Specific Patient-reported Outcome Measures Research Articles

BN SO58 - Patient Reported Outcome Measures in Hiatal Surgery: A Feasibility Study

Abstract Background Hiatal surgery is predominantly a quality-of-life procedure rather than a lifesaving intervention. Whilst procedural complications are well understood, they do not necessarily reflect the patient experience. Traditional indicators of patient care are insufficient. Patient Reported Outcome Measures (PROM) are robust indicators of care quality (1) and are being used across surgical specialities to benchmark success and identify areas for improvement (2). We present a three-year review of our PROM data to demonstrate feasibility of data collection. Method Patient reported outcome measure data were collected from the National Hiatal Surgery Registry (3) for our benign centre over a three-year period (November 2021-January 2024). Consent was obtained from all patients for research purposes. Surgical procedures included: anti-reflux surgery, hiatus hernia repair and cardiomyotomy. All patients were asked to complete pre-operative quality of life surveys. Patients are automatically contacted through the registry at 6months, 1 year and then annually for five years and prompted to complete quality of life surveys. Ekhardt score is used in achalasia. GORD-QoL score was used for anti-reflux procedures, with the Hernia-QoL score for hiatal hernia. Results Data were collected for 20 patients. Mean BMI was 28.2. All completed preoperative surveys. At six months there was attrition of one patient, who has since completed 1-year and 2-year data. At the time of writing, six patients have completed 6month data, 5 patients have completed 1-year data and 3 patients have completed 2-year data. Mean Ekhardt score was 9.25 preoperatively; at six months mean score was 0. Five patients underwent LINX, eleven underwent fundoplication; one Dor, ten Toupet. Mean pre-operative quality of life score was 31.1; at six months mean score was 15.5. At one year this was 4. Conclusion The National Hiatal Surgery Registry facilitates easy acquisition of surgeon specific PROM data over a short- and long-term period. Patient attrition is low, demonstrating acceptability. Our data demonstrate significantly improved patient reported quality of life postoperatively for all hiatal surgery.

Readability of Patient-Reported Outcome Measures Used in Voice Disorders

ObjectivesThis study aims to evaluate Turkish patient-reported outcome measures (PROM) used in voice disorders using different readability formulas and to examine the relationship between text-based features and readability values. Study DesignA search was conducted to identify published Turkish PRO questionnaires related to dysphonia that are completed by patients. MethodsSearch was conducted to identify published PROM related to dysphonia that are completed by patients in a self-administered format. Reading grade levels were analyzed for voice-related PROM using the Ateşman readability and the Bezirci-Yılmaz new readability formulas as computed by a readability calculations software package. Text-based quantitative features are obtained. Descriptive statistics were also computed across the questionnaires. ResultsA total of 14 PROMs were found. In the analysis, the mean of the Ateşman readability values of the PROM was calculated as 70.66 (±5.55). This value is defined as 'easy', according to the Ateşman readability measures. The average Bezirci-Yılmaz new readability value is 6,03 (±1,07). This value corresponds to the 6th grade reading level in the Turkish education system. ConclusionsResults show that more than half of the PROMs in the Turkish language have the recommended reading level. PROMs allow healthcare professionals to objectively understand the patient's symptoms, both qualitatively and quantitatively. Therefore, it is critical that patients accurately understand and respond to these outcome measures. This is why developers should consider reading as a testable structure. Speech and language therapists should consider the reading level required to understand a specific PROMs in order to obtain the most useful information for treatment planning and outcome assessments.

Reducing research participant disparities through use of a co-developed video intervention: A cohort nested pre-post study

Background Evidence on effective approaches for the recruitment of diverse populations remains limited. This study aimed to assess whether a co-developed study information video would improve participant ethnic diversity within a paediatric study cohort. Methods Pre-post study nested within the Uveitis in childhood prospective national inception cohort study, UNICORNS. Intervention of interest: study information and recruitment video with format and content co-developed with a patient expert group. Outcome of interest: the proportion of families from ethnic minority groups, and of families with primary caregivers without a higher educational degree who were recruited to the study, and the proportion of families who engaged in study activities (returned patient reported outcome metrics following recruitment) pre and post intervention implementation. Results During the study period, 191 children were recruited to UNICORNS (110 pre-intervention era, 81 post-intervention era). A larger proportion of families recruited in the post-intervention era were of an ethnic minority background (36% versus 20%, Fisher’s exact test p=0.02). A larger proportion of families returned self-descriptions of their sociodemographic background (100% versus 87%, Fisher’s exact test p=0.0003) and a larger proportion returned study specific patient reported outcome measures (70% versus 55%, Fisher’s exact test p=0.003). Conclusion Co-created digital research information resources can be an effective tool for ensuring better representation in research studies and may also support participant engagement overall. Our reported methods and findings should support other researchers in developing patient-centred and patient co-developed strategies and tools for the purposes of equitable access to medical research.

MORe PREcISE: Longitudinal patient reported outcome measures in stroke at 3 and 6 months.

Background and PurposePost-stroke morbidity is common, but little is known about the burden on patients’ lives from their own perspective. Understanding morbidity from the point of view of the patient may support targeted intervention in post-stroke recovery. This study used a stroke specific Patient Reported Outcome Measure (PROM) containing Mental health (MH) and Physical Health (PH) domains and 5 stroke specific questions. We aimed to consider trends over a 6-month period and further assess the association between the MH and PH measures and common clinical measures. MethodsA multicenter prospective cohort study was conducted at 19 hospital sites across England and Wales. Patients were enrolled from August 2018 to September 2019. Clinical measures and PROMs were assessed at three timepoints: acutely following the index stroke, at 3 and 6-months post-stroke. Clinical measures and PROMs were assessed in each of these points. ResultsPhysical health PROM domains show significant gradual improvement across the study period (χ2 42.6312, p<0.0001), whereas cognitive function domains (χ2 3.7849, p<0.875) did not echo this trend. All clinical measures (GAD-7, PHQ9, MoCA, MRS) were associated with poorer PROM MH outcomes, (aMD -4.4, CI -0.59, -0.29, p≤0.001, aMD -0.45, CI -0.59, -0.32, p=<0.001, aMD 0.75, CI 0.56, 0.95, aMD -1.91, CI -2.41, -1.47, p≤0.001). Clinical measures of disability, as per the MRS, are associated with poor PROM PH scores (aMD -0.57, 95% CI -0.94, -0.20, p=0.003). ConclusionsThis research indicates there is unmet cognitive burden in stroke survivors. PROMs may be able to measure unmet more discretely than common clinical tools that are used post-stroke. Further research and guidance on how to integrate PROMs into current clinical frameworks is essential.

Functional patient-reported outcome measures after hip or knee arthroplasty: A systematic review and content analysis using the International Classification of Functioning, Disability and Health (ICF).

This systematic review aimed to identify the existing patient-reported outcome measures (PROMs) used in hip or knee arthroplasty for adults with osteoarthritis and assess their content validity using the modified International Classification of Functioning, Disability and Health (ICF) core set for osteoarthritis (OA). Four databases were systematically searched to identify disease or joint-specific PROMs evaluating function after hip or knee arthroplasty. Two reviewers independently evaluated the content of PROMs based on established ICF linking rules. From 449 studies included in this review, 50 PROMs were identified. The mobility chapter of activities and participation was the most common component, followed by sensory function and pain chapter of body function and structure. The most frequent ICF activity and participation categories were d451:going up and down stairs, d4701:using private motorized transportation, d4104:standing, and d4154:maintaining a standing position. However, 11 ICF categories of the modified OA coreset were not captured in any PROMs. This ICF-based content analysis of PROMs revealed that included activity and participation categories vary widely, with little overlap between PROMs. The Knee injury and Osteoarthritis Outcome Score and Hip disability and Osteoarthritis Outcome Score had the most coverage for activity and participation (36.7%). Even though our search identified 50 specific PROMs, there remain gaps in content related to activity and participation coverage. By providing a content analysis of the PROMs used after hip or knee arthroplasty, this study may help clinicians select PROMs based on covered categories and relevant clinical objectives.

Responsiveness and minimal important change of specific and generic patient-reported outcome measures for back patients: the Norwegian Neck and Back Register

PurposeTo evaluate responsiveness and minimal important change (MIC) of Oswestry Disability Index (ODI), pain during activity on a numeric rating scale (NRSa) and health related quality of life (EQ-5D) based on data from the Norwegian neck and back registry (NNRR).MethodsA total of 1617 patients who responded to NNRR follow-up after both 6 and 12 months were included in this study. Responsiveness was calculated using standardized response mean and area under the receiver operating characteristic (ROC) curve. We calculated MIC with both an anchor-based and distribution-based method.ResultsThe condition specific ODI had best responsiveness, the more generic NRSa and EQ-5D had lower responsiveness. We found that the MIC for ODI varied from 3.0 to 9.5, from 0.4 to 2.5 for NRSa while the EQ5D varied from 0.05 to 0.12 depending on the method for calculation.ConclusionIn a register based back pain population, the condition specific ODI was more responsive to change than the more generic tools NRSa and EQ5D. The variations in responsiveness and MIC estimates also indicate that they should be regarded as indicative, rather than fixed estimates.

THE IMPACT OF HALLUX VALGUS ON FUNCTION AND QUALITY OF LIFE IN FEMALES

BackgroundThe effect of hallux valgus (HV) on health-related quality of life (HRQOL) and the relationship between radiographic severity of deformity and patient reported outcome measures (PROMs) is poorly understood. The aim of this study was to compare the HRQOL of female patients with HV to the UK population. The secondary aim was to assess the correlation between PROMs, including HRQOL, with radiographic severity of deformity.MethodsWeight bearing radiographic data (hallux valgus (HVA) angle; intermetatarsal (IM) angle) were measured in consecutive female patients presenting with HV. Each patient prospectively completed the Euroqol EQ-5D-5L questionnaire (EQ-5D), Visual Analogue Scale for Pain (VAS-Pain) and Manchester Oxford Foot Questionnaire (MOXFQ). Data were stratified into age ranges and compared with an EQ-5D United Kingdom general population reference dataset. Pearson R correlation values were calculated for the PROMs and radiographic deformity.ResultsBetween July 2015 and March 2020, 425 consecutive female patients presented with HV for consideration for surgery. EQ-5D-5L data were prospectively collected for 396 of these patients (93.2%). Females less than 65 years with HV had a statistically significantly worse quality of life compared with females of the same age group in the general population. Above the age of 65, there was no statistically significant difference in EQ-5D-5L Index score between the two groups. There was no correlation between radiographic HV deformity and HRQOL measures or MOXFQ scores.ConclusionFemale patients presenting with HV deformity have a significantly reduced quality of life compared with the UK general population. The radiographic severity of deformity did not correlate with HRQOL measures or foot and ankle specific PROMs. Foot and ankle specific clinical PROMs moderately correlate with HRQOL and may be a better marker of the negative effect of symptomatic hallux valgus deformity on quality of life.

#1030 Addressing the unmet need of patient reported outcomes in ANCA associated vasculitis, in routine clinical practice

Abstract Background and Aims ANCA associated vasculitis (AAV) is associated with increased morbidity. Recent years has seen improvement in clinical outcomes yet little work has been done to better understand the patient's role within this condition. Patient reported outcome measures (PROMs) are increasingly standard in clinical research outcomes, as well as an overall indicator for good quality patient care. With the exception of Robson et al AAV-PRO [1], little research has been done to develop validated disease specific tools for patients living with AAV. We aim to develop a disease specific tool that can be used in clinical practice to aid patients and clinicians in understanding and track changes in health-related quality of life (HRQoL) over time. Method AAV patients from a single centre were invited to take part in focus groups conducted using a semi-structured topic guide. Participants were asked to discuss topics relating to four broad areas; 1) the effects of AAV on everyday life and living, 2) the effects of treatment on everyday life and living, 3) the role of patients in health management and the patient experience 4) a review of existing generic and disease specific PROM tools (SF-36 and AAV-PRO). Audio transcript of each focus group was transcribed verbatim and data was analysed using a combination of inductive and deductive framework analysis [2]. Results Twenty-eight patients with AAV were invited to participate, 19 attended one of three, 2.5 hour focus groups, 2 patients failed to attend, 4 declined and 3 were unavailable. The mean age of participants was 66.6 +9.4 years and there was a near equal male to female split (10:9). The median time from diagnosis as 7.4 years. Three participants had active or newly diagnosed AAV with BVAS scores of &amp;gt;10. Five participants had previously required dialysis with 1 receiving a renal transplant. All participants received glucocorticoid and immunosuppressive therapy (cyclophosphamide and or Rituximab), 15 (78.9%) were still receiving immunotherapy. Full analysis of the qualitative data is being undertaken. Preliminary findings outlined what was most important to participants when considering HRQoL. Areas acknowledged included; self-identity, social roles e.g. fulfilling caring responsibilities, fears of relapse and disease and treatment burden. Fifty-six deductive and inductive codes were identified which have been further categorised. Participants felt existing tools lacked consideration of some key issues. Scoring systems were considered to be negativity worded e.g. mild, moderate, severe, with the term ‘none’ being the only positive option. Participants reported that existing tools overlooked positive factors which impact HRQoL, such as symptom validation, close clinician monitoring and improvement with treatment. Limitations were noted when trying to address transient or inconsistent symptoms and difficulty separating vasculitis from other health conditions when answering questions. Participants expressed a preference for using a PROM as part of their clinical care and felt that completing it prior to clinic would be most helpful and facilitate discussions with healthcare professionals. The use of an electronic-PROM was widely supported and would support clinicians and patients to monitor changes over time. Conclusion The AAV-PRO represents a significant advancement over generic tools such as the SF-36, however, existing PROMs are not without limitations and are primarily used in research practices. Our study revealed that patients desire active participation in HRQoL discussions and further work to incorporate this into the clinic setting is needed. We are continuing to develop a PROM tool that can be used to assess patients HRQoL in relation to their AAV in the outpatient setting. We believe this will allow a deeper understanding of the disease burden and could support a comprehensive, patient-centred approach to decision-making, offering new insights and identifying the need for supportive care. On a broader scale, these insights can be utilized to compare treatments, shed light on inequalities and facilitate enhancements in services.

How to Raise the Bar in the Capture of Patient-Reported Outcome Measures in Total Joint Arthroplasty: Results from Active and Passive Follow-up Measures.

With the upcoming U.S. Centers for Medicare & Medicaid Services 2027 policy for mandatory reporting of patient-reported outcome measures (PROMs) for total hip or knee arthroplasty (THA or TKA), it is important to evaluate the resources required to achieve adequate PROM collection and reporting at a clinically relevant rate of follow-up. This study aimed to (1) determine follow-up rates for 1-year PROMs when the follow-up was conducted with active methods (attempted contact by staff) and passive (automated) methods, and (2) evaluate factors associated with higher odds of requiring active follow-up or being lost to follow-up following THA or TKA. A prospective cohort of patients undergoing primary elective THA (n = 7,436) or TKA (n = 10,119) between January 2016 and December 2020 at a single institution were included. The primary outcome was the response rate achieved with active and passive follow-up methods at our institution. Patient characteristics, health-care utilization parameters, PROM values, and patient satisfaction were compared between follow-up methods. Passive and active measures were successful for 38% (2,859) and 40% (3,004) of the THA cohort, respectively, while 21% (1,573) were lost to follow-up. Similarly, passive and active measures were successful for 40% (4,001) and 41% (4,161) of the TKA cohort, respectively, while 20% (2,037) were lost to follow-up. Younger age, male sex, Black or another non-White race, fewer years of education, smoking, Medicare or Medicaid insurance, and specific baseline PROM phenotypes (i.e., with scores in the lower half for pain, function, and/or mental health) were associated with loss to follow-up. Older age, male sex, Black race, and a residence with a higher Area Deprivation Index were associated with requiring active follow-up. One of 5 patients were lost to follow-up despite active and passive measures following THA or TKA. These patients were more likely to be younger, be male, be of Black or another non-White race, have fewer years of education, be a smoker, have Medicaid insurance, and have specific baseline PROM phenotypes. Innovative strategies aimed at targeting individuals with these baseline characteristics may help raise the bar and increase follow-up while mitigating costs after total joint arthroplasty. Prognostic Level II . See Instructions for Authors for a complete description of levels of evidence.

Structural validation of the Manchester-Oxford Foot Questionnaire for use in foot and ankle surgery.

The Manchester-Oxford Foot Questionnaire (MOxFQ) is an anatomically specific patient-reported outcome measure (PROM) currently used to assess a wide variety of foot and ankle pathology. It consists of 16 items across three subscales measuring distinct but related traits: walking/standing ability, pain, and social interaction. It is the most used foot and ankle PROM in the UK. Initial MOxFQ validation involved analysis of 100 individuals undergoing hallux valgus surgery. This project aimed to establish whether an individual's response to the MOxFQ varies with anatomical region of disease (measurement invariance), and to explore structural validity of the factor structure (subscale items) of the MOxFQ. This was a single-centre, prospective cohort study involving 6,637 patients (mean age 52 years (SD 17.79)) presenting with a wide range of foot and ankle pathologies between January 2013 and December 2021. To assess whether the MOxFQ responses vary by anatomical region of foot and ankle disease, we performed multigroup confirmatory factor analysis. To assess the structural validity of the subscale items, exploratory and confirmatory factor analyses were performed. Measurement invariance by pathology was confirmed, suggesting the same model can be used across all foot and ankle anatomical regions. Exploratory factor analysis demonstrated a two- to three-factor model, and suggested that item 13 (inability to carry out work/everyday activities) and item 14 (inability to undertake social/recreational activities) loaded more positively onto the "walking/standing" subscale than their original "social interaction" subscale. This large cohort study supports the current widespread use of the MOxFQ across a broad range of foot and ankle pathologies. Our analyses found indications that could support alterations to the original factor structure (items 13 and 14 might be moved from the "social interaction" to the "walking/standing" subscale). However, this requires further work to confirm.

Patient Selection in Randomized Controlled Trials of Total Knee Arthroplasty: A Systematic Review Assessing Inclusion Criteria.

Patients undergo total knee arthroplasty (TKA) at widely disparate stages of osteoarthritis, raising the possibility of high heterogeneity among patients enrolled in TKA research studies. Obscuration of treatment effectiveness and other problems that may stem from cohort heterogeneity can be controlled in clinical studies by rigorously defining target patients. The purpose of this review was to determine the extent to which randomized controlled trials (RCTs) on TKA use osteoarthritis severity, as defined by radiographic grade or patient-reported outcome measures (PROMs), in their inclusion criteria, and to investigate potential impact on outcome. A search of PubMed, Embase, Scopus, Web of Science, and Cochrane databases used a combination of terms involving TKA, PROMs, and radiographic scoring. A total of 1,227 studies were independently reviewed by 2 screeners for the above terms. RCTs with ≥100 patients (236) and with <100 patients (325) undergoing TKA were analyzed with regard to the specific inclusion criteria and recruitment process. Among the identified RCTs with ≥100 patients, 18 (<8%, with a total of 2,952 randomized patients) used specific radiographic scoring or PROM thresholds in their inclusion criteria. Eleven of the 18 studies used specific radiographic scoring, such as the Kellgren-Lawrence or Ahlbäck classifications. Three studies used preoperative PROM thresholds: Knee Society Knee Score of <60, Knee Society Function Score of <60, Oxford Knee Score of <20, and Hospital for Special Surgery Score of <60. Among studies with <100 patients, 48 (<15%) used specific inclusion criteria. The vast majority of RCTs (>85%) did not enroll patients based on disease severity, as measured by PROM score thresholds or radiographic classifications, in their inclusion criteria. The lack of consistent inclusion criteria likely results in heterogeneous cohorts, potentially undermining the validity of RCTs on TKA. Prognostic Level I . See Instructions for Authors for a complete description of levels of evidence.

The Impact of Blood Morphological Parameters on Treatment Outcomes in Tennis Elbow Patients Receiving Platelet-Rich Plasma (PRP) Therapy: A Prospective Study.

Platelet-rich plasma (PRP) therapy holds substantial promise for the treatment of tennis elbow, a complex and challenging musculoskeletal condition. The aim of the study was to assess whether there are correlations between the levels of individual morphotic elements determined in whole blood and the outcomes of tennis elbow treatment with PRP injection, as measured using patient-reported outcome measures (PROMs) such as the Visual Analog Scale (VAS), Quick Disabilities of the Arm, Shoulder, and Hand (QDASH), and Patient-Rated Tennis Elbow Evaluation (PRTEE). A prospective analysis was conducted on 107 patients (132 elbows) undergoing lateral epicondylitis treatment with (PRP) injections. Patients completed VAS, PRTEE, and QDASH questionnaires on the day of PRP administration and at established checkpoints (2, 4, 8, 12, 24, 52, and 104 weeks). Minimal clinically important difference (MCID) was employed to assess the treatment effects. Then, correlations were measured within each PROM, and the impact of the concentration of individual blood parameters on the MCID outcomes was assessed. Analyzing the relationships between the MCID+ and MCID- groups, significant correlations for the VAS and QDASH scales were observed. The level of individual morphotic elements in the blood may have influenced the treatment outcome, as measured using specific patient-reported outcome measures (PROMs). Regarding the VAS scale, factors favoring a positive treatment outcome included higher values of eosinophils (EOS) and basophils (BASO). For the QDASH scale, these factors were a lower value of mean corpuscular volume (MCV) and a higher mean corpuscular hemoglobin (MCH). The levels of certain blood parameters, such as EOS and BASO, in the current study influenced the classification of patients into MCID+ or MCID- groups, based on the VAS and QDASH scales.

Comparison of clinical outcomes between total hip replacement and total knee replacement.

Total hip replacements (THR) and total knee replacements (TKR) are effective treatments for severe osteoarthritis (OA). Some studies suggest clinical outcomes following THR are superior to TKR, the reason for which remains unknown. This study compares clinical outcomes between THR and TKR. To compare the clinic outcomes of THR anad TKR using a comprehensive range of patient reported outcome measures (PROMs). A prospective longitudinal observational study of patients with OA undergoing THR and TKR were evaluated using a comprehensive range of generic and joint specific PROMs pre- and post-operatively. A total of 131 patients were included in the study which comprised the THR group (68 patients) and the TKR group (63 patients). Both groups demonstrated significant post-operative improvements in all PROM scores (P < 0.001). There were no significant differences in post-operative PROM scores between the two groups: Hip and Knee Osteoarthritis Outcome scores (P = 0.140), Western Ontario and McMaster Universities Osteoarthritis Index pain (P = 0.297) stiffness (P = 0.309) and function (P = 0.945), Oxford Hip and Knee Score (P = 0.076), EuroQol-5D index (P = 0.386) and Short-Form 12-item survey physical component score (P = 0.106). Subgroup analyses showed no significant difference (P > 0.05) between cruciate retaining and posterior stabilised prostheses in the TKR group and no significant difference (P > 0.05) between cemented and uncemented fixation in the THR group. Obese patients had poorer outcomes following TKR but did not significantly influence the outcome following THR. Contrary to some literature, THR and TKR are equally efficacious in alleviating the pain and disability of OA when assessed using a comprehensive range of PROMs. The varying knee prosthesis types and hip fixation techniques did not significantly influence clinical outcome. Obesity had a greater influence on the outcome following TKR than that of THR.

Clinical Utility Assessment of a Nursing Checklist Identifying Complex Care Needs Due to Inequities Among Ambulatory Patients With Cancer: Protocol for a Mixed Methods Study.

Disparities in cancer incidence, complex care needs, and poor health outcomes are largely driven by structural inequities stemming from social determinants of health. To date, no evidence-based clinical tool has been developed to identify newly diagnosed patients at risk of poorer outcomes. Specialist cancer nurses are well-positioned to ameliorate inequity of opportunity for optimal care, treatment, and outcomes through timely screening, assessment, and intervention. We designed a nursing complexity checklist (the "Checklist") to support these activities, with the ultimate goal of improving equitable experiences and outcomes of care. This study aims to generate evidence regarding the clinical utility of the Checklist. The primary objectives of this study are to provide qualitative evidence regarding key aspects of the Checklist's clinical utility (appropriateness, acceptability, and practicability), informed by Smart's multidimensional model of clinical utility. Secondary objectives explore the predictive value of the Checklist and concordance between specific checklist items and patient-reported outcome measures. This prospective mixed methods case series study will recruit up to 60 newly diagnosed patients with cancer and 10 specialist nurses from a specialist cancer center. Nurses will complete the Checklist with patient participants. Within 2 weeks of Checklist completion, patients will complete 5 patient-reported outcome measures with established psychometric properties that correspond to specific checklist items and an individual semistructured interview to explore Checklist clinical utility. Interviews with nurses will occur 12 and 24 weeks after they first complete a checklist, exploring perceptions of the Checklist's clinical utility including barriers and facilitators to implementation. Data describing planned and unplanned patient service use will be collected from patient follow-up interviews at 12 weeks and the electronic medical record at 24 weeks after Checklist completion. Descriptive statistics will summarize operational, checklist, and electronic medical record data. The predictive value of the Checklist and the relationship between specific checklist items and relevant patient-reported outcome measures will be examined using descriptive statistics, contingency tables, measures of association, and plots as appropriate. Qualitative data will be analyzed using a content analysis approach. This study was approved by the institution's ethics committee. The enrollment period commenced in May 2022 and ended in November 2022. In total, 37 patients with cancer and 7 specialist cancer nurses were recruited at this time. Data collection is scheduled for completion at the end of May 2023. This study will evaluate key clinical utility dimensions of a nursing complexity checklist. It will also provide preliminary evidence on its predictive value and information to support its seamless implementation into everyday practice including, but not limited to, possible revisions to the Checklist, instructions, and training for relevant personnel. Future implementation of this Checklist may improve equity of opportunity of access to care for patients with cancer. DERR1-10.2196/48432.

Responsiveness of specific and generic patient-reported outcome measures in patients with plantar fasciopathy

Purpose To evaluate and compare responsiveness characteristics for the Foot Function Index revised short form (FFI-RS), RAND-12 Health Status Inventory (RAND-12), and Numeric Rating Scale (NRS), in patients with plantar fasciopathy receiving non-surgical treatment. Materials and methods This study was conducted on a sub-group of patients from an ongoing randomised controlled trial. One-hundred fifteen patients were included. The patient-reported outcome measures (PROMs) were applied at baseline and after 6 months. Responsiveness was calculated using standardised response mean and area under the receiver operating characteristic (ROC) curve. ROC curves were used to compute the minimal important change (MIC) for the outcome measures. Results The region specific FFI-RS had best responsiveness and the NRS at rest had lowest responsiveness. Conclusion FFI-RS were marginally more responsive than the other PROMs. Responsiveness and MIC estimates should be regarded as indicative rather than fixed estimates.

Structural Validation of the Manchester-Oxford Foot Questionnaire (MOxFQ) for use in Foot and Ankle Surgery

Category: Other Introduction/Purpose: The Manchester-Oxford Foot Questionnaire (MOxFQ) is a condition specific patient reported outcome measure (PROM) for foot and ankle surgery. It consists of 16 items across three subscales measuring distinct, but related traits: walking/standing ability, pain, and social interaction. Although it is the most used foot and ankle PROM in the UK, initial MOxFQ validation involved analysis of only 100 individuals undergoing hallux valgus surgery. This project aimed to establish whether an individual’s response to the MOxFQ varies with anatomical region of disease (measurement invariance), and to explore structural validity of the factor structure (subscale items) of the MOxFQ. Methods: This was a single-centre, prospective cohort study involving 6640 patients (mean age 52, range 10-90 years) presenting with a wide range of foot and ankle pathologies between 2013 and 2021. Firstly, to assess whether the MOxFQ responses vary by anatomical region of foot and ankle disease, we performed multi-group confirmatory factor analysis. Secondly, to assess the structural validity of the subscale items, exploratory and confirmatory factor analyses were performed. Results: Measurement invariance by pathology was confirmed suggesting the same model can be used across all foot and ankle anatomical regions. Exploratory factor analysis demonstrated a 2-3 factor model, and suggested that item 13 (inability to carry out my work/everyday activities) and item 14 (inability to undertake social/recreational activities) loaded more positively onto the walking/standing subscale than their original social interaction subscale. Conclusion: This large-cohort study supports the current widespread use of the MOxFQ across a broad range of foot and ankle pathologies. Items 13 and 14 might be better moved from the “social interaction” to the “walking/standing” subscale and this may have future implications for deriving/analysing subscale scores.

Patient-reported outcome measures for transthyretin cardiac amyloidosis: the ITALY study

Background Transthyretin cardiac amyloidosis (ATTR-CA) has a deep impact on the quality of life (QoL), yet no specific patient-reported outcome measures (PROMs) for ATTR-CA exist. Methods The ITALY study involved 5 Italian referral centres (Pisa, Pavia, Ferrara, Florence, Messina) enrolling consecutive outpatients with ATTR-CA. Results Two 30-item questionnaires were created for wild-type (wt) and variant (v) ATTR-CA. Scores ranged from 100 (best condition) to 0 (worst condition). Out of 140 patients enrolled (77% with ATTRwt-CA), 115 repeated the re-evaluation at 6 months. At baseline, only 30% of patients needed help to fill out the questionnaires. Among baseline variables, all KCCQ and SF-36 domains were univariate predictors of ITALY scores in ATTRwt-CA patients, with the KCCQ Symptom Summary score (beta coefficient 0.759), Social Limitations (0.781), and Overall summary score (0.786) being the strongest predictors. The SF-36 Emotional well-being score (0.608), the KCCQ Overall summary score (0.656), and the SF-36 Energy/fatigue score (0.669) were the strongest univariate predictors of ITALY scores in ATTRv-CA. Similar results were found at 6 months. Conclusions The ITALY questionnaires are the first specific PROMs for ATTRwt- and ATTRv-CA. Questionnaire completion is feasible. ITALY scores display close relationships with non-ATTR-specific measures of QoL.

Values derived from patient reported outcomes in spine surgery: a systematic review of the minimal clinically important difference, substantial clinical benefit, and patient acceptable symptom state.

While patient reported outcome measures (PROMs) define value in spine surgery, several values such as minimal clinically important difference (MCID), substantial clinical benefit (SCB), and patient acceptable symptom state (PASS) help guide the interpretation of PROMs and identify thresholds of clinical significance. Significant variation exists in reported values and their calculation, so the primary objective of this study was to systematically review the spine surgery literature for metrics of clinical significance derived from PROMs. We conducted a query of PubMed/MEDLINE and Scopus databases from inception to January 1, 2023, for studies that derived quantitative metrics (e.g., SCB, MCID, PASS) from PROMs in the setting of spine surgery with minimum 1-year follow-up. Details regarding the specific PROMs were collected including which PROM was measured, whether anchor- or distribution-based methods were utilized, the specific calculations, and the recommended value for a given PROM based on all evaluated calculations. Thirty-seven studies of 21,780 patients were included. The most commonly evaluated PROM-derived value was the MCID (n = 28), followed by PASS (n = 6) and SCB (n = 4). Twenty-one studies only utilized anchor-based calculations, 15 utilized both anchor-based and distribution-based methods, and one only utilized distribution-based calculations. The most commonly evaluated legacy PROMs were the Oswestry Disability Index (ODI) (N = 11, MCID range 4-20) and visual analog scale back pain (N = 5, MCID range 0.5-4.6). All 10 studies that derived SCB or PASS utilized the receiver operating characteristic methods. Among the six studies deriving a PASS value, four only evaluated ODI, identifying PASS ranging from 5 to 22. While calculated measures of clinical significance such as MCID, PASS, and SCB exist, significant heterogeneity exists in the current literature. Current shortcomings include a wide variability of reported value thresholds across the literature, and limited applicability to more heterogenous patient populations than the targeted cohorts included in published investigations. Continued investigations that apply these methods to heterogenous, large-scale populations can help increase generalizability and validity of these measures. III.

Do clinical trials prepare to fail by failing to prepare? An examination of MS trials and recommendations for patient-reported outcome measure selection.

Many clinical trials use patient-reported outcome (PRO) measures, which can influence treatment decision-making, drug approval and label claims. Given that many PRO measure options exist, and there are conceptual and contextual complexities with PRO measurement, we aimed to evaluate how and why specific PRO measures have been selected for pivotal multiple sclerosis (MS) clinical trials. Specifically, we aimed to identify the reasons documented for PRO measure selection in contemporary phase III MS disease-modifying treatment (DMT) clinical trials. We searched for phase III clinical trials of MS DMTs published between 2015 and 2021 and evaluated trial protocols, or primary publications where available, for PRO measure selection information. Specifically, we examined study documents for their clarification of clinical concepts measured, definitions of concepts measured, explanations of which PRO measures were considered, why specific PRO measures were chosen, and trade-offs in PRO measure selection. We identified 1705 abstracts containing 61 unique phase III MS DMT clinical trials. We obtained and examined 27/61 trial protocols. Six protocols were excluded: four contained no mention of PRO measures and two contained redacted sections preventing adequate assessment, leaving 21 protocols for assessment. For the remaining 34 trials (61-27), we retrieved 31 primary publications; 15 primary publications mentioned the use of a PRO measure. None of the 36 clinical trials that mentioned the use of PRO measures (21 protocols and 15 primary publications) documented clear PRO or clinical outcome assessment (COA) measurement strategies, provided clear justifications for PRO selection, or reasons why specific PRO measures were selected when alternatives existed. PRO measure selection for clinical trials is not evidence-based or underpinned by structured systematic approaches. This represents a critical area for study design improvement as PRO measure results directly affect patient care, PRO measurement has conceptual and contextual complexities, and there is a wide range of options when selecting a PRO measure. We recommend trial designers use formal approaches for PRO measure selection to ensure PRO measurement-based decisions are optimised. We provide a simple, logical, five-stage approach for PRO measure selection in clinical trials.

Properties of Patient-Reported Outcome Measures for Post-Traumatic Stress Disorder in North Korean Defectors: A Scoping Review.

Posttraumatic stress disorder (PTSD) is highly prevalent among North Korean defectors. This scoping review aimed to appraise the measurement properties of patient-reported outcome measures (PROMs) that assess PTSD among North Korean defectors. A systematic search was conducted using Research Information Sharing Service, ScienceON, PubMed, and Embase from their inception up to September 15, 2022. Each measurement properties of the included PROMs were evaluated using COnsensus-based Standards for the selection of health status Measurement INstruments (COSMIN) methodology. First, the methodological quality of each study on measurement properties was evaluated using the COSMIN Risk of Bias checklist. Second, the result of each single study on a measurement property was rated against the updated criteria for good measurement properties. After screening the articles, nine instruments from 40 studies were included. Among these instruments, Impact of Event ScaleRevised was the most frequently used (13 studies [38.1%]), followed by the Trauma Scale for North Korean Refugees, PTSD Checklist, and Posttraumatic Diagnostic Scale. The theoretical basis of all PROMs was secure in terms of content validity. The most frequently tested characteristics in measurement properties for internal structure were internal consistency, criterion validity, and convergent validity. No study reported sufficient structural validity as confirmatory factor analysis results with a model fit. There is insufficient evidence to support the recommendation of a specific PROM for use among North Korean defectors with PTSD.