Development of a novel patient reported outcome measure for health-related quality of life in amyotrophic lateral sclerosis (PROQuALS): study protocol.

BACKGROUND: Patient reported outcome measures (PROMs) can be used to assess the impact of health conditions upon an individual's health-related quality of life (HRQoL). Whilst PROMs have been used to quantify the HRQoL impact of amyotrophic lateral sclerosis (ALS), existing instruments may not fully capture what matters to people living with ALS (plwALS) or be appropriate to be used directly to inform the cost-effectiveness of new treatments. This highlights a need for a new condition-specific PROM that can both capture what's important to plwALS and be used in economic evaluation. This study has two key aims: 1) to produce a novel PROM for measuring HRQoL in plwALS (PROQuALS). 2) to value a set of items from the novel PROM to generate an associated preference-weighted measure (PWM) that will enable utility values to be generated. METHODS: A mixed-methods study design will be conducted across three stages. Stage 1 involves concept elicitation and the generation of draft PROM content from a robust and comprehensive systematic review of HRQoL in ALS, with input from plwALS. Stage 2 consists of cognitive debriefing of the draft PROM content to ascertain its content validity (Stage 2a), followed by a psychometric survey (Stage 2b) to assess statistical performance. Evidence from Stage 2 will be used to make decisions on the final content and format of the novel PROM. Stage 3 will involve valuation and econometric modeling using health economics methods to generate preference weights, so a PWM derived from the novel PROM can be used in the cost-effectiveness analyses of treatments. Patient and clinical advisory groups will have critical, collaborative input throughout the project. DISCUSSION: The novel PROM will be designed to comprehensively assess important aspects of HRQoL to plwALS and to quantify HRQoL in terms of subjective impact. The PROQuALS measure will be available for use in research and healthcare settings. The associated PWM component will extend and enable the use of PROQuALS in cost-effective analyses of new treatments for ALS. TRIAL REGISTRATION: Not applicable.

A short scale to measure health-related quality of life after traumatic brain injury in children and adolescents (QOLIBRI-OS-KID/ADO): psychometric properties and German reference values.

PURPOSE: The impact of pediatric traumatic brain injury (pTBI) on health-related quality of life (HRQoL) in children and adolescents remains understudied. Short scales have some advantages in terms of economy and administration over longer scales, especially in younger children. The aim of the present study is to psychometrically evaluate the six-item German version of the QOLIBRI-OS-KID/ADO scale for children and adolescents. In addition, reference values from a general German pediatric population are obtained to assist clinicians and researchers in the interpretation of HRQoL after pTBI. METHODS: A total of 297 individuals after TBI and 1997 from a general population sample completed the questionnaire. Reliability, validity, and comparability of the assessed construct were examined. RESULTS: The questionnaire showed satisfactory reliability (α = 0.75 and ω = 0.81 and α = 0.85 and ω = 0.86 for the TBI and general population samples, respectively). The QOLIBRI-OS-KID/ADO was highly correlated with its long version (R2 = 67%) and showed an overlap with disease-specific HRQoL (R2 = 55%) in the TBI sample. The one-dimensional factorial structure could be replicated and tested for measurement invariance between samples, indicating a comparable HRQoL construct assessment. Therefore, reference values and cut-offs indicating clinically relevant impairment could be provided using percentiles stratified by factors significantly associated with the total score in the regression analyses (i.e., age group and gender). CONCLUSION: In combination with the cut-offs, the QOLIBRI-OS-KID/ADO provides a cost-effective screening tool, complemented by interpretation guidelines, which may help to draw clinical conclusions and indications such as further administration of a longer version of the instrument to gain more detailed insight into impaired HRQoL domains or omission of further steps in the absence of an indication.

Patient-reported assessment of compassion in Spanish: a systematic review.

Aims and objectives: This systematic review aims to: (1) explore which tools have been used in Spanish to measure compassion; (2) know which of these tools could be used to assess compassion in healthcare settings from the perspective of patients; (3) evaluate the quality of these patient-reported measures in Spanish contexts; and (4) determine which of these instruments would be best suited to be used in healthcare settings. Background: Compassion has been recognized as a fundamental dimension of quality healthcare. Methods: Several scientific databases were consulted for relevant records published up to December 16th, 2021. In accordance with PRISMA guidelines, 64 studies were included. Results and conclusions: while existing instruments, validated in Spanish, allow for the measurement of self-compassion or compassion to others, there are no valid and reliable measures currently available in Spanish to measure patient-reported compassion. Relevance to clinical practice: In order to ensure and promote compassion in the health care context, it is essential to have a valid and reliable tool to measure this construct in a patient-informed way, and this is currently not possible in the Spanish-speaking context because of the lack of such an instrument in Spanish.

Let's talk about sex! Why should healthcare professionals address sexual distress in breast cancer patients and survivors?

PURPOSE: Sexual distress impacts the quality of life (QoL) of breast cancer patients but is often overlooked in standard care pathways. This study evaluated the prevalence and factors of sexual distress among Dutch breast cancer patients, compared them to the general population, and explored how sexual distress is discussed in clinical settings from the perspectives of patients and healthcare professionals (HCPs). METHODS: Questionnaires containing the Female Sexual Distress Scale (FSDS) and demographic variables were distributed to women with breast cancer. The effect of breast cancer on sexual distress was assessed with a Mann-Whitney U test. Multivariable linear regression was used to analyze variables associated with FSDS. The Sexuality Attitudes and Beliefs Survey (SABS) was sent to HCPs. RESULTS: Breast cancer patients reported significantly higher sexual distress compared to a Dutch non-breast cancer cohort, respectively 16.38 (SD 11.81) and 23.35 (SD 11.39). Factors associated with higher sexual distress were psychological comorbidities, the body image scale, and being diagnosed >10 years ago. Sexual distress was not discussed as often as patients needed. Barriers to addressing sexual distress were time constraints, HCPs' confidence in their ability to address sexual distress, and uncertainty about who is responsible for initiation. CONCLUSIONS: Breast cancer patients showed significantly higher sexual distress compared to the Dutch population. However, it was not frequently addressed in the consultation room. While some barriers have been identified, this study highlights the importance of further exploring obstacles to integrating discussions about sexual distress into routine care to improve QoL of breast cancer patients.

A Comprehensive Approach to PROMs in Elective Orthopedic Surgery: Comparing Effect Sizes across Patient Subgroups.

Background: There is limited knowledge regarding the comparative patient-reported outcomes (PROMs) and effect sizes (ESs) across orthopedic elective surgery. Methods: All patient data between January 2020 and December 2022 were collected, and treatment outcomes assessed as a PROM difference between baseline and one-year follow-up. The cohort was divided into subgroups (hand, elbow, shoulder, spine, hip, knee, and foot/ankle). The PROM ESs were calculated for each patient separately, and patients with ES > 0.5 were considered responders. Results: In total, 7695 patients were operated on. The mean ES across all patient groups was 1.81 (SD 1.41), and the largest ES was observed in shoulder patients and the smallest in hand patients. Overall, shoulder, hip, and knee patients had a larger ES compared to hand, spine, and foot/ankle patients (p < 0.0001). The proportion of positive responders ranged between 91-94% in the knee, shoulder, and hip, and 69-70% in the hand, spine, and foot/ankle subgroups. Conclusions: The ESs are generally high throughout elective orthopedic surgery. However, based on our institutional observations, shoulder, hip, and knee patients experience larger treatment effects compared to hand, spine, and foot/ankle patients, among whom there are also more non-responders. The expected treatment outcomes should be clearly communicated to patients when considering elective surgery. Because of the study limitations, the results should be approached with some caution.

Development of the Sensory-Motor Dysfunction Questionnaire and Pilot Reliability Testing.

Both chronic and recurrent spinal pain alter sensorimotor integration (SMI), which is demonstrated using complex neurophysiological techniques. Currently, there is no patient-reported outcome measure that documents and/or assesses SMI in populations with spinal problems. The purpose of this study was to develop the Sensory-Motor Dysfunction Questionnaire (SMD-Q) and assess its test-retest reliability and internal consistency in individuals with recurrent spinal pain. The SMD-Q was developed based on the existing literature on motor control disturbances associated with disordered SMI. The initial SMD-Q drafts underwent review by two separate panels of subject matter experts and a focus group with subclinical spine pain. Their suggestions were incorporated into the questionnaire prior to reliability testing. The questionnaire was administered twice at a seven-day interval using QualtricsTM. A total of 20 participants (14 females and 6 males; 20.95 ± 2.46 years of age) completed the study. Quadratic weighted kappa (Kw) was used to assess test-retest reliability and Cronbach's alpha (α) was used to assess internal consistency. Four items had a Kw < 0.40, seven had a 0.40 < Kw < 0.75, and one had a Kw > 0.75 (excellent agreement), with excellent internal consistency (α > 0.90). The pilot SMD-Q appears to reliably measure altered SMI, suggesting that revisions and testing with a larger sample are worth pursuing.

Scalability, test-retest reliability and validity of the Brief INSPIRE-O measure of personal recovery in psychiatric services.

Introduction: Mental health services have transitioned from treating symptoms to emphasizing personal recovery. Despite its importance, integrating personal recovery into clinical practice remains work in progress. This study evaluates the psychometric qualities of the Brief INSPIRE-O, a five-item patient-reported outcome measure assessing personal recovery. Method: The study collected data from 2018 to 2020 at the Mental Health Services, Capital Region of Denmark, using an internet-based system examining 8,192 non-psychotic patients - receiving outpatient treatment. Materials: This study evaluated the Brief INSPIRE-O and used measures of symptomatology (SCL-10), well-being (WHO-5), and social functioning (modified SDS). Results: The study population comprised 76.8% females with a mean age of 32.9 years, and diagnoses included anxiety (28%), depression (34%), and personality disorder (19%). The mean Brief INSPIRE-O score (39.9) was lower than the general population norm (71.1). The Brief INSPIRE-O showed acceptable test-retest reliability (0.75), scalability (0.39), and internal consistency (0.73). Correlations with other mental health criteria were in the expected direction for symptomatology (-0.46), well-being (0.60), and social functioning (-0.43) and remained consistent across diagnoses. Discussion: The Brief INSPIRE-O demonstrated strong psychometric qualities and could be recommended as a measure of personal recovery for use in both research and clinical practice. Its strong theoretical basis and short completion time make it suitable for use for research. Incorporating Brief INSPIRE-O into clinical assessment will further support the process of mental health systems re-orientating towards personal recovery.

Long-term patient-reported outcomes among patients undergoing revascularization vs medical therapy for intermittent claudication.

OBJECTIVE: Society for Vascular Surgery guidelines recommend revascularization for patients with intermittent claudication (IC) if it can improve patient function and quality of life. However, it is still unclear if patients with IC achieve a significant functional benefit from surgery compared with medical management alone. This study examines the relationship between IC treatment modality (operative vs nonoperative optimal medical management) and patient-reported outcomes for physical function (PROMIS-PF) and satisfaction in social roles and activities (PROMIS-SA). METHODS: We identified patients with IC who presented for index evaluation in a vascular surgery clinic at an academic medical center between 2016 and 2021. Patients were stratified based on whether they underwent a revascularization procedure during follow-up vs continued nonoperative management with medication and recommended exercise therapy. We used linear mixed-effect models to assess the relationship between treatment modality and PROMIS-PF, PROMIS-SA, and ankle-brachial index (ABI) over time, clustering among repeat patient observations. Models were adjusted for age, sex, diabetes, Charlson Comorbidity Index, Clinical Frailty Score, tobacco use, and index ABI. RESULTS: A total of 225 patients with IC were identified, of which 40% (n = 89) underwent revascularization procedures (42% bypass; 58% peripheral vascular intervention) and 60% (n = 136) continued nonoperative management. Patients were followed up to 6.9 years, with an average follow-up of 5.2 ± 1.6 years. Patients who underwent revascularization were more likely to be clinically frail (P = .03), have a lower index ABI (0.55 ± 0.24 vs 0.72 ± 0.28; P < .001), and lower baseline PROMIS-PF score (36.72 ± 8.2 vs 40.40 ± 6.73; P = .01). There were no differences in patient demographics or medications between treatment groups. Examining patient-reported outcome trends over time; there were no significant differences in PROMIS-PF between groups, trends over time, or group differences over time after adjusting for covariates (P = .07, P = .13, and P =.08, respectively). However, all patients with IC significantly increased their PROMIS-SA over time (adjusted P = .019), with patients managed nonoperatively more likely to have an improvement in PROMIS-SA over time than those who underwent revascularization (adjusted P = .045). CONCLUSIONS: Patient-reported outcomes associated with functional status and satisfaction in activities are similar for patients with IC for up to 7 years, irrespective of whether they undergo treatment with revascularization or continue nonoperative management. These findings support conservative long-term management for patients with IC.

Reference Values for the German Version of the Quality of Life after Brain Injury in Children and Adolescents (QOLIBRI-KID/ADO) from a General Population Sample.

Assessment of health-related quality of life (HRQoL) after pediatric traumatic brain injury (TBI) has been limited in children and adolescents due to a lack of disease-specific instruments. To fill this gap, the Quality of Life after Traumatic Brain Injury for Children and Adolescents (QOLIBRI-KID/ADO) Questionnaire was developed for the German-speaking population. Reference values from a comparable general population are essential for comprehending the impact of TBI on health and well-being. This study examines the validity of the German QOLIBRI-KID/ADO in a general pediatric population in Germany and provides reference values for use in clinical practice. Overall, 1997 children and adolescents aged 8-17 years from the general population and 300 from the TBI population participated in this study. The questionnaire was tested for reliability and validity. A measurement invariance (MI) approach was used to assess the comparability of the HRQoL construct between both samples. Reference values were determined by percentile-based stratification according to factors that significantly influenced HRQoL in regression analyses. The QOLIBRI-KID/ADO demonstrated strong psychometric properties. The HRQoL construct was measured largely equivalently in both samples, and reference values could be provided. The QOLIBRI-KID/ADO was considered reliable and valid for assessing HRQoL in a general German-speaking pediatric population, allowing for clinically meaningful comparisons between general and TBI populations.

EFAS Score - Validation of Danish Version by the Score Committee of the European Foot and Ankle Society (EFAS).

BACKGROUND: The Score Committee of the European Foot and Ankle Society (EFAS) developed, validated, and published the EFAS Score in 13 languages. Currently, the Danish version completed data acquisition and underwent further validation. METHODS: The data were collected pre-operatively and post-operatively at a minimum follow-up of 3 months and mean follow-up of 6 months. Item reduction, scale exploration, confirmatory analyses and responsiveness were executed using classical test theory and item response theory. RESULTS: The internal consistency was confirmed in the Danish version (Cronbach's Alpha 0.88). The Standard Error of Measurement (SEM) was 0.31 and is similar to other language versions. Between baseline and follow-up, 77.2% of patients showed an improvement on their EFAS score, with adequate responsiveness (effect size 1.05). CONCLUSIONS: The Danish EFAS Score version was successfully validated in patients with a wide variety of foot and ankle pathologies. All score versions are freely available at www.efas.net.

Scoring Abdominal Symptoms in People with Cystic Fibrosis.

(1) Background: The introduction of highly effective CFTR-modulating therapies (HEMT) has changed the course of the disease for many people with Cystic Fibrosis (pwCF). Attention previously focused on life-threatening conditions of the respiratory system has broadened, bringing the involvement of the digestive system into the clinical and scientific focus. This emphasized the need for sensitive tools to capture and quantify changes in abdominal symptoms (AS), ideally applying patient-reported outcome measures (PROMs). (2) Methods: The present review focuses on studies addressing AS assessment deriving from the multi-organic abdominal involvement in pwCF. Among 5224 publications retrieved until Nov. 2022, 88 were eligible, and 39 were finally included. (3) Results: The review reveals that for a long time, especially before HEMT availability, AS in pwCF were assessed by single questions on abdominal complaints or non-validated questionnaires. PROMs focusing on quality of life (QOL) including a few GI-related questions were applied. Likewise, PROMs developed and partially validated for other non-CF GI pathologies, such as chronic inflammatory bowel diseases, irritable bowel syndrome, gastroesophageal reflux, constipation, or pancreatitis, were implemented. (4) Conclusions: Only lately, CF-specific GI-PROMs have been developed and validated following FDA guidelines, showing high sensitivity to changes and capturing marked and statistically significant reductions in the burden of AS achieved with HEMT implementation.

Corrigendum: Psychometric evaluation and reference values for the German Postconcussion Symptom Inventory (PCSI-SR8) in children aged 8-12 years.

[This corrects the article DOI: 10.3389/fneur.2023.1266828.].

Minimal clinically important difference in maxillofacial trauma patients: a prospective cohort study.

The present study estimated the minimal clinically important difference (MCID) for pain on a visual analogue scale - numerical rating scale (VAS-NRS) and mean bite force (MBF) in patients treated for maxillofacial trauma (MFT). This cohort study included 120 MFT patients treated according to AO principles. Preoperative and four-week postoperative pain on the VAS-NRS, and MBF were measured to calculate MCIDs as indicators of functional rehabilitation. The patient's perspective of the treatment was assessed using a four-item anchor question. The MCID was determined by two anchor-based approaches, namely, the change difference (CD) method and receiver operating characteristic (ROC) curve method. According to the CD method, the MCID for pain was 2.4 and the MBF was 147.9 N. Based on the ROC curve, the MCID for pain was 2.5 (sensitivity 91.7%, specificity 47.2%) and MBF was 159.1 N (sensitivity 71.4%, specificity 61.1%). This study demonstrated a high sensitivity (>70%) for MCID, which implies that pain reduction of 2.4-2.5 points on the VAS-NRS and a gain in MBF of 147.9-159.1N are clinically relevant for patients treated for MFT.

A systematic review of the measurement properties of the Pennsylvania Shoulder Score.

BACKGROUND: Pennsylvania Shoulder Score (PSS) is a patient-reported outcome measure (PROM) that is gaining use when examining shoulder-specific outcomes. To date, a systematic review of the PSS measurement properties has not been published. PURPOSE: To systematically locate, appraise, and synthesize the evidence concerning the measurement properties of the PSS. STUDY DESIGN: Systematic literature review. METHODS: A pair of raters conducted a literature search using pre-defined keywords from 5 databases (PubMed, CINAHL, SPORTdiscus, PsychINFO, Scopus) during the week of October 23, 2022. Critical appraisal of study design for psychometric articles was used to assess methodological quality and risk of bias. Measurement property data were synthesized with pooled estimates for the indices of test-retest reliability (ICC), standard error of measurement (SEM), minimal detectable change (MDC), Cronbach α (CA), effect size (ES), and standardized response means (SRM) calculated from applicable studies. RESULTS: A total of 13 articles met the inclusion criteria for this review. Methodological quality was agreed upon between the 2 raters with a weighted kappa of 0.77, and 9/13 of the articles were rated above 70%. Test-retest reliability of the PSS total score ranged between 0.90 and 0.94, and construct validity was high when compared to other shoulder-specific PROMs (0.75 < r > 0.96). Responsiveness of the PSS was large across all studies with few PSS subscales dropping to moderate. Pooled MDC90 of the total PSS was 12.13 points, and the pooled ES of the total PSS was 0.85. CONCLUSIONS: The scope of this review demonstrates positive measurement properties of the PSS as a reliable, valid, and responsive measurement of shoulder-specific PROM. However, there are few studies examining the measurement properties of the PSS and more research is needed to assess cross-cultural appropriateness and factor analysis of the questions contained in the PSS.

Total Knee Arthroplasty in the Post-Traumatic Knee: Revision Risks and Functional Outcomes Compared to Osteoarthritic Knees. A Report of Primary Procedures From the Dutch Arthroplasty Register.

BACKGROUND: Total knee arthroplasty (TKA) for post-traumatic arthritis (PTA) poses higher challenges and increased risks of complications compared to TKA for osteoarthritis (OA). This study aimed to compare implant survivorships, reasons for revision, and patient-reported outcome measures between OA and PTA as indications for TKA. METHODS: We selected all primary TKAs for PTA or OA between 2007 and 2020 from the Dutch Arthroplasty Register (Landelijke Registratie Orthopedische Interventies). The study included 3,897 TKA procedures for PTA (median follow-up 4.6 years; interquartile range: 2.2, 7.3) and 255,259 procedures for OA (median follow-up 4.7 years; interquartile range 2.2, 7.6). A total of 10,480 revision procedures were performed across both groups (238 in PTA knees; 10,242 in OA knees). We analyzed the prevalence of preoperative comorbidities and postoperative complications, as well as the reasons for revision, and calculated the implant survival rates. RESULTS: The survival revision rate in the OA group was significantly lower at both follow-up moments (5- and 10- years). The likelihood for revision was increased in TKA for PTA compared to TKA for OA (hazards ratio: 1.16 [95% confidence interval 1.02 to 1.33], P = .03). The most common reason for a revision was instability and arthrofibrosis in the PTA group compared to patellar pain for the OA group. CONCLUSION: This study demonstrated an increased risk for revision for any reason in TKA for PTA compared to OA. Revision for instability and arthrofibrosis were more prevalent in the PTA group, while revision for patellar pain was less prevalent compared to TKA for OA.

Patient-Reported Outcome Measures for Patients With CKD: The Case for Patient-Reported Outcomes Measurement Information System (PROMIS) Tools.

Chronic kidney disease (CKD), kidney failure, and kidney replacement therapies are associated with high symptom burden and impaired health-related quality of life (HRQOL). Symptoms change with disease progression or transition between treatment modalities and frequently go unreported and unmanaged. Tools that reliably monitor symptoms may improve the management of patients with CKD. Patient-reported outcome measures (PROMs) assess symptom severity; physical, psychological, social, and cognitive functioning; treatment-related side effects; and HRQOL. Systematic use of PROMs can improve patient-provider communication, patient satisfaction, clinical outcomes, and HRQOL. Potential barriers to their use include a lack of engagement, response burden, and limited guidance about PROM collection, score interpretation, and workflow integration. Well-defined, acceptable, and effective clinical response pathways are essential for implementing PROMs. PROMs developed by the Patient-Reported Outcomes Measurement Information System (PROMIS) address some challenges and may be suitable for clinical use among patients with CKD. PROMIS tools assess multiple patient-valued, clinically actionable symptoms and functions. They can be administered as fixed-length, customized short forms or computer adaptive tests, offering precise measurement across a range of symptom severities or function levels, tailored questions to individuals, and reduced question burden. Here we provide an overview of the potential use of PROMs in CKD care, with a focus on PROMIS.

Psychometric evaluation and reference values for the German Postconcussion Symptom Inventory (PCSI-SR8) in children aged 8-12 years.

Background: Post-concussion symptoms (PCS) are a common consequence of pediatric traumatic brain injury (pTBI). They include cognitive, emotional, and physical disturbances. To address the lack of age-adapted instruments assessing PCS after pTBI, this study examines the psychometric properties of the German 17-item post-TBI version of the Postconcussion Symptom Inventory (PCSI-SR8) in children aged 8-12 years. The study also aims to establish reference values based on data from a pediatric general population sample to better estimate the prevalence and clinical relevance of PCS after pTBI in clinical and research settings. Methods: A total of 132 children aged 8-12 years from a post-acute TBI sample and 1,047 from a general population sample were included in the analyses. The questionnaire was translated from English into German and linguistically validated using forward and backward translation and cognitive debriefing to ensure comprehensibility of the developed version. Reliability and validity were examined; descriptive comparisons were made with the results of the English study. Measurement invariance (MI) analyses between TBI and general population samples were conducted prior to establishing reference values. Factors contributing to the total and scale scores of the PCSI-SR8 were identified using regression analyses. Reference values were calculated using percentiles. Results: Most children (TBI: 83%; general population: 79%) rated at least one symptom as "a little" bothersome. The German PCSI-SR8 met the psychometric assumptions in both samples and was comparable to the English version. The four-factor structure comprising physical, emotional, cognitive, and fatigue symptoms could be replicated. The MI assumption was retained. Therefore, reference values could be provided to determine the symptom burden of patients in relation to a comparable general population. Clinical relevance of reported symptoms is indicated by a score of 8, which is one standard deviation above the mean of the general population sample. Conclusion: The German version of the PCSI-SR8 is suitable for assessment of PCS after pTBI. The reference values allow for a more comprehensive evaluation of PCS following pTBI. Future research should focus on validation of the PCSI-SR8 in more acute phases of TBI, psychometric examination of the pre-post version, and child-proxy comparisons.

Construct validity and reliability of the Bilateral Vestibulopathy Questionnaire (BVQ).

Background: The Bilateral Vestibulopathy Questionnaire (BVQ) is a recently developed 54-item Patient Reported Outcome Measure (PROM) that evaluates the clinically important symptoms of bilateral vestibulopathy (BVP) and its impact on daily life. This study aimed to assess the construct validity and reliability of the BVQ in a large BVP cohort. Methods: Patients diagnosed with BVP were asked to complete a set of questionnaires, including the BVQ, the EuroQol-5D-5L, the Health Utilities Index, the Dizziness Handicap Inventory, the Hospital Anxiety and Depression Scale, and the Oscillopsia Severity Questionnaire. The construct validity of the BVQ was evaluated by confirmatory and exploratory factor analyses (CFA and EFA), followed by hypotheses testing and known groups validity. Structural properties were explored for each individual item. Reliability was assessed by testing the internal consistency of the BVQ constructs (Cronbach's alpha) and test-retest reliability [intraclass correlation coefficients (ICCs)]. Results: A total of 148 patients with BVP (50% women, mean age 66 years) completed the set of questionnaires. The CFA did not show a satisfactory model in the original BVQ. However, the EFA showed a four-factor solution with 20 Likert-scale items related to oscillopsia, imbalance, emotion, and cognition. The succeeding CFA provided evidence for construct validity and an acceptable model of fit. Hypothesis testing confirmed that this shortened version validly measures the constructs to be measured. Statistically significant differences in scores between known groups were found, providing further support for good construct validity. The structural properties were acceptable. Cronbach's alpha confirmed good internal consistency for the four constructs, ranging from 0.80 to 0.89. The ICCs of the 20 Likert-scale items and four visual analog scale (VAS) items were interpreted as good (range 0.76-0.93). Conclusion: This study showed evidence of good construct validity of the new shortened version of the BVQ, consisting of four constructs with a total of 20 Likert-scale items and four VAS items. The final 24-item BVQ proved to be a reliable and valid multi-item PROM that captures the clinically important symptoms of BVP and evaluates its impact on daily life. Consequently, the BVQ enables the gathering of high-level evidence of treatment effectiveness in a systematic and quantitative manner.

Reduction in abdominal symptoms (CFAbd-Score), faecal M2-pyruvate-kinase and Calprotectin over one year of treatment with Elexacaftor-Tezacaftor-Ivacaftor in people with CF aged ≥12 years - The RECOVER study.

BACKGROUND: RECOVER is a multicentre post-approval study of Elexacaftor/Tezacaftor/Ivacaftor (ETI) in pwCF in Ireland and the UK. The CFAbd-Score is the first validated CF-specific patient reported outcome measure (PROM) focusing on gastrointestinal symptoms; it comprises 28 items in 5 domains. In a preliminary study, we previously reported reductions in abdominal symptoms (AS) in pwCF after 26 weeks of ETI-therapy using the CFAbd-Score. AIM: to assess changes in AS in a second, large cohort and explore novel GI-biomarkers of gut inflammation and cell-proliferation in pwCF over one year of ETI-therapy. METHODS: Participants were recruited as part of the RECOVER study at 8 sites (Ireland&UK). The CFAbd-Score was administered prior to ETI-initiation, and subsequently at 1,2,6 and 12 months on treatment. Faecal M2-pyruvate kinase (M2-PK) and calprotectin (FC) were quantified in samples collected at baseline, 1 and 6 months. RESULTS: 108 CFAbd-Scores and 73 stool samples were collected at baseline. After 12 months of ETI-therapy, total CFAbd-Scores had significantly declined (15.0±1.4→9.8±1.2pts/p<0.001), and so had all its five domains of "pain" (16.9±2.0pts→9.9±1.8pts/p<0.01), "GERD" (14.4±1.8→9.9±1.6/p<0.05), "disorders of bowel movements" (19.2±1.4→14.1±1.5/p<0.01), "appetite" (7.0±1.1→4.6±1.2/p<0.01) and "impaired-QoL" (13.3±1.9→7.5±1.5/p<0.001). Levels of M2-PK and FC significantly decreased during ETI-therapy. DISCUSSION: In-depth analysis of AS with the CFAbd-Score reveals a statistically significant, clinically relevant and sustained improvement with ETI. We attribute this to high sensitivity of the implemented CF-specific PROM, developed and validated following FDA-guidelines. Furthermore, for the first time during ETI-therapy a significant decline in faecal M2-PK, a marker of inflammation and cell-proliferation, was found, in parallel to FC.

Development and validation of a French-language cross-cultural adaptation of the Pedi-IKDC Questionnaire.

BACKGROUND: The Pedi-IKDC is an English-language, knee-specific, paediatric questionnaire used by orthopaedic surgeons around the world as a valuable patient-reported outcome measure (PROM). The objective of this study was thus to extend the applicability of the Pedi-IKDC to French-speaking Canadian patients, for both clinical practice and research, by developing a French-language cross-cultural adaptation of the original version. HYPOTHESIS: The French adaptation of the Pedi-IKDC is valid and reliable for evaluating French-speaking children with knee conditions. PATIENTS AND METHODS: The Pedi-IKDC was translated to French by a panel of orthopaedic surgeons then back-translated by a professional translator. The original English version and the back-translation were compared to assess their similarity and confirm the faithfulness of the French translation. The validity of the French version was then tested at a major paediatric hospital in French-speaking Canada, in 203 children, including 163 with knee pain and 40 without knee symptoms. Internal consistency, construct validity, and discriminant capacity of the French version were assessed. RESULTS: Internal consistency of the Pedi-IKDC adaptation was excellent (Cronbach's alpha, 0.934 in the knee-pain group). Construct validity was robust, with all nine hypotheses adapted from the original Pedi-IKDC article demonstrating strong (n=7) or moderate (n=2) correlations (p<0.001). The evaluation of discriminant capacity identified no statistically significant score differences according to most of the respondent characteristics (body mass index, age group, type of diagnosis, and type of treatment). However, scores differed significantly between females and males. DISCUSSION: The French-language cross-cultural adaptation of the Pedi-IKDC obtained using a universally recognized method for translating PROMs demonstrated good performance, with psychometric properties similar to those of the original Pedi-IKDC and of its Danish, Italian, and Russian adaptations. LEVEL OF EVIDENCE: II.