UNASSIGNED: Chronic cough, a cough lasting >8 weeks, includes refractory chronic cough (RCC) and unexplained chronic cough (UCC). Patient-reported outcome (PRO) measures are needed to better understand chronic cough impacts that matter most to patients. The 19-item Leicester Cough Questionnaire (LCQ), an existing PRO measure of chronic cough, assesses impacts of cough across physical, psychological, and social domains. However, the content validity of the LCQ evaluating these concepts in patients with RCC/UCC had not been established.
UNASSIGNED: To evaluate the content validity of the LCQ in patients with RCC/UCC.
UNASSIGNED: A cross-sectional, qualitative interview study.
UNASSIGNED: First, previously completed qualitative interview results in adults with RCC/UCC (N = 30) were evaluated and mapped to LCQ concepts. Next, a clinical cough expert reviewed each LCQ item and assessed the salience of its concepts for patients with RCC/UCC. Finally, semistructured interviews-including both concept elicitation and cognitive debriefing-were conducted in adults with RCC/UCC (N = 20) to elicit a comprehensive set of participant experiences and to assess the appropriateness of using the LCQ in this population.
UNASSIGNED: Concepts reported in the past and present qualitative interviews were included across all LCQ items, and most impacts reported to be the \"most bothersome\" were assessed in the LCQ. In the current study, all participants indicated that reduced cough frequency would be an important treatment target. During cognitive debriefing, each LCQ item was endorsed by ⩾70% of participants. Additionally, participants were generally able to understand, recall, and select a response for each LCQ item. All participants and the clinical expert indicated that the LCQ was appropriate and assessed the impacts most relevant to patients with RCC/UCC.
UNASSIGNED: Our findings support the content validity of the LCQ and demonstrate that this measure is fit-for-purpose and includes important cough impacts in adults with RCC/UCC.

UNASSIGNED: Loss of mobility is common in persons with multiple sclerosis (PwMS), but little is known about this impairment from the patient\'s perspective.
UNASSIGNED: The aim is to model longitudinal variation in a mobility patient-reported outcome (PRO) and compare trajectories to those observed for Timed 25-Foot Walk (T25FW) in a retrospective cohort.
UNASSIGNED: Latent-class growth analysis was applied to 47,508 measures of Performance Scales© Mobility PRO (PS-Mobility) over ~4 years for 8524 PwMS. For 7347 PwMS, there were 41,988 T25FW measures during this period. Repeated measures correlation and concordance of trajectory assignment were evaluated.
UNASSIGNED: At the group level, PS-Mobility and T25FW linearly worsened and repeated-measures correlation was moderate. Eight latent classes with varying shapes that worsened described PS-Mobility variation, compared to six latent classes for T25FW that differed by intercept. The agreement between PS-Mobility and T25FW cluster assignment was modest. A higher proportion of individuals who were Black/African American, older, Medicaid beneficiaries, living in deprived neighborhoods, had longer disease duration, had progressive disease, and ever smokers were assigned to more impaired clusters.
UNASSIGNED: Cross-sectionally, PS-Mobility and T25FW were highly correlated, but longitudinally correlation was modest to moderate, underscoring the importance of considering both objective and subjective perspectives in evaluating mobility changes in PwMS.

BACKGROUND: Physical activity (PA) measured by accelerometry is proposed as a novel trial endpoint for heart failure (HF). However, standardised methods and associations with established markers are lacking. This study aimed to examine PA measurements and accelerometer repeatability in patients with HF and age- and sex-matched controls, and study correlations with established prognostic HF markers, body composition, and quality of life (QoL).
METHODS: Accelerometry was performed in 105 patients with HF with left ventricular ejection fraction (LVEF) ≤ 40% and in 46 controls. Participants also underwent dual X-ray absorptiometry, cardiopulmonary exercise testing, a six-minute walking test (6MWT), echocardiography, and NT-proBNP measurement, and completed a QoL questionnaire.
RESULTS: Average acceleration was markedly reduced in patients with HF compared with healthy controls (16.1 ± 4.8 mg vs 27.2 ± 8.5 mg, p < 0.001). Healthy controls spent a median daily 56 min (IQR 41-96 min) in moderate-to-vigorous PA (MVPA), whereas HF patients spent only 12 min (IQR 6-24) in MVPA. In HF patients, average acceleration correlated moderately with 6MWT (R = 0.41, p < 0.001) and maximal oxygen uptake (peak VO2) (R = 0.36, p < 0.001) but not with NT-proBNP, LVEF, or QoL. Patients in NYHA class II showed a higher average acceleration than patients in NYHA III (16.6 ± 4.9 mg vs 14.0 ± 3.6 mg, p = 0.01).
CONCLUSIONS: Daily PA was severely reduced in patients with HF compared with healthy controls. In HF patients, we found moderate correlations of accelerometer measurements with markers of physical capacity but not with LVEF or NT-proBNP.
BACKGROUND: NCT05063955. Registered 01 June 2021-retrospectively registered.

OBJECTIVE: Mapping or matching the items in a clinical outcome assessment (COA) to concepts that define a condition is a common method for evaluating a COA\'s concept coverage. The purpose of this research was to address the lack of formal guidance for conducting this task by developing a framework for best practices in COA concept mapping and applying it to a case study.
METHODS: To develop the framework, we examined the literature and created a draft set of best practices which was then reviewed by experienced researchers through focus groups before being finalized. To conduct the case study, we extracted data from a systematic review of knee osteoarthritis (KO) symptoms and impacts and used the framework to map relevant concepts to items in the SF-36v2® Health Survey (SF-36v2).
RESULTS: The framework guides researchers in defining the purpose of and data sources for the mapping, establishing guiding principles and decision-making thresholds, and conducting the mapping exercise. The results of the case study demonstrate the usefulness of the framework in identifying 27/36 items (75%) in the SF-36v2 that addressed concepts that define KO.
CONCLUSIONS: This case study illustrates how the framework for best practices in COA concept mapping may be used, highlighting how establishing clear concept definitions and guiding principles and following a structured process throughout can help produce consistent, reliable, and reproducible results. The results from this rigorous approach can provide valuable evidence to support decisions about the appropriateness of a COA for the intended patient population.
In health-related quality of life research, mapping items in a clinical outcome assessment (COA) to concepts that define a health condition is one way to evaluate an instrument’s content validity (or, how well the instrument addresses the concepts it intends to measure). Without formal guidance on how to do this mapping, researchers can be inconsistent. This article describes the development of a framework for best practices in COA concept mapping. Informed by the literature and input from researchers with expertise in COA development and evaluation, the final framework guides researchers through the mapping process from start to finish, from helping to define the purpose of the task and identify the data sources, to establishing guiding principles and decision-making thresholds, conducting the mapping, and displaying the results. A case study—in which items from the SF-36v2® Health Survey were mapped to concepts from a systematic review of knee osteoarthritis symptoms and impacts—shows the framework in action, demonstrating how following the best practices can lead to consistent results that can support the evaluation of an instrument’s content validity.

OBJECTIVE: To determine the relation between pelvic symmetry, as measured by the cross-measurement technique, and patient-reported outcome measures (PROMs) in terms of functional status and health-related quality of life.
METHODS: In this prospective cohort study, X and Y measurements were taken according to the cross-measurement technique on AP radiographs of patients who were treated for an unstable pelvic ring injury in a level-1 trauma center. Patients completed PROMs at the time of admission (recalled pre-injury score) and one year following their injury, reporting their functional status with the Short Musculoskeletal Function Assessment (SMFA-NL), specifically the lower extremity dysfunction (LED), problems with daily activities (PDA) and mental and emotional problems (MEP) subscales, and quality of life with EuroQol-5D (EQ-5D). Subgroup analyses were also performed. PROMs were used to analyze the relation between pelvic symmetry and patient-reported outcome, using Spearman\'s Rank correlation coefficients.
RESULTS: A total of 130 patients (mean age 58 (SD18) years) with an unstable pelvic ring injury were included, of which 95 (73%) sustained type-B injuries and 35 (27%) type-C injuries. Sixty-three (49%) patients were treated operatively. The median pelvic symmetry ratio was 1.01 (IQR: 0.05). Weak or no correlations were found between the pelvic symmetry scores and the outcome measurements (Spearman\'s correlation coefficients: LED r = 0.09; PDA r = 0.11; MEP r=-0.02; and EQ-5D r=-0.08). Subgroup analyses revealed no correlations.
CONCLUSIONS: No significant relation was found between pelvic symmetry, measured radiologically, and functional status and health-related quality of life, one year following an unstable pelvic ring injury.

BACKGROUND: Validated patient-reported outcome measures to assess disease impact in patients with adult idiopathic inflammatory myopathies (IIMs) are needed. The objective of this study was to assess the construct validity of PROMIS Pain Interference, Fatigue, and Physical Function measures in comparison with core disease activity measures.
METHODS: Adults with IIM, excluding inclusion body myositis, from OMERACT Myositis Working Group (MWG) clinic sites completed PROMIS Short Form v1.0-Pain Interference 6a, PROMIS Short Form v1.0-Fatigue 7a, and PROMIS Short Form v2.0-Physical Function 8b measures. Core disease activity measures including patient and physician global disease activity assessments, manual muscle testing, serum creatine kinase activity, and Health Assessment Questionnaire Disability Index (HAQ-DI) were simultaneously assessed. To evaluate construct validity, a priori hypotheses for the expected correlations between PROMIS measures, age, and core disease measures were determined by >70 % agreement among MWG members and were compared against observed Pearson\'s correlations. Internal consistency of items and floor or ceiling effects for the PROMIS measures were also assessed. Subgroup analysis according to IIM subtype (dermatomyositis vs. non-dermatomyositis IIM) was performed.
RESULTS: 135 adults with IIM from 5 countries across North America, Europe, Asia, and Australia were included. For construct validity, a priori hypotheses were confirmed for 5 of 6 (83 %) PROMIS Pain Interference, 4 of 5 (80 %) PROMIS Fatigue, and 3 of 4 (75 %) PROMIS Physical Function correlations. Internal consistency was high for each PROMIS measure (Cronbach\'s alpha >0.9). Ceiling effects were observed only for PROMIS Pain Interference, with low/no pain in 29 % of patients. Subgroup analysis between dermatomyositis (n = 65) and non-dermatomyositis (n = 70) subtypes demonstrated similar correlations between PROMIS measures and disease activity measures.
CONCLUSIONS: PROMIS Short Form v1.0-Pain Interference 6a, PROMIS Short Form v1.0-Fatigue 7a, and PROMIS Short Form v2.0-Physical Function 8b measures demonstrate strong construct validity when compared to core disease activity measures in IIM, with consistent results across IIM subtypes. These findings support the use of these selected PROMIS measures to assess core domains of interest for measuring life impact in IIMs.

BACKGROUND: Ongoing lower extremity long-bone nonunion is a devastating condition and associated with substantial patient morbidity. There is limited evidence regarding physical and mental function after surgical management of lower extremity nonunions. The purpose of this study was to assess general physical and mental health and lower extremity specific physical function of patients that underwent surgery for a lower extremity long-bone nonunion.
METHODS: One-hundred and twenty-four adult patients who underwent successful surgical management for a lower extremity long-bone nonunion between June 2002 and December 2021 were evaluated at an average follow-up of 8.6 years (interquartile range [IQR]: 4 - 12). General physical and mental health was assessed with the Short-Form 12 (SF-12) physical (PCS) and mental (MCS) component summaries, and lower extremity specific physical function with the Lower Extremity Functional Scale (LEFS). Multivariable linear regression was performed to identify variables that were independently associated with outcomes.
RESULTS: The median LEFS was 50 (IQR: 37 - 63) and the median SF-12 PCS was 43 (IQR: 33 - 52), which are both lower than normative population scores (LEFS: 77 and PCS: 51, p < 0.0001). The median SF-12 MCS was 50, which was comparable to the normative population score of 51 (p < 0.0001). The number of previous surgeries before the index nonunion treatment (p = 0.018 and p = 0.041) and the number of revision surgeries after the index nonunion treatment (p = 0.022 and p = 0.041) were associated with lower LEFS and SF-12 PCS scores.
CONCLUSIONS: At an average of 8.6 years after lower extremity nonunion surgery that led to bone healing, patients continue to report lower general and lower extremity specific physical functioning compared to the normative population. The number of surgical attempts to obtain definitive healing was associated with compromised physical function scores. Mental health scores may return close to normative population scores. These results can be used to inform patients and guide treatment strategies and healthcare policies.

BACKGROUND: Symptom assessment is the key factor in determining disease status and optimal management of exocrine pancreatic insufficiency (EPI). There is a need for a standardized patient-reported outcome (PRO) questionnaire to assess symptoms in patients diagnosed with EPI. The purpose of this qualitative study was to increase understanding of the EPI symptom experience from the patients\' perspective, and to develop and evaluate the content validity of the EPI Symptom Questionnaire (EPI-SQ) in US patients with EPI.
METHODS: Concept elicitation interviews (Phase I) were conducted to understand the symptom experience in patients with a clinical diagnosis of EPI (i.e., fecal pancreatic elastase value of ≤ 200 mcg/g based on most recent value) due to chronic pancreatitis or pancreatectomy. The EPI-SQ was developed based on the data extracted from Phase I interviews and feedback from clinical experts. Next, separate cognitive interviews (Phase II) were conducted to evaluate participants\' understanding of the instructions, items, response scales, and recall periods of the instrument.
RESULTS: During Phase I interviews (n = 21), 19 participants (90%) reported abdominal pain as the most frequent EPI symptom and lifestyle changes were the most frequently endorsed impacts (n = 18; 86%). Phase II results indicated that all participants (n = 7) felt the 12-item EPI-SQ was relevant to their symptom experience and that they understood the items, instructions, and response options as intended.
CONCLUSIONS: The qualitative data from this study support the content validity of the EPI-SQ in measuring EPI symptom severity in US patient populations diagnosed with EPI.

BACKGROUND: Symptom status and treatment changes among patients with chronic obstructive pulmonary disease (COPD) using inhaler treatment in real-world clinical settings are not well understood, particularly according to class of treatment. We investigated the proportion of symptomatic patients among those with COPD using inhaler treatment, based on COPD Assessment Test (CAT) scores in clinical practice, and changes in inhaler treatments and symptoms at 1-year follow-up.
METHODS: This was a retrospective analysis of data from a multicenter, prospective cohort study conducted at medical institutions with respiratory specialists in Japan. The primary endpoint was the proportion of patients with CAT scores ≥ 10 or < 10 in each inhaler treatment group at registration.
RESULTS: Of 414 patients in the full analysis set, 76 (18.4%), 261 (63.0%), and 77 (18.6%) were using long-acting muscarinic antagonist (LAMA), LAMA + long-acting β2-agonist (LABA), and inhaled corticosteroids (ICS) + LABA, respectively, at registration. The proportions of patients with CAT scores ≥ 10 or < 10 per inhaler treatment group at registration, respectively, were 32.9% and 67.1% in the LAMA group, 55.0% and 45.0% in the LAMA + LABA group, and 50.0% and 50.0% in the ICS + LABA group. Most patients (> 75%) in each inhaler treatment group showed no change in inhaler treatment at 1 year, regardless of their CAT score at registration. Approximately 70-80% of patients with CAT scores ≥ 10 at registration still had CAT scores ≥ 10 at 1 year; 10-30% of patients with CAT scores < 10 at registration had CAT scores ≥ 10 at 1 year.
CONCLUSIONS: In real-world Japanese clinical practice, a considerable proportion of patients have persistent symptoms (CAT score ≥ 10) despite using mono or dual inhaler treatment; > 75% of symptomatic patients with COPD using inhaler treatment did not undergo treatment escalation at 1-year follow-up and remained symptomatic.
BACKGROUND: ClinicalTrials.gov identifier, NCT05903989.

BACKGROUND: Patient satisfaction and quality of recovery (QoR) are important patient-reported outcomes and quality metrics. The relationship between these two outcomes is complex, with studies showing a weak correlation between them in the non-obstetric population. We sought to evaluate the correlation between patient satisfaction and QoR scores in the obstetric population after caesarean delivery. As secondary aims, we aimed to determine the influence of urgency of procedure and mode of anaesthetic on patient satisfaction and QoR scores as well as determining drivers of satisfaction and dissatisfaction.
METHODS: Women were invited to complete the Maternal Satisfaction Scale for Caesarean Section (MSCS) and Obstetric Quality of Recovery Score (ObsQoR-11) questionnaires at 24 h after caesarean delivery. Correlations were analysed using Spearman\'s rank tests. Qualitative data were analyzed using thematic content analysis.
RESULTS: Data were collected from 300 women. There was a significant but weak positive correlation between ObsQoR-11 and MSCS scores (r = 0.31, P < 0.001). Correlation was significantly influenced by mode of anaesthesia (P < 0.001) and urgency of procedure (P = 0.005), with greater satisfaction amongst patients receiving spinal anaesthesia and those undergoing scheduled caesarean deliveries for a given QoR score. Quality of communication, interactions with staff and aspects of the postpartum physical environment were significant determinants of satisfaction and dissatisfaction.
CONCLUSIONS: Maternal satisfaction and obstetric QoR are distinct entities with a weak correlation between the two variables. Urgency of procedure and mode of anaesthesia are significant predictors of the correlation between satisfaction and quality of recovery scores. Many of the drivers of satisfaction were modifiable including quality of communication and a comfortable physical space for postpartum recovery.