Patient Related Outcome Measures最新文献

{"title":"Studies on Reliability and Measurement Error of Measurements in Medicine - From Design to Statistics Explained for Medical Researchers.","authors":"Lidwine B Mokkink,&nbsp;Iris Eekhout,&nbsp;Maarten Boers,&nbsp;Cees P M van der Vleuten,&nbsp;Henrica C W de Vet","doi":"10.2147/PROM.S398886","DOIUrl":"https://doi.org/10.2147/PROM.S398886","url":null,"abstract":"<p><p>Reliability and measurement error are measurement properties that quantify the influence of specific sources of variation, such as raters, type of machine, or time, on the score of the individual measurement. Several designs can be chosen to assess reliability and measurement error of a measurement. Differences in design are due to specific choices about which sources of variation are varied over the repeated measurements in stable patients, which potential sources of variation are kept stable (ie, restricted), and about whether or not the entire measurement instrument (or measurement protocol) was repeated or only part of it. We explain how these choices determine how intraclass correlation coefficients and standard errors of measurement formulas are built for different designs by using Venn diagrams. Strategies for improving the measurement are explained, and recommendations for reporting the essentials of these studies are described. We hope that this paper will facilitate the understanding and improve the design, analysis, and reporting of future studies on reliability and measurement error of measurements.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"193-212"},"PeriodicalIF":2.1,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/77/25/prom-14-193.PMC10336232.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9820478","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 Instruments for Clinical Trials in AL Amyloidosis: Report from the Amyloidosis Forum HRQOL Working Group.","authors":"Avery A Rizio,&nbsp;Michelle K White,&nbsp;Anita D'Souza,&nbsp;Kristen Hsu,&nbsp;Paula Schmitt,&nbsp;Tiffany P Quock,&nbsp;James Signorovitch,&nbsp;Isabelle Lousada,&nbsp;Vaishali Sanchorawala","doi":"10.2147/PROM.S399658","DOIUrl":"https://doi.org/10.2147/PROM.S399658","url":null,"abstract":"<p><p>Systemic AL (light chain) amyloidosis is a rare protein misfolding disorder associated with plasma cell dyscrasia affecting various organs leading to organ dysfunction and failure. The Amyloidosis Forum is a public-private partnership between the Amyloidosis Research Consortium and the US Food and Drug Administration Center for Drug Evaluation and Research with the goal of accelerating the development of effective treatments for AL amyloidosis. In recognition of this goal, 6 individual working groups were formed to identify and/or provide recommendations related to various aspects of patient-relevant clinical trial endpoints. This review summarizes the methods, findings, and recommendations of the Health-Related Quality of Life (HRQOL) Working Group. The HRQOL Working Group sought to identify existing patient-reported outcome (PRO) assessments of HRQOL for use in clinical trials and practice deemed relevant across a broad spectrum of patients with AL amyloidosis. A systematic review of the AL amyloidosis literature identified 1) additional signs/symptoms not currently part of an existing conceptual model, and 2) relevant PRO instruments used to measure HRQOL. The Working Group mapped content from each identified instrument to areas of impact in the conceptual model to determine which instrument(s) provide coverage of relevant concepts. The SF-36v2^® Health Survey (SF-36v2; QualityMetric Incorporated, LLC) and Patient-Reported Outcomes Measurement Information System-29 Profile (PROMIS-29; HealthMeasures) were identified as instruments relevant to patients with AL amyloidosis. Existing evidence of reliability and validity was evaluated with a recommendation for future work focused on estimating clinically meaningful within-patient change thresholds for these instruments. For sponsors, the context of use-including specific research objectives, trial population, and investigational product under study-should inherently drive selection of the appropriate PRO instrument and endpoint definitions to detect meaningful change and enable patient-focused drug development.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"153-169"},"PeriodicalIF":2.1,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/6e/58/prom-14-153.PMC10202704.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9530812","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":"Methodological Quality of PROMs in Psychosocial Consequences of Colorectal Cancer Screening: A Systematic Review.","authors":"Emma Grundtvig Gram,&nbsp;Jessica Á Rogvi,&nbsp;Anders Heiberg Agerbeck,&nbsp;Frederik Martiny,&nbsp;Anne Katrine Lykke Bie,&nbsp;John Brandt Brodersen","doi":"10.2147/PROM.S394247","DOIUrl":"https://doi.org/10.2147/PROM.S394247","url":null,"abstract":"<p><strong>Objective: </strong>This systematic review aimed to assess the adequacy of measurement properties in Patient-Reported Outcome Measures (PROMs) used to quantify psychosocial consequences of colorectal cancer screening among adults at average risk.</p><p><strong>Methods: </strong>We searched four databases for eligible studies: MEDLINE, CINAHL, PsycINFO, and Embase. Our approach was inclusive and encompassed all empirical studies that quantified aspects of psychosocial consequences of colorectal cancer screening. We assessed the adequacy of PROM development and measurement properties for content validity using The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) risk of bias checklist.</p><p><strong>Results: </strong>We included 33 studies that all together used 30 different outcome measures. Two PROMs (6.7%) were developed in a colorectal cancer screening context. COSMIN rating for PROM development was inadequate for 29 out of 30 PROMs (97%). PROMs lacked proper cognitive interviews and pilot studies and therefore had no proven content validity. According to the COSMIN checklist, 27 out of 30 PROMs (90%) had inadequate measurement properties for content validity.</p><p><strong>Discussion: </strong>The majority of included PROMs had inadequate development and measurement properties. These findings shed light on the trustworthiness of the included studies' findings and call for reevaluation of existing evidence on the psychosocial consequences of colorectal cancer screening. To provide trustworthy evidence about the psychosocial consequences of colorectal cancer screening, editors could require that studies provide evidence of the methodological quality of the PROM. Alternatively, authors should transparently disclose their studies' methodological limitations in measuring psychosocial consequences of screening validly.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"31-47"},"PeriodicalIF":2.1,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/83/86/prom-14-31.PMC10024469.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9508782","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":"Exploring Disease Remission and Bowel Urgency Severity Among Adults with Moderate to Severe Ulcerative Colitis: A Qualitative Study.","authors":"Marla C Dubinsky, Louise Newton, Laure Delbecque, Theresa Hunter, Aiste Guobyte, April N Naegeli, Shehan McFadden, Jill Donaldson, Tara Symonds, James D Lewis","doi":"10.2147/PROM.S378759","DOIUrl":"10.2147/PROM.S378759","url":null,"abstract":"<p><strong>Purpose: </strong>Achieving and maintaining symptom control is a key treatment goal in ulcerative colitis (UC). Bowel urgency is an important symptom of UC, thus measurement of urgency is critical. This research explored the patient experience of UC and \"remission\" in UC, with a focus on urgency, and cognitively debriefed the Urgency Numeric Rating Scale (NRS), including score interpretation and examination of meaningful improvement.</p><p><strong>Patients and methods: </strong>Semi-structured hybrid concept elicitation and cognitive debriefing interviews with adults with moderately-severely active UC were conducted to explore experiences of UC and urgency, as well as examine meaningful improvement and score interpretation of the Urgency NRS. Purposive sampling was used to identify 20 eligible adult participants with UC. Concept elicitation data were analyzed using thematic analysis, and a deductive approach was used to analyze cognitive debriefing data. Thematic analysis was also applied to meaningful change-related data.</p><p><strong>Results: </strong>Twenty participants were interviewed (average age = 42.6 years old, 50% male); 14 with moderately active (70.0%) and 6 with severely active UC (30.0%). Disease remission was not consistently defined by participants and description varied in terms of definition (absence vs not complete absence of symptoms), duration (months vs days) and key symptoms to consider. Urgency was a prominent symptom for all participants, with 8 (40.0%) identifying it as the most bothersome aspect of UC. No issues were identified with the Urgency NRS. Participants were able to define different levels of urgency severity, describe how they relate to daily life impacts, and score them differently on the Urgency NRS. Participants were also able to reflect urgency improvement on the NRS and discuss how small changes in numeric ratings of urgency can reflect meaningful change in the symptom burden of their UC.</p><p><strong>Conclusion: </strong>The Urgency NRS is a content valid and interpretable measure to assess bowel urgency severity.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"13 ","pages":"287-300"},"PeriodicalIF":2.1,"publicationDate":"2022-12-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/32/61/prom-13-287.PMC9793422.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10459497","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":"Assessing the Validity of the Long-Term Conditions Questionnaire (LTCQ) in Women During Pregnancy and the First Year Following Birth","authors":"L. Kelly, Ray Fitzpatrick, J. Kurinczuk, O. Rivero-Arias, F. Alderdice","doi":"10.2147/PROM.S376070","DOIUrl":"https://doi.org/10.2147/PROM.S376070","url":null,"abstract":"Background The aim of this study was to validate a generic patient-reported outcome measure, the Long-Term Conditions Questionnaire (LTCQ), among pregnant and postpartum women living with a pre-existing long-term condition (LTC). Methods Cognitive interviews were conducted with women who were currently pregnant or had given birth within the past year and living with a pre-existing LTC (n=11) and with healthcare professionals working in maternal care (n=11) to explore the acceptability of LTCQ items. An online survey was subsequently administered among women who were pregnant or had given birth within the past year and living with a pre-existing LTC (n=718). Tests of validity were performed including assessing correlations between the LTCQ and reference measures, the Well-being in Pregnancy (WiP) Questionnaire and the EuroQol EQ-5D-5L. Internal consistency was assessed using the Cronbach’s alpha statistic. Results All LTCQ items were considered relevant and appropriate for use with women who were pregnant or had given birth within the past year. The most commonly reported LTC among the online survey sample (n=718) was a mental health condition (n=350, 48.7%) followed by joint, bone and connective tissues (n= 212, 29.5%) and gastrointestinal (n=143, 19.9%) condition. Data indicated LTCQ scores behaved in a predictable pattern, demonstrating poorer scores for women reporting a greater number of LTCs; mean (SD) scores, one LTC= 61.86 (17.8), two LTCs= 55.29 (16.0), three LTCs= 49.84 (15.52) and four LTCs= 44.94 (12.2). Poorer scores were also reported for women living with at least one mental health condition compared to those reporting no mental health condition, mean score = 66.18 (SD 16.7) v 48.64 (SD 13.3), p<0.001 respectively. As anticipated, LTCQ scores demonstrated significant correlations in the expected direction with both the EQ-5D-5L and WiP scores. For all LTCQ items, the Cronbach’s alpha statistic was 0.93. Conclusion Data presented here indicate that the LTCQ, which assesses living well with one or more LTC, is suitable for use among pregnant and postpartum women, from both the woman’s perspective and from the perspectives of maternity healthcare professionals. Use of the LTCQ would facilitate the identification of unmet needs within this high-risk cohort and support the exploration of how LTCs may affect women throughout the pregnancy and post-natal period. Understanding unmet needs within this cohort of women provides an opportunity to link up specialist care within maternity services and enhance personalised care.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"13 1","pages":"221 - 228"},"PeriodicalIF":2.1,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43884318","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Validity of Clinical Scoring in the Diagnosis of Stroke Subtype: Validation Study","authors":"B. Mekonnen, M. Kebede","doi":"10.2147/PROM.S374473","DOIUrl":"https://doi.org/10.2147/PROM.S374473","url":null,"abstract":"Background The gold standard for distinguishing stroke subtype is non-contrast CT. However, it’s still prohibitively expensive and out of reach for the majority of recourse-constrained settings. Clinically, not all patients will have a definite diagnosis of hemorrhagic/ischemic stroke. To overcome these challenges and improve clinical bedside diagnosis, clinical stroke scores for stroke subtypes have been developed and recommended to be used in the absence of appropriate imaging modality. Methods We conducted a prospective cross-sectional study among stroke patients to compare the accuracy of level of clinical stroke score methods in differentiating stroke type with CT. it was conducted on 140 people at MTU teaching hospital in Bench-Sheko Zone, South-west Ethiopia. Data were collected using check list. Analysis of the data was done using SPSS version 24. Results Our result revealed an incidence of hemorrhagic stroke were 50%, ischemic stroke were 48.6% by CT evaluation. Specificity, sensitivity, positive predictive value, negative predictive value and the overall accuracy of Siriraj stroke score for differentiation of hemorrhage from ischemic stroke were 68.6%, 83.9%, 74.6%, 79.5%, and 82% respectively, the Guys score were 89.7%, 47.8%, 73.3%, 74.5% and 74.5% respectively and while the Bensson score were 88.6%, 35.3%, 75%, 58.5%, and 62.3% respectively. Conclusion We conclude that Siriraj stroke score showed good sensitivity and fair overall accuracy for hemorrhagic stroke even if it had poor specificity.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"13 1","pages":"209 - 219"},"PeriodicalIF":2.1,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48275362","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Potential Role of Dental Patient-Reported Outcomes (dPROs) in Evidence-Based Prosthodontics and Clinical Care: A Narrative Review.","authors":"Cláudio Rodrigues Leles, Jésio Rodrigues Silva, Thalita Fernandes Fleury Curado, Martin Schimmel, Gerald McKenna","doi":"10.2147/PROM.S256724","DOIUrl":"10.2147/PROM.S256724","url":null,"abstract":"<p><p>Oral health problems are associated with poor quality of life, with the potential to cause functional, aesthetic, nutritional, and psychological difficulties, in addition to pain and suffering. Traditionally, dental treatment outcomes are measured using purely clinical parameters; however, this may be ineffective as these parameters cannot adequately capture the full impact of poor oral health on the patient, or their respective coping strategies. From this perspective, there are significant benefits when the patient's perception of their care is considered, and included in treatment planning and delivery. The impacts perceived by the patient on their treatment outcomes can be measured using patient-reported outcomes (PROS), or more specifically with dPROS, focused on dental patient-reported outcomes. Although there are some instruments available for measuring these outcomes in clinical trials, very little information is available for explaining the context in which these outcomes are considered, and also how to capture this information using appropriate instruments, specially in evidence-based dental practice. This article aims to review the literature, seeking to describe what has been considered about assessing patient's outcomes, as well as how to measure them, and explore the potential benefits of using dPROS in evidence-based prosthodontics and clinical care of partially and fully edentulous patients.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"13 1","pages":"131-143"},"PeriodicalIF":1.8,"publicationDate":"2022-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9172924/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46972083","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 Inclusion of Patients' Reported Outcomes to Inform Treatment Effectiveness Measures in Opioid Use Disorder. A Systematic Review.","authors":"Nitika Sanger, Balpreet Panesar, Michael Dennis, Tea Rosic, Myanca Rodrigues, Elizabeth Lovell, Shuling Yang, Mehreen Butt, Lehana Thabane, Zainab Samaan","doi":"10.2147/PROM.S297699","DOIUrl":"10.2147/PROM.S297699","url":null,"abstract":"<p><strong>Introduction: </strong>Patient centred care is needed now more than ever in the treatment of opioid use disorder. Trials, policy makers, and service providers have most often used treatment retention and opioid urine screens as measures of treatment effectiveness. However, patients receiving medication for opioid use disorder treatment (MOUD) may prioritise the use of different ways to assess treatment success.</p><p><strong>Objective: </strong>The aim of this review is to synthesize literature examining the self-reported goals patients would like to achieve in MOUD for opioid use disorder.</p><p><strong>Methods: </strong>We searched MEDLINE, EMBASE, PsycINFO, Cumulative Index to Nursing and Allied Health Literature, Web of Science, Cochrane Library, Cochrane Clinical Trials Registry, the National Institutes for Health Clinical Trials Registry, and the WHO International Clinical Trials Registry Platform from inception until April 30th, 2021. No restrictions were placed on language, age, or type of MOUD. A qualitative synthesis is presented given that a meta-analysis was not possible.</p><p><strong>Results: </strong>The search yielded a total of 21,082 records from which 8 met criteria for inclusion in the qualitative synthesis. We identified a total of 43 patient-reported treatment goals from the 8 studies. Twelve domains were created from the 43 goals reported. These domains cover a range of important areas for patients' goals related to living a normal life, physical health, mental health, treatment, and substance use specific areas.</p><p><strong>Conclusion: </strong>This review highlights several patient goals that they would like to achieve during treatment for opioid use disorder that are not commonly considered as markers of treatment effectiveness. Goals related to health, living a normal life, and overall substance use concerns by patients should be taken into consideration by clinical trialists, researchers, policy makers, service providers, patients, and communities engaged in developing and tailoring treatment plans for opioid use disorder.</p><p><strong>Systematic review registration: </strong>PROSPERO CRD42018095553.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"13 1","pages":"113-130"},"PeriodicalIF":1.8,"publicationDate":"2022-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9165704/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49280323","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":"Evaluating the Impact of Therapy on Quality of Life in Type 2 Diabetes: A Literature Review of Utilities Associated with Treatment-Related Attributes","authors":"W. Valentine, K. Norrbacka, K. Boye","doi":"10.2147/PROM.S322390","DOIUrl":"https://doi.org/10.2147/PROM.S322390","url":null,"abstract":"Introduction Treatment-related attributes and process characteristics such as dosing frequency, timing flexibility, ease of use of injection devices and unpleasant side-effects may have small but measurable effects on quality of life (QoL) in people with type 2 diabetes (T2D). A literature review was performed to identify recently published utility values quantifying the effect of treatment-related attributes on QoL. Methods Literature search strategies were designed using high-level medical subject heading (MeSH) terms supplemented with free-text terms and searches were run in March 2020 in the PubMed, Embase and Cochrane Library databases. For inclusion, studies were required to be published in full-text form, in English, since 2010 and report utility values (elicited using either direct or indirect methods) for treatment-related attributes or process characteristics including side effects, change in weight/body mass index (BMI), dosing frequency and timing flexibility, device attributes (e.g. needle handling, requirement for reconstitution) and convenience (e.g. waiting time). Results A total of 30 studies were included in the review, of which all but three were conducted in people with T2D. The EQ-5D was the most commonly used elicitation method (fourteen studies), followed by time tradeoff (TTO) methodology. Treatment-related adverse events and inconveniences such as needle handling in administration devices and waiting time were consistently associated with lower QoL, whereas lower dosing frequency and increased timing flexibility with dosing were consistently associated with utility benefits. The relationship between change in BMI and QoL was non-linear and influenced by baseline BMI. Conclusion Treatment-related attributes and process characteristics are associated with minor changes in QoL, which should be taken into account in long-term health economic modeling of new treatments and administration devices.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"13 1","pages":"97 - 111"},"PeriodicalIF":2.1,"publicationDate":"2022-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45521946","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient-Reported Outcomes in Rheumatoid Arthritis: A Key Consideration for Evaluating Biosimilar Uptake?","authors":"Gabriel Horta-Baas","doi":"10.2147/PROM.S256715","DOIUrl":"10.2147/PROM.S256715","url":null,"abstract":"<p><strong>Purpose: </strong>This review aims to provide an overview of the impact of TNFis biosimilars, with marketing authorization, in patient-reported outcome measures (PROMs) scores and explore how PROMs endpoints might add value in biosimilars uptake in RA patients.</p><p><strong>Patients and methods: </strong>A comprehensive search of Medline, Scopus, Lilacs, and CINAHL databases was performed for papers published between January 2012 and December 2021. For inclusion, studies had to be prospective, published in a peer-reviewed journal, published in English or Spanish language; studies using PROMs as an outcome measure. After screening title and abstracts and assessing the remaining full texts fulfilling the inclusion criteria, 31 papers were used in this narrative review.</p><p><strong>Results: </strong>PROMs were used as secondary outcomes in included studies. The most frequently employed domains to assess biosimilar efficacy include physical function, patient global assessment (PtGA), health-related quality of life (HRQoL), and fatigue. The results of randomized clinical trials uniformly showed that mean change in PROMs scores is comparable between biosimilar and reference biologic treatment groups. However, open-label and real-world studies revealed high rates of discontinuation of therapy, mainly for subjective worsening of disease activity or non-specific adverse events. Even without objective clinical evidence of inflammation, patients who are considered to have active disease (higher scores on PtGA) have higher discontinuation rates of biosimilars. The available information suggests that the nocebo effect is the most likely cause for the discontinuation of biosimilars.</p><p><strong>Conclusion: </strong>There is scarce literature surrounding the impact of biosimilars in PROMs, especially in open-label studies. In real-life studies, biosimilars have a higher discontinuation rate than reference products. TNFis biosimilars treatment efficacy in RA depends on disease activity and other factors such as PtGA and fatigue. The nocebo effect is the best explanation for biosimilar's discontinuation.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"13 1","pages":"79-95"},"PeriodicalIF":1.8,"publicationDate":"2022-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8977480/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47718087","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}