Specific Patient-reported Outcome Measures Research Articles

Measurement properties of patient-reported outcome measures for women with dysmenorrhea: A systematic review.

Dysmenorrhea, or menstrual pain, is a subjective experience, and can only be assessed by patient-reported outcomes. These instruments should be reliable, valid and responsive. To identify and critically appraise the available evidence for the measurement properties of specific patient-reported outcome measures used for dysmenorrhea. The PRISMA statement was used to report this systematic review. Databases searched were PubMed, SCOPUS, CINAHL, Web of Science, ScienceDirect and Google Scholar (April 2021; updated on February 2023). Original studies with primary data collection, with no restriction on language and publication date that reported psychometric properties of one or more dysmenorrhea-related patient-reported outcome measure. The literature searches, selection of studies, data extraction and assessment of the risk of bias were performed independently by two reviewers and followed the COSMIN guidelines. Thirty studies were analysed in this review, and 19 patient-reported outcome measures were evaluated. The instruments varied in relation to the measured construct and measurement properties (validity, reliability and responsiveness). The methodological quality of the studies and the quality of evidence of the patient-reported outcome measures were variable. Among the 13 studies that reported the development of patient-reported outcome measures, most had inadequate methodological quality, and the overall rating was insufficient or inconsistent. The Dysmenorrhea Symptom Interference (DSI) scale was the only identified patient-reported outcome measure that has the potential to be recommended because of its sufficient rating combined with moderate quality of evidence for content validity. Future studies should further evaluate the measurement properties of the existing patient-reported outcome measures, or develop new patient-reported outcome measures following the COSMIN methodology. Not applicable as this is a systematic review. PROSPERO protocol: CRD42021244410. Registration on April 22, 2021.

#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 >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.

A systematic review of patient reported outcome measures in patients with anti-neutrophil cytoplasmic antibody associated vasculitis.

ANCA associated vasculitis (AAV) is associated with significant morbidity, fatigue, pain and poor health-related quality of life (HRQoL). This review aims to assess the comprehensiveness of existing patient reported outcome measures (PROMs) used in AAV and identify associations with poorer HRQoL outcomes. A literature review of studies using PROMs, including those labelled HRQoL in people with AAV as a primary or secondary study outcome were screened and reviewed up to July 2023. Quality was assessed using the Critical Appraisal Skills Programme. A total of 30 articles were included which utilised 22 different PROM tools. 76.7% (n = 23) used the SF-36 or a variation as a generic measure of health status and or HRQoL. Two studies developed a disease specific PROM. The AAV-PRO showed good psychometric properties but potential limitations in capturing all relevant aspects of the disease experience for AAV patients. Factors associated with poorer HRQoL included: neurological and sinonasal involvement, women and younger patients. 86.6% of studies showed no meaningful relationships between the SF-36 and BVAS, VDI or disease duration. Depression and anxiety were common and socioeconomic factors such as unemployment were significantly associated with poorer mental health outcomes. Glucocorticoids were found to be independently associated with worse SF-36 scores. Generic PROMs are useful in measuring significant changes but lack sensitivity to specific symptoms and unique AAV-related issues, while existing disease specific PROMs have limitations and may not fully capture AAV patient's perspective on disease and treatment burden.

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.

Hyaluronic Acid Fillers to Correct Cleft Lip Asymmetry in Adults: Description of Technique and Patient Reported Outcome Measure Using CLEFT-Q.

To determine the efficacy of hyaluronic acid (HLA) lip filler to correct subtle cleft lip asymmetries in adults using a validated patient reported outcome measure (PROM). Prospective cross sectional. UK National Health Service. Over 18 years old with repaired cleft lip and dissatisfaction of their lip appearance. HLA lip filler injection. A validated, cleft specific PROM, the lip module of CLEFT-Q™ prior to treatment and again after six weeks. 24 patients included. A mean total CLEFT-Q™ score pre-procedure was 14.9 (Stand deviation (SD) = 4.91) and 24 (SD = 6.08) post-procedure. Difference in mean total score pre- and post-procedure were statically significant (P = .0001) in all domains on the CLEFT-Q™. No adverse outcomes. HLA filler to correct subtle cleft lip asymmetries in adults is a simple low risk technique, which can significantly improve the patient's perception of lip appearance. HLA lip filler to correct subtle cleft lip asymmetries in adults is a simple technique, low risk procedure which can significantly improve the patient's perception of lip appearance.

GD03 Identifying core outcome domains and development of a cutaneous leishmaniasis core outcome measure for use in clinical practice and trials

Abstract Cutaneous leishmaniasis (CL) is caused by an intracellular protozoan parasite transmitted through a sand fly bite. A review of clinical trials examining different treatment modalities for CL found studies to be deficient in design, execution, analysis and reporting. Core outcome sets (COS), including agreed domains and measures, have been developed for several dermatological disorders. The aims of this study were to reach consensus on the most important core outcome domains to measure when assessing CL, agree on parameters to measure the domains and develop a clinical tool to assess CL. Potential outcome domains for CL were identified through a collaborative international approach involving virtual discussions and e-consultations with stakeholders, including teams caring for CL from Sri Lanka, India, Brazil and the UK. A subsequent judgement process was then undertaken, using the multidisciplinary stakeholder panel of international experts, adhering to nominal group technique to reach final agreement on the core outcome domains. Clinicians presented cases representing the clinical and psychosocial impact of CL to ensure the patient’s perspective was considered. Established core outcome domains included signs and symptoms (objective and subjective), treatment efficacy, treatment impact, clinical sequelae (scarring and pigment) and health-related quality of life (HRQoL). For each core outcome domain, a review of previous approaches to measure the domain was considered. Items to measure the domains were then evaluated by multiple rounds until the panel of experts reached consensus. It was agreed that ‘signs and symptoms’ should capture clinical morphology, diameter and induration of an index lesion with the aid of a palpability score; ‘treatment efficacy and impact’ should capture patient and investigator views through visual analogue scores, percentage change in size of the lesion and re-epithelialization compared with baseline. Sequelae assessment rated pigment, as well as atrophic and hypertrophic/keloid scars. Two open-ended questions were included as a means of capturing some aspects of HRQoL. CutLeishCOM was then generated to capture demographic details, reflect the core outcome domains and assess outcome measurements in each patient. CutLeishCOM has been validated in a small cohort in this study; further validation is required. A specific patient-reported outcome measure should also be considered to ensure adverse effects from treatments, and negative impacts are captured when assessing CL, thus enabling a patient-focused and empathic approach to management.

P355 PATIENT–REPORTED OUTCOME MEASURES IN WILD–TYPE AND VARIANT CARDIAC TRANSTHYRETIN AMYLOIDOSIS: THE ITALY (IMPACT OF TRANSTHYRETIN AMYLOIDOSIS ON LIFE QUALITY) STUDY

Abstract Background Transthyretin amyloidosis (ATTR) is due to TTR tissue accumulation. Both patients without mutations in the TTR gene (wild–type ATTR, ATTRwt) and those with such mutations (variant ATTR, ATTRv) may develop cardiac amyloidosis (CA). ATTR–CA has a deep impact on the quality of life. The ITALY study aimed 1) to create the first patient–reported outcome measures (PROMs) for ATTRv– and ATTRwt–CA, 2) to assess the feasibility of their administration, 3) to investigate the relationship between score values, clinical and laboratory findings, the Kansas City Cardiomyopathy Questionnaire (KCCQ) and Short Form Health Survey 36 (SF–36) scores. Methods Five Italian referral centers were involved (Pisa, Pavia, Ferrara, Florence, Messina). Item selection involved expert clinicians and 2 groups of 10 patients. The questionnaires were administered to consecutive patients evaluated at baseline and 6 months. Results Two 30–items questionnaires were created. Each item has 5 answers graded from 4 (best option) to 0 (worst option). The score ranged from 100 (best condition) to 0 (worst condition). As of November 2022, 82 patients (65 ATTRwt–CA, 17 ATTRv–CA) have completed the follow–up. At baseline, 30% of patients needed help to fill the questionnaire, and 20% at 6 months; 65% completed the questionnaire at baseline and 63% at 6 months. At baseline, median score values were 64 (interquartile range [IR] 54–74) in ATTRwt–CA and 59 (IR 51–71) in ATTRv–CA. ITALY score were correlated with global KCCQ and SF–36 scores at baseline and 6 months (r values from 0.7 to 0.8 in ATTRwt–CA and from 0.5 to 0.7 in ATTRv–CA). In patients with ATTRwt–CA, all KCCQ and SF–36 domains were univariate predictors of ITALY score values at baseline. All the KCCQ and SF–36 domains were independently predictive of ITALY score values in models including age, NYHA class, 6MWT, NT–proBNP; these results were confirmed at 6 months. In ATTRv–CA, All the KCCQ and SF–36 domains were independently predictive of ITALY score values in models including LV ejection fraction, at baseline and at 6 months. Conclusions The ITALY questionnaires are the first specific PROMs for ATTRwt– and ATTRv–CA. They have been created by experts in CA and patients and validated in a multicenter Italian cohort with 2 assessments over 6 months. Questionnaire completion is usually feasible. ITALY scores display close relationships with non–ATTR–specific measures of quality of life (KCCQ and SF–36).

The effectiveness of criteria-based, publicly funded abdominoplasty after massive weight loss: a prospective cohort study

**Background**: With the rising incidence of obesity and bariatric surgery, there has been an increase in body contouring procedures. Abdominoplasty, following massive weight loss, has been shown to improve body image and health-related quality of life. However, the inclusion criteria for this surgery is varied, and the role for public funding is controversial. The aim of this pilot study was to evaluate the benefits of strict, criteria-based and publicly funded abdominoplasty following massive weight loss using patient-reported outcome measures (PROMs).**Methods**: This was a prospective cohort study. Patients who qualified for abdominoplasty completed a general health PROM (SF-36) and a body contouring specific PROM (BODY-Q) pre- and postoperatively at three, six and twelve months. Complications were recorded. PROM scores were analysed using the Wilcoxon Signed Rank Test (p &lt; .05). The study was approved by the Central Adelaide Health Network Human Research Ethics Committee [HREC/16/TQEH/133].**Results**: Twenty-three patients were included in the analysis. The mean follow-up time was four months. The body contouring-specific (BODY-Q) scores improved in eight of nine domains. The general (SF-36) scores improved in six of nine domains. The complication rate was low and minor in nature.**Conclusion**: This pilot study suggests that publicly funded abdominoplasty with specific criteria restrictions has a role in significantly improving body satisfaction and health-related quality of life in patients after massive weight loss.

Measuring the impact of steroid therapy on health-related quality of life in patients with rheumatic diseases: international development of a glucocorticoid treatment-specific patient-reported outcome measure.

Glucocorticoids (GCs) ('steroids') are used to treat rheumatic diseases but adverse effects are common. We aimed to explore the impact of GC therapy on health-related quality of life (HRQoL), to inform the development of a treatment-specific patient-reported outcome measure (PROM) for use in clinical trials and practice. Semi-structured qualitative interviews were conducted with patients from the UK, USA and Australia, treated for a rheumatic condition with GCs in the last two years. Purposive sampling was used to select participants with a range of demographic and disease features. An initial conceptual framework informed interview prompts and cues. Interviews elicited GC-related physical and psychological symptoms and salient aspects of HRQoL in relation to GC therapy. Interview data were analysed inductively to develop initial individual themes and domains. Candidate questionnaire items were developed and refined. Sixty semi-structured qualitative interviews were conducted (UK n = 34, USA n = 10, Australia n = 16). Mean age 58 years; 39/60 female; 18 rheumatic diseases were represented. 126 individual themes were identified and organised into six domains: physical symptoms; psychological symptoms; psychological impact of steroids; impact of steroids on participation; impact of steroids on relationships; and benefits of steroids. Candidate questionnaire items were tested and refined by piloting with patient research partners, iterative rounds of cognitive interviews, and linguistic translatability assessment, informing a draft questionnaire. We describe an international qualitative study to develop candidate items for a treatment-specific PROM for patients with rheumatic diseases. A future survey will enable the validation of a final version of the PROM.

A Delphi Consensus Study on Undergoing Carotid Endarterectomy: Patient Reported Outcome Measures

Currently, evidence is lacking for disease specific patient reported outcome measures (PROMs) for use in atherosclerotic carotid artery stenosis (either symptomatic or asymptomatic) submitted to carotid endarterectomy (CEA). This study aimed to obtain expert consensus on the most important items to include in a PROM designed to capture the impact of atherosclerotic carotid artery stenosis and its treatment on health related quality of life. A three round modified Delphi consensus study was performed. A mixed expert Delphi panel of doctors (international panel of dedicated vascular surgeons and neurologists) and patients (either symptomatic or asymptomatic patients meeting criteria for carotid artery revascularisation) was implemented. The aim was to obtain pre-defined consensus on items in four pre-defined domains: generic, quality of life, symptom related, and treatment related. Consensus was reached in rounds 2 and 3 with > 70% overall expert agreement. The experts agreed on 23 items (out of 49) which were distributed as follows: five in the generic, six in the quality of life, six in the symptom, and six in the treatment related domain. Interestingly, comparing the items that reached consensus in this study, with the generic and disease specific PROMs previously used in carotid artery disease investigation, the only constant items were "difficulty with walking" and "ability to perform daily activities" included in the symptom domain. Considering the items that reached expert consensus in the additional domains, emphasis was given to the impact of the diagnosis, treatment and follow up, and to fear/concern "about the future" and "about severe stroke". In the treatment domain emphasis was also attained on the side effects, long term patient satisfaction, and on the information provided regarding treatment options. As hard clinical outcomes become increasingly rare, assessment of the impact of CEA becomes increasingly difficult. The consensus reached provides a newly defined disease specific PROM that warrants independent validation in specific populations in the future.