Rasch Measurement Theory Analysis Research Articles

The WOUND-Q Function and Symptoms Scales for Chronic Lower Extremity Wounds: A Validation study.

Determine the validity and reliability of the LIMB-Q scales, Function and Symptoms, in patients with chronic lower extremity wounds. Cognitive debriefing interviews with people with current or previous wounds were conducted to examine content validity. Scales were field-tested in an international sample of people with chronic lower extremity wounds sourced from an online platform (i.e., Prolific). Psychometric properties were examined using Rasch Measurement Theory analysis. A test-retest reproducibility study was performed, and construct validity examined. Content validity was established after 10 cognitive interviews. A total of 233 people with lower extremity wounds (age 19-80 years, mean 39.3) participated in the field-test. All 25 items tested demonstrated good fit to the Rasch model with ordered thresholds. One item had a fit residual outside ± 2.5, but no items had significant χ2 values after Bonferroni adjustment. Reliability was high with Person Separation Index, Cronbach alpha, and intraclass correlation coefficients values >0.8. Strong correlations were found between the Function and Symptoms scales and EQ-5D dimensions measuring similar constructs as well as the EQ-5D global score. All hypothesis for construct validity were confirmed. PROMs are an important component of patient-centered care, as they capture the patient's perspective in a rigorous and reproducible way. Adding these two scales to the WOUND-Q provides a means to measure function and symptoms associated with lower extremity wounds. These new WOUND-Q scales can be used to measure outcomes important to patients with lower extremity wounds in clinical settings and research studies.

Extending the Range of Measurement for Minimally Invasive Treatments by Adding New Concepts to FACE-Q Aesthetics Scales.

The Satisfaction with Face Overall and Psychological Function scales are the most frequently used FACE-Q Aesthetics module scales. This study aimed to extend their range of measurement by adding and testing new concepts. We aimed to create FACE-Q Aesthetics item libraries. In-depth concept elicitation interviews were conducted. Concepts were formed into items and refined through multiple rounds of patient and expert input. The items were tested with people living in the United States, Canada, and the United Kingdom who had minimally invasive facial aesthetic treatments. Participants were recruited through an online platform (ie, Prolific). Psychometric properties were examined using Rasch measurement theory analysis, test-retest reliability, and construct validity. We conducted 26 interviews. New concepts were developed into items and refined with input from 12 experts, 11 clinic patients, and 184 Prolific participants. A sample of 1369 Prolific participants completed 52 appearance and 22 psychological items. After removing 10 and 2 items respectively, the psychometric tests provided evidence of reliability with the person separation index, Cronbach alpha, and test-retest reliability values without extremes of 0.88 or more. For validity, lower scores were associated with looking older than one's age, being more bothered by facial skin laxity, treatment wearing off, and having deeper lines on Merz Assessment scales. Short-form scales formed from the 42 appearance items provide examples of item library application. This study provides an innovative means to customize scales to measure appearance and psychological function that maximizes content validity and minimizes respondent burden in the context of minimally invasive treatments.

Mixed-methods research to support the use of new lymphoma-specific patient-reported symptom measures derived from the EORTC item library

BackgroundNo specific measures exist to assess patient-reported symptoms experienced by individuals with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) or mantle cell lymphoma (MCL). This study was conducted to elicit patient-reported CLL/SLL- and MCL-related symptoms and their impact on patients’ lives. The study qualitatively and quantitatively evaluated sets of conceptually-selected EORTC Item Library items for assessing CLL/SLL- and MCL-related symptoms.MethodsThe qualitative component of the research included a literature review, clinician consultations, and patient interviews. Concepts important to patients were identified and coded; cognitive debriefing of the selected library items was completed with patients. CLL/SLL and MCL-related symptoms and impacts were organized in a structured conceptual model, which was mapped to item sets from the Item Library. The quantitative component comprised exploratory macro-level Rasch measurement theory (RMT) analysis conducted to provide supportive quantitative insight on the item sets.Results41 patients (21-MCL; 20-CLL/SLL) and 5 clinicians participated in the qualitative study; 57 unique patients (30-MCL; 27-CLL/SLL) completed the EORTC items. The conceptual models generated from the qualitative work included symptoms and functional impacts of CLL/SLL and MCL. Symptom domains included swollen lymph nodes, B symptoms, abdominal issues, pain, fatigue, subjective cognitive impairment, anemia-related symptoms, bleeding, infection, and other issues (appetite loss, temperature fluctuation, rash, weight gain, sleep problems, cough). Impacts included physical function, role function, and other functions (psychological, social). Cognitive debriefing demonstrated that the separate item sets for CLL/SLL and MCL-related symptoms were well understood and aligned with patients’ experiences. All selected items were included in the conceptual models. The exploratory RMT analysis showed that the item sets provided adequate coverage of the continuum of CLL/SLL- and MCL-related symptom severity.ConclusionsThis study gathered qualitative and early quantitative evidence supporting use of the EORTC Item Library to assess CLL/SLL- and MCL-related symptoms and impacts. These items are promising candidates for measurement of patient-reported disease symptoms in these populations. A larger sample size will be essential to establish the psychometric properties necessary to support use in clinical trials.Plain English summaryPatients who suffer from rare cancers of the blood, bone marrow, and lymph nodes can experience chronic and debilitating symptoms. At present, however, there are no dedicated instruments for assessing the patient’s experience of symptoms of conditions like chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) or mantle cell lymphoma (MCL), or for assessing their impact on patients’ lives. This research project aimed to address that need. The researchers selected relevant and clinically meaningful symptoms from the EORTC Item Library that assess fatigue, B symptoms, and CLL/SLL- and MCL-specific symptoms. Using patients and clinician interviews as well as quantitative analyses, the research revealed no major concerns with using these item sets to assess symptoms of CLL/SLL and MCL. Interviews with patients demonstrated that the separate item sets for CLL/SLL and MCL-related symptoms were well understood and aligned with patients’ experiences. All selected items were included in the conceptual models. Item sets identified in this study can potentially be used to assess patient-reported symptom endpoints in clinical trial settings in these disease areas.

Further refinement of the Patient-Reported Impact of Dermatological Diseases (PRIDD) measure using classical test theory and item response theory.

Existing dermatology-specific Patient-Reported Outcome Measures (PROMs) do not fully capture the substantial physical, psychological and social impact of dermatological conditions on patients' lives and are not recommended for use according to the COSMIN criteria. Most were developed with insufficient patient involvement and relied on classical psychometric methods. We are developing the new Patient-Reported Impact of Dermatological Diseases (PRIDD) measure for use in research and clinical practice in partnership with patients. To examine the factor structure of PRIDD, determine the definitive selection of items for each subscale, and establish structural validity and internal consistency through classical and modern psychometric methods. Two cross-sectional online surveys were conducted. Adults (≥ 18 years) worldwide living with a dermatological condition were recruited through the membership network of the International Alliance of Dermatology Patient Organizations (GlobalSkin). They completed the PRIDD questionnaire and a demographics questionnaire via an online survey. We examined missing data and distribution of scores for each item. The factor structure was assessed using confirmatory and exploratory factor analysis (Survey 1). Internal consistency was examined using Cronbach's α. Rasch measurement theory analyses were conducted, including iterative assessment of rating scale function, fit to the Rasch model, unidimensionality, reliability, local dependence, targeting and differential item functioning (DIF) (Surveys 1 and 2). Participants in Surveys 1 and 2 numbered 483 and 504 people, respectively. All items had ≤ 3% missing scores and all five response options were used. A four-factor model showed the best fit. PRIDD and all four subscales were internally consistent but showed some misfit to the Rasch measurement model. Adjustments were made to rectify disordered thresholds, remove misfitting items, local dependency and DIF, and improve targeting. The resulting 16-item version and subscales fit the Rasch model, showed no local dependency or DIF at the test level, and were well targeted. This field test study produced the final PRIDD measure, consisting of 16 items across four domains. The data triangulated and refined the conceptual framework of impact and provide evidence of PRIDD's structural validity and internal consistency. The final step in the development and validation of the PRIDD measure is to test the remaining measurement properties.

Psychometric Evaluation of the PNH Symptom Questionnaire (PNH-SQ) Among Patients with Paroxysmal Nocturnal Hemoglobinuria from Three Phase 2 Clinical Trials with Pozelimab Monotherapy or in Combination with Cemdisiran

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease caused by chronic dysregulation of the complement system. PNH has a high symptom burden with significant impacts on the life of a patient. The PNH Symptom Questionnaire (PNH-SQ) is a PNH-specific patient-reported outcome (PRO) questionnaire designed to assess daily symptom severity in patients with PNH. The objective of this current study was to evaluate PNH-SQ psychometric properties among patients with PNH from three phase 2 clinical trials with pozelimab monotherapy or in combination with cemdisiran. Methods: PRO data from three phase 2 PNH clinical trials, study 1852 (NCT03946748; pozelimab monotherapy; n=24), study 2092 (NCT04811716; combination therapy; n=24), and study 20105 (NCT04888507; combination therapy; n=6), were used for analysis. The PNH-SQ is a patient-reported daily diary that assesses 10 core symptoms of PNH using a five-point severity scale (none; very mild; mild; moderate; severe; very severe) for fatigue, shortness of breath, muscle weakness, headaches, abdominal pain, pain in back or legs, and chest discomfort, and a five-point difficulty scale (none, not at all, a little bit, somewhat, quite, very) for difficulty sleeping, difficulty thinking clearly, and difficulty swallowing. Prespecified psychometric analyses were performed after study database lock using blinded data. Analyses included quality of completion (ie ePRO prevented item-level missingness) and item responses over time. Reliability and validity of daily assessments with classical test theory (internal consistency and test-retest reliability, construct validity by observing correlations between pairs of PNH-SQ items and of the PNH-SQ daily score with other PRO scores from the EORTC QLQ-C30, FACIT-Fatigue and EQ-5D) and Rasch measurement theory (RMT) analyses were conducted. Test-retest reliability (within stable patients 4 weeks apart) and construct validity of various exploratory methods for aggregating daily scores over time were assessed. Results: Completion rates of PNH-SQ items were higher in studies 20105 and 2092 (at Day 1: 54.2% in 1852 vs 83.3% in 20105 and 91.7% in 2092). Fatigue was consistently the most experienced symptom at baseline of the three studies, while difficulty swallowing was not experienced by any patient. A high proportion of ‘none’ responses were observed across all assessments. Cronbach's alpha calculated using all daily assessments was 0.75, indicating modest reliability. The correlation between each pair of items at Day 1 ranged between 0.11 and 0.93, with the lowest correlations being observed between pain items and sleep/cognitive items, and between abdominal pain and fatigue/dyspnea. At Day 1, PNH-SQ daily score trended higher according to the Patient Global Impression of Severity (PGIS)-symptoms categories and showed moderate correlations in expected direction with fatigue and pain, as assessed by FACIT-Fatigue and EORTC QLQ-C30, and lower correlations with dyspnea and overall health status (EQ-5D visual analog scale). RMT analyses of the daily PNH-SQ data highlighted that for response options “Very mild” for severity/“Not at all” for difficulty did not provide any useful information. RMT also uncovered a meaningful hierarchy of symptoms ranging from fatigue to difficulty swallowing. A fixed 7-day average led to the best test-retest reliability (intraclass correlation coefficient, 0.76). Conclusions: Our analyses documented the PNH-SQ as an instrument to capture symptoms of PNH. The most frequent symptoms reported in our sample using the PNH-SQ, fatigue, dyspnea, and headaches, were aligned with observational study data. The sample of observations from the three trials was strongly skewed towards absence of symptoms, probably reflecting the low symptomatic severity of patients included in the study. Despite this restriction, early supportive data for the scoring of the PNH-SQ from a sample of patients with PNH were obtained, generating useful insights into the function of the response scales, opportunities for the creation of daily assessments of symptom severity, and for the creation of a measure reflecting severity over a longer period, for which a fixed 7-day average appeared as a potential good scoring approach. Generating further evidence, especially in a sample of more symptomatic patients, is recommended to substantiate these results.

BODY-Q Normative Scores: Psychometric Validation of the BODY-Q in the General Population in Europe and North America.

BODY-Q is a rigorously developed patient-reported outcome measure designed to measure outcomes of weight loss and body contouring patients. To allow interpretation and comparison of BODY-Q scores across studies, normative BODY-Q values were generated from the general population. The aim of this study was to examine the psychometric properties of BODY-Q in the normative population. Data were collected using two crowdsourcing platforms (Prolific and Amazon Mechanical Turk) in 12 European and North American countries. Rasch measurement theory (RMT) was used to examine reliability and validity of BODY-Q scales. RMT analysis supported the psychometric properties of BODY-Q in the normative sample with ordered thresholds in all items and nonsignificant chi-square values for 167 of 176 items. Reliability was high with person separation index of greater than or equal to 0.70 in 20 of 22 scales and Cronbach alpha values of greater than or equal to 0.90 in 17 of 22 scales. Mean scale scores measuring appearance, health-related quality of life, and eating-related concerns scales varied as predicted across subgroups with higher scores reported by participants who were more satisfied with their weight. Analysis to explore differential item functioning by sample (normative versus field-test) flagged some potential issues, but subsequent comparison of adjusted and unadjusted person estimates provided evidence that the scoring algorithm worked equivalently for the normative sample as in the field-test samples. The BODY-Q scales showed acceptable reliability and validity in the normative sample. The normative values can be used as reference in research and clinical practice in combination with local estimates for parallel analysis and comparison.

Establishing Reliability and Validity of the FACE-Q Craniofacial Module for Pediatric Head and Neck Cancer.

Purpose: We aimed to establish content validity and assess the psychometric properties of the FACE-Q Craniofacial Module, a patient-reported outcome measure, for use in pediatric and adolescent patients with head and neck cancer (HNC). Methods: To establish content validity (Part 1), between June 2017 and August 2019, cognitive interviews were conducted with survivors of pediatric HNC (n = 15), and input was obtained from clinical experts (n = 21). To examine item and scale performance (Part 2), Rasch Measurement Theory (RMT) analysis was performed using data from two international studies (n = 121). Results: Part 1: Qualitative data from 15 survivors and input from 21 experts provided evidence to support the use of the FACE-Q Craniofacial Module in pediatric HNC. Part 2: The field-test study sample included 121 survivors of pediatric HNC. RMT analysis provided evidence of reliability and validity for 10 FACE-Q scales. Data for each scale fit the RMT model. Scale reliability was high, with Person Separation Index and Cronbach's alpha values ≥0.82 for 9 scales. Mean scores on the Appearance, Psychological, and Social scales were higher for those who liked aspects of their face more. For participants with (vs. without) a facial difference, mean scores were lower for the Face, Jaws, Psychological, and Social scales. Conclusion: The FACE-Q Craniofacial Module evidenced reliability and validity for HNC survivors aged 8-29 years and can be used in research and clinical care to measure quality of life of pediatric survivors with HNC.

Development and Validation of a Patient-Reported Outcome Measure for Fingernail and Toenail Conditions: The NAIL-Q.

Patient-reported outcome measures (PROMs) are needed to measure outcomes that matter to people with nail conditions, from their perspective. To design a comprehensive new PROM (NAIL-Q) to measure outcomes important in toenail and fingernail conditions. A mixed methods iterative approach was used. Phase 1 involved concept elicitation interviews that were audio-recorded, transcribed, and coded line-by-line. Concepts were developed into scales and refined through cognitive debriefing interviews with patients and expert input. Data was then collected from an international sample using a crowdsource platform. Eligible participants were aged ≥18 years with a nail condition for at least 3 months. Rasch Measurement Theory (RMT) analysis was used to examine item and scale performance. Other psychometric tests included test-retest reliability, and convergent and construct validity. Phase 1 interviews involved 23 patients with 10 nail conditions and input from 11 dermatologists. The analysis led to the development of 84 items for field-testing. In Phase 2, 555 participants completed the survey. Toenail conditions (n = 441) were more common than fingernail conditions (n = 186). The RMT analysis reduced the number of items tested to 45 in 7 scales measuring nail appearance, health-related quality of life concerns, and treatment outcomes. All items had ordered thresholds and nonsignificant chi-square p values. Reliability statistics with and without extremes for the Person Separation Index were ≥0.79 and Cronbach's alpha were ≥0.83, and for intraclass correlation coefficients were ≥0.81. Construct validity was further supported in that most participants agreed that the NAIL-Q was easy to understand, asked relevant and important questions in a respectful way, and that it should be used to inform clinical care. The NAIL-Q is a rigorously designed and tested PROM that measures nail appearance, health-related quality of life and treatment outcomes. This PROM can be used in clinical practice to inform patient care and to include the patient perspective in research.

Does the Multiple Sclerosis Impact Scale-29 (MSIS-29) have the range to capture the experience of fully ambulatory multiple sclerosis patients? Learnings from the ASCLEPIOS studies.

Trials of disease-modifying therapies (DMTs) for multiple sclerosis (MS) often include patients with minimal disability. Patient-reported outcome instruments used in these trials have often not captured physical and psychological treatment effects concomitant with observed clinical benefits. To examine whether the Multiple Sclerosis Impact Scale-29 (MSIS-29) captures changes in the impact of MS in a sample of patients enrolled in the Phase 3 ASCLEPIOS studies (ofatumumab vs. teriflunomide). Measurement properties (i.e. item fit, reliability, and targeting) of the MSIS-29 were analyzed using Rasch measurement theory (RMT) in data from two phase 3 ofatumumab clinical trials including patients with relapsing-remitting or secondary progressive MS (N = 1882). Targeting of the MSIS-29 items to the patient population was explored within groups categorized by Expanded Disability Status Scale (EDSS) scores. Under RMT analyses, both the Physical and Psychological Impact scales of the MSIS-29 were not appropriately targeted to the overall sample of patients. In particular, 49% and 30% of patients with an EDSS score ≤ 2.5 had fewer physical and psychological impacts, respectively, than would typically be captured by these MSIS-29 items compared to patients with EDSS scores of ≥ 3. The MSIS-29 is commonly used to evaluate the patient-reported physical and psychological impact of MS. However, it may be limited in evaluating changes associated with DMTs in patients with minimal disability.

P035 Patient-reported outcome measures for rheumatoid arthritis disease activity: using Rasch measurement theory to achieve more meaningful measurement

Abstract Background/Aims Disease activity (DA) monitoring is a standard of care in rheumatoid arthritis (RA), and there is demand for achieving this through patient-reported outcome measures (PROMs). A systematic review of PROMs for RA DA following COSMIN guidelines demonstrated a lack of sufficient evidence for content validity for the 10 existing PROMS, thus none could be recommended for use. The aim of this study was to use Rasch measurement theory (RMT) to develop a valid item pool for measurement of DA in RA. Methods Paper questionnaires were sent to people aged 18 or over with RA from four South Wales University Health Boards between September 2020 and November 2021. The questionnaire included 268 individual RA DA items extracted from the 10 PROMs identified by the systematic review, another four PROMs and a non-measurement group of items. Further items suggested by patient feedback were incorporated, including a Pain Activity Scale, discomfort when walking, standing, and exercising, plus fear of falling when walking. Items were grouped into domains established by OMERACT: tenderness and swelling, patient global, pain, fatigue, physical functioning; and additionally stiffness, swelling, discomfort/fear and mood. Exploratory factor analyses (EFA) were used to indicate which items from which domains loaded onto factors together. For each separate domain, psychometric properties were assessed by RMT analyses. This provided results on targeting, model fit, internal consistency, local dependency, unidimensionality and item threshold ordering. Results A test dataset of n = 398 and a validation dataset of n = 293 were available. EFA of the test dataset showed that 30 items from across the tenderness and swelling, patient global, pain, fatigue, physical functioning and stiffness domains loaded together. RMT analyses of these items in the test dataset indicated that the patient global domain comprised two distinct domains, which followed a clear content difference of general health and disease activity items. In assessing the best items to measure the now 7 domains, 12 items were discarded. These 7 domains were confirmed in the validation data. Subtest analyses indicated that patient global general health and fatigue did not measure the same overall construct as the other 5 domains, and instead worked together to measure a separate construct. This structure was confirmed using bi-factor model confirmatory factor analyses in the validation data. Conclusion Patient global items relating to general health and disease activity were two separate domains. RA DA can be measured using tenderness and swelling, patient global disease activity, pain, physical functioning and stiffness items, whilst fatigue and patient global general health items measure a health-related quality of life construct. The next steps are to undertake cognitive interviews to establish content validity and to develop a computer adaptive test based on anchored locations calculated using these data. Disclosure T. Pickles: None. M. Horton: None. K.B. Christensen: None. R. Phillips: None. D. Gillespie: None. N. Mo: None. E. Choy: None.

Development and Preliminary Validation of an Instrument to Measure Symptoms and Impacts in Patients with Proliferative Diabetic Retinopathy.

Following a review of patient-reported outcome (PRO) instruments in the literature, existing PRO instruments may not adequately capture the experience of receiving treatment for proliferative diabetic retinopathy (PDR). Therefore, this study aimed to develop a de novo instrument to comprehensively assess the patient experience of PDR. This qualitative, mixed-methods study comprised item generation for the Diabetic Retinopathy-Patient Experience Questionnaire (DR-PEQ), content validation in patients with PDR, and preliminary Rasch measurement theory (RMT) analyses. Adult patients with diabetes mellitus and PDR who received aflibercept and/or panretinal photocoagulation within 6months of study initiation were eligible for participation. The preliminary DR-PEQ comprised four scales: Daily Activities, Emotional Impact, Social Impact, and Vision Problems. DR-PEQ items were generated using existing knowledge of patient experiences in PDR and conceptual gaps identified from existing PRO instruments. Patients indicated the level of difficulty conducting daily activities and frequency experiencing emotional impacts, social impacts, and vision problems attributed to diabetic retinopathy and its treatment in the past 7days. Content validity was evaluated in two rounds of in-depth, semi-structured patient interviews. Measurement properties were investigated via RMT analyses. The preliminary DR-PEQ comprised 72 items. Overall, mean (SD) patient age was 53.7 (14.7) years. Forty patients completed the first interview; of these, 30 completed the second interview. Patients reported thatthe DR-PEQ was easily understood and relevant to their experience. Minor revisions, including removal of the Social Impact scale and addition of a Treatment Experience scale, were implemented to generate 85 items spanning four scales: Daily Activities, Emotional Impact, Vision Problems, and Treatment Experience. RMT analyses provided preliminary evidence that the DR-PEQ performed as intended. The DR-PEQ evaluated a broad spectrum of symptoms, functional impacts, and treatment experiences relevant to patients with PDR. Additional analyses are warranted to evaluate psychometric properties in a larger patient population.

A Mixed-Methods Study to Better Measure Patient-Reported Pain and Fatigue in Soft Tissue Sarcoma.

Pain and fatigue are commonly reported by patients with soft tissue sarcoma (STS) as distressing symptoms, yet no patient-reported outcome (PRO) measures have been validated or developed specifically for STS. This study aimed to develop novel PRO scales using existing item banks to measure pain and fatigue in STS. A three-stage mixed-methods approach was used. Stage 1: a literature review examined the development and validation of the European Organization for Research and Treatment of Cancer (EORTC) library, Patient-Reported Outcomes Measurement Information System (PROMIS) pain/fatigue item banks, Functional Assessment of Cancer Therapy-General, and FACIT-Fatigue. Conceptual models were developed for pain and fatigue. Stage 2: semi-structured interviews were conducted with clinical experts (n = 3) and STS patients (n = 28) to ensure conceptual coverage and cognitively debrief the selected PRO items. Stage 3: exploratory Rasch measurement theory (RMT) analyses were performed to examine the measurement properties of the proposed scales. Stage 1: The conceptual model for fatigue was organized into two overarching domains: fatigability and fatigue, further split into two subdomains: symptoms and impact. The conceptual model for pain had one overarching domain split into two subdomains: descriptors and impact. Pain (n = 56) and fatigue (n = 40) items were selected from the EORTC item library. Stage 2: qualitative findings ensured conceptual coverage, provided insight into the relevance and comprehension of the items, and informed subsequent item reduction. Stage 3: The total item number was reduced to 43 (pain n = 18, fatigue n = 25). Exploratory RMT analyses supported the final scales' psychometric properties. This mixed-methods research generated important information on the experience of pain and fatigue in specific subtypes of STS. Five novel PRO scales have been developed through careful item selection in consultation with experts and supported by qualitative and quantitative evidence. These scales may be of value to future clinical trials for STS.

Dutch translation and cultural adaptation of the LYMPH-Q, a new patient-reported outcome measure for breast cancer-related lymphedema

BackgroundThe LYMPH-Q Upper Extremity module is a new patient-reported outcome measure (PROM) developed to assess patient outcomes of breast cancer-related arm lymphedema (BCRL). Content for the LYMPH-Q Upper Extremity Module was developed from the extensive input of patients and experts in the field of breast surgery and breast cancer-related lymphedema. Rasch Measurement Theory analysis was used to assess psychometric properties. The aim of this study was to perform a Dutch translation and cultural adaptation of the LYMPH-Q Upper Extremity Module.MethodsThe translation process was performed in accordance with the guidelines of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR).The process included two forward translations, two back translations, and cognitive debriefing interviews with patients with BCRL.ResultsComparison of the two forward translations showed that the translations for most items (n = 60; 88.2%) were conceptually consistent between the two translators. Translations of the remaining items were reviewed and discussed until consensus was reached. Three items in the back translation had a different meaning when compared to the original English version and required re-translation. The resultant Dutch version of the LYMPH-Q was tested in a series of cognitive debriefing interviews with seven patients and showed good content validity.ConclusionsThe translation and cultural adaptation process resulted in a conceptually equivalent Dutch version of the LYMPH-Q Upper Extremity Module. This new PROM can now be used in clinical practice and research settings to evaluate outcomes in patients with BCRL.Level of evidence: Not gradable

PCR46 A Mixed-Methods Study Using Cognitive Debriefing to Assess the Content Validity of a New Health and Functioning Measure for Tobacco and/or Nicotine Product Users

To inform tobacco harm reduction strategies, a new consumer reported outcome measure (CROM) was developed to assess perceived Health and Functioning (H&F) among users of tobacco and/or nicotine products (TNP). This study assessed the face, content validity, and early measurement properties of the draft H&F CROM. Cognitive debriefing interviews were conducted in two waves in the UK and US with adult users of: cigarettes (n=20), e-cigarettes (n=20), heated tobacco products (IQOSTM) (n=20), smokeless tobacco products (n=10), and nicotine replacement therapy products (n=10). Participants completed the draft measure (195 items) electronically through a “think-aloud” process. Different response options were tested. Transcripts were analyzed thematically using Atlas.ti. Exploratory psychometric analysis was conducted by Rasch Measurement Theory (RMT) analysis. Expert panel meetings were held to inform result interpretation and updating the CROM after each wave. The participants generally endorsed the content of the CROM, while the interim analysis allowed for some early item and scale refinements. The RMT analysis largely supported the scales’ reliability, sample-to-scale targeting, and discrimination potential across the TNP user groups. Conceptual overlap between some items was identified through both qualitative and quantitative evidence. Mixed-methods evidence was used to guide the selection of response scale, e.g., severity difficulty or frequency that could better match the underlying concept of each scale, while the 7-day recall period was found to have less of a ceiling effect. This study provides early supporting evidence of the new H&F CROM’s content relevance, clarity, and ease of completion. It comprises ten scales (146 items) covering different health concepts (including oral, lung, and general health and breathing difficulty), appearance, bother with TNP smell, as well as different functioning concepts (including work, social, and well-being). A psychometric validation study is being conducted to inform the item content, scoring structure, and measurement properties of the CROM.

The HAND-Q: Psychometrics of a New Patient-reported Outcome Measure for Clinical and Research Applications.

Background:The perspective of the patient in measuring the outcome of their hand treatment is of key importance. We developed a hand-specific patient-reported outcome measure to provide a means to measure outcomes and experiences of care from the patient perspective, that is, HAND-Q.Methods:Data were collected from people with a broad range of hand conditions in hand clinics in six countries between April 2018 and January 2021. Rasch measurement theory analysis was used to perform item reduction and to examine reliability and validity of each HAND-Q scale.Results:A sample of 1277 patients was recruited. Participants ranged in age from 16 to 89 years, 54% were women, and a broad range of congenital and acquired hand conditions were represented. Rasch measurement theory analysis led to the refinement of 14 independently functioning scales that measure hand appearance, health-related quality of life, experience of care, and treatment outcome. Each scale evidenced reliability and validity. Examination of differential item functioning by age, gender, language, and type of hand condition (ie, nontraumatic versus traumatic) confirmed that a common scoring algorithm for each scale could be implemented.Conclusions:The HAND-Q was developed following robust psychometric methods to provide a comprehensive modular independently functioning set of scales. HAND-Q scales can be used to assess and compare evidence-based outcomes in patients with any type of hand condition.

The FATIGUE-PRO: a new patient-reported outcome instrument to quantify fatigue in patients affected by systemic lupus erythematosus.

ObjectivesThis study aimed to implement a patient-centred and evidence-based approach to develop a novel patient-reported outcome (PRO) instrument to measure fatigue in patients with SLE.MethodsA three-step mixed methods psychometric (MMP) approach was followed. Steps comprised first draft item generation and review using interview data; evaluation and refinement of second draft items using mixed methods data, including interview and quantitative data from a phase 2 clinical study in SLE analysed using Rasch Measurement Theory (RMT) analysis; and evaluation of the final FATIGUE-PRO items using RMT and complementary Classical Test Theory (CTT) analyses. Guided by MMP criteria, a team of clinicians and outcome-measurement experts assessed evidence to inform instrument development.ResultsStep 1 culminated in 55 items (n = 39 patients interviewed). Their refinement in step 2 using mixed methods evidence led to the final FATIGUE-PRO instrument comprising 31 items across three scales of fatigue: physical fatigue (9 items), mental and cognitive fatigue (11 items) and susceptibility to fatigue (11 items). Qualitative (n = 43 patients) and quantitative (n = 106 patients) evidence strongly supported the scales’ content comprehensiveness and targeting, item quality and fit, conceptual uniqueness and appropriateness of the response scale. The FATIGUE-PRO further benefited from excellent reliability (RMT: 0.92–0.94 and CTT: 0.95–0.96) and supportive evidence of construct validity from assessments against other PROs.ConclusionThe conceptual advances, comprehensive coverage and strong psychometric properties of the FATIGUE-PRO will significantly advance the measurement and management of fatigue in SLE, both in clinical trials and routine practice.Trial registrationClinicalTrials.gov (https://clinicaltrials.gov), NCT02804763

Development and Psychometric Validation of a Patient-Reported Outcome Measure for Arm Lymphedema: The LYMPH-Q Upper Extremity Module

BackgroundA multiphased mixed-methods study was performed to develop and validate a comprehensive patient-reported outcome measure (PROM) for arm lymphedema in women with breast cancer (i.e., the LYMPH-Q Upper Extremity Module).MethodsQualitative interviews (January 2017 and June 2018) were performed with 15 women to elicit concepts specific to arm lymphedema after breast cancer treatment. Data were audio-recorded, transcribed, and coded. Scales were refined through cognitive interviews (October and Decemeber 2018) with 16 patients and input from 12 clinical experts. The scales were field-tested (October 2019 and January 2020) with an international sample of 3222 women in the United States and Denmark. Rasch measurement theory (RMT) analysis was used to examine reliability and validity.ResultsThe qualitative phase resulted in six independently functioning scales that measure arm symptoms, function, appearance, psychological function, and satisfaction with information and with arm sleeves. In the RMT analysis, all items in each scale had ordered thresholds and nonsignificant chi-square p values. For all the scales, the reliability statistics with and without extremes for the Person Separation Index were 0.80 or higher, Cronbach’s alpha was 0.89 or higher, and the Intraclass Correlation Coefficients were 0.92 or higher. Lower (worse) scores on the LYMPH-Q Upper Extremity scales were associated with reporting of more severe arm swelling, an arm problem caused by cancer and/or its treatment, and wearing of an arm sleeve in the past 12 months.ConclusionsThe LYMPH-Q Upper Extremity Module can be used to measure outcomes that matter to women with upper extremity lymphedema. This new PROM was designed using a modern psychometric approach and, as such, can be used in research and in clinical care.

FACE-Q Craniofacial Module: Part 1 validation of CLEFT-Q scales for use in children and young adults with facial conditions

The CLEFT-Q includes 12 independently functioning scales that measure appearance (face, nose, nostrils, teeth, lips, jaws), health-related quality of life (psychological, social, school, speech distress), and speech function, and an eating/drinking checklist. Previous qualitative research revealed that the CLEFT-Q has content validity in noncleft craniofacial conditions. This study aimed to examine the psychometric performance of the CLEFT-Q in an international sample of patients with a broad range of facial conditions. Data were collected between October 2016 and December 2019 from 2132 patients aged 8 to 29 years with noncleft facial conditions. Rasch measurement theory (RMT) analysis was used to examine Differential Item Function (DIF) by comparing the original CLEFT-Q sample and the new FACE-Q craniofacial sample. Reliability and validity of the scales in a combined cleft and craniofacial sample (n=4743) were examined. DIF was found for 23 CLEFT-Q items when the datasets for the two samples were compared. When items with DIF were split by sample, correlations between the original and split person locations showed that DIF had negligible impact on scale scoring (correlations ≥0.995). In the combined sample, RMT analysis led to the retention of original content for ten CLEFT-Q scales, modification of the Teeth scale, and the addition of an Eating/Drinking scale. Data obtained fit with the Rasch model for 11 scales (exception School, p=0.04). Person Separation Index and Cronbach alpha values met the criteria. The scales described in this study can be used to measure outcomes in children and young adults with cleft and noncleft craniofacial conditions.

Development and Validation of New BODY-Q Scales Measuring Expectations, Eating Behavior, Distress, Symptoms, and Work Life in 4004 Adults From 4 Countries.

The BODY-Q is a rigorously developed patient-reported outcome measure (PROM) for patients seeking treatment for obesity and body contouring surgery. A limitation of the uptake of the BODY-Q in weight management treatments is the absence of scales designed to measure eating-specific concerns. We aimed to develop and validate 5 new BODY-Q scales measuring weight loss expectations, eating behaviors, distress, symptoms, and work life. In phase 1 (qualitative), patient and expert input was used to develop and refine the new BODY-Q scales. In phase 2 (quantitative), the scales were field-tested in bariatric and weight management clinics in the United States (US), The Netherlands, and Denmark between June 2019 and January 2020. Data were also collected in the US and Canada in September 2019 through a crowdworking platform. Rasch measurement theory (RMT) analysis was used for item reduction and to examine reliability and validity. The new BODY-Q scales were refined through qualitative input from 17 patients and 20 experts (phase 1) and field-tested in 4004 participants (phase 2). All items showed ordered thresholds and good fit to the Rasch model. The RMT analysis provided evidence of reliability, with PSI values ≥0.72, Cronbach alpha values ≥0.83, and test-retest values ≥0.79. Better scores on 4 scales (exception expectations scale) correlated with lower BMI, with the strongest correlation between the eating-related distress scale scoresand BMI (r= -0.249, P < 0.001). The new BODY-Q scales can be used in research and clinical practice to assess weight loss treatments from the patient perspective.

Development and Psychometric Validation of BREAST-Q Scales Measuring Cancer Worry, Fatigue, and Impact on Work.

The BREAST-Q is a patient-reported outcome measure for women with breast cancer. The aim of this study was to develop new BREAST-Q scales to measure Cancer Worry, Fatigue and Impact on Work. Data were collected between January 2017 and November 2019. Phase 1 (qualitative) included participants from Canada and the USA, pre/post any type of breast cancer treatment (surgery, adjuvant, neoadjuvant). Interviews were audio-recorded, transcribed verbatim and coded line-by-line. New scales were drafted and refined through cognitive interviews and expert input. Phase 2 (field-test study) involved USA members of the Love Research Army (LRA). Rasch measurement theory analysis was used to examine reliability and validity. In phase 1, 57 women were interviewed. Three concepts were identified as important to the breast cancer experience that are not currently covered in the BREAST-Q and developed into scales, i.e., Cancer Worry, Fatigue and Impact on Work. Feedback from nine women and 23 experts was used to establish content validity. The scales were field-tested in the LRA sample (n=1680), of whom 1006 completed a test-retest. Reliability was >0.81 for the person separation index, >0.89 for Cronbach's alpha and >0.83 for interclass correlation coefficients. Lower scores on all three scales were significantly associated with being closer in time to diagnosis and having a higher cancer stage at diagnosis (p<0.001 on ANOVA). These new scales expand the BREAST-Q measurement system and provide a means to evaluate additional important outcomes for breast cancer patients in clinical care and research.