Patient Related Outcome Measures最新文献

{"title":"Validating the Emotional Well-Being Questionnaire in Type 2 Diabetes: A Pilot Confirmatory Factor Analysis.","authors":"Maarja Randväli, Jekaterina Šteinmiller, Kay Sundberg, Toomas Toomsoo","doi":"10.2147/PROM.S538578","DOIUrl":"10.2147/PROM.S538578","url":null,"abstract":"<p><strong>Purpose: </strong>The Emotional Well-Being Questionnaire (EWQ) assesses a broad spectrum of mental health conditions and related symptoms-depression, anxiety, asthenia, and insomnia-highly relevant in type 2 diabetes (T2D), where emotional distress can impair adherence and outcomes. This pilot cross-sectional study provides preliminary validation evidence for the EWQ six-factor model in a T2D cohort using confirmatory factor analysis (CFA), evaluating reliability and convergent validity for mental-health screening.</p><p><strong>Patients and methods: </strong>A sample of 240 adults (T2D n = 122; control n = 118) with T2D completed the EWQ and nine-item Patient Health Questionnaire (PHQ-9). Confirmatory factor analysis (CFA) was conducted to assess the six-factor model fit, and convergent validity was evaluated through analysis of EWQ scores with PHQ-9 scores.</p><p><strong>Results: </strong>Confirmatory factor analysis indicated acceptable-borderline fit for a pilot sample (CFI = 0.886; TLI = 0.883; RMSEA = 0.071), providing preliminary support for the six-factor structure. The EWQ demonstrated reliability in the T2D group (Cronbach's α = 0.79). Convergent validity was supported by a significant positive correlation with PHQ-9 scores (r = 0.652, p < 0.001), confirming the EWQ's capacity to assess depressive symptoms in this population.</p><p><strong>Conclusion: </strong>The EWQ's six-factor structure showed preliminary adequacy in adults with T2D. While internal consistency and convergent validity with the PHQ-9 were supportive, overall model fit indices were moderate; therefore, findings should be interpreted with caution and replicated in larger, more diverse samples before clinical implementation. Future studies should focus on cross-cultural validation, measurement invariance, and longitudinal assessment to refine its clinical utility.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"129-145"},"PeriodicalIF":1.8,"publicationDate":"2025-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12442912/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145085573","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Paravertebral Muscle Degeneration on Residual Low Back Pain Following Percutaneous Kyphoplasty for Osteoporotic Vertebral Fractures: A Retrospective Study.","authors":"Tao Feng, Shengyang Jin, Junjie Niu, Qi Yan, Dawei Song, Jinning Wang","doi":"10.2147/PROM.S529923","DOIUrl":"10.2147/PROM.S529923","url":null,"abstract":"<p><strong>Background: </strong>Residual low back pain (LBP) is frequently reported after percutaneous kyphoplasty (PKP) for osteoporotic vertebral fractures (OVFs), yet its underlying mechanisms remain unclear. Paravertebral muscles (PVMs) degeneration, particularly fat infiltration and atrophy may contribute to persistent postoperative pain.</p><p><strong>Objective: </strong>To evaluate the association between PVMs degeneration and residual LBP after PKP and identify imaging-based predictors for risk stratification.</p><p><strong>Methods: </strong>This retrospective cohort study included 213 patients (mean age 70.88 ± 8.58 years; 82.2% female) with single-level OVFs who underwent PKP between January 2021 and June 2023. Patients with multiple-level fractures, chronic LBP, neurological deficits, prior spinal surgery, incomplete imaging, or inadequate follow-up were excluded. Fat infiltration percentage (FI%) and cross-sectional area of the multifidus (MF), erector spinae (ES), and psoas major (PS) were measured at the L4 level using transverse T2-weighted MRI. Residual LBP was defined as postoperative VAS ≥3.5 at 12-month follow-up. Logistic regression and ROC analyses were conducted and appropriate univariate tests (<i>t</i>-test or Mann-Whitney <i>U</i>-test) were performed.</p><p><strong>Results: </strong>Residual LBP occurred in 13.6% of patients and was associated with higher VBQ scores (3.14 ± 0.38 vs 2.57 ± 0.25, P=0.001), greater postoperative kyphosis (16.03 ± 6.69° vs 6.70 ± 4.80°, <i>P</i>=0.001), increased FI% of ES/MF (57.28 ± 5.63% vs 43.40 ± 14.93%, <i>P</i>=0.001), reduced PS area (10.74 ± 4.23 cm² vs 16.15 ± 3.71 cm², <i>P</i>=0.001), and concentrated cement distribution (11.5% vs 73.6%, P=0.001). Independent predictors included elevated VBQ (OR=85.2, 95% CI 7.006-1036.458), kyphosis (OR=1.14, 95% CI 1.017-1.276), FI% of ES/MF (OR=1.082, 95% CI 1.008-1.160), and PS area (OR=0.509, 95% CI 0.285-0.910). ROC analysis identified FI% ≥49.78% and PS area ≤11.937 cm² as optimal cutoffs.</p><p><strong>Conclusion: </strong>Preoperative magnetic resonance imaging assessment of paravertebral muscle may help identify patients at risk for residual low back pain after kyphoplasty. Incorporating preoperative imaging and postoperative physical therapy referral may improve patient outcomes.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"117-127"},"PeriodicalIF":1.8,"publicationDate":"2025-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12368434/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144963925","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Impact of Self-Perceived Burden on Loneliness in Stroke Patients: The Mediating Role of Rumination.","authors":"Xinxin Zhou, Lina Guo, Yuanli Guo, Genoosha Namassevayam, Peng Zhao, Mengyu Zhang, Yuying Xie, Yanjin Liu","doi":"10.2147/PROM.S528307","DOIUrl":"10.2147/PROM.S528307","url":null,"abstract":"<p><strong>Background: </strong>Stroke outcomes are often measured through objective scales, which may neglect subtle cognitive changes and fail to capture patients' subjective experiences of recovery and quality of life. This study aimed to examine the interrelations among self-perceived burden, loneliness, and rumination in stroke survivors through the patient-reported outcomes and to provide theoretical insights and intervention strategies for improving psychological well-being and quality of life in stroke patients.</p><p><strong>Methodology: </strong>Data from 1024 stroke patients who aged 18 years and above were prospectively collected in September 2022 in Zhengzhou, China. PROs included Self-Perceived Burden Scale, UCLA-Loneliness Scale, and Event-Related Rumination Inventory. Statistical methods employed included correlation analysis and mediation effect analysis.</p><p><strong>Results: </strong>A total of 1024 participants completed this survey (90.9%), with 56.2% males and a mean age of 62.22 (SD = 13.60) years. Approximately 84.28% of stroke patients felt moderate loneliness. Self-perceived burden was positively correlated with rumination (<i>r</i> = 0.516, 95% CI [0.460, 0.574]) and loneliness (<i>r</i> = 0.370, 95% CI [0.307, 0.431]). Rumination was also positively associated with loneliness (<i>r</i> = 0.493, 95% CI [0.443, 0.541]). Both intrusive and deliberate rumination served as mediators in the relationship between SPB and loneliness (<i>b</i> = 0.119, 55.09%, <i>b</i> = 0.031, 14.35%, respectively).</p><p><strong>Conclusion: </strong>Intrusive and deliberate rumination mediated the relationship between self-perceived burden and loneliness in stroke patients. Rumination in stroke patients should be emphasized as a modifiable factor to reduce loneliness and improve quality of life.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"105-115"},"PeriodicalIF":1.8,"publicationDate":"2025-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12301111/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144732632","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Agreement Between Child Self- and Parent Proxy-Reports of Health-Related Quality of Life in Spinal Muscular Atrophy: Preliminary Insights from a Nationwide Patient Registry in Germany.","authors":"Erik Landfeldt, Berenike Leibrock, Justine Hussong, Simone Thiele, Maggie C Walter, Eva Moehler, Michael Zemlin, Ulrich Dillmann, Marina Flotats-Bastardas","doi":"10.2147/PROM.S517362","DOIUrl":"10.2147/PROM.S517362","url":null,"abstract":"<p><strong>Objective: </strong>The objective of this pilot study was to examine the agreement between child self- and parent proxy-assessment of health-related quality of life (HRQoL) in spinal muscular atrophy (SMA) in the era of disease-modifying therapy.</p><p><strong>Methods: </strong>Children with SMA and one of their parents were recruited via the German national TREAT-NMD SMA patient registry. HRQoL was measured using the Pediatric Quality of Life Inventory 3.0 Neuromuscular Module (PedsQL 3.0 NMM), KIDSCREEN-27, and the Health Utilities Index (HUI). Agreement between child self- and parent proxy-ratings of ordinal measures was estimated using Cohen's κ, and for continuous measures using intraclass correlation coefficients (ICCs) from one-way random-effects models.</p><p><strong>Results: </strong>The final sample comprised 17 children with SMA (mean age: 9.88 years, SD: 4.33 years, range: 5-16 years; 59% female) and one of their parents. All but two patients (88%) were receiving disease-modifying therapy (nusinersen or risdiplam). The ICC for the total PedsQL 3.0 NMM score was estimated at 0.85 (95% CI: 0.64-0.94, p < 0.001) (indicative of excellent agreement). The corresponding estimate for the KIDSCREEN total score was 0.27 (95% CI: 0.00-0.75, p = 0.197) (poor/fair agreement) and the global HUI utility 0.98 (95% CI: 0.93 to 0.9952, p < 0.001) (excellent agreement). The lowest levels of concordance were found for school and family life, as well as mental well-being, as opposed to physical functioning and disability.</p><p><strong>Conclusion: </strong>We show that the agreement between child self- and parent proxy-reports of HRQoL in SMA varies markedly across HRQoL measures and examined domains, ranging from poor/fair to excellent. Compared with previous research, agreement for the PedsQL 3.0 NMM was markedly higher in our contemporary cohort of patients treated with novel therapies. These preliminary findings will be helpful in informing the design of future research of HRQoL in SMA.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"93-103"},"PeriodicalIF":1.8,"publicationDate":"2025-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12212432/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144541813","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Translation and Cross-Cultural Adaptation into French of the Mother-to-Infant Bonding Scale.","authors":"Anthony Pernoud, Alyx Taylor, Roberta De Luca, Roberto Marci, Elodie Timmins, Katherine Potter, Hugo Bothorel","doi":"10.2147/PROM.S524248","DOIUrl":"10.2147/PROM.S524248","url":null,"abstract":"<p><strong>Purpose: </strong>Patient-reported outcome measures (PROMs) are relevant for assessing the bond between a mother and her child, both before and after childbirth. Several questionnaires have been developed with the Mother-to-Infant Bonding Scale (MIBS) prominent among them, as it is a valid and easy-to-administer questionnaire owing to its length. Even though, this PROM has been adapted in Japanese, Indonesian and Swedish, it remains to be translated into French. The objective of this study was to translate and cross-culturally adapt the MIBS into French (MIBS-Fr).</p><p><strong>Patients and methods: </strong>The translation and cultural-adaptation of the questionnaire were performed following a 10-step process as recommended by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). During the cognitive debriefing, each item of the questionnaire was rated between 1 to 10 according to the comprehension level by 11 mothers.</p><p><strong>Results: </strong>The French version was very well understood with a mean level of comprehension of 9.7 ± 1.4 out of 10. The one-word descriptor used to express feelings in the original version was replaced by phrases in the French version for a better language adaptation. Slight modifications were made by the original developer, and back translations were found to be very consistent.</p><p><strong>Conclusion: </strong>This study reports the development of a French version of the MIBS (MIBS-Fr) following the ISPOR's recommendations for the translation and intercultural adaptation of a questionnaire. The MIBS-Fr provides French-speaking healthcare professionals with a practical and standardized tool to assess mother-to-infant bonding, facilitating early identification of bonding difficulties and supporting appropriate interventions in postpartum care.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"85-92"},"PeriodicalIF":1.8,"publicationDate":"2025-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12182736/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144369117","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pharmacist Led Telephonic Insulin Titration: A Pilot Study on A1C Control in a Family Medicine Residency.","authors":"Priya Raghavan, Shaunta Chamberlin, Robert Eric Heidel, Garland Anthony Wilson","doi":"10.2147/PROM.S502402","DOIUrl":"10.2147/PROM.S502402","url":null,"abstract":"","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"79-84"},"PeriodicalIF":1.8,"publicationDate":"2025-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11913038/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143649841","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychometric Validation of Patient Satisfaction Assessment Tool for Alternative Medicine Healthcare Facility in India.","authors":"Divya Taneja, Renu Mittal, Shashank Atrey, Nitu Barod, Shweta Singh, Niharika Jain, Roshni, Subhash Kaushik","doi":"10.2147/PROM.S506607","DOIUrl":"10.2147/PROM.S506607","url":null,"abstract":"<p><strong>Purpose: </strong>Good health status has traditionally been linked to greater levels of patient satisfaction. With the emergence and integration of various medical modalities, including Homeopathy, Ayurveda, and other complementary and alternative medical treatments, patients are likely to have different satisfaction levels during their interaction with different practitioners. Amidst this diversity, the efficacy, accessibility, and cost-effectiveness of these treatment approaches have garnered significant attention. Hence, a tool was developed to assess the patient's interaction with Complementary and Alternative Medicine treatments specifically Homeopathy. The objective of this study is to evaluate the validity and reliability of the Patient Satisfaction Assessment Tool (PSAT) developed to assess patient interaction with homeopathic outreach services.</p><p><strong>Methods: </strong>A new structured questionnaire was developed using a standardized procedure. After obtaining clearance from the scientific and ethics committee of the Central Council for Research in Homoeopathy, data was collected from consenting participants above 18 years of age attending the Dr. D.P. Rastogi Central Research Institute for Homoeopathy, Noida, Uttar Pradesh, India. Data was then computerized and analyzed by principal component analysis as the extraction method and orthogonal varimax as the rotation method.</p><p><strong>Results: </strong>A total of 285 participants were enrolled for psychometric validation, and 254 participants were included in the final analyses after exclusion. The mean (±Standard Deviation) age of participants was 37.63 (±12.9) years (range 18-79). The final 34-item questionnaire was arranged into nine domains as per rotated component matrix analysis. The overall internal consistency of the final questionnaire, as calculated by Cronbach's alpha, was 0.79, and the measure of sampling adequacy was 0.85 (Kaiser-Meyer-Olkin test).</p><p><strong>Conclusion: </strong>Initial results from the pilot tests suggest that Patient Satisfaction Assessment Tool (PSAT) is effective in capturing key aspects of patient satisfaction, from homeopathic clinical services which can guide future quality improvement initiatives in alternative medicine healthcare facilities.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"67-78"},"PeriodicalIF":1.8,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11847423/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143483610","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health-Related Quality of Life in Long COVID: Mapping the Condition-Specific C19-YRSm Measure Onto the EQ-5D-5L.","authors":"Adam B Smith, Darren C Greenwood, Paul Williams, Joseph Kwon, Stavros Petrou, Mike Horton, Thomas Osborne, Ruairidh Milne, Manoj Sivan","doi":"10.2147/PROM.S490870","DOIUrl":"10.2147/PROM.S490870","url":null,"abstract":"<p><strong>Background: </strong>Long COVID (LC) is a clinical syndrome with persistent, fluctuating symptoms subsequent to COVID-19 infection. LC has significant detrimental effects on health-related quality of life (HRQoL), activities of daily living (ADL), and work productivity. Condition-specific patient-reported outcome measures (PROMs), such as the modified COVID-19 Yorkshire Rehabilitation Scale (C19-YRSm) do not provide the health utility data required for cost-utility analyses of LC interventions. The aim of this study was to derive a mapping algorithm for the C19-YRSm to enable health utilities to be generated from this PROM.</p><p><strong>Methods: </strong>Data were collected from a large study evaluating LC services in the UK. A total of 1434 people with LC had completed both the C19-YRSm and the EQ-5D. Correlation and linear regression analyses were applied to determine items from the C19-YRSm and covariates for inclusion in the algorithm. Model fit, mean differences across the range of EQ-5D-3L utility scores, and Bland-Altman plots were evaluated. Responsiveness (standardised response mean; SRM) of the mapped utilities was investigated on a subset of participants with repeat assessments.</p><p><strong>Results: </strong>There was a strong level of association between 8 items and one domain on the C19-YRSm with the EQ-5D single-item dimensions. Model fit was good (R² = 0.7). The mean difference between observed and mapped scores was <0.10 for the range from 0 to 1 indicating good targeting for positive values of the EQ-5D-3L. The SRM for the mapped EQ-5D-3L was 0.37 compared to 0.17 for the observed utility scores, suggesting the mapped EQ-5D-3L is more responsive to change.</p><p><strong>Conclusion: </strong>A simple, responsive, and robust mapping algorithm was developed to generate enable EQ-5D-3L health utilities from the C19-YRSm. This will facilitate economic evaluations of LC interventions, treatment, and management, as well as further helping to describe and characterise patients with LC irrespective of any treatment and interventions.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"55-66"},"PeriodicalIF":1.8,"publicationDate":"2025-01-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11776923/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143067082","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluation of the Content Validity of the COVID-19 Symptoms Daily Diary.","authors":"Jennifer Dine, Yanfen Guan, Mirline Milien, Claire Ervin, Dana D Byrne, Michelle L Brown, Carisa De Anda, Josephine M Norquist","doi":"10.2147/PROM.S488914","DOIUrl":"10.2147/PROM.S488914","url":null,"abstract":"<p><strong>Introduction: </strong>The COVID-19 Symptoms Daily Diary (CSDD) is a patient-reported outcome measure designed to assess the severity of core COVID-19 symptoms in clinical trials. The preliminary version of the CSDD was developed based on regulatory guidance and the hallmark COVID-19 symptoms identified by the CDC. This study aimed to evaluate CSDD content validity, to determine whether it is fit for the purpose of supporting efficacy endpoints in clinical trials of treatments for COVID-19. This research also sought to evaluate the appropriateness of a newly developed Pre-COVID-19 Symptoms Questionnaire.</p><p><strong>Methods: </strong>A targeted literature review was completed to evaluate the relevance of concepts included in the diary and to identify any important symptoms that may have been missing. Hybrid (concept elicitation and cognitive debriefing) semistructured qualitative interviews were then conducted across 3 iterative rounds with 30 adults in the United States recently diagnosed with COVID-19.</p><p><strong>Results: </strong>The CSDD included concepts that were most frequently reported by interview participants, including those identified as most bothersome and most important to treat. During cognitive debriefing, participants described the diary concepts as salient and reported the instructions, recall period, and response options as clear and appropriate. Only 2 of 15 CSDD items were modified across 3 interview rounds; specifically, definitions for the vomiting and diarrhea frequency items were clarified for consistent interpretation and response. Interview participants also reported general ease in understanding and responding to the Pre-COVID-19 questionnaire, with feedback resulting in only minor changes to the reference period and instructions.</p><p><strong>Conclusion: </strong>The findings of the current study provide strong evidence for the content validity of the CSDD and the appropriateness of each of the items assessed. This rigorous evaluation (aligned with regulatory guidance) indicates that the CSDD is fit for the purpose of supporting efficacy endpoints in planned clinical trials evaluating medications for COVID-19 treatment.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"37-53"},"PeriodicalIF":1.8,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11748756/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009429","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Is It Time Alpha-1 Antitrypsin Deficiency Had a Specific Patient Reported Outcome Measure? A Review.","authors":"Joshua De Soyza, Hung-Yeh Chien, Adeola Ayodotun Onasanya, Alice M Turner","doi":"10.2147/PROM.S490849","DOIUrl":"10.2147/PROM.S490849","url":null,"abstract":"<p><p>Alpha-1 antitrypsin deficiency (AATD) is a rare cause of chronic lung and liver disease without its own patient reported-outcome measure (PROM). PROMs for Chronic Obstructive Pulmonary Disease (COPD) are commonly used instead, but AATD differs from COPD in several ways. We reviewed whether the PROMs used in the AATD literature adequately assess quality-of-life in these patients. 11 studies used PROMs as their primary outcomes; 21 included them as secondary outcomes. The St George's Respiratory Questionnaire (SGRQ) was the most commonly used PROM, used by 7 of the 11 primary outcome studies. Others included the COPD Assessment Tool, SF-36, LCOPD, EQ-5D, and the Chronic Respiratory Diseases Questionnaire. Several studies assessed SGRQ as being associated with respiratory disease severity as measured by FEV1% predicted, exacerbation rate, oxygen use and exercise tolerance. However, no studies used PROMs which included assessment of liver-related symptoms, other extra-pulmonary manifestations of AATD, or concerns related to genetics or finances. These factors are likely to have an impact on quality of life in AATD. A specific AATD-PROM is therefore required to holistically address the quality of life effects of an AATD diagnosis.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"16 ","pages":"23-35"},"PeriodicalIF":1.8,"publicationDate":"2025-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11742593/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009430","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}