Acceptability Of Outcome Measures Research Articles

Ulnohumeral Cross-Pinning in the Management of Traumatic Elbow Instability

Traumatic elbow instability (subluxation and dislocation) represents a challenging clinical entity that can be complicated by persistent intraoperative instability after attempted stabilization or recurrent postoperative instability. Both static and dynamic supplemental stabilization procedures have been described for cases where fracture fixation and ligament repair fail to restore stability. There is a paucity of prior studies involving ulnohumeral cross-pinning (UCP), which is a type of static supplemental stabilization. Our purpose was to assess complications and outcomes after UCP. We reviewed all surgical cases involving primary and revision UCP for traumatic elbow instability at a single center from 2017-2023. Baseline demographics were recorded. Outcomes including radiographs, range of motion, patient-reported outcome measures, and surgical complications were analyzed. Fourteen patients undergoing UCP were included with a mean follow-up of 27 months. Five cross-pinnings (36%) were performed during revision procedures. Mean visual analog scale (VAS) pain, Disabilities of the Arm, Shoulder, and Hand (QuickDASH), and the Single Assessment Numeric Evaluation (SANE) scores were 2.4, 34, and 69, respectively. The mean flexion-extension and pronation-supination arcs were 114° and 140°, with 85% achieving at least a 100° arc for flexion and forearm rotation. Five patients (36%) had complications, all of which required reoperation. Two complications involved postoperative instability: one radial head subluxation and one radial head dislocation. Both occurred in revision UCP cases indicated for recurrent postoperative instability. Ulnohumeral cross-pinning for persistent and recurrent elbow instability results in maintained ulnohumeral joint alignment, functional arcs of elbow range of motion, and acceptable patient-reported outcome measures, particularly in the setting of a primary procedure indicated for persistent intraoperative instability. Ulnohumeral cross-pinning is a reasonable supplemental stabilization procedure for complex elbow instability. Therapeutic IV.

Is the Rehabilitation Complexity Scale Useful in Individuals Undergoing In-Hospital Pulmonary Rehabilitation?

ObjectiveTo assess validity and responsiveness of the Extended Rehabilitation Complexity Scale (RCS-E v13) to in-hospital pulmonary rehabilitation (PR) in individuals with chronic respiratory diseases (CRD). DesignCross-sectional, multicentric study. Assessments in individuals attending units on 2 nonconsecutive days. SettingFourteen in-hospital PR units. ParticipantsFive hundred forty-seven individuals (59.2% male, age 72y [range, 65-78y]): 317 with chronic respiratory failure because of various causes (CRF); 96 with chronic obstructive pulmonary disease without CRF (COPD), 39 tracheostomized and ventilated (TX/V), and 95 with other diseases (Miscellaneous). InterventionAssessment of RCS-E v13 before and after the PR program. Main Outcome MeasuresRCS-E v13 and outcome measures: Barthel Index (BI), Barthel Index Dyspnea (BiD), Medical Research Council scale for dyspnea (MRC), COPD Assessment Test (CAT), Short Physical Performance Battery (SPPB), and 6-minute walk test (6MWT). ResultsThe highest RCS-E v13 admission values (median [interquartile range]) were found in TX/V (17 [15-18]) as compared with other groups (8 [7-10], 10 [9-12], and 8 [8-10] in COPD, CRF, and Miscellaneous, respectively, P<.001). At admission and discharge, RCS-E v13 correlated strongly with BI, 6MWT, and SPPB and moderately with MRC and BiD (r=.43-.60). After the program, RCS-E v13 as well as all outcome measures improved significantly in all groups (P<.001 for all). The size of improvement was different among groups according to the different variables. In the overall group, the effect size was high for changes in RCS-E v13 (Cohen's d=−2.0984), CAT (d=−1.1937), MRC (d=−1.0505), BiD (d=−.9364), and SPPB (d=.9231) whereas it was moderate for 6MWT (d=.7670) and BI (d=.6574). ConclusionsRCS-E v13 varies according to different CRD, is responsive to PR, has good construct and concurrent validity, and correlates with most of the accepted outcome measures of PR. Its scoring may provide useful information on the care burden of individuals undergoing PR.

High-frequency ultrasound evaluation of morphea: Retrospective analytical study.

To date, there are no accepted outcome measures to monitor morphea, and consensus on specific monitoring criteria for morphea remains elusive. A few studies have assessed the criterion validity of skin ultrasound in morphea. So, in this study, we approach ultrasound findings in morphea lesions. This was a retrospective-analytical study conducted between December 2021 and May 2023. Patients were clinically evaluated at a dermatology outpatient clinic and then referred for high-frequency ultrasound (HF-US) evaluation and were selected to be included in this study. The lesions were confirmed by histopathology as well. Sonographic evaluations were performed on the lesion site and the symmetrical uninvolved other side. Dermal thickness and dermal echogenicities were recorded. Statistical analysis of group differences was performed by using the 2-tailed Student t-test. A p-value of less than 0.05 was considered statistically significant. Forty-one morphea lesions in the inflammatory phase of 27 patients were included in the study. The mean dermal thickness of morphea lesions was 1107.97±414.3 and the mean dermal thickness of the control side was 1094.65±331.06, The difference between these two variables was not statistically significant. The mean dermal density of lesions was 49.13±18.97 and the mean dermal density of the control side was 52.22±25.33. The difference between these two variables was not statistically significant. This study shows that HF-US indicated increasing dermal thickness and reducing the dermal density of the morphea lesions in the inflammatory phase confirmed with the histopathology.

Cognitive Functional Therapy compared with usual physiotherapy care in people with persistent low back pain: a mixed methods feasibility randomised controlled trial in the United Kingdom National Health Service

ObjectivesTo determine the feasibility of completing a definitive randomised controlled trial (RCT), evaluating the clinical and cost-effectiveness of Cognitive Functional Therapy (CFT) in comparison to usual physiotherapy care (UPC), for people with persistent low back pain (LBP). Design and settingA two-arm parallel feasibility RCT completed in a United Kingdom (UK) Secondary Care National Health Service (NHS) physiotherapy service. ParticipantsSixty adult participants who reported LBP lasting for more than three months, that was not attributable to a serious (e.g. cancer) or specific (e.g. radiculopathy) underlying cause, were invited to participate. Participants were allocated at random to receive CFT or UPC. InterventionsCognitive Functional Therapy and Usual Physiotherapy Care for persistent LBP. Main outcome measuresThe primary outcome was the feasibility of completing a definitive RCT, defined by recruitment of at least 5 participants per month, delivery of CFT per protocol and securing relevant and acceptable outcome measures. Data concerning study processes, resources, management and participant reported outcome measures were collected at baseline, 3, 6 and 12-month follow-up. ResultsSixty participants (n = 30 CFT and n = 30 UPC) were recruited with 80% (n = 48), 72% (n = 43) and 53% (n = 32) retained at 3, 6 and 12-month follow-up respectively. NHS physiotherapists were trained to competence and delivered CFT with fidelity. CFT was tolerated by participants with no adverse events. Relevant and clinically important outcome data were collected at all time points (0.4%, 3%, 1% and 0.8% of data was missing from the returned outcome measure booklets at baseline and 3, 6 and 12-month follow-up respectively). The Roland-Morris disability questionnaire was considered the most suitable primary outcome measure with a proposed sample size of 540 participants for a definitive cluster RCT. ConclusionIt is feasible to conduct a randomised study of CFT in comparison to UPC for NHS patients. A future study should incorporate an internal pilot to address aspects of feasibility further, including participant retention strategies. Clinical trial registration numberISRCTN12965286 Contribution of the paper•Cognitive Functional Therapy (CFT) has shown encouraging results for reducing pain and disability in people with persistent LBP in previous clinical trials. The clinical and cost-effectiveness of CFT has not previously been evaluated in a definitive trial in the United Kingdom National Health Service. This is the first study to evaluate the feasibility of completing such a trial.•The findings of this study confirmed that;•NHS patients can be recruited on time and to target, with no safety concerns regarding a randomised controlled trial.•NHS physiotherapists can be trained to competence to deliver CFT with fidelity.•Clinically relevant and important data can be collected using standardised outcome measures.•New knowledge about the feasibility of completing a definitive trial of CFT in the UK NHS has been gained.

Implementing early rehabilitation and mobilisation for children in UK paediatric intensive care units: the PERMIT feasibility study.

Early rehabilitation and mobilisation encompass patient-tailored interventions, delivered within intensive care, but there are few studies in children and young people within paediatric intensive care units. To explore how healthcare professionals currently practise early rehabilitation and mobilisation using qualitative and quantitative approaches; co-design the Paediatric Early Rehabilitation and Mobilisation during InTensive care manual of early rehabilitation and mobilisation interventions, with primary and secondary patient-centred outcomes; explore feasibility and acceptability of implementing the Paediatric Early Rehabilitation and Mobilisation during InTensive care manual within three paediatric intensive care units. Mixed-methods feasibility with five interlinked studies (scoping review, survey, observational study, codesign workshops, feasibility study) in three phases. United Kingdom paediatric intensive care units. Children and young people aged 0-16 years remaining within paediatric intensive care on day 3, their parents/guardians and healthcare professionals. In Phase 3, unit-wide implementation of manualised early rehabilitation and mobilisation. Phase 1 observational study: prevalence of any early rehabilitation and mobilisation on day 3. Phase 3 feasibility study: acceptability of early rehabilitation and mobilisation intervention; adverse events; acceptability of study design; acceptability of outcome measures. Searched Excerpta Medica Database, Cumulative Index to Nursing and Allied Health Literature, MEDLINE, PEDro, Open grey and Cochrane CENTRAL databases. Narrative synthesis. In the scoping review we identified 36 full-text reports evaluating rehabilitation initiated within 7 days of paediatric intensive care unit admission, outlining non-mobility and mobility early rehabilitation and mobilisation interventions from 24 to 72 hours and delivered twice daily. With the survey, 124/191 (65%) responded from 26/29 (90%) United Kingdom paediatric intensive care units; the majority considered early rehabilitation and mobilisation a priority. The observational study followed 169 patients from 15 units; prevalence of any early rehabilitation and mobilisation on day 3 was 95.3%. We then developed a manualised early rehabilitation and mobilisation intervention informed by current evidence, experience and theory. All three sites implemented the Paediatric Early Rehabilitation and Mobilisation during InTensive care manual successfully, recruited to target (30 patients recruited) and followed up the patients until day 30 or discharge; 21/30 parents consented to complete additional outcome measures. The findings represent the views of National Health Service staff but may not be generalisable. We were unable to conduct workshops and interviews with children, young people and parents to support the Paediatric Early Rehabilitation and Mobilisation during InTensive care manual development due to pandemic restrictions. A randomised controlled trial is recommended to assess the effectiveness of the manualised early rehabilitation and mobilisation intervention. A definitive cluster randomised trial of early rehabilitation and mobilisation in paediatric intensive care requires selection of outcome measure and health economic evaluation. The study is registered as PROSPERO CRD42019151050. The Phase 1 observational study is registered Clinicaltrials.gov NCT04110938 (Phase 1) (registered 1 October 2019) and the Phase 3 feasibility study is registered NCT04909762 (Phase 3) (registered 2 June 2021). This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/21/06) and is published in full in Health Technology Assessment; Vol. 27, No. 27. See the NIHR Funding and Awards website for further award information.

Group-based PFMT programme for preventing and/or treating UI in pregnant women: protocol of a randomized controlled feasibility study

BackgroundUrinary incontinence (UI) is a prevalent health problem in women worldwide. Many women experience UI during pregnancy. The National Institute for Health and Care Excellence (NICE) recommended pelvic floor muscle training (PFMT) as the first-line conservative treatment for UI. However, it is not widely implemented due to the limited number of healthcare trainers. Group-based PFMT has been used with older women and a limited number of maternity studies. But the effectiveness of the group-based PFMT needs to be investigated because the overall quality of the studies is low. Therefore, this study aims to assess the feasibility of delivering a group-based PFMT programme for pregnant women in Nanjing city.MethodsThis feasibility study will be conducted in Nanjing Maternity and Child Health Care Hospital in China, using a mixed methods design to investigate the feasibility and acceptability of delivering group-based PFMT to pregnant women. Pregnant women with or without the symptoms of UI will be included. This study aims to recruit 48 pregnant women with 24 in each arm. Participants will receive either the group-based PFMT delivered by a midwife or usual antenatal care which includes only verbal instruction on PFMT. The study will assess the completion rates, acceptability of outcome measures, recruitment and retention rate and calculate an appropriate sample size for a future study.DiscussionThe results of this study will inform the design and implementation of a definitive randomized clinical trial to explore the effectiveness of the intervention.Trial registrationClinicalTrials.gov, NCT05242809.

Current Validated Clinical and Patient Reported Disease Outcome Measures in Juvenile Idiopathic Arthritis.

Juvenile idiopathic arthritis (JIA) is a common chronic illness in childhood and comprises seven categories based on the International League of Associations for Rheumatology (ILAR) criteria. Accurate assessment and measurement of the clinical, functional, and quality of life outcomes of patients with JIA are paramount for understanding the disease course and formulating effective treatment strategies. Interest in the development and use of outcome measurements specifically focused on rheumatologic conditions has greatly expanded over the last two decades, adding to and improving upon the established disease measures. Furthermore, many of these measures have been validated using the widely accepted Outcome Measures in Rheumatology (OMERACT) core principles of instrument validation, allowing researchers and clinicians to gain confidence in these tools. This review summarizes the current validated disease outcome measures in JIA, including clinical, imaging, patient-reported, and functional outcome measurement tools, and highlights ongoing work that continues to refine and improve upon the available tools. The clinical disease outcome measures discussed in this review include physician global assessment (PhGA), American College of Rheumatology (ACR, Wallace) criteria for clinical inactive disease and clinical remission, juvenile arthritis disease activity scores (JADAS), juvenile spondyloarthritis disease activity index (JSPaDA), juvenile arthritis damage index (JADAI), and the ACR pediatric response scores. The imaging outcome measures discussed include the Dijkstra composite scores, childhood arthritis radiographic score of the hip (CARSH), and Poznanski Score. The patient-reported disease outcome measures discussed include patient global assessment (PtGA), patient-reported outcome measurement information system for JIA (PROMIS), juvenile arthritis parent/child centered disease assessment index (JAPAI, JACAI), juvenile arthritis multidimensional assessment report (JAMAR), and the Pediatric quality of life inventory rheumatology module (PedsQL). The functional outcome tools discussed include the Childhood Health Assessment Questionnaire (CHAQ), juvenile arthritis functionality scale and index (JAFS and JASI), and Juvenile Arthritis Functional Assessment Report and Scale (JAFAS and JAFAR).

A rehabilitation intervention to improve recovery after an episode of delirium in adults over 65 years (RecoverED): study protocol for a multi-centre, single-arm feasibility study

BackgroundDelirium affects over 20% of all hospitalised older adults. Delirium is associated with a number of adverse outcomes following hospital admission including cognitive decline, anxiety and depression, increased mortality and care needs. Previous research has addressed prevention of delirium in hospitals and care homes, and there are guidelines on short-term treatment of delirium during admission. However, no studies have addressed the problem of longer-term recovery after delirium and it is currently unknown whether interventions to improve recovery after delirium are effective and cost-effective. The primary objective of this feasibility study is to test a new, theory-informed rehabilitation intervention (RecoverED) in older adults delivered following a hospital admission complicated by delirium to determine whether (a) the intervention is acceptable to individuals with delirium and (b) a definitive trial and parallel economic evaluation of the intervention are feasible.MethodsThe study is a multi-centre, single-arm feasibility study of a rehabilitation intervention with an embedded process evaluation. Sixty participants with delirium (aged > 65 years old) and carer pairs will be recruited from six NHS acute hospitals across the UK. All pairs will be offered the intervention, with follow-up assessments conducted at 3 months and 6 months post-discharge home. The intervention will be delivered in participants’ own homes by therapists and rehabilitation support workers for up to 10 intervention sessions over 12 weeks. The intervention will be tailored to individual needs, and the chosen intervention plan and goals will be discussed and agreed with participants and carers. Quantitative data on reach, retention, fidelity and dose will be collected and summarised using descriptive statistics. The feasibility outcomes that will be used to determine whether the study meets the criteria for progression to a definitive randomised controlled trial (RCT) include recruitment, delivery of the intervention, retention, data collection and acceptability of outcome measures. Acceptability of the intervention will be assessed using in-depth, semi-structured qualitative interviews with participants and healthcare professionals.DiscussionFindings will inform the design of a pragmatic multi-centre RCT of the effectiveness and cost-effectiveness of the RecoverED intervention for helping the longer-term recovery of people with delirium compared to usual care.Trial registrationThe feasibility study was registered: ISRCTN15676570

Protocol for the feasibility and implementation study of a model of best practice in primary care led postdiagnostic dementia care: PriDem

IntroductionCare is often inadequate and poorly integrated after a dementia diagnosis. Research and policy highlight the unaffordability and unsustainability of specialist-led support, and instead suggest a task-shared model, led by...

Understanding clinical outcome measures reported in HIV pregnancy studies involving antiretroviral-naive and antiretroviral-experienced women.

HIV infection is a clinically significant public health disease and contributes to increased risk of maternal and fetal morbidity and mortality. HIV pregnancy studies use outcome measures as metrics to show how people with HIV feel, function, or survive. These endpoints are crucial for tracking the evolution of HIV illness over time, assessing the effectiveness of antiretroviral therapy (ART), and comparing outcomes across studies. Although the need for ideal outcome measures is widely acknowledged, selecting acceptable outcome measures for these HIV pregnancy studies can be challenging. We discuss the many outcome measures that have been implemented over time to assess HIV in pregnancy studies, their benefits, and drawbacks. Finally, we offer suggestions for improving the reporting of outcome measures in HIV in pregnancy studies. Medical professionals can best care for pregnant women living with HIV receiving ART by having a thorough understanding of these outcome metrics.

A personhood and citizenship training workshop for care home staff to potentially increase wellbeing of residents with dementia: intervention development and feasibility testing of a cluster randomised controlled trial

BackgroundIn the UK, one third of people with dementia live in residential care homes, a sector where high staff turnover negatively affects continuity of care. To examine the effect of including personhood and citizenship principles in training, interventions need to be robustly tested, with outcomes relevant to residents with dementia.MethodsPhase one intervention development: The training intervention (PERSONABLE) comprised five reflective exercises facilitated by a mental health nurse/researcher. PERSONABLE was informed by four focus groups, and one field exercise, consisting of care home staff and family members. Phase two feasibility testing: Participants were (i) care home residents with dementia and (ii) care home staff working in any role. After baseline measurements, care homes were randomly allocated to (i) staff receiving PERSONABLE training or (ii) training as usual. Feasibility outcomes were the recruitment and attrition of care homes, residents and staff members (measured ten weeks between randomisation and follow-up), the acceptability of the training intervention PERSONABLE, and acceptability of outcome measures. The care home environment was evaluated, at baseline, using the Therapeutic Environment Screening Survey for Residential Care Homes. Measurements conducted at baseline and follow-up were resident wellbeing (Dementia Care Mapping™), staff knowledge of and confidence with personhood and citizenship (Personhood in Dementia Questionnaire and a perceived ability to care visual analogue scale). Inter-rater agreement for Dementia Care Mapping™ was undertaken at follow-up in one intervention and one training as a usual care home.ResultsPhase one: The developed reflective approach to the PERSONABLE exercises appeared to give staff a holistic understanding of residents living with dementia, seeing them as autonomous people rather than reductively as persons with a condition. Phase two: Six care homes, 40 residents and 118 staff were recruited. Four residents were lost to follow-up. Twenty-nine staff in the PERSONABLE arm of the study received the training intervention. In the PERSONABLE arm, 26 staff completed both baseline and follow-up measurements compared to 21 in the training as the usual arm. The most common reason for the loss to follow-up of staff was leaving employment. For the outcome measure Dementia Care Mapping™, the proportion of overall agreement between the two observers was 18.6%. High attrition of staff occurred in those homes undergoing leadership changes.ConclusionWith the right approach, it is possible to achieve good engagement during trial recruitment and intervention delivery of care home managers, staff and residents. Organisational changes are a less controllable aspect of trials but having a visible researcher presence during data collection helps to capitalise the engagement of those staff remaining in employment. Tailored, brief and flexible training interventions encourage staff participation. Simplification of study methods helps promote and retain sufficient staff in a definitive randomised controlled trial. This study found that some components of Dementia Care Mapping™ work effectively as an outcome measure. However, inter-rater reliability was poor, and the practical implementation of the measurement would need a great deal of further refinement to accurately capture the effect of a training intervention if delivered across a large number of clusters. The Dementia Care Mapping™ measurement fidelity issue would be further complicated if using multiple different unacquainted observers.Trial registrationRegistered with the ISRCTN under the title: Does a dementia workshop, delivered to residential care home staff, improve the wellbeing of residents with dementia? Trial identifier: ISRCTN13641553. Registered: 30/05/2017 http://www.isrctn.com/ISRCTN13641553.

Using the Vitiligo Noticeability Scale in clinical trials: construct validity, interpretability, reliability and acceptability.

Validated outcome measures are needed for vitiligo trials. To assess construct validity, interpretability, reliability and acceptability of the Vitiligo Noticeability Scale (VNS). We used images of vitiligo before and after treatment, plus outcome data, from the HI-Light Vitiligo trial. We compared outcome assessments made by trial participants with assessments of images by clinicians and people with vitiligo who were not trial participants [Patient and Public Involvement (PPI) panel]. Hypothesis testing assessed psychometric properties of the VNS, with κ statistics to assess agreement between outcomes. Three focus groups and two online discussion groups provided insight into the use of VNS by people with vitiligo. Our hypothesis of a positive association between VNS and participant-reported global treatment success was supported for trial participants (κ = 0·41 if VNS success was defined as ≥ 4; κ = 0·71 if VNS success was defined as ≥ 3), but not for the blinded PPI panel (κ = 0·28). As hypothesized, the association with participant-reported global success was higher for VNS (κ = 0·41) than for clinician-reported percentage repigmentation (κ = 0·17). Seventy-five per cent of trial participants valued a VNS of 3 (partial response) as a treatment success. Test-retest reliability was good: κ = 0·69 (95% confidence interval 0·63-0·74). Age and skin phototype did not influence interpretation of the VNS scores. To people with vitiligo, the VNS is an acceptable and meaningful patient-reported outcome measure. Trial participants may assess their vitiligo differently compared with blinded assessors. A VNS score of 3 may be more highly valued by people undergoing vitiligo treatment than was previously thought. What is already known about this topic? Vitiligo is a common condition, and can have a considerable psychological impact. A Vitiligo Core Outcome Set is being developed, to enable the results of vitiligo trials to be compared and combined more easily. The Vitiligo Noticeability Scale (VNS) is a patient-reported outcome measure (PROM) developed in partnership with people with vitiligo; initial validation studies have been promising. What does this study add? The VNS shows good construct validity, reliability and acceptability; it can be used in all ages and skin phototypes. All five levels of the VNS scale should be reported for transparency, to aid interpretation of trial findings, and to facilitate meta-analysis in systematic reviews. VNS assessments made by trial participants and independent observers are likely to be qualitatively different, making blinded assessment of VNS by independent observers difficult to interpret. Blinding of participants to trial interventions is recommended whenever possible. What are the clinical implications of the work? The VNS can be used as a PROM to assess the cosmetic acceptability of repigmentation at individual patches of vitiligo. A VNS score of 3 or more is likely to be valued by patients as a treatment success.

Hyperoxic exposure monitoring in diving: A farewell to the UPTD

Depending on pO2 and exposure time hyperoxic breathing gas may cause injury in many organs including the lungs. Pulmonary oxygen toxicity (POT) may be asymptomatic, but will initially present as a tracheobronchitis in symptomatic subjects. A number of objective measurements of POT have been investigated, but the decrement in vital capacity (VC) has remained the most accepted outcome measure. The unit pulmonary toxic dose (UPTD) has been established as the most common exposure index for POT in diving. UPTD is calculated based on the pO2 and exposure time. A literature search identified five models predicting POT, but no model would accurately predict VC change for the full range of pO2 variation and exposure time relevant for surface-oriented diving. Nevertheless, compared to UPTD, the K-index(K = t2*pO24.57, where t = time (hours) and pO2 = inspired pO2 (atm)) suggested by Arieli performed better for pO2 &gt; 150 kPa and allowed estimation of recovery. We recommend that the Arieli K-index should replace UPTD as the POT exposure index for all surface-oriented diving. Based on the limited data available we suggest a daily threshold of K = 120 for a maximum of two diving days followed by two days of recovery. For five consecutive days of diving, we recommend that the threshold should not exceed K=70 and two recovery days should be allowed. For multiday diving without days of recovery, the daily exposure should probably be limited to K = 40-50.

Validation studies of rheumatoid arthritis patient-reported outcome measures in populations at risk for inequity: A systematic review and analysis using the OMERACT summary of measurement properties equity table

BackgroundExisting patient-reported outcome measures (PROMs) in rheumatoid arthritis (RA) may be limited in their applicability to populations that are at risk for inequities. We conducted a systematic review to identify and rate evidence in the validation studies for PROMs in populations at risk for inequity. MethodsA systematic review of MEDLINE and EMBASE was completed. The search strategy was developed to identify measurement property studies for PROMs of interest (selected pain, disease activity, global evaluation and quality of life scales) in patients with RA. We identified experimental, observational, and qualitative studies reporting analysis of feasibility, construct validity and discriminant ability metrics for populations at risk for inequity by various factors including race, ethnicity, culture or language; employment status; sex and gender identity; education level; socioeconomic status; social support; age; health literacy and disability. These were rated based on the OMERACT Summary of Measurement Properties Equity table. ResultsFrom 19,786 titles and abstracts screened, we identified 14 unique studies reporting validation metrics for pain (n = 3), DAS28-ESR or DAS28-CRP (n = 2), ACR20 (n = 1), patient global assessment (n = 2), EQ5D (n = 4), and PROMIS® (n = 3) by race (n = 10 studies), age (n = 6 studies), sex (n = 5 studies), education level (n = 2 studies), and disability, literacy, employment status, social support level and socioeconomic status (n = 1 study each). Five studies reported on feasibility, 12 reported construct validity metrics, and 4 studies reported on discriminant validity metrics. All studies by culture or language were rated as having good measurement property metrics. There was limited assessment of measurement property metrics for other populations at risk for inequity. ConclusionOur study highlights important gaps in patient representation in rheumatology research for accepted outcome measures. New outcome measures being developed for research purposes and clinical practice should ensure and report representation of patients from populations at risk for inequities in the testing of metrics of feasibility, construct validity and discriminant ability metrics.

Cross-cultural adaptation and validation of the Arabic version of oxford ankle foot questionnaire for children

Purpose The purpose of this study was to translate and cross-culturally adapt the original English version of the Oxford Ankle Foot Questionnaire (OxAFQ-c) into the Arabic language, and to evaluate its psychometric properties among Arabic speaking children aged from 5 to 16 years in Saudi Arabia. Materials and methods An Arabic OxAFQ-c for children was developed according to established guidelines (ISPOR). The Arabic OxAFQ-c version was completed by eighty-seven patients with foot and ankle problems and their caregivers. Construct validity of the Arabic OxAFQ-c was also examined. Results The reliability analysis of OxAFQ-Ar exhibited good internal consistency in both children's and parent's versions for all domains (α = 0.80–0.89) and excellent test-retest reliability in both versions for all domains. (ICC = 0.87–0.94). A moderate correlation between the OxAFQ-Ar and PedsQL 4.0 was observed indicating moderate construct validity. Conclusions The OxAFQ-c was successfully translated and cross-cultural adapted into the Arabic language. The OxAFQ-Ar is a valid, reliable and useful quality of life questionnaire for evaluating children’s ankle foot problems. IMPLICATION OF REHABILITATION OF OxAFQ-c ARABIC VERSION The Arabic version of the OxAFQ-c is an acceptable, clear and comprehensible outcome measure. The Arabic version of the OxAFQ-c demonstrated evidence supporting its internal consistency, test-retest reliability and construct validity as a measure to evaluate foot and ankle pathologies in patients aged 5–16 years. The Arabic OxAFQ-c has very good internal consistency, test-retest reliability, and acceptable measurement error with no floor and ceiling effects. The Arabic version of the OxAFQ-c can be used in daily clinical practice and in research studies to assess children aged from 5 to 16 years in Arabic speakers with ankle-foot conditions.

Adapted cardiac rehabilitation for people with sub-acute, mild-to-moderate stroke: a mixed methods feasibility study

ObjectiveTo determine the recruitment strategy, acceptability, adherence, outcome measures, and adverse events for a definitive study that will explore adapted cardiac rehabilitation (CR) for people post-stroke with mild-to-moderate severity stroke in the sub-acute stage of recovery. DesignMixed methods feasibility study. SettingAcute hospital setting, neurology outpatients and community hospitals. Participants32 participants with stroke (mean age: 64.4 years) of median National Institutes of Health Stroke Scale (NIHSS) score 2 (range: 0 to 6) within six months of stroke. InterventionAll participants attended six weeks, adapted CR within one to six months after a stroke. A combined class with people post cardiac event. Main outcome measuresIncremental shuttle walk test (ISWT), blood pressure, heart rate, weight, body mass index, quality of life, fatigue, anxiety and depression, tone, falls, stroke attitude and knowledge, physical activity (accelerometry) and functional ability. QualitativeInterviews with participants, non-participants and people post-cardiac event. Focus groups with Stroke and CR teams. Results32 participants were recruited. The programme was acceptable to people with mild stroke (NIHSS<3) and people post cardiac events; 80% of classes attended, a mean of 9.6 classes, with six drop-outs. The ISWT was an acceptable outcome measure (for NIHSS<3) and most measures showed positive changes. There was one adverse event. ConclusionA definitive study to determine the effect of six weeks of adapted CR on cardiorespiratory fitness (CRf) in people who have had a mild severity stroke (NIHSS<3) in the sub-acute phase of recovery, is feasible. Teams need specialist education and support. A more specialist service may be needed for people with a stroke severity defined by NIHSS>2. Clinical Trial Registration NumberISRCTN14861846.

Facilitating Perinatal Access to Resources and Support (PeARS): a feasibility study with external pilot of a novel intervention

BackgroundUp to 50% of women in areas of high socio-economic deprivation are at risk of developing depressive symptoms in pregnancy. Feeling well supported, can facilitate good mental health perinatally. A brief, innovative intervention to facilitate access to support and resources was developed and tested. This included one antenatal and one postnatal session, each with three evidence-based components: i) support from a non-professional peer to enable a woman to identify her needs; ii) information about local community services and signposting; and iii) development of a personalised If–Then plan to access that support. The aims were to evaluate the intervention and research methods for feasibility and acceptability for perinatal women, maternity care providers and peers, and provide preliminary effectiveness indications.MethodsPregnant women living in an area of high deprivation were recruited from community-based antenatal clinics and randomised to intervention or control condition (a booklet about local resources). Outcome measures included women’s use of community services by 34 + weeks gestation and 6 months postnatally; mental health and wellbeing measures, and plan implementation. Interviews and focus groups were conducted with women participants, providers, and peers. Data were analysed using framework analysis. Recruitment and retention of peers and participants, intervention fidelity, and acceptability of outcome measures were recorded.ResultsPeer facilitators could be recruited, trained, retained and provide the intervention with fidelity. One hundred twenty six women were recruited and randomised, 85% lived in the 1% most deprived UK areas. Recruitment constituted 39% of those eligible, improving to 54% after midwifery liaison. Sixty five percent were retained at 6 months postnatally. Women welcomed the intervention, and found it helpful to plan access to community services. Providers strongly supported the intervention philosophy and integrated this easily into services. The study was not powered to detect significant group differences but there were positive trends in community service use, particularly postnatally. No differences were evident in mental health and wellbeing.ConclusionsThis intervention was well received and easily integrated into existing services. Women living in highly deprived areas could be recruited, randomised and retained. Measures were acceptable. Peer facilitators were successfully trained and retained. Full effectiveness studies are warranted.

Cross-cultural adaptation and validation of the Arabic version of the upper extremity functional index

Purpose The purpose of this study was to translate and cross-culturally adapt the UEFI into Modern Standard Arabic language and to examine its psychometric properties among patients with upper extremity musculoskeletal disorders. Materials and Methods Translation and cross-cultural adaptation were done following Beaton’s guidelines. Internal consistency, test-retest reliability, measurement error and floor and ceiling effects for the Arabic UEFI were tested among 109 patients with upper extremity musculoskeletal disorders. Construct validity of the Arabic UEFI was also examined. Results Translation and cross-cultural adaptation processes were generally smooth with no major issues. The Arabic UEFI was considered appropriate and comprehensible by the participants. Internal consistency for Arabic UEFI was adequate (Cronbach's alpha = 0.96). Test-retest reliability for Arabic UEFI was excellent with ICC2,1=0.92. Measurement error was acceptable with a standard error of measurement of 5.5 and minimal detectable change of 12.8 points. Arabic UEFI shows no floor or ceiling effects. The results supported the majority of the construct validity predefined hypotheses (78%) supporting the construct validity of Arabic UEFI as a measure of upper extremity function. Conclusion The Arabic UEFI is an appropriate, valid and reliable outcome measure for Arabic-speaking patients with upper extremity musculoskeletal disorders. Implication for Rehabilitation: The Arabic UEFI is an acceptable, clear and comprehensible outcome measure. The Arabic UEFI has excellent internal consistency, test-retest reliability, and acceptable measurement error with no floor and ceiling effects. The Arabic UEFI is a valid measure of upper extremity activity limitation.

Response (minimum clinically relevant change) in ASD symptoms after an intervention according to CARS-2: consensus from an expert elicitation procedure

The lack of consensual measures to monitor core change in Autism Spectrum Disorder (ASD) or response to interventions leads to difficulty to prove intervention efficacy on ASD core symptoms. There are no universally accepted outcome measures developed for measuring changes in core symptoms. However, the CARS (Childhood Autism Rating Scale) is one of the outcomes recommended in the EMA Guideline on the clinical development of medicinal products for the treatment of ASD. Unfortunately, there is currently no consensus on the response definition for CARS among individuals with ASD. The aim of this elicitation process was to determine an appropriate definition of a response on the CARS2 scale for interventions in patients with Autism Spectrum Disorder (ASD). An elicitation process was conducted following the Sheffield Elicitation Framework (SHELF). Five experts in the field of ASD and two experts in expert knowledge elicitation participated in an 1-day elicitation workshop. Experts in ASD were previously trained in the SHELF elicitation process and received a dossier of scientific evidence concerning the topic. The response definition was set as the mean clinically relevant improvement averaged over all patients, levels of functioning, age groups and clinicians. Based on the scientific evidence and expert judgment, a normal probability distribution was agreed to represent the state of knowledge of this response with expected value 4.03 and standard deviation 0.664. Considering the remaining uncertainty of the estimation and the available literature, a CARS-2 improvement of 4.5 points has been defined as a threshold to conclude to a response after an intervention. A CARS-2 improvement of 4.5 points could be used to evaluate interventions' meaningfulness in indivudals. This initial finding represents an important new benchmark and may aid decision makers in evaluating the efficacy of interventions in ASD.

Physical activity monitoring to assess disability progression in multiple sclerosis.

BackgroundClinical outcome measurement in multiple sclerosis (MS) usually requires a physical visit. Remote activity monitoring (RAM) using wearable technology provides a rational alternative, especially desirable when distance is involved or in a pandemic setting.ObjectiveTo validate RAM in progressive MS using (1) traditional psychometric methods (2) brain atrophy.Methods56 people with progressive MS participated in a longitudinal study over 2.5 years. An arm-worn RAM device measured activity over six days, every six months, and incorporated triaxial accelerometry and transcutaneous physiological variable measurement. Five RAM variables were assessed: physical activity duration, step count, active energy expenditure, metabolic equivalents and a composite RAM score incorporating all four variables. Other assessments every six months included EDSS, MSFC, MSIS-29, Chalder Fatigue Scale and Beck’s Depression Inventory. Annualized brain atrophy was measured using SIENA.ResultsRAM was tolerated well by people with MS; the device was worn 99.4% of the time. RAM had good convergent and divergent validity and was responsive, especially with respect to step count. Measurement of physical activity over one day was as responsive as six days. The composite RAM score positively correlated with brain volume loss.ConclusionRemote activity monitoring is a valid and acceptable outcome measure in MS.