Patient-reported knee-related rating scores and scales are widely used in reporting the clinical outcomes of anterior cruciate ligament (ACL) surgery. Understanding the psychometric properties of such measures is vital to recognizing the limitations that such measures may confer. The aim of this study was to review the available evidence as to the psychometric properties of patient-reported outcome measures (PROMs) used in ACL surgery. Eleven studies were identified, the majority being prospective cohort studies. Eight English, ACL-specific patient-reported outcome measures were identified and evaluated: Lysholm score, Tegner Activity Scale (TAS), Cincinnati score, ACL-Quality of Life (QOL) score, International Knee Documentation Committee (IKDC) Subjective Knee Form (SKF), Knee Injury and Osteoarthritis Outcome Score (KOOS)-ACL score, and ACL-Return to Sport Injury (RSI) scale. Only the Lysholm score, ACL-QOL, IKDC SKF, and ACL-RSI were evaluated for internal consistency, having an acceptable Cronbach\'s α (α>0.70). Most of the scoring systems were assessed for test-retest reliability, with four of them (Lysholm score, TAS, Cincinnati score, and IKDC SKF) having acceptable intraclass correlation coefficient (ICC) values (ICC > 0.70). Criterion validity was assessed for most measures with a good correlation with the IKDC. Effect sizes and standardized response means were large for three instruments that measured responsiveness (Lysholm score, TAS, and Cincinnati score) and moderate for one (ACL-QOL). Evidence is stronger and more robust for the Lysholm score, TAS, ACL-QOL, and IKDC SKF. However, there is variation in their psychometric properties as well as the aspect of knee-related health they are assessing. Hence, none can be universally applicable to all patients with ACL injuries. Recognizing these parameters is vital when choosing which instrument to use in reporting the outcomes of ACL injury or ACL surgery studies.

BACKGROUND: Breast cancer is a prevalent cancer characterized by its aggressive nature and potential to cause mortality among women. The rising mortality rates and women\'s inadequate perception of the disease\'s severity in developing countries highlight the importance of screening using conventional methods and reliable scales. Since the validity and reliability of the breast cancer perception scale (BCPS) have not been established in the Iranian context. Therefore, this study aimed to determine the measurement properties of the BCPS in women residing in Tabriz, Iran.
METHODS: The present study comprised a cross-sectional design, encompassing a sample of 372 Iranian women. The participants were selected through a multi-stage cluster random sampling technique conducted over a period spanning from November 2022 to February 2023. The measurement properties of the Iranian version of BCPS were assessed following the guidelines outlined in the COSMIN checklist. This involved conducting various steps, including the translation process, reliability testing (internal consistency, test-retest reliability, and measurement error), and methodological tests for validity (content validity, face validity, construct validity, and hypothesis testing). The study also investigated the factors of responsiveness and interpretability. The presence of floor and ceiling effects was assessed.
RESULTS: The internal consistency of the scale was assessed using Cronbach\'s alpha, yielding a satisfactory value of 0.68. Additionally, McDonald\'s omega (95% CI) was computed, resulting in a value of 0.70 (0.66 to 0.74). Furthermore, the test-retest reliability was evaluated, revealing a high intraclass correlation coefficient (ICC) of 0.97 (95% CI: 0.94 to 0.99). The CVI, CVR, and impact scores of the BCPS were determined to be 0.98, 0.95, and 3.70, respectively, indicating favorable levels of content and face validity. To assess construct validity, an examination of the Exploratory Factor Analysis (EFA) was conducted on a set of 24 items. This analysis revealed the presence of six distinct factors, which collectively accounted for 52% of the cumulative variance. The fit indices of the validity model (CFI = 0.91, NFI = 0.96, RFI = 0.94, TLI = 0.90, χ2/df = 2.03, RMSEA = 0.055 and SRMR = 0.055) were confirmed during the confirmatory factor analysis (CFA). The overall score of BCPS exhibited a ceiling effect of 0.3%. The floor effect observed in the overall score (BCPS) was found to be 0.5%. Concerning the validation of the hypothesis, Spearman\'s correlation coefficient of 0.55 was obtained between the BCPS and the QLICP-BR V2.0. This correlation value signifies a statistically significant association. Furthermore, it is worth noting that the minimum important change (MIC) of 3.92 exhibited a higher value compared to the smallest detectable change (SDC) of 3.70, thus suggesting a satisfactory level of response.
CONCLUSIONS: The obtained findings suggest that the Iranian version of the BCPS demonstrates satisfactory psychometric properties for assessing the perception of breast cancer among Iranian women. Furthermore, it exhibits favorable responsiveness to clinical variations. Consequently, it can serve as a screening instrument for healthcare professionals to comprehend breast cancer and as a reliable tool in research endeavors.

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.

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The Critical Path Institute convened the Support Flexible Approaches to PRO Data Collection project as part of the eCOA: Getting Better Together Initiative which was instigated to identify and address common challenges and drive positive change with eCOA implementation in clinical trials. The project aimed to identify clinical trial stakeholders\' concerns related to electronic PRO (ePRO) implementation and propose areas of improvement via simplification and flexibility. One workstream focused on patient-/site-centric approaches for simplification and surveyed representatives of clinical sites and site monitors for their perspectives. A semi-structured questionnaire was developed and distributed via snowball sampling to site professionals and clinical research associates (CRAs) that had ePRO experience who had been identified via representative groups or sponsor-led site networks. Responses were received from various site roles across a range of global regions; the largest contribution was from the United States. Topics raised included helpdesk capabilities, technical concerns, device types, and user interfaces among others and are discussed further in this paper. The feedback derived from the questionnaire provided the basis for concrete ideas that sponsors should consider incorporating into protocol design for participant visits, technology use, devices, and methods of back-up data collection.

BACKGROUND: Pediatric asthma has been identified by regulators, clinicians, clinical trial sponsors, and caregivers as an area in need of novel fit-for-purpose clinical outcome assessments (COAs) developed in accordance with the U.S. Food and Drug Administration\'s (FDA\'s) regulatory guidance for evaluating clinical benefit in treatment trials. To address this gap, the Patient-Reported Outcome (PRO) Consortium\'s Pediatric Asthma Working Group has continued development of 2 COAs to assess asthma signs and symptoms in pediatric asthma clinical trials to support efficacy endpoints: a PRO measure, the Pediatric Asthma Diary-Child (PAD-C) for children 8-11 years old (y.o.) and an observer-reported outcome measure, the Pediatric Asthma Diary-Observer (PAD-O) for caregivers of children 4-11 y.o. This qualitative research aimed to generate evidence regarding the content validity of the PAD-C and PAD-O.
METHODS: Semi-structured combined concept elicitation and cognitive interviews were conducted with a diverse sample of U.S. participants (15 children 8-11 y.o. and 30 caregivers of children 4-11 y.o.). All children had clinician-diagnosed mild to severe asthma. Interviews explored the experience of pediatric asthma and assessed the understanding and relevance of both measures. Interviews were conducted across 3 iterative rounds to allow for modifications.
RESULTS: Concept elicitation findings demonstrated that the core sign/symptom and impact concepts assessed in the PAD-C (cough, hard to breathe, out of breath, wheezing, chest tightness, and nighttime awakenings/symptoms) and PAD-O (cough, difficulty breathing, short of breath, wheezing, and nighttime awakenings/signs) correspond to those most frequently reported by participants; concept saturation was achieved. All PAD-C and PAD-O instructions and core items were well understood and considered relevant by most participants. Feedback from participants, the Pediatric Asthma Working Group, advisory panel, and FDA supported modifications to the measures, including addition of 1 new item to both measures and removal of 1 caregiver item.
CONCLUSIONS: Findings provide strong support for the content validity of both measures. The cross-sectional measurement properties of both measures and their user experience and feasibility in electronic format will be assessed in a future quantitative pilot study with qualitative exit interviews, intended to support the reliability, construct validity, final content, and, ultimately, FDA qualification of the measures.
Pediatric asthma is one of the most common chronic diseases in children. However, there are problems of underdiagnosis, poor disease management, and undertreatment for many pediatric asthma patients, pressuring healthcare systems worldwide. Evaluating asthma symptoms is an important part of the development of treatments for pediatric asthma. However, there are few clinical outcome assessments (COAs) developed in line with regulatory guidance to directly assess symptom severity and evaluate the benefit of new treatments in children with asthma. In this study, we continued the development of the Pediatric Asthma Diary—Child (PAD-C) and the Pediatric Asthma Diary—Observer (PAD-O), according to regulatory guidance, to assess asthma signs and symptoms in children 4 through 11 years old and address this unmet need. The study aimed to explore the experience of pediatric asthma and assess how well-understood and relevant the measures are. Three rounds of qualitative interviews were conducted with 15 children 8 through 11 years old and 30 caregivers of children 4 through 11 years old with asthma. Results show that both measures are well-understood and assess the relevant and important aspects of pediatric asthma reported by children and caregivers. Findings provide evidence supporting the PAD-C and PAD-O as measures of symptom severity and their future use in pediatric asthma treatment trials. Further research is underway to evaluate their measurement properties and assess the user experience and feasibility of electronic completion, to ultimately support the PAD-C and PAD-O in an ongoing COA qualification process by the United States Food and Drug Administration.

Currently, there are no patient-reported outcome tools specifically validated for use in people living with human immunodeficiency virus (PLHIV) to measure treatment injection acceptance and experience. The Perception of Injection (PIN) questionnaire was modified with consent from the Vaccinees\' Perception of Injection (VAPI), a validated instrument developed by Sanofi Pasteur. The objective of developing the PIN was to provide information on participant experience with injectable therapies, including acceptance of pain, injection-site reactions, and tolerability following injections in PLHIV.
This post hoc analysis used data from participants who received the long-acting intramuscular cabotegravir plus rilpivirine combination treatment every 4 weeks, as part of the ATLAS (NCT02951052) and FLAIR (NCT02938520) studies, to evaluate the psychometric properties of the PIN questionnaire.
These findings support the reliability, validity, and responsiveness to change for the PIN questionnaire in PLHIV.
As a clinical trial endpoint, the PIN questionnaire could provide valuable evidence around the acceptance and experience of injections in PLHIV which could have implications for treatment adherence in this population.
ATLAS (NCT02951052); 1 November, 2016. FLAIR (NCT02938520); 19 October, 2016.

When analyzing patient-reported outcome (PRO) data, sources of differential item functioning (DIF) can be multiple and there may be more than one covariate of interest. Hence, it could be of great interest to disentangle their effects. Yet, in the literature on PRO measures, there are many studies where DIF detection is applied separately and independently for each covariate under examination. With such an approach, the covariates under investigation are not introduced together in the analysis, preventing from simultaneously studying their potential DIF effects on the questionnaire items. One issue, among others, is that it may lead to the detection of false-positive effects when covariates are correlated. To overcome this issue, we developed two new algorithms (namely ROSALI-DIF FORWARD and ROSALI-DIF BACKWARD). Our aim was to obtain an iterative item-by-item DIF detection method based on Rasch family models that enable to adjust group comparisons for DIF in presence of two binary covariates. Both algorithms were evaluated through a simulation study under various conditions aiming to be representative of health research contexts. The performance of the algorithms was assessed using: (i) the rates of false and correct detection of DIF, (ii) the DIF size and form recovery, and (iii) the bias in the latent variable level estimation. We compared the performance of the ROSALI-DIF algorithms to the one of another approach based on likelihood penalization. For both algorithms, the rate of false detection of DIF was close to 5%. The DIF size and form influenced the rates of correct detection of DIF. Rates of correct detection was higher with increasing DIF size. Besides, the algorithm fairly identified homogeneous differences in the item threshold parameters, but had more difficulties identifying non-homogeneous differences. Over all, the ROSALI-DIF algorithms performed better than the penalized likelihood approach. Integrating several covariates during the DIF detection process may allow a better assessment and understanding of DIF. This study provides valuable insights regarding the performance of different approaches that could be undertaken to fulfill this aim.

Dengue is the most prevalent arboviral infection causing an estimated 50-60 million cases of febrile illness globally per year, exacting considerable disease burden. Few instruments exist to assess the patient illness experience, with most based on healthcare provider assessment, lacking standardization in timepoints and symptom assessment. This study aimed to evaluate the content validity of the novel \'Dengue Virus Daily Diary (DENV-DD)\', designed to measure symptom intensity and disease burden within outpatient infant to adult populations.
The Dengue Illness Index Report Card was used as a foundation to create the DENV-DD, consisting of patient- and observer-reported outcome (PRO/ObsRO) instruments. In two South American dengue-endemic communities, qualitative combined concept elicitation and cognitive debriefing interviews were conducted among individuals and caregivers of children with symptomatic laboratory-confirmed dengue. Interviews were conducted across two rounds allowing DENV-DD modifications. A small-scale quantitative assessment of the DENV-DD was also conducted with data from an independent Dengue Human Infection Model (DHIM) to generate early evidence of feasibility of DENV-DD completion, instrument performance and insight into the sign/symptom trajectory over the course of illness.
Forty-eight participants were interviewed (20 adults, 20 older children/adolescents with their caregivers, 8 caregivers of younger children). A wide spectrum of signs/symptoms lasting 3-15 days were reported with fever, headache, body ache/pain, loss of appetite, and body weakness each reported by > 70% participants. DENV-DD instructions, items and response scales were understood, and items were considered relevant across ages. DHIM data supported feasibility of DENV-DD completion.
Findings demonstrate content validity of the DENV-DD (PRO/ObsRO instruments) in dengue-endemic populations. Psychometric and cultural validity studies are ongoing to support use of the DENV-DD in clinical studies.
Dengue is the most common viral infection transmitted to humans by mosquitos, and affects an estimated 50–60 million individuals globally per year. However, there are few resources for understanding and capturing the patient experience of dengue throughout illness. Most research studies are based on healthcare provider assessment, which lack consistency in terms of assessment time points and the signs/symptoms assessed. The ‘Dengue Illness Index Report Card (DII-RC)’ was used as a foundation to create the new ‘Dengue Virus Daily Diary (DENV-DD)’ to better capture the patient experience of symptom intensity and dengue disease burden for the duration of illness. Forty-eight individuals and caregivers of younger children from Peru and Ecuador who recently had symptomatic dengue were interviewed to understand the patient experience over the time of illness and to test whether the DENV-DD is understood by patients and caregivers and includes all relevant and important signs/symptoms and health-related quality of life impacts. Nine individuals with active dengue infection also completed the DENV-DD daily for 28-days as part of a clinical study. We found that > 70% of patients experienced fever, headache, body ache/pain, loss of appetite and body weakness. The DENV-DD instructions, questions and response option(s) were well understood, feasible to complete and the concepts assessed by the DENV-DD were relevant to the dengue experience. Our study adds to the understanding of the dengue illness experience and supports the DENV-DD for use in future dengue studies as an assessment of signs/symptoms throughout the duration of illness.

For many years, outcomes such as mortality and morbidity were the standard for evaluating oncological treatment effectiveness. With the introduction of patient-reported outcome measures (PROMs), the focus shifted from a mere extension of a patient\'s life or release from disease to the improvement of a multilayered concept of health, decisively affecting life satisfaction. In this study, we deal with the topic of PROMs in liver and gastrointestinal randomized controlled trials.
The final database included 43 papers reporting results of randomized controlled trials (RCTs) for liver or gastrointestinal cancer interventions where one of the primary or secondary outcomes was a health-related quality of life measure. The most often used PROM was the European Organization for Research and Treatment of Cancer Quality of Life questionnaire (EORTC QLQ-C30) for both liver cancer and gastrointestinal cancer (in 62% of gastrointestinal cancer studies and 57% of liver cancer studies). For the gastrointestinal cancer group, the QLQ-STO22, a cancer-specific extension of the QLQ-C30, was the second most commonly used PROM. In liver cancer, the generic PROM Short Form 36 and the EORTC QLQ-HCC18, a cancer-specific extension of the QLQ-C30, were the second most commonly used PROMs.
We found that RCTs often do not include comprehensive quality-of-life measures. When quality of life is part of an RCT, it is often only a secondary outcome. For a holistic view of the patient, a stronger integration and weighting of patient-reported outcomes in RCTs would be desirable.