and collect information about preferences for on-site or at-home treatment. Trial participants will be randomly assigned to an active condition or active control condition in a 1:1 ratio. Inuit aged 14 to 60 years will receive a culturally adapted and therapist-guided VR-CBT with biofeedback or a VR relaxation program with nonpersonalized guided components over a 10-week period. We will collect pre- and posttreatment measures of emotion regulation and biweekly assessments over the treatment and at 3-month follow-up. The primary outcome will be measured by the Difficulties in Emotion Regulation Scale (DERS-16) and a novel psychophysiological reactivity paradigm. Secondary measures include psychological symptoms and well-being via rating scales (eg, anxiety or depressive symptoms). As this is the prospective registration of an RCT protocol, we do not yet report any results from the trial. Funding was confirmed in January 2020, and recruitment is expected to start in March 2023 and is set to finish in August 2025. The expected results are to be published in spring 2026. The proposed study responds to the community's desire for accessible and appropriate resources for psychological well-being, as it was developed in active collaboration with the Inuit community in Québec. We will test feasibility and acceptance by comparing a culturally adapted, on-site psychotherapy with a commercial self-management program while incorporating novel technology and measurement in the area of Indigenous health. We also aim to fulfill the needs for RCT evidence of culturally adapted psychotherapies that are lacking in Canada. International Standard Randomized Controlled Trial Number (ISRCTN) 21831510; https://www.isrctn.com/ISRCTN21831510. PRR1-10.2196/40236.

A cross-sectional study to assess the clinical utility of modern visual function assessments in patients with inherited retinal disease: a mixed methods observational study protocol

BackgroundTreatment options for patients with inherited retinal disease are limited, although research into novel therapies is underway. To ensure the success of future clinical trials, appropriate visual function outcome measures that can assess changes resulting from therapeutic interventions are urgently required. Rod-cone degenerations are the most common type of inherited retinal disease. Visual acuity is a standard measure but is typically preserved until late disease stages, frequently making it an unsuitable visual function marker. Alternative measures are required. This study investigates the clinical utility of a range of carefully selected visual function tests and patient reported outcome measures. The aim is to identify suitable outcome measures for future clinical trials that could be considered for regulatory approval.MethodsThis cross-sectional study involves two participant groups, patients with inherited retinal disease (n = 40) and healthy controls (n = 40). The study has been designed to be flexible and run alongside NHS clinics. The study is split into two parts. Part one includes examining standard visual acuity, low luminance visual acuity, the Moorfields acuity chart visual acuity, mesopic microperimetry and three separate patient reported outcome measures. Part two involves 20 min of dark adaptation followed by two-colour scotopic microperimetry. Repeat testing will be undertaken where possible to enable repeatability analyses. A subset of patients with inherited retinal disease will be invited to participate in a semi-structured interview to gain awareness of participants’ thoughts and feelings around the study and different study tests.DiscussionThe study highlights a need for reliable and sensitive validated visual function measures that can be used in future clinical trials. This work will build on work from other studies and be used to inform an outcome measure framework for rod-cone degenerations. The study is in keeping with the United Kingdom Department of Health and Social Care research initiatives and strategies for increasing research opportunities for NHS patients as part of their NHS care.Trial registrationISRCTN registry, ISRCTN24016133, Visual Function in Retinal Degeneration, registered on 18th August 2022.

Theories for interventions to reduce physical and verbal abuse: A mixed methods review of the health and social care literature to inform future maternity care.

Despite global attention, physical and verbal abuse remains prevalent in maternity and newborn healthcare. We aimed to establish theoretical principles for interventions to reduce such abuse. We undertook a mixed methods systematic review of health and social care literature (MEDLINE, SocINDEX, Global Index Medicus, CINAHL, Cochrane Library, Sept 29th 2020 and March 22nd 2022: no date or language restrictions). Papers that included theory were analysed narratively. Those with suitable outcome measures were meta-analysed. We used convergence results synthesis to integrate findings. In September 2020, 193 papers were retained (17,628 hits). 154 provided theoretical explanations; 38 were controlled studies. The update generated 39 studies (2695 hits), plus five from reference lists (12 controlled studies). A wide range of explicit and implicit theories were proposed. Eleven non-maternity controlled studies could be meta-analysed, but only for physical restraint, showing little intervention effect. Most interventions were multi-component. Synthesis suggests that a combination of systems level and behavioural change models might be effective. The maternity intervention studies could all be mapped to this approach. Two particular adverse contexts emerged; social normalisation of violence across the socio-ecological system, especially for 'othered' groups; and the belief that mistreatment is necessary to minimise clinical harm. The ethos and therefore the expression of mistreatment at each level of the system is moderated by the individuals who enact the system, through what they feel they can control, what is socially normal, and what benefits them in that context. Interventions to reduce verbal and physical abuse in maternity care should be locally tailored, and informed by theories encompassing all socio-ecological levels, and the psychological and emotional responses of individuals working within them. Attention should be paid to social normalisation of violence against 'othered' groups, and to the belief that intrapartum maternal mistreatment can optimise safe outcomes.

A qualitative exploration of speech-language pathologists' approaches in treating spoken discourse post-traumatic brain injury.

Spoken discourse impairments post-traumatic brain injury (TBI) are well-documented and heterogeneous in nature. These impairments have chronic implications for adults in terms of employment, socializing and community involvement. Intervention delivered by a speech-language pathologist (SLP) is recommended for adults with discourse impairments post-TBI, with an emphasis on context-sensitive treatment. The developing evidence base indicates a wide array of treatment components for SLPs to evaluate and implement within their clinical practice. However, there is limited insight into how SLPs are currently treating discourse impairments and the rationales informing clinical practice. To explore the under-researched area of clinical practice for spoken discourse interventions with adults post-TBI, including treatment components and clinician rationales, and to contribute towards a shared knowledge base. Participants were recruited via purposeful sampling strategies. Six SLPs participated from Australia, the United Kingdom (UK) and the United States (US). Semi-structured interviews were conducted via Zoom. Interviews were manually transcribed, coded and analysed via a qualitative content analysis approach. Participants described discourse treatment practices across various settings and TBI recovery stages. Results indicated that SLPs used numerous treatment activities, resources and outcome measures. Intervention approaches primarily targeted social communication skills, strategy development/utilization and insight-building. Clinical practice conformed to available guidelines where possible, reflected best practice and incorporated components of the research literature. Participants reported using individualized treatment activities aimed at addressing client-specific factors and rationales prioritized tailored, context-sensitive and goal-directed treatment. This study provided insight into a previously under-researched area. It highlighted a wide range of treatment activities and factors informing current SLPs' treatment of spoken discourse impairment post-TBI. Overall, clinical practice and rationales discussed in this study were aligned with best practice and emphasized a contextualized, individualized approach to discourse treatment across service settings and stages of recovery. Participants identified areas requiring further support, including access to training, resources and research, and the challenge of finding suitable outcome measures. Further investigation into discourse management post-TBI, from initial assessment to outcome measurement, may help inform clinical decision-making and the transfer of research to practice. What is already known on the subject Spoken discourse impairments occur in dialogic and monologic productions post-TBI. Interventions targeting both genres are detailed within the research literature; however, studies exploring clinical practice and decision-making for discourse interventions post-TBI are limited. What this paper adds to existing knowledge This study provides new insight into the current treatment targets, activities, resources and outcome measures employed by clinicians supporting adults with discourse impairment post-TBI. It details the factors that influence clinical decision-making for this caseload and identifies an emphasis on client priorities and the value of clinician experience. What are the potential or actual clinical implications of this work? This study identifies the broad and complex considerations required to deliver context-sensitive discourse intervention post-TBI. It indicates the need for an in-depth review from assessment to treatment outcomes to better understand and support this area of practice and to direct future research. This study also highlighted the role of clinician experience in discourse intervention and the value of sharing clinical knowledge and resources within and across the profession to support all levels of clinician experience.

Blood and Cerebrospinal Fluid Biomarkers of Inflammation in Parkinson's Disease.

Given the clear role of inflammation in the pathogenesis of Parkinson’s disease (PD) and its impact on incidence and phenotypical characteristics, this review provides an overview with focus on inflammatory biofluid markers in blood and cerebrospinal fluid (CSF) in PD patient cohorts. In preparation for clinical trials targeting the immune system, we specifically address the following questions: 1) What evidence do we have for pro-inflammatory profiles in blood and in CSF of sporadic and genetic PD patients? 2) Is there a role of anti-inflammatory mediators in blood/CSF? 3) Do inflammatory profiles in blood reflect those in CSF indicative of a cross-talk between periphery and brain? 4) Do blood/CSF inflammatory profiles change over the disease course as assessed in repeatedly taken biosamples? 5) Are blood/CSF inflammatory profiles associated with phenotypical trajectories in PD? 6) Are blood/CSF inflammatory profiles associated with CSF levels of neurodegenerative/PD-specific biomarkers? Knowledge on these questions will inform future strategies for patient stratification and cohort enrichment as well as suitable outcome measures for clinical trials.

Patient-Reported Outcome Measures of Quality of Life in People Affected by Diabetic Foot: A Psychometric Systematic Review

This psychometric systematic review aimed to identify the most suitable patient-reported outcome measures (PROMs) of quality of life (QoL) in people affected by diabetic foot. We performed a literature search in MEDLINE (PubMed), CINAHL (EBSCOhost), and PsycINFO (EBSCOhost) databases from inception to February 1, 2022. We also searched gray literature databases. Eligible studies were full-text reports developing a QoL condition-specific PROM or assessing one or more of its measurement properties in people affected by diabetic foot. We assessed the methodological quality of included studies independently using the "Consensus-Based Standards for the Selection of Health Measurement Instruments Risk of Bias" checklist. The measurement properties were evaluated using specific criteria. We graded the quality of the evidence using a "Grading of Recommendations Assessment, Development and Evaluation" approach modified by Consensus-Based Standards for the Selection of Health Measurement Instruments. Forty-three reports (46 studies) providing information on the measurement properties of 10 different PROMs were included. We did not identify any instruments that could be recommended for use. We identified 2 PROMs that were not recommended for use and 8 that were potentially recommended but would require further investigation. Of these 8 PROMs, 4 had better evidence for content validity. Available PROMs to measure QoL in people affected by diabetic foot have limited evidence for their measurement properties. There is no fully suitable PROM. Pending further evidence, 4 PROMs could potentially be recommended for use.

A Narrative Review on Translational Research in Acute Brain Injury

AbstractThere has been a constant endeavor to reduce the mortality and morbidity associated with acute brain injury. The associated complex mechanisms involving biomechanics, markers, and neuroprotective drugs/measures have been extensively studied in preclinical studies with an ultimate aim to improve the patients' outcomes. Despite such efforts, only few have been successfully translated into clinical practice. In this review, we shall be discussing the major hurdles in the translation of preclinical results into clinical practice. The need is to choose an appropriate animal model, keeping in mind the species, age, and gender of the animal, choosing suitable outcome measures, ensuring quality of animal trials, and carrying out systematic review and meta-analysis of experimental studies before proceeding to human trials. The interdisciplinary collaboration between the preclinical and clinical scientists will help to design better, meaningful trials which might help a long way in successful translation. Although challenging at this stage, the advent of translational precision medicine will help the integration of mechanism-centric translational medicine and patient-centric precision medicine.

Customized Information and Communication Technology for Reducing Social Isolation and Loneliness Among Older Adults: Scoping Review.

BackgroundAdvancements in science and various technologies have resulted in people having access to better health care, a good quality of life, and better economic situations, enabling humans to live longer than ever before. Research shows that the problems of loneliness and social isolation are common among older adults, affecting psychological and physical health. Information and communication technology (ICT) plays an important role in alleviating social isolation and loneliness.ObjectiveThe aim of this review is to explore ICT solutions for reducing social isolation or loneliness among older adults, the purpose of ICT solutions, and the evaluation focus of these solutions. This study particularly focuses on customized ICT solutions that either are designed from scratch or are modifications of existing off-the-shelf products that cater to the needs of older adults.MethodsA scoping literature review was conducted. A search across 7 databases, including ScienceDirect, Association for Computing Machinery, PubMed, IEEE Xplore, PsycINFO, Scopus, and Web of Science, was performed, targeting ICT solutions for reducing and managing social isolation and loneliness among older adults. Articles published in English from 2010 to 2020 were extracted and analyzed.ResultsFrom the review of 39 articles, we identified 5 different purposes of customized ICT solutions focusing on reducing social isolation and loneliness. These were social communication, social participation, a sense of belonging, companionship, and feelings of being seen. The mapping of purposes of ICT solutions with problems found among older adults indicates that increasing social communication and social participation can help reduce social isolation problems, whereas fulfilling emotional relationships and feeling valued can reduce feelings of loneliness. In terms of customized ICT solution types, we found the following seven different categories: social network, messaging services, video chat, virtual spaces or classrooms with messaging capabilities, robotics, games, and content creation and management. Most of the included studies (30/39, 77%) evaluated the usability and acceptance aspects, and few studies (11/39, 28%) focused on loneliness or social isolation outcomes.ConclusionsThis review highlights the importance of discussing and managing social isolation and loneliness as different but related concepts and emphasizes the need for future research to use suitable outcome measures for evaluating ICT solutions based on the problem. Even though a wide range of customized ICT solutions have been developed, future studies need to explore the recent emerging technologies, such as the Internet of Things and augmented or virtual reality, to tackle social isolation and loneliness among older adults. Furthermore, future studies should consider evaluating social isolation or loneliness while developing customized ICT solutions to provide more robust data on the effectiveness of the solutions.

Quantifying test-retest reliability of repeated objective attentional measures in Lewy body dementia

Objective cognitive impairment is a feature of Lewy body dementia (LBD), and computerised attentional tasks are commonly used as outcome measures in interventional trials. However, the reliability of these measures, in the absence of interventions, are unknown. This study examined the reliability of these attentional measures at short-term and longer-term follow-up stages. LBD patients (n = 36) completed computerised attentional tasks [simple and choice reaction time, and digit vigilance (SRT, CRT, DV)] at short-term (Day 0–Day 5) and longer-term (4 and 12 weeks) follow-up. Intra-class correlations (ICCs) were calculated to assess test–retest reliability. At short-term, the reciprocal SRT, CRT and DV mean reaction time to correct answers, the reciprocal DV coefficient of variation, and reciprocal power of attention (PoA) all showed excellent levels of reliability (all ICCs > 0.90). The reciprocal PoA showed the highest level of reliability (ICC = 0.978). At longer-term follow-up, only the reciprocal PoA had excellent levels of reliability (ICC = 0.927). Reciprocal SRT, CRT and DV reaction time to correct answers, and the CRT coefficient of variation values, showed good levels of test–retest reliability (ICCs ≥ 0.85). Contrary to expectations, most attentional measures demonstrated high levels of test–retest reliability at both short-term and longer-term follow-up time points. The reciprocal PoA composite measure demonstrated excellent levels of test–retest reliability, both in the short-term and long-term. This indicates that objective attentional tasks are suitable outcome measures in LBD studies and that the composite PoA measure may offer the highest levels of reliability.

Neuroprotective Effects of Novel Treatments on Acute Optic Neuritis-A Meta-Analysis.

Optic neuritis, inflammation of the optic nerve, can cause visual impairment through retinal nerve fiber layer (RNFL) degeneration. Optical coherence tomography could serve as a sensitive noninvasive tool for measuring RNFL thickness and evaluating the neuroprotective effects of treatment. We conducted a meta-analysis to compare RNFL loss between novel add-on treatments and corticosteroid therapy at least 3 months after acute optic neuritis. The outcome measures were mean differences (MDs) in (1) RNFL thickness compared with the baseline in the affected and unaffected eye and (2) LogMAR visual acuity (VA). Seven studies involving five novel agents (memantine, erythropoietin, interferon-beta, phenytoin, and clemastine) were analyzed. When compared with the baseline RNFL thickness of the affected eye, the neuroprotective effects of novel add-on treatments could not be demonstrated. The difference in visual outcomes was also not significant between the two treatment groups. One study revealed that phenytoin has the potential to alleviate RNFL loss when the baseline thickness of the unaffected eye is considered. Larger randomized controlled trials with suitable outcome measures are warranted to evaluate the neuroprotective effects of novel treatments. Further studies should also tailor therapies to specific patient populations and investigate a more targeted treatment for acute optic neuritis.

Oral morphine versus transmucosal diamorphine for breakthrough pain in children: methods and outcomes: UK (DIPPER study) consensus

ObjectivesNo randomised controlled trials have been conducted for breakthrough pain in paediatric palliative care and there are currently no standardised outcome measures. The DIPPER study aims to establish the feasibility...

Strategies for Empowering activities in Everyday life (SEE 1.0): study protocol for a feasibility study of an Internet-based occupational therapy intervention for people with stroke

BackgroundRehabilitation after stroke seldom focuses on needs related to an active everyday life and the process of change that people must undergo to adapt to an altered capacity and life situation. In particular, occupational therapy in the late phase needs to support clients in adopting sustainable self-initiated management strategies to regain daily activities and an active everyday life. To improve access to rehabilitation, the use of digital solutions has been suggested. This study aims to evaluate the feasibility of the Internet-based occupational therapy intervention “Strategies for Empowering activities in Everyday life” (SEE, version 1.0). We will investigate the feasibility of the intervention process in terms of acceptability and adherence as well as the most suitable outcome measures to evaluate SEE and improve the knowledge about the potential changes and outcomes of SEE for clients with stroke.MethodsThis feasibility study is based on a pretest posttest design without a control group. Quantitative and qualitative data will be collected from clients and staff concurrently embedded in a mixed-method design during the entire study.DiscussionThe project is a first test of a novel Internet-based occupational therapy intervention, and the research will contribute to the continued development and evaluation of the SEE programme. SEE can provide people with strategies in daily activities that can support them to live an active everyday life despite changed capacity and to improve access to rehabilitation interventions.Trial registrationNCT04588116. Name of the registry: Strategies Empowering Activities in Everyday Life (SEE 1.0). A Web-based Occupational Therapy Intervention. URL of trial registry record. Date of registry: Trial first posted: October 19, 2020; first submitted: October 2, 2020

Natural history study of spinal muscular atrophy with respiratory distress type 1 (SMARD1) in a cohort of European patients

Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is a rare childhood autosomal recessive motor neuron disease, caused by IGHMBP2 gene mutations. The main hallmarks of the disease are diaphragmatic palsy and progressive distal muscular atrophy and paralysis. Currently, there is no treatment and very little data are available on the disease progression. We aim to determine the disease natural course and to define the most suitable functional outcome measures, in the perspective of a future clinical trial.