Patient Related Outcome Measures最新文献

Background: 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).

Methods: 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.

Results: 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.

Discussion: 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.

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.

Purpose: Cost-effectiveness evaluations of interventions require health utility data. However, in medical conditions, such as aromatic L-amino acid decarboxylase (AADC) deficiency, this presents problems due to the rarity of the disease. The study aim therefore was to employ a discrete choice experiment (DCE) to generate health utilities for AADC deficiency.

Methods: A previous literature review, clinician and parent interviews had identified six key AADC deficiency attributes: mobility, muscle weakness, oculogyric crises (OCG), feeding ability, cognitive impairment and screaming. A representative sample of the French general population was recruited. Participants rated 5 health state vignettes describing AADC deficiency using time-trade-off (TTO) and standard gamble (SG). Additionally, participants rated the worst health state using the Health Utility Index version 3 (HUI3). Subsequently, participants completed DCE 11 choice sets. Indirect DCE part-worth utilities were converted to health utilities using the anchors from the TTO, SG and HUI3.

Results: The DCE was completed online by 1001 participants (50.9% female; mean age 45.7 years). Most participants (596, 59.5%) provided consistent responses to the repeated choice task. Five models were evaluated, and one preference reversal ("head control"/"sitting unaided") was identified in all models. The rescaled utilities ranged from 0.3891 to 0.5577 (difference of 0.17 utilities) for TTO anchors corresponding to the worst (633233) and best (111111) health states. Health utilities ranged from 0.5534 to 0.7093 for the SG anchors. The disutility associated with a transition from "no problems walking" to "bedridden" was -0.0533, whereas disutility of moving from "constant screaming" relative to "no screaming" was -0.0248. The disutility associated with daily OCG was -0.0167. Disutilities for the other attributes were small although there were exceptions.

Conclusion: A DCE was used to derive health utilities for AADC deficiency. These health utilities will subsequently be used in an economic model evaluating an AADC deficiency intervention.

Introduction: Non-spontaneous vaginal delivery, such as cesarean delivery and operative vaginal deliveries, provides births other than regular vaginal pregnancy. In taking effective steps to minimize the caesarean section rate and the issues associated with it, it would be useful to examine the determinants of instrumental deliveries and their key indications. Therefore, this study aimed to determine magnitude and related factors of non-spontaneous vaginal delivery.

Methods: Facility-based analytical cross-sectional study design was used. A total sample size of 383 was used. All mothers who gave birth during data collection period and fulfill inclusion criteria were included in the study. Interviewer-administered data collection method was used. Data were entered in Epi-data and exported to SPSS to analyze both descriptive and advanced analysis.

Results: About 24.4% of mothers' mode of delivery was non-spontaneous vaginal delivery. Those mothers whose occupation was housewives were 2.8 times more likely to give birth through non-spontaneous vaginal delivery than mothers whose occupation was teachers (AOR = 2.8 95% CI 1.103-7.261). Mothers with grand multipara were less likely to give birth through non-spontaneous vaginal delivery than nulliparous (AOR = 0.10 95% CI 0.022-0.468) and primipara (AOR = 0.17 95% CI 0.041-0.671). Mothers with complications during pregnancy were 3 times more likely to give birth via non-spontaneous vaginal delivery than mothers without complications during pregnancy. Mothers with non-macrosomic neonates and female neonatal sex were less likely experiencing to give birth through non-spontaneous vaginal delivery as compared to their respective reference groups.

Conclusion: Magnitude of non-spontaneous vaginal delivery was high in this study. Being a housewife, having complications during pregnancy, nullipara and primipara, macrosomic fetus and male neonate were associated with outcome variable. Attention should be given for the housewives, experiencing complication during pregnancy, a fetus with big weight and male neonatal sex in order to minimize risks of non-spontaneous delivery.

Inclusion of the patient perspective in the reporting of symptomatic adverse events provides different and complementary information to clinician reporting using the Common Terminology Criteria for Adverse Events (CTCAE). The National Cancer Institute's Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE^®) is designed for patients to self-report their symptomatic adverse events in a manner that complements CTCAE reporting. Using CTCAE and PRO-CTCAE together offers the potential to refine our understanding of the prevalence and trajectory of lower grade AEs that can lead to elective discontinuation of therapy and diminished quality of life. This review addresses the development of PRO-CTCAE with an emphasis on the differences between PRO-CTCAE scores and CTCAE severity grades. This distinction is important when evaluating, grading and reporting toxicity and tolerability in cancer clinical trials.

Background: Patient engagement is increasingly considered to be an important element in the treatment of brain disorders to optimise outcomes for patients, society, and healthcare systems. Nonetheless, scientific research examining methodologies to engage patients with brain diseases in Research and Innovation (R&I) is scarce.

Aim: To review existing scientific evidence regarding the engagement of patients with brain disorders in research and innovation.

Methods: Studies were retrieved from several bibliographic databases (publication date between January 2016 and April 2019) with pre-specified selection criteria.

Results: In total, 49 articles were identified as meeting the inclusion criteria and were reviewed systematically. Results showed that there is limited evidence available on the impact and (cost-) effectiveness of patient engagement in (brain) research and innovation. Most published studies are protocols, guidelines, and discussion articles for patient engagement in health research and innovation. Overall, there exists a general consensus to engage patients in every step of the research procedure. Relevant evidence identified includes principles of engagement, definitions of stakeholder types, key considerations for planning, conducting and disseminating engaged research, potential engagement activities, and examples of promising practices.

Discussion: Findings are inconclusive due to methodological differences. Comparison between studies was difficult due to differences in patients, form of engagements, and total duration of engagement of patients. Experiences of patient engagement mainly concern adherence to medical treatments or participation of "expert patients" in clinical trials, but very rarely the governance of R&I according to the dictates of Responsible Research and Innovation (RRI). More structuralized, well-conducted and comparable Randomized Controlled Trials (RCTs) are needed to be able to make evidence-based recommendations on how to increase effective patient engagement in research and innovation and assess the impact and (cost)-effectiveness.

Purpose: To understand women's perspectives, attitudes, and beliefs surrounding menopause transition and increase understanding of digital technology use for symptom management.

Patients and methods: Information was obtained using three studies of women aged 40-65 years experiencing menopause transition symptoms. The HealthyWomen online cross-sectional survey was designed to reflect an inclusive sociodemographic sample representative of the US population. BECOME was a blinded, ethnographic, qualitative research study of women's menopause transition experiences and comprised facilitator-led online asynchronous discussions, online homework entries, and audio-only teleconferences. The NODE.Health online, two-part, cross-sectional patient survey was designed to capture patient and healthcare provider (HCP) sentiment about the use of digital health technologies to address gaps in perimenopausal symptom knowledge and management.

Results: The HealthyWomen survey included 1045 participants, 37 were included in BECOME, and 100 completed the NODE.Health survey. Hot flashes, night sweats, and sleep problems were the most frequently experienced symptoms in the HealthyWomen survey, and over half of participants experiencing symptoms felt the need to seek relief. Whether menopause was considered a medical problem or natural process differed by self-identified race, culture, and ethnicity, as did the likelihood of consulting a HCP over symptoms. Participants preferred to discuss menopause transition with HCPs who did not rush them, were good listeners, and had expertise in the area. Most technology experience was with health websites, but nearly half were unsatisfied with online resources describing menopause-related symptoms. Convenience, ease of use, and accessibility were the most common reasons for pursuing digital health technology.

Conclusion: Factors such as cultural beliefs, values and attitudes towards menopause determine personal experiences. More open discussions with friends, family, and HCPs may raise awareness and reduce barriers to seeking help. To provide optimal care throughout the menopause transition, HCPs should consider patients' psychosocial and cultural backgrounds, and personal and subjective perspectives.

Background: Light chain (AL) amyloidosis is a rare and progressive disease that affects myriad organs and systems. Patients with cardiac involvement have the highest risk of death. This report compiles findings across three cohorts of patients with AL amyloidosis to understand patterns of employment and work impacts.

Methods: Data came from three cohorts recruited through patient advocacy organizations in the US. Patients in Cohort 1 completed the SF-36v2^® Health Survey (SF-36v2), the Work Productivity and Activity Impairments - Specific Health Problem (WPAI) questionnaire, and the 12-item Kansas City Cardiomyopathy Questionnaire (KCCQ-12). The relationship between work impacts (WPAI scores) and HRQoL (SF-36v2 scores) was investigated using multivariable logistic regression and summarized according to cardiac severity using New York Heart Association (NYHA) classes estimated from KCCQ-12 scores. Changes in employment, days of missed work, and long-term disability due to AL amyloidosis were summarized for patients diagnosed in the past 24 months and stratified by NYHA class (Cohort 2). Findings were contextualized using patient interviews (Cohort 3).

Results: Work-related impacts, especially reduced productivity, were common among patients with AL amyloidosis. WPAI scores were significantly related to HRQoL (p<0.05 for all models). Among patients with cardiac involvement, the greatest degree of work impacts was observed for those in NYHA class 3 or 4. Changes in employment, missed work, and long-term disability were common among newly diagnosed patients, especially among those in NYHA class 3 or 4. Patient interviews supported the survey findings; patients described absences, reduced productivity at work, and loss of employment due to the disease and its treatment.

Conclusion: Patients with AL amyloidosis, particularly those with more advanced disease, experience impacts across a range of employment-related outcomes. These findings highlight the need for more effective treatments and interventions which may improve functioning and patient outcomes, while reducing indirect costs associated with the disease.

Purpose: To identify factors that contribute to adverse events among older adults during short stays at skilled nursing facilities (SNFs) for rehabilitation (ie, up to 100 resident days). Adults aged 65 years and older are at serious risk for adverse events throughout their continuum of care. Over 33% of older adults admitted to SNFs experienced an adverse event (eg, falls) within the first 35 days of their stay.

Design: A scoping review.

Setting and participants: Older adults admitted for short stays in SNFs.

Methods: Eligibility criteria were peer-reviewed original articles published between 1 January 2015 and 30 May 2021, written in English, and containing any of the following key terms and synonyms: "skilled nursing facilities", "adverse events", and "older adults". These terms were searched in PubMed, MEDLINE, CINAHL, EBSCOHost, and the ProQuest Nursing and Allied Health Database. We summarized the findings using the Joanna Briggs Institute and PRISMA-ScR reporting guidelines. We also used the Capability-Opportunity-Motivation-Behavior (COM-B) model of health behavioral change as a framework to guide the content, thematic, and descriptive analyses of the results.

Results: Eleven articles were included in this scoping review. Intrinsic and extrinsic contributors to adverse events (ie, falls, medication errors, pressure ulcers, and acute infections) varied for each COM-B domain. The most frequently mentioned capacity-related intrinsic contributors to adverse events were frailty and reduced muscle strength due to advancing age. Inappropriate medication usage and polypharmacy were the most common capacity-related extrinsic factors. Opportunity-related extrinsic factors contributing to adverse events included environmental hazards, poor communication among SNF staff, lack of individualized resident safety plans, and overall poor care quality owing to racial bias and organizational and administrative issues.

Conclusion: These findings shed light on areas that warrant further research and may aid in developing interventional strategies for adverse events during short SNF stays.