Background:Trust building is essential for effective healthcare, including care delivered using digital health technologies (DHTs). This analysis aimed to develop an understanding of the mechanisms of trust building in relation to DHTs for diabetes using tracking and measuring tools. Methods:This was a multimethod qualitative analysis conducted in Germany, Spain, and the United States, using a behavioral science approach. Interviews were conducted with different groups of participants from June 2022 to August 2022. The interviews consisted of one-on-one interviews (90 min) with experts in the diabetes support system, online dyad interviews (120 min), and online homework (120 min) with adults with type 2 diabetes (T2D) with different mind states (struggling, juggling, and controlling) and their caregivers. Focus group discussions were conducted with healthcare professionals (HCPs; each 150 min), including general physicians, specialists, and nurses, representing a conservative or innovator persona. Results:Nine interviews were conducted with experts (n=9), 28 dyad interviews were conducted with people with T2D (n=38) and caregivers (n=18), and 15 focus group discussions were conducted with 59 HCPs. Building trust in DHTs appeared to be created differently from building trust in medications, and requires activating, motivating, and sustaining desired behavior change. DHT trust builds systematically in three levels: trust in individual's own role in diabetes management (self-efficacy), trust in data as a tool to create empowerment, and trust in viability of sustained behavior change. Conclusion:People who had built trust on all three levels felt in control of their diabetes and were motivated and engaged in DHTs. Focus on trust building is important for digital healthcare launch strategies, particularly in user conversion from consideration phase to actual initiation of use of DHTs.
Objectives:This study aimed to assess the validity, reliability and responsiveness of the Senegalese kidney disease quality of life (KDQOL-36). Methods:This cross-sectional and multicenter psychometric evaluation was conducted between June and November 2024. The Wolof version of the KDQOL-36 scores was assessed in 575 Senegalese patients aged 18 years or older with stage 3-4 CKD or on dialysis, who had been followed for at least three months and not hospitalized during the previous three months. The factor structure was evaluated using confirmatory factor analysis. We assessed internal consistency and test-retest reliability using α and ω coefficients, the intraclass correlation coefficient (ICC) and Bland-Altman analysis. Construct validity was examined using "known group analyses", and responsiveness was evaluated using the paired Wilcoxon test, standardized response means and effect size. Results:A bifactor structure was retained for the generic module, comprising overall, physical and mental health. A three-factor structure was confirmed for the kidney disease targeted scale, including the burden, symptoms/problems and effects. The scale and subscales showed sufficient internal consistency. Test-retest reliability was moderate to good. Known group analyses revealed significant differences in the scale scores between patients who received dialysis and who did not receive dialysis, and between men and women. Overall health, physical health, symptoms/problems and effects demonstrated moderate to high sensitivity to change. Conclusions:This study provides evidence supporting the structural validity, construct validity, reliability, and responsiveness of the Senegalese Wolof version of the KDQOL-36 for assessing health-related quality of life.
Background:Adherence is a key determinant of treatment success in oncology and, according to the World Health Organization (WHO), describes how closely patients follow the recommendations agreed upon with their healthcare provider, including taking medications, adhering to dietary plans, or making lifestyle changes. Patient-reported outcome measures (PROMs) provide insights into patients' perceptions, barriers, and beliefs. However, a comprehensive overview of adherence-related PROMs in oncology is lacking. Methods:We conducted a systematic umbrella review in PubMed and Embase up to October 2024 in adult cancer patients. A qualitative synthesis was performed to classify adherence dimensions according to the WHO framework and to evaluate the validation status. Results:Of 6,073 records screened, 89 systematic literature reviews (SLR) were included, encompassing 599 PROM applications. Most PROMs assessed medication adherence (41.1%). 4.8% of PROMs measured multiple adherence dimensions. 49.7% of PROMs were validated, while for 39.7% of PROMs validation status was not reported. Conclusion:Adherence-related PROMs in oncology predominantly focus on medication, while lifestyle and multidimensional aspects remain underrepresented. Validation status is frequently unclear. Standardized and validated modular PROMs are needed to inform the choice of PROMs for monitoring patient adherence in clinical practice and to enhance health outcomes.
Background:Treatment of tenosynovial giant cell tumor with TURALIO (pexidartinib) is associated with serious hepatotoxicity risk. To ensure its safe use, a Risk Evaluation and Mitigation Strategy (REMS) program was implemented, incorporating structured education, mandatory enrollment, and liver function monitoring. The assessment of patient and caregiver knowledge, attitudes, and behaviors (KAB) is a central component of evaluating REMS effectiveness. Methods:Four waves of TURALIO Patient KAB Survey were conducted between 2020 and 2024 among REMS-enrollees. Surveys assessed understanding of two Key Risk Messages (KRMs): (KRM 1) hepatotoxicity risk and symptom recognition, and (KRM 2) liver monitoring requirements before and during treatment. Response rates and demonstrated understanding of KRMs were summarized descriptively. Results:Across four waves of this voluntary survey, 239 participants completed KAB surveys. Survey response rates ranged from approximately one-third to one-half of invited participants across waves. Correct response rates for individual knowledge items were consistently high. Demonstrated understanding of hepatotoxicity risk remained at ≥80% (the predefined threshold) across waves (87.5% in Wave 1; 80.0% in Wave 4). Understanding liver monitoring requirements improved over time, reaching 100% in Wave 3. Combined demonstrated understanding of KRM1 and KRM2 was <80% in three survey waves, particularly in Wave 2 (73.4%). Demonstrated understanding of KRMs remained generally stable despite evolving patient demographics and treatment duration over time. Conclusion:Across KAB Survey waves, patients receiving TURALIO demonstrated sustained knowledge of hepatotoxicity risk and monitoring requirements supporting the effectiveness of the tREMS program in ensuring the safe real-world use of pexidartinib.
Objective:Severe hypertriglyceridemia (sHTG) and familial chylomicronemia syndrome (FCS) are metabolic conditions associated with an increased risk of adverse health outcomes. The FCS Symptoms and Impacts Scale (FCS-SIS) was developed to assess the symptoms and impacts of FCS. This study assessed the content validity of the 4 FCS-SIS symptom items in adults with sHTG. Methods:Two iterative rounds of qualitative interviews were conducted with adults (aged ≥ 18 years) who had clinician-confirmed sHTG (triglycerides ≥ 500 mg/dL) and recently experienced ≥ 2 symptoms of sHTG. Participants described their initial experiences and symptoms of sHTG during concept elicitation. Cognitive debriefing of the 4 FCS-SIS symptom items assessed relevance, interpretability, answerability, and meaningful change. Results:Among 20 participants, the most frequently reported, most bothersome, and most important-to-treat sHTG symptoms were difficulty thinking (n = 20), physical fatigue (n = 20), diarrhea (n = 19), and abdominal pain (n = 18), which the 4 FCS-SIS symptom items assess. Participants reported these items to be easy to understand (n = 20) and, for most items, reported that a 1- to 2-point improvement would be meaningful. Conclusion:FCS-SIS symptom items are content-valid items for the assessment of sHTG symptoms in adults with sHTG.
PurposeGoal Attainment Scaling (GAS) is a personalized outcome measure that evaluates treatment response based on patient- and caregiver-prioritized goals, offering a structured yet flexible framework for assessing meaningful change. Although GAS is increasingly applied in clinical trials, its implementation remains inconsistent, and the perspectives of clinicians and researchers who use it in practice remain poorly understood. This study explored the experiences of clinicians and researchers with GAS to better understand how it is operationalized, the challenges encountered, and opportunities to improve its consistency and utility in patient-centered care and research.Participants and MethodsSemi-structured interviews were conducted with 11 clinicians and academics from Canada, the United States, the United Kingdom, and Australia who had experience using GAS in clinical or research settings. Data were analyzed thematically using the Framework Method, with coding conducted inductively and supported by NVivo 12.ResultsParticipants valued GAS for capturing outcomes most meaningful to patients and demonstrating treatment responsiveness in heterogeneous conditions. However, clinician resistance, time demands, limited familiarity, and variability in implementation across settings were identified as key challenges. Education, peer advocacy, and digital tools were suggested as strategies to improve feasibility and consistency.ConclusionClinicians and researchers view GAS as a valuable patient-centered outcome measure but highlight variability in implementation as a barrier to broader adoption. Guidelines for GAS implementation, training, and supportive tools may strengthen the use of GAS in both clinical practice and research, ultimately improving the measurement of outcomes that matter most to patients.
This perspective article outlines the recommendations for the analysis and presentation of data collected via patient-reported outcome measures (PROMs), to promote good practice across Wales. We do not present detailed instructions or repeat what can be easily found in statistics texts, but rather focus on the overall approach that should be adopted when dealing with such data. Throughout the article, we introduce the importance of patient-reported outcome data within the National Health Service (NHS) Wales. We then briefly describe how PROM questionnaires are validated. We then introduce the different data types and describe how PROM questionnaires are structured. Following this, we highlight how the way that patient-reported outcome data is collected can affect appropriate analysis and what should be kept in mind when comparing different items within a questionnaire. We then provide some recommendations as to how to present such data and how to go about choosing appropriate inferential statistics tests for further analysis. Finally, we go over some of the potential consequences of not analysing the data appropriately. Additionally, we also include a simplified version of these recommendations that can be shared with a wider audience.
Purpose:Combination antiretroviral therapy (cART) has improved outcomes in individuals living with HIV. This study investigated quality of life, mental health, and sexual function in virologically suppressed men. Patients and methods:This case-control study compared quality of life, depression, and sexual function of 39 heterosexual men on cART with viral suppression and 60 healthy male controls. Participants completed the EUROHIS-QOL 8, Beck Depression Inventory (BDI), and Arizona Sexual Experience Scale (ASEX) questionnaires. Results:HIV group's mean age was 39 years, controls 41.5 years, with similar demographics. Median EUROHIS-QOL8 and BDI scores were similar between groups. While ASEX scores were higher in HIV patients (p = .032), this difference did not result in a higher prevalence of sexual dysfunction based on established ASEX criteria. BDI scores strongly negatively correlated with EUROHIS-QOL8 in the HIV group (r = -.731, p < .001) and moderately in controls (r = -.475, p < .001). Quality of life decreased with depressive symptoms in both groups. Conclusion:Among men with HIV who achieved viral suppression, no significant differences were found versus healthy controls in quality of life, depression, or sexual dysfunction. These findings support individual evaluation rather than HIV status-based assumptions.
Objective To assess the psychometric properties of a respiratory symptom score from the Quality of Life-Bronchiectasis Questionnaire in adults with Mycobacterium avium complex (MAC) lung disease. Methods Structural validity, internal consistency, test-retest reliability, and known-groups validity were assessed using data from the ARISE (NCT04677543) and ENCORE (NCT04677569) phase 3 trials. Convergent validity was assessed using Exacerbations of Chronic Pulmonary Disease Tool (EXACT), EXACT Respiratory Symptoms (E-RS), St. George Respiratory Questionnaire (SGRQ), and Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale. Anchor-based meaningful within-patient change (MWPC) thresholds were estimated using the Patient Global Impression of Severity Respiratory scale. Results In total, 362 patients were included for cross-sectional (ARISE: 97, ENCORE: 265) and longitudinal (ARISE: 99) validation analyses. Modern psychometric methods supported item relevance and an essentially unidimensional mean score. The respiratory symptom score demonstrated adequate internal consistency (McDonald's omega: 0.86, Cronbach's alpha: 0.81), test-retest reliability (intraclass correlation coefficient: 0.73), convergent validity (EXACT: -0.72, E-RS: -0.72, SGRQ: -0.77, FACIT-Fatigue: 0.51), and known-groups validity. The estimated MWPC threshold was a 16.67-point median change from baseline (95% CI: 8.33-16.83 points). Conclusions The respiratory symptom score is a robust, sensitive, and responsive measure of respiratory symptoms in adults with a new or recurrent MAC lung disease diagnosis.
Purpose:The Birth Satisfaction Scale - Revised (BSS-R) is a widely used patient-reported outcome measure (PROM) for assessing childbirth experience. However, no French translation and adaption currently exists, limiting its use in French-speaking populations. The objective of this study was to translate and adapt the BSS-R into French (Fr-BSS-R). Patients and Methods:The questionnaire was translated and culturally adapted using a structured 10-step procedure in line with the guidelines of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). During the cognitive debriefing stage, 15 mothers evaluated each item, scoring their level of comprehension on a scale from 1 to 10. Results:The French version was very well understood by the 15 mothers, with a mean comprehension score of 9.5 ± 1.1 out of 10. Both forward and backward translations were very consistent and considered accurate by the original developer. Five ratings out of 150 (3%) were < 7 and concerned items 1, 3, 8 and 9. Comments prompted the authors to slightly modify the French translation produced to improve semantic equivalence. Conclusion:This work presents the creation of a French adaptation of the BSS-R (Fr-BSS-R), developed in accordance with ISPOR guidelines for translation and cross-cultural adaptation, thereby enabling its application in French-speaking populations. Future research should investigate the psychometric properties of the Fr-BSS-R to support its broader use in French-speaking populations.
PurposeHealth-related quality of life (HRQoL) measures in the form of health utilities are valuable for economic evaluations of the effectiveness of food allergy interventions. However, traditional HRQoL instruments lack the sensitivity to generate health utilities that capture the impact that food allergies, such as peanut allergies, may have on children's mental health and daily activities. This study used mapping and discrete choice experiment (DCE) methods to generate health utilities from the Food Allergy Quality of life Questionnaire-Parent Form (FAQLQ-PF), which were then applied to clinical trial data.Patients and MethodsHealth utilities (HU) were generated using two methods: mapping and DCE. Parents of children with peanut allergies (N=159) completed the FAQLQ-PF and EQ-5D-Y-Proxy-1 questionnaires. Mapping algorithms were developed once the FAQLQ-PF responses were mapped onto the EQ-5D-3L utilities. A composite DCE with time trade-off and a vignette was conducted among parents without peanut allergic children (N=767). The utilities derived from the mapping and DCE methods were applied to clinical trial data (PEPITES and PEOPLE) for an epicutaneous peanut patch (DBV712).ResultsThe mapping algorithm showed an association of 0.199 between FAQLQ-PF and EQ-5D-3L utilities. The DCE disutilities were highest for severe food-related anxiety, emotional distress, and social limitations. Once applied to the clinical trial data, the HU derived from the mapping algorithm demonstrated statistically significant HRQoL improvements for the intervention group at 36 months. Using the DCE-derived utilities, statistically significant HRQoL improvements for the intervention group were demonstrated at both 24- and 36-months. The effect size analysis demonstrated that the DCE-derived utilities were more responsive than mapped utilities.ConclusionDCE-derived utilities demonstrated greater responsiveness to changes in HRQoL compared with mapped utilities, suggesting their potential use in economic evaluations and HTA submissions for peanut allergy interventions. The DCE-derived health utilities showed greater sensitivity to changes in HRQoL. These utilities can be used in health technology assessments to better capture the impact of peanut allergy treatments on children's quality of life.
Purpose:Speed of clinical response to a new therapeutic intervention is a critical determinant of the overall treatment outcomes, but tools focused on asthma response speed are currently unavailable. This study aimed to validate the psychometric properties of a new questionnaire initially written in Italian, the Speed of Change in Feeling of Health (SCFH), in patients with asthma. Patients and Methods:Two hundred and seventy subjects with not-well- or poorly controlled asthma, enrolled in the Italian sites of the NEWTON real-world study, were asked to complete the provisional version of SCFH and three validated questionnaires, the Stanford Expectation of Treatment Scale (SETS), the 5-item version of the Asthma Control Questionnaire (ACQ-5), and the Global Rating Scale (GRS). Internal consistency and validity were determined. Moreover, we assessed the minimal clinically important difference (MCID) using anchor-based method. Results:One hundred and ninety-six patients completed the questionnaire at least once at 7 (±1), 14 (±2), or 30 (±3) days after starting BDP/FF NEXThaler® 100/6 μg treatment. We started from SCFH provisional questionnaire of 30 items, the internal consistency of which revealed a high redundancy (Cronbach's alpha of 0.98), prompting its reduction to an 8-item questionnaire (SCFH-8; Cronbach's alpha = 0.90). Known-group validity indicated significant differences in SCFH-8 scores (p = 0.0003) at 30 (±3) days after enrollment between improved and non-improved subjects, as defined by changes in ACQ-5 scores. The study confirmed the convergent validity through comparisons with the SETS and the ACQ-5 questionnaires. A clinically relevant change in feeling of health was observed in 43.3% of participants at 7 days, while the cumulative frequency of patients reporting a clinically relevant change in their feeling of health at 30 days after enrollment was 70.4%. Conclusion:The Italian version of SCFH-8 is a valid, short tool with good psychometric properties for determining the speed of change in health perception after starting treatment.
Health and medical journals have a mission to evaluate and disseminate information generated from rigorous and peer reviewed scientific enquiry. As journal editors, we are entrusted with safeguarding the integrity of the scientific record, supporting the researchers who advance discovery, and honoring the excellence of academic institutions that cultivate rigorous and unbiased inquiry. Maintaining the integrity of the information published in our journals helps ensure that scientists, health professionals, policymakers and the public can benefit from information that is accurate, trustworthy, accessible, and understandable.
Purpose:To assess the psychometric properties of the disease-specific Hypoparathyroidism Symptom Diary (HypoPT-SD) patient-reported outcome (PRO) tool, which consists of a 7-item symptom subscale, a 4-item impact subscale, a single item for anxiety, and a single item for sadness or depression, using data from the BALANCE randomized, placebo-controlled Phase 3b/4 study (NCT03324880). Methods:Eligible patients had symptomatic hypoparathyroidism (HypoPT) at baseline and were aged 18-85 years (inclusive). Patients received recombinant human parathyroid hormone (1-84) or placebo. The HypoPT-SD was filled in daily; data recorded at baseline and Weeks 4, 12, and 26 (end of treatment [EOT]) were included in this analysis. Inter-item and item-total correlations were used to assess HypoPT-SD structure; Cronbach's coefficient α was used to analyze the internal consistency and reliability, and intraclass correlations were used to measure test-retest reliability. Construct validity was determined using correlational analyses between HypoPT-SD scores and scores from other conceptually similar PRO tools. Ability to detect change was assessed and thresholds for meaningful within-patient change were established. Results:The psychometric analysis population (N=93) was predominantly female (88.2%) and white (96.8%), with a mean age of 48.5 years. Inter-item correlations ranged from 0.35 to 0.85 at baseline and from 0.49 to 0.93 at EOT. Item-total correlations ranged from 0.57 to 0.83 at baseline and from 0.69 to 0.88 at EOT. Cronbach's α values at baseline were 0.90 (symptom subscale) and 0.88 (impact subscale). Intraclass correlation coefficients for both subscales in stable patients exceeded 0.70. Significant cross-sectional correlations were observed with most of the conceptually linked PRO tools analyzed, and HypoPT-SD scores were responsive to change. Potential changes of 1.5 (symptom subscale) and 0.8 (impact subscale) were determined as meaningful change thresholds for within-patient improvements. Conclusion:The HypoPT-SD is a reliable measure of key symptoms and impacts of HypoPT.
Purpose: Given the importance of managing chest pain, anxiety, and cardiac arrhythmia in cardiac care unit patients this study aim to determine the effect of a structured supportive and palliative care approach on these three critical aspects. Patients and Methods: A randomized, double-blind, clinical trial with two groups was conducted in the second half of 2025. The research population included patients who were hospitalized in the cardiac care unit, with a final sample size of 36 people in each group. The data collection instruments included personal information, an electrocardiogram machine, a Visual Analog Scale, and the Spielberger anxiety questionnaire. The variables were measured before the intervention. Routine treatments were then continued for the control group, and in the intervention group, in addition to the routine treatments, a structured supportive and palliative care approach was implemented. Three and seven days after the intervention, these variables were measured. Results: Seventy-two patients participated in this study. As the results showed, chest pain, arrhythmia incidence, and anxiety in both groups decreased over time from the first to the third time; however, this difference was statistically significant in the intervention group for all three variables (p=0.044, 0.012, and 0.03, respectively), whereas in the control group, it was only significant in the anxiety variable (p=0. 045). The findings also showed that the mean scores of chest pain, arrhythmia incidence, and anxiety before the intervention in the two groups did not differ significantly; however, there was a significant difference in the three variables three and seven days after the intervention. Conclusion: As the results showed, supportive and palliative care approaches are effective in reducing chest pain, arrhythmia incidence, and anxiety levels in cardiac care unit patients. Therefore, it is necessary to prepare guidelines in this field based on the results of this study and other published studies.
Background:Patient-reported outcomes (PROs) are important for understanding patients' experiences with their health conditions; however, individual perspectives of meaningful change for personalized treatment goals are rarely captured. Consequently, treatment outcomes assessed in clinical trials or routine practice may not align with real-world priorities or reflect individual perspectives. To bridge this gap, we developed the 2-part treatment goal evaluation scale (TGES) to assess individuals' perspectives of whether meaningful within-patient change has occurred in their self-identified treatment goal. Methodology:The draft TGES measure included the TGES - Baseline-a single, open-ended item to identify patients' primary treatment goal when starting a new treatment for a new or ongoing condition-and the TGES - Follow-Up-2 items designed to assess patients' perspectives of whether meaningful change has occurred in their self-identified goal and overall condition. Three rounds of qualitative interviews with adults aged ≥18 years with either atopic dermatitis (AD) or migraine were conducted to revise the draft TGES and assess content validity. Results:A total of 28 participants with AD (n=15) or migraine (n=13) were included; most identified as female (60.7%) and White (78.6%). Most participants interpreted the TGES - Baseline instructions (82.1%) and item (96.4%) as intended, whereas 39.3% suggested revisions to the instructions. Overall goal categories were identified: 3 AD-specific (improving physical symptoms, sensory symptoms, and choice of clothing) and 4 migraine-specific (reduced migraine frequency, impact on activities, migraine severity, and associated symptoms other than headache). Most participants interpreted the TGES - Follow-Up instructions (100.0%) and items (96.4%; 75.0%) as intended, with minor revisions suggested to improve clarity. Conclusion:Qualitative interviews supported the content validity of the TGES for identifying individual treatment goals and assessing perspectives of meaningful change. With future quantitative evaluation, the patient-centric and condition-agnostic TGES may help evaluate personalized endpoints and inform within-patient meaningful change in clinical trial and real-world settings.
Purpose:Validated patient-reported outcome measures (PROMs) are required for use in clinical trials of celiac disease (CeD) therapies. The Celiac Disease Symptom Diary 2.1© (CDSD 2.1©), which measures the daily severity of core CeD symptoms (abdominal pain, bloating, diarrhea, nausea, tiredness), was developed according to the latest regulatory guidelines for fit-for-purpose PROMs. This study evaluated the psychometric properties of CDSD 2.1. Methods:Psychometric properties of CDSD 2.1 were evaluated using data from a 12-week US observational study, the Virtual Celiac Symptoms Study (NCT05309330), in patients with CeD maintaining a gluten-free diet. Participants completed CDSD 2.1 daily and other PROMs (Patient Global Impression of Severity [PGIS], Gastrointestinal Symptom Rating Scale [GSRS], and Celiac Symptom Index [CSI]) at specified time points to evaluate the reliability, validity, and responsiveness of CDSD 2.1. Results:Overall, 480 participants (338 adults, 142 adolescents) completed the study. Cronbach's alpha (baseline = 0.77 adults/adolescents) indicated high internal consistency reliability of weekly average gastrointestinal (GI; abdominal pain, bloating, nausea, diarrhea) CDSD 2.1 scores. An intraclass correlation coefficient of 0.89 (adults)/0.88 (adolescents) demonstrated high test-retest reliability among stable patients on PGIS. Moderate-to-strong correlations between weekly average GI CDSD 2.1 scores and GSRS domains at baseline and CSI at Week 3 confirmed construct validity (r = 0.44-0.76; p<0.05). Weekly average GI CDSD 2.1 score changes followed expected patterns based on PGIS change groups, demonstrating responsiveness. Conclusion:This evaluation provides evidence to support the use of CDSD 2.1 in clinical trials as a reliable and responsive measure of CeD symptom severity.
This systematic review aims to explore the utilization of patient-reported measures (PRMs) in primary care, focusing on healthcare providers' use of PRM data and factors influencing its effectiveness. Following the PRISMA 2020 guidelines, we registered the review in PROSPERO (CRD420251030695) and screened 2465 records, ultimately including eight studies. Data extraction and quality appraisal were conducted using a structured approach and the Mixed Methods Appraisal Tool (MMAT). Findings suggest that effective PRM data use depends on integration into electronic health records (EHRs) and clinical workflows, with barriers including technical limitations and organizational culture. PRM data can support clinical decision-making, shared decision-making, and communication and support professional empowerment and resource optimization. However, evidence quality was moderate, and conclusions should be interpreted with caution due to the small number of studies. Differences in health systems and study heterogeneity further constrain generalizability. This review highlights the need for seamless EHR integration, streamlined instruments, and active professional engagement to optimize PRM implementation, while identifying a critical research gap and calling for future studies on cost-effectiveness and equity impacts.
Background:Patient Acceptable Symptom State (PASS) describes the highest level of symptom beyond which patients consider themselves well. PASS can be used to establish what patients' view as an acceptable outcome after surgery. The Oxford Elbow Score (OES) is one of the most used elbow-specific patient-reported outcome measure (PROM), but a PASS-value has not yet been established for the OES after elbow arthroplasty. The primary purpose of this study was to determine the PASS for the OES after primary elbow arthroplasty due to acute distal humeral fracture. Methods:This is a retrospective multi-center cohort study. All nationwide patients treated with elbow arthroplasty due to acute distal humeral fracture from January 1, 2008, until December 1, 2021, were invited to participate. Data were collected retrospectively using electronic health records. Study participants answered the OES, and a PASS-anchor question was used to assess if patients were in an acceptable state at follow-up. Logistic modelling was used to determine the PASS-value, defined as the value of OES needed to achieve an acceptable postoperative result with at least 95% probability. A PASS-value was estimated for the total population, for patients with <5 years of follow-up, and patients with ≥5 years of follow-up. Results:The OES and PASS-anchor was answered by 159 (62%) patients. Median OES was 41 (0-48) in the total population. One hundred and thirty-nine patients (87%) reported an acceptable result. The 95% PASS-value for the OES was 33 (95% CI 29.6-38.5) in the total population. Conclusion:The PASS-value for the OES after elbow arthroplasty due to acute distal humeral fracture was 33 for the total population and similar for patients with short-term and long-term follow-up. We recommend using the PASS-value to aid in interpretation of clinical trials and registry-based studies as well as identifying clinical failures in registries.
