Patient Related Outcome Measures最新文献

{"title":"Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy.","authors":"Jennifer Quinn,&nbsp;Kathleen A Delaney,&nbsp;Wing Yen Wong,&nbsp;Wolfgang Miesbach,&nbsp;Monika Bullinger","doi":"10.2147/PROM.S357555","DOIUrl":"https://doi.org/10.2147/PROM.S357555","url":null,"abstract":"<p><strong>Purpose: </strong>The hemophilia-specific health-related quality of life (HRQOL) questionnaire (Haemo-QOL-A) is validated for detecting QOL changes following standard therapy for hemophilia A, but has not been rigorously evaluated after gene therapy. This post hoc analysis evaluated the psychometric properties of Haemo-QOL-A in adult people with severe hemophilia A (PWSHA) receiving valoctocogene roxaparvovec (AAV5-hFVIII-SQ) in 2 clinical trials (phase 1/2, NCT02576795; phase 3, NCT03370913).</p><p><strong>Patients and methods: </strong>Adult PWSHA (factor VIII levels ≤1 IU/dL) received 1 AAV5-hFVIII-SQ infusion (6×10¹³ vg/kg). Participants were assessed using the Haemo-QOL-A and the EuroQOL (EQ)-5D-5L and visual analog scale (VAS) questionnaires pre- and post-infusion. Psychometric analyses included convergent and discriminant validity, internal consistency, and reliability. Clinically important difference (CID) was estimated using 3-point change in EQ-5D-5L VAS as anchor.</p><p><strong>Results: </strong>Haemo-QOL-A data were analyzed from 7 (phase 1/2, 3-year follow-up) and 16 participants (phase 3, 26-week analysis). Change in Haemo-QOL-A Total Scores correlated with EQ-5D-5L VAS score change at 26 weeks (Pearson's correlation 0.77). At 26 weeks, increased Haemo-QOL-A Physical Functioning was associated with decreased EQ-5D-5L Pain and Discomfort and decreased Anxiety and Depression (Spearman's Rank correlations -0.73 and -0.62, respectively, <i>P</i> <0.01). Internal consistency analysis showed good reliability for all domains (Cronbach's alpha >0.7) except Treatment Concern (Cronbach's alpha = 0.31). Anchor-based CID estimates were met for Haemo-QOL-A Total Score (≥5.5) and domain scores (≥6) for Consequences of Bleeding, Physical Functioning, Role Functioning, and Worry.</p><p><strong>Conclusion: </strong>Our preliminary results suggest that the Haemo-QOL-A is a valid, reliable instrument for HRQOL assessment in PWSHA undergoing gene therapy. Future research should be undertaken to confirm these findings in a larger number of participants.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/8f/95/prom-13-169.PMC9307866.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40539666","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":"Patients' Experiences with Management of Benign Paroxysmal Positional Vertigo: Insights from the Vestibular Disorders Association Registry.","authors":"Colin R Grove,&nbsp;Wagner Henrique Souza,&nbsp;Patricia L Gerend,&nbsp;Cynthia A Ryan,&nbsp;Michael C Schubert","doi":"10.2147/PROM.S370287","DOIUrl":"https://doi.org/10.2147/PROM.S370287","url":null,"abstract":"<p><strong>Introduction: </strong>Benign paroxysmal positional vertigo (BPPV) is the most frequently occurring peripheral vestibular disorder. Clinical practice guidelines (CPG) for BPPV exist; however, little is known about how affected patients perceive their condition is being managed. We aimed to leverage registry data to evaluate how adults who report BPPV are managed.</p><p><strong>Material and methods: </strong>We retrospectively analyzed of data from 1,262 adults (58.4 ± 12.6 years old, 81.1% female, 91.1% White) who were enrolled in the Vestibular Disorders Association Registry from 2014 to 2020. The following patient-reported outcomes were analyzed by proportions for those who did and did not report BPPV: symptoms experienced, falls reported, diagnostics undertaken, interventions received (eg, canalith repositioning maneuvers [CRMs], medications), and responses to interventions.</p><p><strong>Results: </strong>Of the 1,262 adults included, 26% reported being diagnosed with BPPV. Many adults who reported BPPV (83%) also endorsed receiving additional vestibular diagnoses or may have had atypical BPPV. Those with BPPV underwent magnetic resonance imaging and were prescribed medications more frequently than those without BPPV (76% vs 57% [χ²=36.51, p<0.001] and 85% vs 78% [χ²=5.60, p=0.018], respectively). Falls were experienced by similar proportions of adults with and without BPPV (55% vs 56% [χ²⁼=11.26, p=0.59]). Adults with BPPV received CRMs more often than those without BPPV (86% vs 48%, χ²=127.23, p<0.001). More registrants with BPPV also endorsed benefit from CRMs compared to those without BPPV (51% vs 12% [χ²=105.30, p<0.001]).</p><p><strong>Discussion: </strong>In this registry, BPPV was often reported with other vestibular disorders. Healthcare utilization was higher than would be expected with care based on the CPG. The rates of falls in those with and without BPPV are higher than previously reported. Adults with BPPV reported significant differences in how their care is managed and their overall outcomes compared to those without BPPV.</p><p><strong>Conclusion: </strong>Patient-reported outcomes provide useful information regarding the lived experience of adults with BPPV.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/be/0f/prom-13-157.PMC9271286.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40516321","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":"Predictive Value of the <i>(Quick)</i>DASH Tool for Upper Extremity Dysfunction Following Percutaneous Coronary Intervention.","authors":"Eva Zwaan,&nbsp;Elena Cheung,&nbsp;Alexander IJsselmuiden,&nbsp;Carlo Holtzer,&nbsp;Ton Schreuders,&nbsp;Marcel Kofflard,&nbsp;Marco Alings,&nbsp;J Henk Coert","doi":"10.2147/PROM.S353895","DOIUrl":"https://doi.org/10.2147/PROM.S353895","url":null,"abstract":"<p><strong>Purpose: </strong>The use of the Disability of the Arm, Shoulder and Hand (DASH) questionnaire and its shortened version, the <i>Quick</i>DASH, have been used to assess upper extremity function following a transradial percutaneous coronary intervention (TR-PCI). However, the use of these scores has not yet been validated for TR-PCI induced complications in the upper extremity. The aim of this study was to establish the validity of the DASH and the <i>Quick</i>DASH, for the assessment of upper extremity dysfunction following a transradial percutaneous coronary intervention (TR-PCI).</p><p><strong>Patients and methods: </strong>This study was a diagnostic retrospective analysis of the ARCUS study, of whom 440 underwent TR-PCI and 62 control patients were treated via the transfemoral approach. All participants completed the DASH and <i>Quick</i>DASH questionnaire prior to the procedure and at each follow-up visit up to six months of follow-up. Receiver operating characteristics (ROC) were constructed to determine the validity of the questionnaires, using physical examinations to determine the occurrence of upper extremity dysfunction, according to the ARCUS study.</p><p><strong>Results: </strong>At each follow-up moment, the area under the curve (AUC) showed a poor ability of the DASH and <i>Quick</i>DASH to discriminate between patients with and without upper extremity dysfunction (AUC: 0.565-0.586). There was no significant difference between the questionnaires (p > 0.05).</p><p><strong>Conclusion: </strong>The DASH and <i>Quick</i>DASH questionnaires are both equally incapable of discriminating between patients with and without upper extremity dysfunction following a TR-PCI. Study results suggest that the DASH and <i>Quick</i>DASH questionnaires are incapable of discerning changes in upper extremity function as a result of procedural complications following a TR-PCI vs cardiac induced activity cessation.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/06/83/prom-13-145.PMC9249092.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40559418","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 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":null,"pages":null},"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":null,"pages":null},"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":null,"pages":null},"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":null,"pages":null},"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}

{"title":"How to Improve Interpretability of Patient-Reported Outcome Measures for Clinical Use: A Perspective on Measuring Abilities and Feelings","authors":"J. Kopec","doi":"10.2147/PROM.S355679","DOIUrl":"https://doi.org/10.2147/PROM.S355679","url":null,"abstract":"Abstract Two general classes of concepts measured by patient-reported outcome measures (PROMs) are abilities and feelings. Over the past several decades, there has been a significant progress in measuring both. Nevertheless, current multi-item scales are subject to criticism related to scale length, score dimensionality, interpretability, cultural bias, and insufficient detail in measuring specific domains. To address some of these issues, the author offers an alternative perspective on how questions about abilities and feelings could be formulated. Abilities can be defined in terms of a relationship between the level of performance and the associated perception of difficulty, and represented graphically by an ability curve. For feelings, it may be useful to measure frequency and intensity jointly to determine the proportion of time in each level of intensity. The resultant frequency × intensity matrix can be presented as a bar graph. Empirical data to support the feasibility and validity of these approaches to PROM design are provided, potential advantages and limitations are discussed, and some future research avenues are suggested.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43028294","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 Outcome Measures in Adult Patients Diagnosed with Epilepsy Being Treated with Perampanel.","authors":"Brian D Moseley,&nbsp;Shaloo Gupta,&nbsp;Nate Way,&nbsp;Jonathon Wright,&nbsp;John C Rowland,&nbsp;Victoria E Barghout,&nbsp;Feride Frech,&nbsp;Craig Plauschinat","doi":"10.2147/PROM.S343302","DOIUrl":"https://doi.org/10.2147/PROM.S343302","url":null,"abstract":"<p><strong>Background: </strong>Epilepsy is a complex disorder that can affect patients' medical, psychological, and social well-being. The purpose of this study was to evaluate the patient-reported outcome (PRO) measures of health-related quality of life (HRQoL), satisfaction, and adherence in adult patients diagnosed with epilepsy treated with perampanel in the United States (US).</p><p><strong>Methods: </strong>A US-based, multicenter, observational cross-sectional survey was completed by 61 patients taking perampanel with or without other antiseizure medications (ASMs). Respondents were ≥18 years old, had a physician-confirmed diagnosis of epilepsy, used perampanel for ≥4 months, and provided informed consent. Patients responded to questions concerning their demographic characteristics, treatment history, experiences before perampanel, experiences while taking perampanel, HRQoL, treatment satisfaction, and medication adherence.</p><p><strong>Results: </strong>Patients (N=61) were 42.8 years old on average; majority were female (63.9%) and white (75.4%). Mean time on perampanel was 2.5 years, with sodium channel blockers often (55.7%) used concomitantly with perampanel. Patients reported, on average, 5.5 (standard deviation [SD]=13.2) seizures/month after initiating perampanel, whereas these same patients reported experiencing 20.4 (SD=60.0) seizures/month prior to perampanel. When comparing their experience on perampanel with their experience with previous ASMs, more patients \"strongly agreed\" that perampanel allowed them to live a more normal life (36.1% vs 27.5%) and worked as intended if they missed taking a dose (16.4% vs 7.8%). Average satisfaction scores were high, with ratings of 71.8 for effectiveness, 84.0 for convenience, and 71.9 for global satisfaction (0-100 scores). Perampanel use was associated with improvements in HRQoL and fewer symptoms of depression and anxiety. The majority of patients were adherent (62.3%) to perampanel.</p><p><strong>Discussion: </strong>Perampanel use was associated with reductions in number of seizures, better HRQoL, and high adherence rates. These results provide initial evidence that perampanel can be an effective, tolerable, and valid option for patients with epilepsy in the real world.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/fa/6d/prom-13-39.PMC8841652.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39638048","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":"Patient and Caregiver Insights into the Disease Burden of Myelodysplastic Syndrome.","authors":"John Soper,&nbsp;Islam Sadek,&nbsp;Alyson Urniasz-Lippel,&nbsp;Deborah Norton,&nbsp;Marina Ness,&nbsp;Ruben Mesa","doi":"10.2147/PROM.S346434","DOIUrl":"https://doi.org/10.2147/PROM.S346434","url":null,"abstract":"<p><p>A diagnosis of myelodysplastic syndrome (MDS) is typically unexpected and can be difficult for patients to grasp. Not only is MDS a complicated disease to understand, which can contribute to stress and anxiety, but it also has an uncertain prognosis, which can be emotionally paralyzing. Not surprisingly, emotional distress and the symptom burden of MDS, including extreme fatigue due to cytopenias, negatively impact a patient's quality of life (QOL). Studies have shown that patient-centered care-including greater physician understanding of the disease burden their patients experience, discussing and establishing agreed-on treatment goals, and including patients in the decision-making process about their care-may help improve patient QOL. To better understand patient and caregiver experiences with MDS and how the disease impacts QOL, a small survey was conducted of patients with MDS or leukemia and their caregivers on an online health network. Among the 30 respondents who completed the survey, four had MDS and one was a caregiver for a patient with MDS. Here we focus on the five MDS respondents and contextualize the findings with personal experiences from a patient and physician perspective. The patient perspective was provided by John Soper, PhD, DABCC, who was diagnosed with MDS in 2019. Dr Soper is a retired board-certified clinical chemist and a member of the MDS Foundation. The physician perspective was provided by Dr Ruben Mesa, Executive Director of the Mays Cancer Center at UT Health San Antonio MD Anderson. The survey responses and the accompanying patient and physician perspectives highlight the importance of open communication between patients and their healthcare provider to better serve those with MDS and improve their QOL.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":null,"pages":null},"PeriodicalIF":2.1,"publicationDate":"2022-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/54/df/prom-13-31.PMC8824781.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39620378","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}