Patient Related Outcome Measures最新文献

{"title":"The Pattern of Admission, Clinical Characteristics, and Outcomes Among Patients Admitted to the Intensive Care Unit of a Tertiary Hospital in Tanzania: A 5-Year Retrospective Review","authors":"Nadeem Kassam, Philip Adebayo, Iris Matei, Eric L. Aghan, Samina S Somji, Samwel Kadelya, Yasson Abha, Frank Swai, Mangaro Mabusi, Kamran Hameed, Hanifa Mbithe, Alyyah Thawer, Mandela Makakala, Fatma Bakshi, Harrison Chuwa, M. Ng'wanasayi, C. Wambura, R. Mvungi, James Orwa, Munish Sharma, G. Udeani, S. Surani","doi":"10.2147/PROM.S441293","DOIUrl":"https://doi.org/10.2147/PROM.S441293","url":null,"abstract":"Introduction Despite the implementation of complex interventions, ICU mortality remains high and more so in developing countries. The demand for critical care in Sub-Saharan Africa is more than ever before as the region experiences a double burden of rising rates of non-communicable diseases (NCD) in the background battle of combating infectious diseases. Limited studies in Tanzania have reported varying factors associated with markedly high rates of ICU mortality. Investigating the burden of ICU care remains crucial in providing insights into the effectiveness and challenges of critical care delivery. Material and Methods A single-center retrospective study that reviewed records of all medically admitted patients admitted to the ICU of the Aga Khan Hospital, Dar-es-Salaam, from 1st October 2018 to 30th April 2023. To define the population in the study, we used descriptive statistics. Patients’ outcomes were categorized based on ICU survival. Binary logistic regression was run (at 95% CI and p-value < 0.05) to identify the determinants for ICU mortality. Results Medical records of 717 patients were reviewed. The cohort was male (n=472,65.8%) and African predominant (n=471,65.7%) with a median age of 58 years (IQR 45.0–71.0). 17.9% of patients did not survive. The highest mortality was noted amongst patients with septic shock (29.3%). The lowest survival was noted amongst patients requiring three organ support (n=12,2.1%). Advanced age (OR 1.02,CI 1.00–1.04), having more than three underlying comorbidities (OR 2.50,CI 1.96–6.60), use of inotropic support (OR 3.58,CI 1.89–6.80) and mechanical ventilation (OR 9.11,CI 4.72–18.11) showed association with increased risk for mortality in ICU. Conclusion The study indicated a much lower ICU mortality rate compared to similar studies conducted in other parts of Sub-Saharan Africa. Advanced age, underlying multiple comorbidities and organ support were associated with ICU mortality. Large multi-center studies are needed to highlight the true burden of critical care illness in Tanzania.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":" 6","pages":"383 - 392"},"PeriodicalIF":2.1,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138612769","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":"Psychometric Analysis of the Patient-Reported Hypoparathyroidism Symptom Diary Symptom Subscale Using Data from Two Clinical Trials.","authors":"Lauren Nelson, Steven W Ing, Mishaela R Rubin, Jia Ma, Susan Martin, Rohini Sen, Olulade Ayodele","doi":"10.2147/PROM.S414794","DOIUrl":"10.2147/PROM.S414794","url":null,"abstract":"Purpose The hypoparathyroidism symptom diary (HypoPT-SD) is a disease-specific patient-reported outcome (PRO) tool comprising a 7-item symptom subscale, a 4-item impact subscale and 1-item anxiety, and sadness or depression components. This analysis assessed the psychometric properties of the HypoPT-SD symptom subscale scores using data from two open-label, single arm, Phase 4 studies (Study 402 and Study 404). Patients and Methods Eligible patients were aged 18 years or older with a confirmed diagnosis of hypoparathyroidism. All patients received recombinant human parathyroid hormone (1–84) during the analysis period. Scores were recorded at baseline, and at months 6, 30 and 36 (end of treatment [EOT]) in Study 402, and at baseline and week 52 (EOT) in Study 404. The structure of the HypoPT-SD Symptom subscale was analyzed by measuring correlations between pairs of item scores; internal consistency and reliability were evaluated using Cronbach’s coefficient α; test-retest reliability was assessed using intraclass correlation; and construct validity was determined by performing correlational analyses between scores recorded using the HypoPT-SD and those for other conceptually similar PRO tools. Results A total of 60 patients were included in the analysis. Inter-item pairwise correlations were strong for all but 5 of the item pairs analysed. Cronbach’s α values for the HypoPT-SD Symptom subscale were 0.88 using data from Study 402 and 0.92 using data from Study 404. In general, the HypoPT-SD Symptom subscale scores had moderate or strong correlations with scores recorded using PRO tools. Intraclass correlation coefficients exceeded 0.70 using test–retest data from all patients in Study 402 and from a subgroup of patients with stable disease from Study 404. Conclusion This analysis demonstrated the test-retest reliability, internal consistency and construct validity of the HypoPT-SD using data from longitudinal prospective studies and supports the use of the HypoPT-SD in future clinical studies.","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"355-367"},"PeriodicalIF":2.1,"publicationDate":"2023-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10693201/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138478324","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 Burden of Sickle Cell Disease on Children and Their Caregivers: Caregiver Reports of Children's Health-Related Quality of Life and School Experiences, Caregiver Burden, and Their Association with Frequency of Vaso-Occlusive Crises.","authors":"Andrew Campbell, Avery A Rizio, Kristen L McCausland, Serban Iorga, Glorian P Yen, Jincy Paulose, Soyon Lee","doi":"10.2147/PROM.S419607","DOIUrl":"10.2147/PROM.S419607","url":null,"abstract":"<p><strong>Background: </strong>Children with sickle cell disease (SCD) experience a multiplex of disease-related symptoms and complications, including vaso-occlusive crises (VOCs), episodes characterized by extreme pain.</p><p><strong>Methods: </strong>A cross-sectional observational survey examined the health-related quality of life (HRQoL) and school experiences of children with SCD 2 months-11 years, burden experienced by their caregivers, and associations between these outcomes and VOC frequency. Caregivers (N=167) of children with SCD in the US completed the Infant-Toddler Quality of Life-Short Form 47 (ITQoL-SF47) for children 2 months-4 years, the Child Health Questionnaire-Parent Form 50 (CHQ-PF50) and PROMIS Pain Interference and Sleep Disturbance Parent Proxy short forms for children 5-11 years, and a study-specific survey of school experiences.</p><p><strong>Results: </strong>Children with SCD 2 months-4 years had lower ITQoL-SF47 scores (ie, worse HRQoL, p<0.001) than a normative sample of children; across domains, differences ranged from 18.73-45.03 points and exceeded minimal important difference (MID) thresholds. Except for the behavior domain, children with SCD 5-11 years had lower scores on all CHQ-PF50 domains than the normative sample (p<0.001); differences ranged from 6.78-36.37 points and exceeded MID thresholds. Children with more frequent VOCs had lower HRQoL and worse school experiences than children with less frequent VOCs (p<0.05, except for behavior domains). The largest differences based on VOC frequency were observed for overall health and bodily pain/discomfort among children 2 months-4 years (differences=40.88 and 32.50 points, respectively), and bodily pain and role/social limitations due to physical health among children 5-11 years (differences=38.99 and 37.80, respectively). Caregivers of children with more frequent VOCs experienced greater burden than caregivers of children with less frequent VOCs, though specific areas of impact (eg, caregiver emotions, time) differed across child age groups.</p><p><strong>Conclusion: </strong>VOC frequency is negatively associated with HRQoL, highlighting the burden experienced by children with SCD and their caregivers.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"369-381"},"PeriodicalIF":2.1,"publicationDate":"2023-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10693247/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138478325","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":"Qualitative Exploration in Exit Interviews of Changes Observed in Clinical Trials for Individuals with Autism Spectrum Disorder Without Intellectual Disability.","authors":"Michael Chladek, Claire Burbridge, Elizabeth Gibbons, Tom Willgoss, Janice Smith, Susanne Clinch","doi":"10.2147/PROM.S385682","DOIUrl":"https://doi.org/10.2147/PROM.S385682","url":null,"abstract":"<p><strong>Purpose: </strong>To explore, from the perspective of Study Partners (SPs; eg, caregivers) of clinical trial participants with autism spectrum disorder (ASD), any changes experienced in socialization and communication over the clinical trial, how these changes manifested, and the impact these changes had on the autistic individual, the SP, and family. This helps interpret whether changes in trial outcomes were meaningful.</p><p><strong>Patients and methods: </strong>Interviews were conducted with the SPs of individuals with ASD, without intellectual disability, from 2 clinical trials: 86 children (aged 5-12 years) or adolescents (aged 13-17 years) who took part in the aV1ation trial (83.7% male), and 41 adults (aged 18+ years) who took part in the V1aduct trial (80.5% male). The primary endpoint for both trials was change from baseline in the Vineland^TM-II two-domain composite, consisting of the mean of the Socialization and Communication domains. In these interviews the participants verbally indicated level of change for each of these key domains on 7-point change scales.</p><p><strong>Results: </strong>Improvements in the Socialization domain enabled greater awareness of the feelings of others and allowed for stronger empathy and kindness. Improvements in the Communication domain allowed for the autistic individual to be better at listening and better at self-expression. Together, changes in these two domains, which were considered most important, allowed for richer, deeper relationships. Study Partners noted that improvements in these domains allowed for better integration within the family unit, decreased stress, and increased optimism about the autistic individual's future.</p><p><strong>Conclusions: </strong>The impacts of changes in either domain were synergistic, combining together to create positive experiences which in turn led to further positive impacts in other skills. These qualitative insights provide context to the changes that were observed during the clinical trial and captured using the Vineland^TM-II, illustrating the meaning of these changes to the individuals with ASD without intellectual disability and their families, and the impact that they have on people's everyday lives and overall health-related quality of life.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"313-335"},"PeriodicalIF":2.1,"publicationDate":"2023-11-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10676099/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138461409","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":"Quantitative and Qualitative Exploration of Meaningful Change on the Vineland Adaptive Behavior Scales (Vineland™-II) in Children and Adolescents with Autism Without Intellectual Disability Following Participation in a Clinical Trial.","authors":"Susanne Clinch, Stacie Hudgens, Elizabeth Gibbons, Tom Willgoss, Janice Smith, Ela Polek, Claire Burbridge","doi":"10.2147/PROM.S385542","DOIUrl":"https://doi.org/10.2147/PROM.S385542","url":null,"abstract":"<p><strong>Purpose: </strong>The Vineland^TM Adaptive Behavior Scale is often used in autism spectrum disorder (ASD) trials. The Adaptive Behavior Composite Score (VABS-ABC) is the standardized overall score (the average of the Socialization, Communication and Daily Living skills domains), and the standardized 2-Domain Composite Score (VABS-2DC) is a novel outcome measure (average of the Socialization and Communication domains). A within-person meaningful change threshold (MCT) has not been established for the VABS-2DC. This paper presents a quantitative and qualitative interpretation of what constitutes a meaningful change in these scores to individuals with ASD without Intellectual Disability (ID; IQ≥70) and their families, as reported by their study partners (SPs).</p><p><strong>Participants and methods: </strong>Data were obtained from the aV1ation clinical trial in children and adolescents with ASD and associated exit interviews. The intent-to-treat (ITT) clinical trial population included 308 individuals with autism (85.4% male; average age: 12.4 years [standard deviation (SD)=2.97]); 124 in the child cohort (aged 5 to 12 years; average age: 9.4 years [SD=1.86]), and 184 in the adolescent cohort (aged 13 to 17 years; average age: 14.5 years [SD=1.39]). Study partners of 86 trial participants were included in the Exit Interview Population (EIP): participants represented were 83.7% male, average age: 12.3 years [SD=2.98]). Anchor and distribution-based methods were used to estimate within-person change to support a responder definition, to aid interpretation of the clinical trial data; qualitative data were used to contextualize the meaning of changes observed.</p><p><strong>Results: </strong>A within-person MCT range of 4 to 8 points was proposed for both VABS-ABC and VABS-2DC, which was associated with at least a 1-point improvement on 4 different anchors. Evidence for this within-person MCT was further supported by qualitative data, which suggested any change was considered meaningful to the individual with ASD, as reported by their SP, no matter what the magnitude.</p><p><strong>Conclusion: </strong>A change in standardized score of 4 to 8 points constitutes a within-person MCT on both VABS-ABC and novel VABS-2DC in those with ASD and no ID. A change of this, or more, was reported by the SPs in this trial to be meaningful and highly impactful upon the individuals with ASD and their family.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"337-354"},"PeriodicalIF":2.1,"publicationDate":"2023-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10675867/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138461410","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 Ravulizumab on Patient Outcomes and Quality of Life in Generalized Myasthenia Gravis.","authors":"Carlo Antozzi, Renato Mantegazza","doi":"10.2147/PROM.S408175","DOIUrl":"10.2147/PROM.S408175","url":null,"abstract":"<p><p>Myasthenia gravis (MG) is an autoimmune ion channel disorder in which antibodies to different end-plate antigens impair neuromuscular transmission, ultimately leading to muscle weakness and fatigability. In about 85% of patients with MG, autoantibodies against the acetylcholine receptor (AChR) activate the complement cascade, causing damage to the neuromuscular junction. MG is a chronic disorder for which standard therapies with corticosteroids, immunosuppressive drugs, and immunomodulation with plasma exchange or intravenous immunoglobulins modify the course of the disease, but the residual burden of physical, psychological, and social disability highlights several unmet needs, among these the need for specific, targeted, and well tolerated therapies able to improve the patients' quality of life. Complement inhibition paved the way to precision medicine in MG since, for the first time, a specific therapy targeting a crucial pathogenetic step has been designed, tested, and proven to be effective in a controlled fashion. Ravulizumab represents the first long-acting complement inhibitor approved for treatment of patients with generalized MG, able to provide rapid, complete, and sustained complement inhibition. Ravulizumab improved the MG Activity of Daily Living scale and other clinical parameters up to 26 weeks as shown by the CHAMPION MG trial, and by its open label extension, with the added value of being administered every 8 weeks. The schedule of administration is likely to improve patients' adherence and hence their quality of life. The introduction of complement inhibition will considerably change the traditional therapeutic strategy for MG.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"305-312"},"PeriodicalIF":1.8,"publicationDate":"2023-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/db/f9/prom-14-305.PMC10590807.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"50158496","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":"Association Between Hematologic Response and Change in Health-Related Quality of Life Among Patients with Light-Chain (AL) Amyloidosis.","authors":"Avery A Rizio,&nbsp;Kristen L McCausland,&nbsp;Michelle K Carty,&nbsp;Ansgar Conrad,&nbsp;Tiffany P Quock","doi":"10.2147/PROM.S421211","DOIUrl":"10.2147/PROM.S421211","url":null,"abstract":"<p><strong>Purpose: </strong>The purpose of this secondary data analysis was to examine the association between hematologic response and health-related quality of life (HRQoL) among patients with light-chain (AL) amyloidosis.</p><p><strong>Patients and methods: </strong>Data for this secondary analysis were collected through a non-interventional, longitudinal, online self-report survey of patients with AL amyloidosis. Patients completed an initial online survey, with follow-up surveys administered 1, 6, 12, 18, and 24 months after completion of the initial survey. The online survey included an assessment of patients' most recent self-reported hematologic response status. Eight domains and 2 summary components of HRQoL were evaluated with the SF-36v2^® Health Survey. A series of logistic regression models were used to examine the association between self-reported hematologic response at 24 months (dichotomized as new or maintained complete hematologic response; less than a complete response) and change in HRQoL from baseline to 24 months (dichotomized as meaningful worsening; improvement or preservation).</p><p><strong>Results: </strong>For all measured domains of HRQoL except physical functioning, there was no statistically significant relationship between meaningful worsening in HRQoL and hematologic response status at 24 months. Patients without a complete hematologic response had an odds of experiencing meaningful worsening of HRQoL that was similar to that of patients with a complete hematologic response.</p><p><strong>Conclusion: </strong>Among patients with AL amyloidosis, change in HRQoL was generally not associated with hematologic response. Achieving a complete hematologic response does not necessarily mean that a patient will experience increased or stable HRQoL. When defining treatment success, it is important to recognize that clinical markers such as hematologic response may not fully encapsulate the patient experience.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"297-304"},"PeriodicalIF":2.1,"publicationDate":"2023-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/f9/4a/prom-14-297.PMC10576564.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41237433","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":"Erratum: Development and Validation of a Revised Instrument to Measure Burden of Long-Term Medicines Use: The Living with Medicines Questionnaire Version 3 [Corrigendum].","authors":"","doi":"10.2147/PROM.S436333","DOIUrl":"10.2147/PROM.S436333","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.2147/PROM.S151143.].</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"283-284"},"PeriodicalIF":2.1,"publicationDate":"2023-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/bf/00/prom-14-283.PMC10576452.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41237434","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":"Generating Items for Measuring Needs-Based Quality of Life and Self-Perceived Health Inequity in Patients with Multimorbidity: Development of the MultiMorbidity Questionnaire (MMQ).","authors":"Kristine Henderson Bissenbakker,&nbsp;Anne Møller,&nbsp;Alexandra Brandt Ryborg Jønsson,&nbsp;John Brandt Brodersen","doi":"10.2147/PROM.S427183","DOIUrl":"10.2147/PROM.S427183","url":null,"abstract":"<p><strong>Purpose: </strong>To describe the processes of developing domains and items for the MultiMorbidity Questionnaire (MMQ), a multimorbidity-specific PROM for the assessment of Needs-based QoL.</p><p><strong>Patients and methods: </strong>We developed items and domains for the MMQ through 17 qualitative content validity questionnaire interviews with adults with multimorbidity by testing items from an item bank (covering items with content inspired by existing Needs-based QoL measures for single diseases). The interviews alternated between an explorative part and more focused cognitive interview techniques.</p><p><strong>Results: </strong>Testing the 47 items from the first draft of the MMQ items showed that the Needs-based approach as a framework did not cover all the QoL aspects our informants stated as being important. Therefore, the conceptual framework was supplemented by Self-perceived health inequity, and new items were generated. MMQ, measuring Needs-based QoL (MMQ1) and Self-perceived health inequity (MMQ2), was assembled. MMQ1 covers the domains: \"Physical ability\" (10 items), \"Limitations in everyday life\" (15 items), \"Worries\" (11 items), \"My social life\" (11 items), \"Self-image\" (12 items), and \"Personal finances\" (2 items). Self-perceived health inequity proved to be a relevant framework for other aspects of QoL not covered by the Needs-based approach to QoL. MMQ2 covers the domains: \"Experiences of being stigmatized\" (five items), \"Experiences of not being seen and heard\" (four items), \"Insufficient understanding of the burden of disease\" (three items) and \"Experiences of feeling powerless\" (five items).</p><p><strong>Conclusion: </strong>We have developed the final MMQ draft, a multimorbidity-specific PROM for the assessment of Needs-based QoL (MMQ1) and Self-perceived health inequity (MMQ2) with high content validity (regarding content relevance and comprehensiveness). The final MMQ draft will be assessed for its psychometric properties using Modern Test Theory.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"269-282"},"PeriodicalIF":2.1,"publicationDate":"2023-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e2/fa/prom-14-269.PMC10576455.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41237435","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 Safinamide on Patient-Reported Outcomes in Parkinson's Disease.","authors":"Christian Espinoza-Vinces,&nbsp;Rafael Villino-Rodríguez,&nbsp;Ainhoa Atorrasagasti-Villar,&nbsp;Gloria Martí-Andrés,&nbsp;María-Rosario Luquin","doi":"10.2147/PROM.S369590","DOIUrl":"10.2147/PROM.S369590","url":null,"abstract":"<p><strong>Introduction: </strong>Parkinson's disease (PD) is a chronic and progressive neurodegenerative condition due to the degeneration of nigral dopaminergic cells. Both motor and non-motor symptoms (NMS) of PD produce a marked impairment in PD patients' quality of life (QoL), but contrary to motor features, NMS do not improve with dopamine replacement. Novel therapeutic interventions for PD have successfully controlled most motor manifestations of PD, but the management of NMS is still challenging. Since NMS have a negative impact on the QoL of PD patients, researchers are currently looking for drugs that can modulate the activity of neurotransmitter systems other than dopamine in the hope that can alleviate NMS in PD. Among the recently approved drugs for patients experiencing fluctuations in motor symptoms, safinamide stands out as an effective add-on therapy to levodopa. Safinamide is a monoamine oxidase type-B inhibitor (MAOB-I), with proven efficacy in reducing motor fluctuations. Its distinctive mechanism of action impacts dopaminergic pathways via MAOB inhibition and glutamatergic pathways by blocking sodium and calcium channels. Findings from Phase III clinical trials, meta-analysis, post-hoc analysis, and real-life experiences indicate that safinamide benefits motor symptoms such as tremor, bradykinesia, rigidity, and gait. Additionally, it shows promise for improving NMS like fatigue, pain, mood, and sleep disturbances in patients with PD.</p><p><strong>Areas covered: </strong>In this article, the authors explore the impact of safinamide on patient-reported outcomes in PD. A thorough search was conducted on PubMed focusing on studies published between 2018 and 2023 in English. The inclusion criteria encompassed clinical trials, randomized controlled trials, systematic reviews, meta-analyses, and reviews. The search strategy revolved around the implementation of MeSH terms related to safinamide and its impact on the quality of life in PD.</p><p><strong>Conclusion: </strong>Our data strongly support the improving effect on QoL, reducing the disabling NMS reported in patients with PD.</p>","PeriodicalId":19747,"journal":{"name":"Patient Related Outcome Measures","volume":"14 ","pages":"285-295"},"PeriodicalIF":2.1,"publicationDate":"2023-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b9/e0/prom-14-285.PMC10576461.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41237436","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}