Evidence For Measurement Properties Research Articles

Young Infants Clinical Signs Study 8-sign Algorithm for Identification of Sick Infants Adapted for Routine Home Visits: A Systematic Review and Critical Appraisal of its Measurement Properties.

Objective. The 8-sign algorithm adapted from the Young Infants Clinical Signs Study (YICSS) is widely used to identify sick infants during home visits (YICSS-home algorithm). We aimed to critically appraise the development and evidence of measurement properties, including sensibility, reliability, and validity, of the YICSS-home algorithm. Methods. Relevant studies were identified through a systematic literature search. Results. The YICSS-home algorithm has good sensibility. The algorithm demonstrated at least moderate inter-rater reliability and sensitivity ranging from 69% to 80%. However, the algorithm was developed among sick infants brought for care to a health facility and not initially developed for use by community health workers (CHWs) during home visits. Some important risk factors were omitted at item generation. Inter-CHW reliability and construct validity have not been estimated. Conclusion. Future research should build on the strengths of the YICSS-home algorithm and address its limitations to develop a new algorithm with improved predictive accuracy.

The measurement of fatigue in clinicians within hospital settings: A systematic review of measurement properties.

Medical staff fatigue leads to accidents and mistakes and puts patient safety at risk. A measure of fatigue in the workplace may help to quantify, predict, and manage fatigue. This review aimed to evaluate instruments used to measure fatigue in medical staff within hospitals. A systematic review following the JBI methodology was undertaken. A search for articles was conducted in 2021. Included articles (all validation studies) were assessed for methodological quality using the COSMIN checklist. Measurement property data was evaluated for Quality of Evidence using GRADE methodology. Ten studies representing five instruments were reviewed: Occupational Fatigue Exertion and Recovery scale (now superseded); Occupational Fatigue Exertion and Recovery scale (15-item); Multidimensional Fatigue Inventory; Need for Recovery Scale; and the Swedish Occupational Fatigue Inventory. Four instruments show promise for measuring fatigue in hospital medical staff, however, there is limited certainty in the measure property estimates. The Quality of Evidence for measurement properties for all instruments is insufficient. Further validation studies following the COSMIN standards are needed before recommendations for use can be made.

Updated recommendations on measures for clinical trials in pediatric chronic pain: a multiphase approach from the Core Outcomes in Pediatric Persistent Pain (Core-OPPP) Workgroup.

Many gaps remain in finding effective, safe, and equitable treatments for children and adolescents with chronic pain and in accessing treatments in different settings. A major goal of the field is to improve assessment of pain and related experience. Valid and reliable patient-reported outcome measures are critical for advancing knowledge of clinical interventions for pediatric chronic pain. Building on the work of the Ped-IMMPACT group, we previously updated a core outcome set (COS) for pediatric chronic pain clinical trials using stakeholder feedback from providers, youth, and parents. The new COS includes 3 mandatory domains: pain severity, pain-related interference with daily living, and adverse events and 4 optional domains: overall well-being, emotional functioning, physical functioning, and sleep quality. The aim of this study was to use a multiphased approach to recommend specific measures for each of the 7 domains identified in our new COS for pediatric chronic pain. We synthesized evidence through conducting the following: (1) a Delphi study of experts to identify candidate measures for the new COS domains, (2) a review phase to gather evidence for measurement properties for candidate measures, and (3) an expert consensus conference to reach agreement on measurement recommendations. Final recommendations included 9 patient-reported measures. Important contextual considerations are discussed, and guidance is provided regarding strengths and limitations of the recommendations. Implementation of these recommendations may be enhanced by widespread dissemination and ease of access to measurement tools.

Are Ecological Momentary Assessments of Pain Valid and Reliable? A Systematic Review and Meta-analysis.

This systematic review and meta-analysis aimed to determine the level of evidence for the psychometric properties of Ecological Momentary Assessment (EMA) in populations with persistent pain. Five databases were searched from 1980 to December 2021. Two reviewers independently screened the titles, abstracts, and full text, extracted data, and assessed adherence to reporting standards and methodological rigor before evaluating the quality of evidence. A meta-analysis, including the pooling of correlations for the relevant EMA pain outcomes, was completed. Overall, 3270 studies were identified, with 14 studies meeting inclusion criteria. Meta-analyses confirmed good to excellent relationships demonstrated between EMA and recalled pain intensity and interference across different timeframes. Many of the included studies did not fully adhere to recommended reporting standards, and the quality of included studies was either doubtful or inadequate due to methodological flaws. The level of evidence for measurement properties of pain outcomes was low for the criterion validity of pain intensity and interference and very low for reliability and construct validity of pain intensity and interference. Ecological momentary assessment of pain experience appears both valid and reliable. Although the levels of evidence were low or very low, these findings provide preliminary support for the use of EMA in clinical practice and research settings. Potential strengths of EMA include providing measures with greater ecological validity while also reducing recall bias, both pertinent in pain outcome measurement. More research, including higher-quality studies, is needed to demonstrate further support for EMA, including the need for establishing other types of validity.

10.3 Achieving Meaningful Health Outcome Measurement in Pediatric Care: Are Our Tools Fit for a Purpose?

Standard measurement sets are increasingly being developed and implemented to facilitate routine outcome monitoring and value-based clinical care. The International Consortium for Health Outcomes Measurement (ICHOM) recently developed a standard set to benchmark overall pediatric health outcomes in routine care, which includes measures of foundational aspects of child and youth mental health and functioning. The aim of this study was to determine if these measures have sufficient measurement property evidence to be recommended for implementation.

Investigating quality of life instrument measurement properties for adults with active venous leg ulcers: A systematic review.

The primary objective of this systematic review was to identify which quality of life instruments have been applied in published studies of patients with active venous leg ulcers. Our secondary objective was to map the measurement properties of each identified quality of life instrument and to inform future recommendations for clinical practice and research. We searched CINAHL, Ovid Medline, Ovid Emcare and ProQuest to identify studies published from 1 January 2000 to 31 July 2021. Eleven studies that utilised quality of life instruments in adults with active venous leg ulcers met the inclusion criteria. Thirteen quality of life instruments were identified as some studies utilised both generic and condition‐specific quality of life instruments. Six out of nine (6/9) instruments were rated ‘very good’ of methodological quality on internal consistency; 1/7 studies rated ‘adequate’ on reliability; 2/4 rated ‘adequate’ on content validity; 3/6 studies rated ‘adequate’ on structural validity; 5/6 rated ‘adequate’ on hypotheses testing for construct and 2/6 studies rated ‘adequate’ on responsiveness. There is limited evidence of measurement properties of quality of life instruments for people with active venous leg ulcers. The Venous Leg Ulcer Quality of Life Questionnaire (VLU‐QoL) could be provisionally recommended for use although from our review it is clear further studies to assess VLU‐QoL measurement properties are needed to inform future recommendations for clinical practice and research.

Outcomes Evaluating Quality of Life and Their Measurement Properties in Early-onset Scoliosis: A Systematic Review.

Early-onset scoliosis (EOS) is a spinal deformity affecting children under the age of 10. Understanding the impact of EOS on quality of life can be achieved through appropriate patient/carer-reported outcome measures (PROMs). The aim of this systematic review was to identify, evaluate and summarize the evidence for the measurement properties of health-related quality of life (HR-QoL) outcome measures in the EOS population. A 2-stage search methodology was conducted across the PubMed, MEDLINE, EMBASE, EMCARE, PsychINFO, and CINAHL databases. Search 1 identified PROMs used to evaluate the quality of life in EOS, and search 2 identified studies of the measurement properties of these PROMs. Two reviewers performed searching, study screening and selection and assessed studies for risk of bias using the Consensus-based Standards for the selection of health Measurement INstruments (COSMIN) Risk of Bias checklist. One reviewer performed data extraction. Evidence for each measurement property per PROM was summarized and evaluated using a modified GRADE approach. Search 1 identified 19 (10 disease-specific, 9 generic) PROMs that have been used to assess HR-QoL or a relevant domain in the EOS population. Search 2 identified only one PROM (the 24-item Early-Onset Scoliosis Questionnaire, EOSQ) with eligible studies (n=4) evaluating measurement properties. There is low-quality evidence for sufficient content validity, very low-quality evidence for sufficient reliability and low-quality evidence for sufficient criterion validity, specifically regarding the pulmonary function domain) of the EOSQ. Construct validity and responsiveness were rated as indeterminate. There were no studies on measurement error. Our search strategy found that measurement properties have only been evaluated for one PROM, the EOSQ. There is low-quality evidence for sufficient measurement properties for the EOSQ in patients under the age of 10 with EOS. Further research is needed to improve the quality of evidence for EOSQ measurement properties and investigate other PROMs in this population. Level II.

Identification of tools used to assess the external validity of randomized controlled trials in reviews: a systematic review of measurement properties

BackgroundInternal and external validity are the most relevant components when critically appraising randomized controlled trials (RCTs) for systematic reviews. However, there is no gold standard to assess external validity. This might be related to the heterogeneity of the terminology as well as to unclear evidence of the measurement properties of available tools. The aim of this review was to identify tools to assess the external validity of RCTs. It was further, to evaluate the quality of identified tools and to recommend the use of individual tools to assess the external validity of RCTs in future systematic reviews.MethodsA two-phase systematic literature search was performed in four databases: PubMed, Scopus, PsycINFO via OVID, and CINAHL via EBSCO. First, tools to assess the external validity of RCTs were identified. Second, studies investigating the measurement properties of these tools were selected. The measurement properties of each included tool were appraised using an adapted version of the COnsensus based Standards for the selection of health Measurement INstruments (COSMIN) guidelines.Results38 publications reporting on the development or validation of 28 included tools were included. For 61% (17/28) of the included tools, there was no evidence for measurement properties. For the remaining tools, reliability was the most frequently assessed property. Reliability was judged as “sufficient” for three tools (very low certainty of evidence). Content validity was rated as “sufficient” for one tool (moderate certainty of evidence).ConclusionsBased on these results, no available tool can be fully recommended to assess the external validity of RCTs in systematic reviews. Several steps are required to overcome the identified difficulties to either adapt and validate available tools or to develop a better suitable tool.Trial registrationProspective registration at Open Science Framework (OSF): https://doi.org/10.17605/OSF.IO/PTG4D.

Developing a Conceptual Disease Model of Patient Experiences and Identifying Patient-Reported Clinical Outcome Assessments for Use in Trials of Treatments for Focal Onset Seizures.

PurposeTo identify concepts important to understanding the experiences of adults with focal onset seizures (FOS) and evaluate clinical outcome assessments (COAs) for measuring these concepts in clinical trials of treatments for FOS.MethodsA search of published qualitative research, clinical trials, and approved product labels for FOS treatments was performed to develop a conceptual disease model (CDM) of patients’ experience of living with FOS. Concepts of interest (COI) were selected, and a second literature search was conducted to identify COAs measuring these concepts. Ten COAs were selected and reviewed to document their development process, evidence of measurement properties, and methods for interpreting change scores using criteria proposed in regulatory guidelines for patient-reported outcomes to support label claims.ResultsConcepts identified from the published literature (13 articles, 1 conference abstract), 24 clinical trials, and 8 product labels were included in a novel CDM. Impacts on physical, cognitive, and social and emotional function were chosen as COI for evaluating treatment outcomes for FOS; the additional concept of social support and coping strategies was chosen to understand patients’ lived experiences. From 51 unique COAs identified, 10 were selected based on their potential coverage of the COI; some symptom severity and health-related quality of life (HRQoL) COAs covered multiple COI. Of these 10, 8 COAs evaluated impacts/limitations on physical function, 8 measured social and emotional impacts, and 5 assessed social support and coping strategies. While most assessments had gaps in evidence validating their measurement properties, 2 COAs measuring symptom severity and 1 COA measuring HRQoL had evidence confirming their potential utility in clinical trials to support label claims.ConclusionThis research provides insights into the experience of patients with FOS and identifies COAs that measure concepts considered to support endpoints in clinical trials for FOS.

Measurement properties of grimace scales for pain assessment in nonhuman mammals: a systematic review.

Facial expressions of pain have been identified in several animal species. The aim of this systematic review was to provide evidence on the measurement properties of grimace scales for pain assessment. The protocol was registered (SyRF#21-November-2019), and the study is reported according to the PRISMA guidelines. Studies reporting the development, validation, and the assessment of measurement properties of grimace scales were included. Data extraction and assessment were performed by 2 investigators, following the COnsensus-based Standards for the Selection of Health Measurement INstruments guidelines. Six categories of measurement properties were assessed: internal consistency, reliability, measurement error, criterion and construct validity, and responsiveness. Overall strength of evidence (high, moderate, and low) of each instrument was based on methodological quality, number of studies, and studies' findings. Twelve scales for 9 species were included (mice, rats, rabbits, horses, piglets, sheep or lamb, ferrets, cats, and donkeys). Considerable variability regarding their development and measurement properties was observed. The Mouse, Rat, Horse and Feline Grimace Scales exhibited high level of evidence. The Rabbit, Lamb, Piglet and Ferret Grimace Scales and Sheep Pain Facial Expression Scale exhibited moderate level of evidence. The Sheep Grimace Scale, EQUUS-FAP, and EQUUS-Donkey-FAP exhibited low level of evidence for measurement properties. Construct validity was the most reported measurement property. Reliability and other forms of validity have been understudied. This systematic review identified gaps in knowledge on the measurement properties of grimace scales. Further studies should focus on improving psychometric testing, instrument refinement, and the use of grimace scales for pain assessment in nonhuman mammals.

A systematic review of utility-based and disease-specific quality of life measurement instruments for women with urinary incontinence.

This systematic review aims to identify disease-specific and generic quality of life (QoL) outcome measurement instruments used in populations of women with urinary incontinence (UI) and to determine the most psychometrically robust and appropriate disease-specific and generic tools for measuring the quality of life in this population. A systematic search was conducted of PubMed, Embase, SCIELO, and CINAHL databases for studies evaluating measurement properties of QoL instruments in women with UI. The methodological quality of studies and the quality of measurement properties were evaluated using the COnsensus-based Standards for the selection of health status Measurement INtruments (COSMIN) checklist and quality criteria. Overall, evidence for measurement properties was graded using the modified grading of recommendations, assessment, development & evaluation approach. A total of 73 studies were included, and 27 specific and 6 generic instruments were identified. The Incontinence QoL questionnaire (IQoL) had the highest overall psychometric quality for English-speaking populations and was the most widely translated tool. Evidence for generic QoL tools in this population is limited. Few studies evaluated measurement error or cross-cultural validity. The IQoL is the most psychometrically robust disease-specific tool for use in this population. More research is needed to determine the most psychometrically robust generic tool. Future studies should also evaluate measurement error and cross-cultural validity as evidence for these properties is particularly lacking.

The Withdrawal Assessment Tool to identify iatrogenic withdrawal symptoms in critically ill paediatric patients: A COSMIN systematic review of measurement properties

The Withdrawal Assessment Tool (WAT-1) is one of the most widely used clinician-reported outcome measures to evaluate iatrogenic withdrawal symptoms (IWS) in critically ill children. However, the WAT-1's measurement properties have not been aggregated. Aggregating psychometric research on the WAT-1 will enhance appropriate use, and outline gaps for future empirical research. The aim of this systematic review is to critically appraise, compare, and summarize the measurement properties and evidence quality, and describe the interpretability and feasibility of the WAT-1 for identifying IWS symptoms in critically ill children. A systematic search of Medline, Embase and CINAHL was conducted from inception to 15 April 2020. Study inclusion/exclusion, data extraction, and measurement property evidence and the modified GRADE quality scoring were applied according to the COnsensus-based Standards for the selection of health Measurement Instruments (COSMIN) guidelines. Six studies were included in the review. There was sufficient, high-quality evidence for reliability, structural validity, criterion validity, measurement error, construct validity, and feasibility. More information is required to support the WAT-1's content validity, responsiveness, internal consistency, cross-cultural validity, and interpretability according to COSMIN guidelines. The results of this review indicate that the WAT-1 is a precise, easy to use measure of IWS in critically ill children despite some measurement property inconsistencies and gaps in the publication record. More information is required to support its content validity, responsiveness, internal consistency, cross-cultural validity, and interpretability.

Validity of Clinical Measurement Instruments Assessing Scapular Function: Insufficient Evidence to Recommend Any Instrument for Assessing Scapular Posture, Movement, and Dysfunction-A Systematic Review.

To determine the construct validity, criterion validity, and responsiveness of measurement instruments evaluating scapular function. Systematic review of measurement properties. The MEDLINE, Embase, CINAHL, and SPORTDiscus databases were systematically searched from inception until March 2019. Studies published in Dutch, English, or German were included when they evaluated at least 1 of the measurement properties of interest. No restrictions were made regarding participants' health status. Two reviewers independently evaluated study quality using the COSMIN checklist and extracted and analyzed data. Quality of evidence was graded by measurement property for each distinctive type of measurement. Thirty-one measurement instruments in 14 studies were categorized into instruments to measure scapular posture and movement, and to assess scapular dyskinesis. Quality of evidence was at most moderate for 4 instruments with respect to criterion validity. Of these, criterion validity for instruments measuring scapular protraction/retraction posture and rotation angles up to 120° of thoracohumeral elevation was sufficient. Criterion validity for instruments measuring asymmetrical scapular posture, range of motion, and the lateral scapular slide test was insufficient. Quality of evidence for measurement properties of all other instruments was graded lower. There is currently insufficient evidence to recommend any instrument for the clinical examination of scapular function. Measurement instruments to assess scapular dyskinesis are prone to misinterpretation and should therefore not be used as such. J Orthop Sports Phys Ther 2020;50(11):632-641. doi:10.2519/jospt.2020.9265.

Measurement properties of handheld dynamometry for assessment of shoulder muscle strength: A systematic review.

Like any assessment tool, handheld dynamometry (HHD) must be valid and reliable in order to be meaningful in clinical practice and research. To summarize the evidence of measurement properties of HHD for the assessment of shoulder muscle strength. Cochrane Central Register of Controlled Trials (CENTRAL), PubMed, EMBASE, and PEDro were searched up to February 2020. Inclusion criteria were studies (a) evaluating HHD used on the glenohumeral joint, (b) evaluating measurement properties, and (c) included individuals≥18years old with or without shoulder symptoms. Exclusion criteria were studies (a) including patients with neurologic, neuromuscular, systemic diseases, or critical illness or bed-side patients and (b) that did not report the results separately for each movement. In total, 28 studies with 963 participants were included. The reliability results showed that 98% of the intraclass correlation coefficient (ICC) values were ≥0.70. The measurement error showed that the minimal detectable change in percent varied from 0% to 51.0%. The quality of evidence was high or moderate for the majority of movements and type of reliability examined. Based on the evidence of low or very low quality of evidence, the convergent validity and discriminative validity of HHD were either sufficient, indeterminate, or insufficient. The reliability of HHD was overall sufficient, and HHD can be used to distinguish between individuals on the group level. The measurement error was not sufficient, and evaluation of treatment effect on the individual level should be interpreted with caution.

Self-efficacy instruments for type 2 diabetes self-care: A systematic review of measurement properties.

To identify currently available self-efficacy instruments for type 2 diabetes self-care and to evaluate the evidence for their measurement properties. Systematic review of measurement properties. The PubMed, Embase and CINAHL databases were searched from their inception to 27 May 2019. The updated COnsensus-based Standards for the selection of health Measurement INstruments methodology were applied. Twelve instruments were identified from 3,665 records. Eight instruments were related to self-efficacy in performing a comprehensive set of the tasks/behaviours for diabetes self-care and the other four addressed specific behaviours related to diabetes self-care, such as insulin management. Most of the 12 instruments were developed based on Bandura's self-efficacy theory as their theoretical background. Overall, sufficient high-quality evidence for measurement properties was seldom identified. The Diabetes Management Self-Efficacy Scale is currently the best instrument, with particularly the 4-factor, 16-item version being demonstrated to have sufficient high-quality evidence for structural and internal consistency and sufficient moderate-quality evidence for reliability and convergent validity. None of the instruments evaluated all of the relevant measurement properties. The Diabetes Management Self-Efficacy Scale is currently the most suitable instrument for potential use in practice and research. The measurement invariance across languages, measurement error and responsiveness of this instrument still need be evaluated. This systematic review provides clinicians and researchers with a comprehensive list of available instruments for measuring self-efficacy in diabetes self-care, as well as evidence for helping them to select the most appropriate instrument. Using a psychometrically sound instrument will accurately inform practitioners about the levels of self-efficacy in self-care among patients with type 2 diabetes in practice and avoid threatening the credibility of research studies on self-efficacy in diabetes self-care.

Is the Patient Activation Measure a valid measure of osteoarthritis self-management attitudes and capabilities? Results of a Rasch analysis

BackgroundThe Patient Activation Measure (PAM-13) was developed using Rasch analysis to assess knowledge, skills and confidence in the management of one’s health. Previous studies report positive relationships between PAM-13 scores, self-management behaviours and longitudinal health outcomes in adults with chronic disease. There is little extant measurement property evidence for the use of PAM-13 in specific osteoarthritis (OA) populations. This study tested measurement properties of the PAM-13 in people living with hip and knee OA.MethodsItem response frequency analysis was conducted. Rasch analysis evaluated the fit of the PAM-13 data to the Rasch model. Model-data fit was evaluated using infit and outfit statistics; person/item reliability and person separation indices were computed. Unidimensionality was evaluated using Principal Components Analysis of Rasch residuals and the data were assessed for item redundancy. Differential Item Functioning (DIF) examined bias in respondent subgroups and correlations tested relationships between PAM-13 and other patient-reported outcomes.ResultsTwo-hundred-and-seventeen PAM-13 surveys were completed; there were no missing responses, floor or ceiling effects. Person and item reliability were acceptable (0.98 and 0.87 respectively) with good separation (person separation index 2.58). Unidimensionality was evaluated, with 49.4% of the variance explained by the first eigenvector. There was evidence of potential local response-dependence. The Rasch fit statistics were acceptable (except for item-2). There were some issues identified with targeting of the PAM-13 items to people with higher ability and the item difficulty order was different to that proposed in original cohorts. Significant DIF was identified for sex and educational level for a small number of items. PAM-13 scores were moderately correlated with depressive symptoms on the Depression Anxiety Stress Scale and Assessment of Quality of Life-6D. There were small correlations between PAM-13 and Knee injury and Osteoarthritis Outcome Score pain and activities of daily living scores.ConclusionsThis study provides some evidence of adequate person and item reliability, unidimensionality, and construct validity to support the use of PAM-13 to measure patient activation in people living with hip and knee OA. Possible limitations regarding targeting, different item difficulty order, DIF and local response dependence should be investigated in future research.

Assessing participation of children with acquired brain injury and cerebral palsy: a systematic review of measurement properties.

AimTo examine which instruments used to assess participation of children with acquired brain injury (ABI) or cerebral palsy (CP) align with attendance and/or involvement constructs of participation; and to systematically review measurement properties of these instruments in children with ABI or CP, to guide instrument selection.MethodFive databases were searched. Instruments that quantified ‘attendance’ and/or ‘involvement’ aspects of participation according to the family of participation‐related constructs were selected. Data on measurement properties were extracted and methodological quality of the studies assessed.ResultsThirty‐seven instruments were used to assess participation in children with ABI or CP. Of those, 12 measured attendance and/or involvement. The reliability, validity, and responsiveness of eight of these instruments were examined in 14 studies with children with ABI or CP. Sufficient measurement properties were reported for most of the measures, but no instrument had been assessed on all relevant properties. Moreover, most psychometric studies have marked methodological limitations.InterpretationInstruments to assess participation of children with ABI or CP should be selected carefully, as many available measures do not align with attendance and/or involvement. Evidence for measurement properties is limited, mainly caused by low methodological study quality. Future studies should follow recommended methodological guidelines.What this paper adds Twelve instruments used to assess participation of children with acquired brain injury (ABI) or cerebral palsy (CP) aligned with attendance/involvement.Seven instruments have some psychometric evidence supporting their use with children with CP.For children with ABI, only the Child and Adolescent Scale of Participation has shown preliminary evidence of measurement properties.

Concepts and Instruments for Patient-Reported Outcome Assessment in Celiac Disease: Literature Review and Experts’ Perspectives

BackgroundIn diseases where there is a large subjective component, such as celiac disease (CD), patient reported-outcomes (PRO) endpoints are highly relevant. However, there is a gap in knowledge about which PRO endpoints and instruments should be used for clinical trials for treatment of celiac disease. ObjectivesTo identify patient-centered symptom, impact, and health-related quality of life (HRQoL) concepts in CD and relevant PRO instruments, and to gather expert input on concepts and instruments to inform selection of PRO endpoints for use in clinical trials of new CD treatments. MethodsA targeted literature review was conducted to identify symptom, impact, and HRQoL concepts, including those captured in PROs further reviewed against U.S. Food and Drug Administration standards for development and validation as endpoints. US and European clinicians, payers, and a patient advocate (n = 21) were interviewed to assess the identified concepts’ relative importance in measuring treatment benefit and to gauge the value of potential PROs as endpoints for market access/reimbursement. ResultsThirty-four published studies were identified: 27 elucidated patient-centered concepts and 7 detailed the development or validation of PRO instruments. The Celiac Disease Symptom Diary and Celiac Disease Patient Reported Outcome instrument were deemed most appropriate for use as endpoints; however, each had limitations related to conceptual coverage, evidence for measurement properties, and feasibility for use in clinical trials. Experts reported gastrointestinal symptoms as most important to treat, with extra-intestinal symptoms burdensome from the patient perspective as well. Payers emphasized measuring both frequency and severity of symptoms and targeting patients nonresponsive to the gluten-free diet for treatment. ConclusionsWith emerging treatment options for CD, further work is needed to operationalize PRO symptom endpoints that are meaningful to patients, valued by payers, and acceptable to regulators in demonstrating efficacy.

Measurement properties of patient-reported outcome measures in radiotherapy-induced trismus.

Trismus or lockjaw is the reduction in mouth opening. Radiotherapy-induced trismus occurs when the masticatory muscles are within range of radiation treating head and neck cancer, resulting in muscular fibrosis and trismus. We aim to assess the measurement properties of available validated patient-reported outcome measures relevant to radiotherapy-induced trismus in head and neck cancer patients. Systematic electronic searches were performed in MEDLINE and EMBASE databases to identify studies validating radiotherapy-induced trismus with patient-reported outcome measures up to January 2018. Two reviewers independently rated the methodological quality of identified validation studies, using the consensus-based standards for the selection of health status measurement instrument (COSMIN) checklist. The level of evidence for measurement properties was then summarised based upon pre-defined criteria. Only one of the 280 retrieved studies were deemed fit for inclusion. The Gothenburg Trismus Questionnaire was assessed and found to have a good methodological quality of measurement properties except for reliability, which was poor. There was a moderate level of evidence supporting internal consistency, content validity, structural validity and hypothesis testing. The evidence for test-retest reliability remains unknown in the population of interest. The Gothenburg Trismus Questionnaire is the only patient-reported outcome measure in the field of showing some good measurement properties, and it may therefore represent a useful instrument for the assessment of radiotherapy-induced trismus in head and neck cancer patients. However, further validation studies are needed especially with respect to the reliability, measurement error and the responsiveness of this instrument.

Measurement of Physical Activity in Adults with Muscular Dystrophy: A Systematic Review

There is little consensus about measurement of physical activity in adults with muscular dystrophy. This systematic review summarizes evidence for measurement properties of direct and indirect measures of physical activity in adults with muscular dystrophy. A two-phase search for peer-reviewed articles identified firstly, studies which measured physical activity in this population and secondly, studies reporting the measurement properties of activity measures. Methodological quality was assessed using COSMIN guidelines and a best evidence synthesis conducted. Phase 1 included 53 studies identifying 63 measures including accelerometers, direct observation, heartrate monitors, calorimetry, positional sensors, activity diaries, single scales and questionnaires. Phase 2 included 26 studies of measurement properties for 32 measures. Methodological quality of the included studies was low, only 2 were rated good. There was insufficient evidence to robustly recommend any physical activity measures and further research is required to validate measures of physical activity for adults with muscular dystrophy. Based on the findings of this review, measures with potential for further study have been highlighted.