Reliable Patient-reported Outcome Measure Research Articles

Convergent construct validity and test-retest reliability of both German versions of the original and the revised Niigata PPPD Questionnaire: NPQ and NPQ-R

BackgroundPersistent Postural-Perceptual Dizziness (PPPD) is a frequent chronic functional disorder that manifests with dizziness, unsteadiness, or non-spinning vertigo present for at least 3 months. Characteristic provocation factors are moving or complex visual stimuli and exclusion of organic diseases. To assess the severity and impact of PPPD, Japanese researchers developed the Niigata PPPD Questionnaire (NPQ). The study's aim was to evaluate the concurrent construct validity and reliability [including test-retest reliability, internal consistency, standard error of measurement (SEM), and minimal detectable change (MDC)] of the German version of the NPQ (12 items) and its revised version, NPQ-R, which contains 19 items addressing additional symptoms and symptom behavior.MethodsThe Swiss Reha Rheinfelden and the German Center for Vertigo and Balance Disorders included 265 PPPD patients (mean age 50.2 ± 16.8 years, disease duration 46.3 ± 76.6 months). Patients completed the NPQ and the NPQ-R (twice), the DHI and potentially related constructs: anxiety (ABC-Scale, VSS), depression (HADS), and general health (SF-36) once. To assess the questionnaires' reliability and validity, several statistical measures were calculated, including Spearman's rank correlation coefficients, Intraclass Correlation Coefficients (ICC2, 1), Cronbach's alpha, SEM, and MDC.ResultsOn average, patients scored 29.9 ± 13.2 for NPQ and 52.3 ± 19.6 for NPQ-R. Correlations between NPQ/NPQ-R and (1) disease-specific questionnaires were rs= 0.712 and rs= 0.752 (DHI), rs=0.426 and rs= 0.0.462 (VSS-V), rs= -0.500 and rs= -0.545 (ABC-Scale), (2) anxiety-specific subscales rs = 0.394 and rs = 0.430 (VSS-A) and rs= 0.354 and rs= 0.430 (HADS-A), (3) depression-related subscales rs=0.438 and rs= 0.487 (HADS-D), and (4) general health rs ranged between rs= -0.216 and −0.578 (all SF-36 subscales). Internal consistency, test-retest reliability, SEM and MDC calculated for NPQ/NPQ-R were α = 0.88/α = 0.91, ICC=0.83 (CI 0.77 to 0.0.87), SEM 5.55/8.37, and MDC 15/23 points.ConclusionThe German versions of NPQ and NPQ-R are valid and reliable patient-reported outcome measures for assessing PPPD, demonstrating satisfactory psychometric measurement properties including convergent construct validity and reliability parameters: internal consistency, test-retest reliability, SEM, and MDC as an evaluative measure. The NPQ-R, with its additional subscales addressing associated symptoms and symptom behavior, represents both the patient and clinician perspective on PPPD-specific problems. Therefore, we recommend utilizing the NPQ-R for a comprehensive assessment of PPPD.

Optimising patient engagement for assessing total joint arthroplasty outcomes – a randomised controlled trial

IntroductionPatient satisfaction is a critical outcome in total joint arthroplasty (TJA), yet assessing it effectively remains a challenge due to limitations in patient-reported outcome measures (PROMS). While these measures are commonly gathered in clinical settings, additional contact through mail or phone is often needed, and low response rates can affect the validity and reliability of collected data. To improve response rates, this study evaluated various methods of incentivizing patient participation in a randomized trial format, focusing on postal questionnaires.Patients and methodsThe study investigated three methods to improve response rates: including a gift card with the questionnaire, promising a gift card upon questionnaire completion, and offering no incentive. It also examined whether different monetary values and the inclusion of the surgeon’s name on materials impacted response rates. We tried to determine factors that could improve follow up telephone response rates in the group of patients that failed to return their questionnaires.ResultsHigher response rates were observed with monetary incentives (P = 0.056), larger amounts of money offered (P = 0.3839) for filling out the questionnaire, and if the surgeon’s details were on the cover letter or questionnaire (P = 0.632). There was no correlation between age and sex and participation. We did find a statistically significant difference in total participation and poorer total knee arthroplasty outcomes scores (P < 0.001).ConclusionOur study supports findings from prior research indicating that monetary incentives and personalized materials can improve response rates, although in this cohort, results were modest. Follow-up calls further boosted response rates, suggesting that multi-modal engagement may be beneficial. Although the response improvements were limited and lacked statistical significance, the study highlights the importance of refining strategies to ensure reliable PROMS data, which is vital for understanding patient outcomes in TJA. Future studies might consider demographic factors and other outreach methods to enhance PROMs data collection.

The Mastocytosis Control Test: A Patient-Reported Outcome Measure Assessing Disease Control

BACKGROUNDMastocytosis is characterized by expanding neoplastic mast cells in organs such as the skin, bone marrow, and gastrointestinal tract. The release of mast cell mediators triggers cutaneous, gastrointestinal, and other symptoms. Currently, no validated mastocytosis-specific patient-reported outcome measure (PROM) exists to assess disease control. OBJECTIVEHere, we developed a disease-specific instrument, the Mastocytosis Control Test (MCT), for evaluating and monitoring disease control in patients with non-advanced disease. METHODSSix potential MCT items were generated using a combined approach consisting of a literature review, patient interviews, and expert input. Item selection and reduction were performed by impact analysis and inter-item correlation, followed by expert reviews and cognitive debriefings. In a validation study, the resulting MCT was tested for validity and reliability by assessing internal consistency, test-retest reliability, convergent validity, known-groups validity, and receiver operating characteristics (ROC) analysis. RESULTSTen patients participated in the item generation phase and 101 in the item reduction and validation phase. The final MCT consisted of five items. Our methods showed a valid total score, high internal consistency, and test-retest reliability. Convergent and known-groups validity demonstrated a strong correlation with related anchors. The ROC curve analysis suggested a cutoff value of ≥13 points to identify patients with good disease control. CONCLUSIONSThe MCT is a disease-specific, valid, and reliable PROM for adult patients with non-advanced disease. It may measure and monitor disease control in routine daily practice and clinical trials.

Patients' joint perception after anterior cruciate ligament reconstruction as a reliable patient-reported outcome measure.

This study aims to evaluate and compare patients' joint perception (PJP) with other commonly employed patient-reported outcome measures (PROMs) in a cohort of patients following anterior cruciate ligament reconstruction (ACLR). A retrospective study was conducted on 108 patients who underwent ACLR between 2017 and 2021, with complete data available for visual analogue scale (VAS) pain, PJP, Forgotten Joint Score (FJS), Tegner Activity Scale, Lysholm Knee Scoring Scale, Anterior Cruciate Ligament Return to Sport After Injury (ACL-RSI), Single Assessment Numeric Evaluation (SANE), Cincinnati Knee Rating System (CKRS)and The Hospital for Special Surgery Anterior Cruciate Ligament Satisfaction Survey (HSS ACL-SS) at a mean follow-up of 3 years. Spearman's rank-order correlation was used to determine the correlations between clinical scores. A receiver operating characteristic (ROC) curve analysis was used to assess the discriminatory power of the chosen PROMs in detecting natural joint perception. Natural joint perception was defined as patients responding 'like a native or natural joint' or PJP = 1. This study hypothesized that PJP would demonstrate significant correlations with established PROMs, indicating its reliability and validity as a tool for assessing outcomes following ACLR. PJP showed significant correlations with all the reported PROMs, suggesting its potential as a valid and reliable assessment tool for evaluating ACLR outcomes. The ROC curve analysis indicated a good area under the curve for identifying natural joint perception using various PROMs. According to the PJP, nearly 20% of patients reported natural joint perception. This study evaluates PJP as a reliable tool for assessing patient outcomes following ACLR. Its significant correlation with other established PROMs underscores its potential as a valuable addition to the existing array of PROMs in ACLR. Level III.

Development of a new health-related quality of life measure for people with diabetes who experience hypoglycaemia: the Hypo-RESOLVE QoL

Aims/hypothesisValid and reliable patient-reported outcome measures are vital for assessing disease impact, responsiveness to healthcare and the cost-effectiveness of interventions. A recent review has questioned the ability of existing measures to assess hypoglycaemia-related impacts on health-related quality of life for people with diabetes. This mixed-methods project was designed to produce a novel health-related quality of life patient-reported outcome measure in hypoglycaemia: the Hypo-RESOLVE QoL.MethodsThree studies were conducted with people with diabetes who experience hypoglycaemia. In Stage 1, a comprehensive health-related quality of life framework for hypoglycaemia was elicited from semi-structured interviews (N=31). In Stage 2, the content validity and acceptability of draft measure content were tested via three waves of cognitive debriefing interviews (N=70 people with diabetes; N=14 clinicians). In Stage 3, revised measure content was administered alongside existing generic and diabetes-related measures in a large cross-sectional observational survey to assess psychometric performance (N=1246). The final measure was developed using multiple evidence sources, incorporating stakeholder engagement.ResultsA novel conceptual model of hypoglycaemia-related health-related quality of life was generated, featuring 19 themes, organised by physical, social and psychological aspects. From a draft version of 76 items, a final 14-item measure was produced with satisfactory structural (χ2=472.27, df=74, p<0.001; comparative fit index =0.943; root mean square error of approximation =0.069) and convergent validity with related constructs (r=0.46–0.59), internal consistency (α=0.91) and test–retest reliability (intraclass correlation coefficient =0.87).Conclusions/interpretationThe Hypo-RESOLVE QoL is a rigorously developed patient-reported outcome measure assessing the health-related quality of life impacts of hypoglycaemia. The Hypo-RESOLVE QoL has demonstrable validity and reliability and has value for use in clinical decision-making and as a clinical trial endpoint.Data availabilityAll data generated or analysed during this study are included in the published article and its online supplementary files (https://doi.org/10.15131/shef.data.23295284.v2).Graphical

Salivary, lacrimal and nasal (SALANS) measure to assess side effects following radioactive iodine treatment: development, psychometric properties, and factor structure.

This study aimed to develop and psychometrically evaluate a patient-reported outcome measure (PROM), SAlivary, LAcrimal, NaSal (SALANS), to document patients' symptoms after radioactive iodine (RAI) treatment for differentiated thyroid cancer (DTC). We generated and iteratively revised SALANS items based on expert input, focus group discussions and feedback from cognitive testing (n = 17). We administered an initial SALANS measure with 39 items to patients diagnosed with DTC in the past two years (n = 105). Exploratory factor analysis (EFA) examined the factor structure of the SALANS items. We assessed the consistency reliability and related the total and subscale scores of the final SALANS to existing PROMs to assess validity. The final SALANS consisted of 33 items and six subscales (sialadenitis, taste, xerostomia, dry eyes, epiphora, and nasal) with six factors extracted by EFA. The six subscales demonstrated good internal reliability (α range = 0.87-0.92). The SALANS total score showed good convergent validity with the Xerostomia Inventory (r = 0.86) and good discriminant validity with a measure of spirituality (r = - 0.05). The mean SALANS total score was significantly higher (d = 0.5, p < 0.04) among patients who had RAI compared to those who did not have RAI. Preliminary evidence suggests that SALANS is a novel and reliable PROM to assess the type and frequency all symptoms experienced after RAI treatment for DTC. Future work is needed to further validate and develop the scale.

Pelvic floor muscle training patient reported Outcome Measures (PROM) (PFMT-P): Turkish adaptation and its validity and relibiality

ObjectiveAlthough there are several patient reported outcome measures (PROM) regarding knowledge, source of knowledge and practice about pelvic floor muscle training (PFMT), there is a lack of a valid and reliable PROM that includes the practice component of PFMT along with knowledge and source of knowledge. There is no valid and reliable measurement tool in Turkish used for this purpose. In this context, the aim of our study is to evaluate the psychometric properties of Pelvic Floor Muscle Training Patient Reported Outcome Measures (PROM) (PFMT-P) and to test whether it is a valid and reliable measurement tool for Turkish women. Study designThis study has been carried out psychometric testing (validity and reliability) of a new assessment tool concerning PFMT-P. A total of 170 female healthcare professionals (midwives, nurses, doctors, physiotherapists) who were between the ages of 23 and 49 and who volunteered to participate were included in the study. SPSS 25.0 and LİSREL 8.80 statistical programs were used for the analysis of data. Descriptive statistics were evaluated with numbers, percentages and means. Content validity index and confirmatory factor analysis were used for validity. Cronbach Alpha value and test–retest were used for reliability. Level of significance was p < 0.05. ResultsContent validity index (CVI) was found to be 0.96 for the overall scale. Model fit indices were found as perfect and good matched. Cronbach’s alpha coefficient was found as 0.813 for the overall scale. Test-retest correlation was found as 0.658, it was 0.998 for practice component and 0.997 for source of knowledge component and a positive, significant and high correlation was found. ConclusionsIt has been determined that PFMT-P is a valid and reliable measurement tool suitable for Turkish culture. With this, it is a short, comprehensive and useful tool that can be used in both pregnancy and the postpartum period for women of reproductive age or menopause.

Facioscapulohumeral muscular dystrophy Health Index: Japanese translation and validation study

Purpose The Facioscapulohumeral Muscular Dystrophy Health Index (FSHD-HI) is a patient-reported outcome measure developed for patients with FSHD. This study aimed to translate the FSHD-HI into Japanese (FSHD-HI-J), evaluate cultural adaptation, and examine its psychometric properties. Materials and methods We created two forward translations, integrated them into a single Japanese version, and evaluated the back-translated version of the FSHD-HI. After finalizing the translation and cultural adaptation, we conducted a survey of 66 patients with FSHD to examine the reliability and validity of the FSHD-HI-J. For psychometric evaluations, we used Cronbach’s alpha to assess internal consistency, the intraclass correlation coefficient (ICC) for test-retest reliability, and assessed validity based on the associations between FSHD-HI-J, clinical variables, and quality of life measures. Results The FSHD-HI-J was found to be clinically relevant, indicating high internal consistency and test-retest reliability (ICC = 0.92 [95% confidence interval: 0.86-0.95] for the total score), as well as significant associations with clinical variables (D4Z4 repeats and functional impairment) and other quality of life measures (|rho| = 0.25-0.73). Conclusions The FSHD-HI-J is a valid and reliable patient-reported outcome measure for Japanese patients with FSHD. This validated, disease-specific patient-reported outcome is essential for future clinical practice and clinical trials.

Development and evaluation of a patient-reported outcome measure specific for Gaucher disease with or without neurological symptoms in Japan

BackgroundPatients with Gaucher disease (GD), a rare lysosomal storage disorder, have reduced health-related quality of life (HRQOL). A patient-reported outcome measure (PROM) for HRQOL developed for type 1 GD (GD1) is not appropriate for patients with neuronopathic GD (nGD) types 2 (GD2) and 3 (GD3). In this study, we developed a new PROM for use in all GD types. We previously reported the qualitative analysis of interviews with Japanese patients with nGD, which was used to create nGD-specific PROM items. Here we evaluated the full PROM combining the type 1 questionnaire with the new nGD-specific items.MethodsPatients with confirmed GD were recruited (Association of Gaucher Disease Patients in Japan or leading doctors) for pre-testing (May 2021) or the main survey (October–December 2021). The PROM had three parts: Parts 1 and 2 were translated into Japanese from the pre-existing GD1 PROM, whereas Part 3 was newly developed. Patients (or their caregivers, where necessary) completed the PROM questionnaire on paper and returned it by mail. Mean scores were determined overall and by GD type. Inter-item correlations, content consistency (Cronbach’s alpha), and test–retest reliability (Cohen’s kappa; main survey only, taken 2 weeks apart) were calculated.ResultsSixteen patients (three with GD1; six with GD2; seven with GD3) and 33 patients (nine with GD1; 13 with GD2; 11 with GD3) participated in the pre-test and main survey, respectively. All GD2 patients and one-third (6/18) of GD3 patients required caregivers to complete the questionnaire. Mean scores indicated that the burden was highest in GD2 and lowest in GD1. In the main survey, internal consistency was high (Cronbach’s alpha = 0.898 overall, 0.916 for Part 3), and test–retest reliability was high for Part 3 (kappa > 0.60 for 13/16 items) but low for Part 1 (kappa < 0.60 for 12/15 items).ConclusionsWe have developed a flexible and reliable PROM that can be tailored for use in all types of GD and propose using Parts 1 and 2 for GD1, Parts 2 and 3 for GD2, and Parts 1, 2, and 3 for GD3.

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.

Piloting patient-reported outcome measures for evaluation of the Irish Chronic Disease Programme

Abstract Background The Irish National Integrated Care Programme for Chronic Disease (ICPCD) aims to deliver end-to-end, patient-centred care across three clinical programmes; Heart, Respiratory and Diabetes. Patient-reported outcome measures (PROMs) are a means of objectively measuring changes in patients’ subjectively-perceived health status over time in response to services. As such, PROMs facilitate service monitoring and evaluation and are an important tool for the ICPCD to ensure optimal, patient-centred, value-based healthcare delivery. Methods To select the most appropriate PROMs instrument for use across ICPCD, comprehensive literature review was undertaken followed by key stakeholder consultation in winter 2022. Following consideration against a set of pre-determined criteria the EQ-5D-5L, a well-validated, reliable PROMs instrument, was selected as the most appropriate PROMs instrument for use. Study design involved pilot implementation of EQ-5D-5L in one ICPCD ambulatory care hub service followed by mixed methods evaluation of staff experience of the pilot. Results The pilot was conducted from April to June 2023 among a socio-demographically diverse sample of patients attending their first and last episode of care with the ICPCD diabetic patient education service in Galway. Following service attendance there was a significant aggregate improvement in self-reported overall health rating but no significant change in self-reported mobility, self-care, usual activities, pain/discomfort or anxiety/depression. Predominant staff concerns included staff and patient burden and the need for adequate staff resourcing and information technology support. Conclusions In conjunction with key national stakeholders findings from the pilot will be used to support the development of a PROMs implementation plan at scale for adoption across all ICPCD services. Ultimately national implementation will support the ongoing delivery of high quality patient-centred care within ICPCD services. Key messages • PROMs facilitate service evaluation and are an important tool for the Irish National Integrated Care Programme for Chronic Disease (ICPCD) to ensure optimal patient-centred healthcare delivery. • This pilot will support PROMs implementation at scale across ICPCD services and ultimately reinforce the ongoing delivery of high-quality, value-based, integrated healthcare.

Hyperacusis Assessment Questionnaire-A New Tool Assessing Hyperacusis in Subjects with Tinnitus.

Hyperacusis, a kind of decreased sound tolerance, is difficult to measure objectively. It often co-occurs with tinnitus. There is a need for valid and reliable patient-reported outcome measures to capture this subjective phenomenon. The aim of the study was to create a questionnaire capturing hyperacusis in terms of loudness, fear, and pain and to evaluate its psychometric properties. The study sample consisted of 106 adult patients with hyperacusis and tinnitus with a mean age of 45.2 years. A medical interview, an audiological examination, and several questionnaires (the Tinnitus Handicap Inventory, the Hyperacusis Questionnaire, the State-Trait Anxiety Inventory, and Visual Analog Scales) were applied. The final 14-item Hyperacusis Assessment Questionnaire showed an appropriate three-factor structure with 70.5% of the variance explained. Convergent and divergent validity were confirmed by correlations with other measures of hyperacusis, anxiety, tinnitus severity, misophonia, and hearing thresholds. The internal consistency assessed with Cronbach's alpha was excellent (α = 0.91), as was reproducibility (intraclass correlation, ICC = 0.96). The new Hyperacusis Assessment Questionnaire is a psychometrically sound and brief tool assessing the severity of hyperacusis in terms of loudness, fear, and pain. It can be used in clinical practice and scientific research for patients with hyperacusis and tinnitus.

Patient Reported Outcome Measures in the Foot and Ankle Literature: A Systematic Review

Category: Other Introduction/Purpose: Optimising clinical practice requires emphasis on patient-orientated care and therefore patient reported outcome measures (PROMs) play a key role in shaping evidence-based clinical guidelines. As such, it is crucial that we use appropriate, valid and reliable PROMs to quantify outcomes in orthopaedic research. A previous systematic review evaluating evidence published between 2002-2011 identified 139 different PROMs used in the foot and ankle literature and found the most common PROM was The American Orthopaedic Foot and Ankle Society (AOFAS) score. However, in 2011, AOFAS issued a position statement outlining the lack of reliability and validity of the AOFAS score, describing it as a ‘less than ideal’ measure. This systematic review aims to provide an updated analysis of the PROMS used in the foot and ankle literature. Methods: This systematic review was performed according to PRIMSA guidelines. An electronic title/abstract screen of every article published between January 2012- December 2022 in Foot and ankle international, Clinical Orthopaedics and Related Research, Knee Surgery Sports Traumatology and Arthroscopy, The Bone and Joint Journal and The American Journal of Sports Medicine was performed by two reviewers. Articles with a foot and ankle focus were selected for full text screening and papers reporting at least one PROM were included in the final review. The following data was extracted from each article: journal of publication, month/year of publication, pathology/intervention described, PROMS reported and use of the minimally clinically important difference (MCID) to evaluate post intervention changes in PROMs. The number of studies using each individual PROM was calculated and a subgroup analysis was performed to investigate variation in PROM selection according to year of publication, journal of publication and the condition being investigated. Results: A total of 3,174 foot and ankle articles were identified, of which 1,168 (36.8%) articles reporting at least one PROM were included. The most common PROM was the AOFAS score, reported in 50.3% of studies, followed by visual analogue scale (VAS) pain score (44.4%) and 36-item short form survey (SF-36) (15.3%). Use of the AOFAS score declined across the review period. Between 2012-2016 AOFAS was the most common PROM, whilst VAS pain was most common in four of the last six years. Although some variation in PROMs according to clinical pathology exists, AOFAS score is featured in the top 3 most common PROMs for every analysed condition. Only 8/1,168 (0.7%) articles reported the number of patients achieving a minimally clinically important difference (MCID) post-intervention. Conclusion: Whilst use of the AOFAS score declined over the review period, it remains the most common PROM overall across all included journals and many conditions. There exists a lack of consensus regarding the most suitable alternative PROMs, with multiple validated instruments including FAOS, FAAM and FFI used inconsistently with relatively low frequency. There is a heavy reliance on statistical significance to evaluate and compare post-intervention PROM changes. However, it is important to quantify PROMs according to the patient perspective. Reporting the proportion of patients achieving MCID will allow clinicians to interpret PROMs and develop management guidelines in a patient-focused manner.

Rasch Analysis of the Korean Version of the Tinnitus Handicap Inventory.

Tinnitus is the perception of abnormal sounds in the ears or head without external auditory stimulation. While classical test theory is often used in tinnitus questionnaire development, it has limitations in assessing item characteristics. Item response theory (IRT) offers more precise individual ability estimations and identifies key and less important items, making it superior for reliable measurement tools. This study investigated the suitability of the Korean version of the Tinnitus Handicap Inventory (K-THI) as a patient-reported outcome measure (PROM) for clinical trials. Using Rasch analysis based on IRT, we evaluated K-THI's measurement of tinnitus-related disability in 545 patients (40.4% men, 59.6% women). Five items (2, 7, 8, 19, and 24) did not fit the Rasch model, yet a unidimensional scale and good fit for person and item data emerged (person: 0.89; item: 0.98). The three-point rating scale in K-THI proved suitable. IRT allowed precise evaluation of K-THI's properties, vital for reliable PROMs in patient-centered care. Our findings highlight IRT's role in questionnaire development, contributing to the advancement of PROMs.

Validation of the Peak Pruritus Numerical Rating Scale as a Patient-Reported Outcome Measure in Prurigo Nodularis.

Validated patient report tools for quantifying patient experiences of itch in prurigo nodularis (PN) are limited. This study aimed to evaluate the validity of the 11-point peak pruritus numerical rating scale (PP NRS) as a single-item patient-reported outcome (PRO) measure for assessing itch severity in PN. Content validity of the PP NRS was evaluated through qualitative interviews with adults with PN. The PP NRS was then psychometrically evaluated using data from a placebo-controlled trial of nemolizumab in adults with PN, during which patients completed the PP NRS daily. Meaningful within-patient change was estimated from the qualitative interviews and by anchor- and distribution-based analyses of trial data. The interview participants (N = 21) all understood the PP NRS and reported itch as their worst symptom overall. The PP NRS showed good test-retest reliability and demonstrated convergent validity and known-groups validity. PP NRS scores improved more in patients classified as "improved" on other clinical outcome measures than in those classified as "worsened/unchanged". Triangulation of the different estimates identified a 2- to 5-point decrease in PP NRS score as a meaningful within-patient change threshold. The PP NRS is a content-valid and reliable PRO measure for quantifying itch severity in adults with PN in clinical trials. NCT03181503.

Bowel Urgency in Ulcerative Colitis: Current Perspectives and Future Directions.

Bowel urgency (BU), the sudden or immediate need for a bowel movement, is one of the most common and disruptive symptoms experienced by patients with ulcerative colitis (UC). Distinct from the separate symptom of increased stool frequency, BU has a substantial negative impact on quality of life and psychosocial functioning. Among patients with UC, BU is one of the top reasons for treatment dissatisfaction and one of the symptoms patients most want improved. Patients may not discuss BU often due to embarrassment, and healthcare providers may not address the symptom adequately due to the lack of awareness of validated tools and/or knowledge of the importance of assessing BU. The mechanism of BU in UC is multifactorial and includes inflammatory changes in the rectum that may be linked to hypersensitivity and reduced compliance of the rectum. Responsive and reliable patient-reported outcome measures of BU are needed to provide evidence of treatment benefits in clinical trials and facilitate communication in clinical practice. This review discusses the pathophysiology and clinical importance of BU in UC and its impact on the quality of life and psychosocial functioning. Patient-reported outcome measures developed to assess the severity of BU in UC are discussed alongside overviews of treatment options and clinical guidelines. Implications for the future management of UC from the perspective of BU are also explored.

Using Rasch Analysis to Evaluate the Psychometric Properties of the Brief MHQ in Patients with Traumatic Finger Amputations.

Background: Digit amputations affect 45,000 Americans each year and are associated with substantial healthcare expenditures and loss of wages. Few patient-reported outcome measures (PROMs) are validated in patients with digit amputations. The brief Michigan Hand Outcomes Questionnaire (bMHQ) is a 12-item PROM used in several hand conditions. However, its psychometric properties have not been investigated in patients with digit amputations. Methods: The reliability and validity of the bMHQ was investigated using Rasch analysis. Data were collected from the Finger Replantation and Amputation Challenges in Assessing Impairment, Satisfaction, and Effectiveness (FRANCHISE) study. Participants were divided into replantation and revision amputation cohorts and then further separated into single-digit amputation (excluding thumb), thumb-only amputation and multiple-digit amputation (excluding thumb) subgroups. Each of the six subgroups were analysed for item fit, threshold ordering, targeting, differential item functioning (DIF), unidimensionality and internal consistency. Results: All treatment groups demonstrated high unidimensionality (Martin-Löf test = 1) and internal consistency (Cronbach's α > 0.85). The bMHQ is not a reliable PROM in individuals with single-digit or multiple-digit amputations. The aesthetics, satisfaction and two-handed activities of daily living (ADLs) items had the poorest fit to the Rasch model across all categories. Conclusions: The bMHQ is not well-suited for measuring outcomes in patients with digit amputations. We recommend clinicians use more comprehensive assessment tools, such as the complete MHQ, to measure outcomes in these complex patient populations. Level of Evidence: Level III (Diagnostic).

Development of a standard set of PROs and generic PROMs for Dutch medical specialist care

PurposeThe added value of measuring patient-reported outcomes (PROs) for delivering patient-centered care and assessment of healthcare quality is increasingly evident. However, healthcare system wide data collection initiatives are hampered by the proliferation of patient-reported outcome measures (PROMs) and conflicting data collection standards. As part of a national initiative of the Dutch Ministry of Health, Welfare and Sport we developed a consensus-based standard set of generic PROs and PROMs to be implemented across Dutch medical specialist care.MethodsA working group of mandated representatives of umbrella organizations involved in Dutch medical specialist care, together with PROM experts and patient organizations worked through a structured, consensus-driven co-creation process. This included literature reviews, online expert and working group meetings, and feedback from national patient- and umbrella organizations. The ‘PROM-cycle’ methodology was used to select feasible, valid, and reliable PROMs to obtain domain scores for each of the PROs included in the set.ResultsEight PROs across different domains of health were ultimately endorsed: symptoms (pain & fatigue), functioning (physical, social/participation, mental [anxiety & depression]), and overarching (quality of life & perceived overall health). A limited number of generic PROMs was endorsed. PROMIS short forms were selected as the preferred instruments for all PROs. Several recommendations were formulated to facilitate healthcare system level adoption and implementation of the standard set.ConclusionsWe developed a consensus-based standard set of Generic PROMs and a set of recommendations to facilitate healthcare system wide implementation across Dutch medical specialist care.

Validation of the European Portuguese Language Version of the Standardized Cosmesis and Health Nasal Outcomes Survey.

The Standardized Cosmesis and Health Nasal Outcomes Survey (SCHNOS) questionnaire is a tool developed to evaluate functional and aesthetic components of rhinoplasty. It is a reliable patient-reported outcome measure, not available in the European Portuguese language. Our goal was to translate and culturally adapt the SCHNOS questionnaire to the European Portuguese language. The questionnaire was forward and backward translated and culturally adapted to the European Portuguese language following international guidelines. The authors evaluated internal consistency, correlation, and reproducibility to determine the validity of the questionnaire. The final European Portuguese version of the SCHNOS was administered to 58 native European Portuguese speakers. Both the SCHNOS-O (obstructive) and SCHNOS-C (cosmetic) showed high internal consistency with Cronbach's α of 0.93 and 0.95, respectively. Also, for the entire SCHNOS, Cronbach's α was 0.96. All the items demonstrated good item-test and item-rest correlations with the differences between pre- and postestimates being nonsignificant. The translation, adaption, and validation of the SCHNOS into European Portuguese were successfully performed. This provides another tool to help evaluate the functional and aesthetic outcomes of rhinoplasty patients.

Arabic translation, cultural adaptation, and validation of the BDDQ-AS for rhinoplasty patients

BackgroundBody Dysmorphic Disorder (BDD) is a significant aspect that compromises patient satisfaction after rhinoplasty. BDDQ-AS (Body Dysmorphic Disorder Questionnaire-Aesthetic Surgery) is a validated, simple, reliable patient-reported outcome measure. It is a screening tool to detect body dysmorphic disorder in rhinoplasty patients. This study aimed to translate, culturally adapt, and validate BDDQ-AS to Arabic as a novel tool for screening and detecting BDD in Arabic rhinoplasty individuals.MethodsBDDQ-AS was translated from English to Arabic following the international consensus guidelines. We tested the translation on ten Arabic-speaking rhinoplasty patients to ensure that the final version was understandable and acceptable. The proposed Arabic version was then completed by 112 patients whose average age was 28.79 ± 9.32 years. The screening is assumed positive if the patients expressed bother and preoccupation about their appearance (questions 1 and 2 “yes”), as well as a moderately disrupted everyday life (question 7 "yes" or questions 3, 4, 5, or 6 are equal to or greater than “3′′). The internal consistency, test–retest reliability, and item-response theory (IRT) were used to evaluate psychometric validations.ResultsThe Arabic BDDQ had a high level of internal consistency, as measured by Cronbach's alpha 0.995. The A-BDDQ-AS was deemed reliable with an Intraclass Correlation Coefficient (ICC) of 0.989. A-BDDQ had good discrimination scores (above 2.0) with adequate difficulty parameters. The overall scale content validity average was 0.83, affirming that all items were relevant, clear, and straightforward.ConclusionThe Arabic version of the BDDQ-AS is reliable, culturally adapted, and psychometrically validated to be readily used and incorporated into clinical practice. It is a beneficial tool that can guide the screening of Arabic rhinoplasty patients suffering from body dysmorphic disorder and be utilized in further studies to optimize patient outcomes.Graphical