Patient Related Outcome Measures最新文献

Introduction: The COVID-19 Symptoms Daily Diary (CSDD) is a patient-reported outcome measure designed to assess the severity of core COVID-19 symptoms in clinical trials. The preliminary version of the CSDD was developed based on regulatory guidance and the hallmark COVID-19 symptoms identified by the CDC. This study aimed to evaluate CSDD content validity, to determine whether it is fit for the purpose of supporting efficacy endpoints in clinical trials of treatments for COVID-19. This research also sought to evaluate the appropriateness of a newly developed Pre-COVID-19 Symptoms Questionnaire.

Methods: A targeted literature review was completed to evaluate the relevance of concepts included in the diary and to identify any important symptoms that may have been missing. Hybrid (concept elicitation and cognitive debriefing) semistructured qualitative interviews were then conducted across 3 iterative rounds with 30 adults in the United States recently diagnosed with COVID-19.

Results: The CSDD included concepts that were most frequently reported by interview participants, including those identified as most bothersome and most important to treat. During cognitive debriefing, participants described the diary concepts as salient and reported the instructions, recall period, and response options as clear and appropriate. Only 2 of 15 CSDD items were modified across 3 interview rounds; specifically, definitions for the vomiting and diarrhea frequency items were clarified for consistent interpretation and response. Interview participants also reported general ease in understanding and responding to the Pre-COVID-19 questionnaire, with feedback resulting in only minor changes to the reference period and instructions.

Conclusion: The findings of the current study provide strong evidence for the content validity of the CSDD and the appropriateness of each of the items assessed. This rigorous evaluation (aligned with regulatory guidance) indicates that the CSDD is fit for the purpose of supporting efficacy endpoints in planned clinical trials evaluating medications for COVID-19 treatment.

Alpha-1 antitrypsin deficiency (AATD) is a rare cause of chronic lung and liver disease without its own patient reported-outcome measure (PROM). PROMs for Chronic Obstructive Pulmonary Disease (COPD) are commonly used instead, but AATD differs from COPD in several ways. We reviewed whether the PROMs used in the AATD literature adequately assess quality-of-life in these patients. 11 studies used PROMs as their primary outcomes; 21 included them as secondary outcomes. The St George's Respiratory Questionnaire (SGRQ) was the most commonly used PROM, used by 7 of the 11 primary outcome studies. Others included the COPD Assessment Tool, SF-36, LCOPD, EQ-5D, and the Chronic Respiratory Diseases Questionnaire. Several studies assessed SGRQ as being associated with respiratory disease severity as measured by FEV1% predicted, exacerbation rate, oxygen use and exercise tolerance. However, no studies used PROMs which included assessment of liver-related symptoms, other extra-pulmonary manifestations of AATD, or concerns related to genetics or finances. These factors are likely to have an impact on quality of life in AATD. A specific AATD-PROM is therefore required to holistically address the quality of life effects of an AATD diagnosis.

Purpose: Growth hormone deficiency (GHD) causes decreased growth rate in children, resulting in short stature in childhood and adulthood. Daily subcutaneous injections with growth hormone (GH) have been standard treatment. Newer weekly GH formulations now exist. This study estimates utilities associated with GHD treatment for both people with the disease and caregivers by employing time trade-off (TTO) methodology.

Methods: Three online surveys were conducted amongst the general population in the UK and Canada. Based on a pilot, data collection was conducted in two surveys only (Survey A and Survey B). In Survey A, adults aged ≥18 years evaluated health states as if they were receiving injections themselves. In Survey B, adults with a child <15 years evaluated health states as if they were administering injections to a child. The surveys assessed device complexity, injection frequency, injection pain, needle visibility and storage possibilities.

Results: 2026 and 2028 respondents completed Survey A and Survey B, respectively. Of these, 1782 respondents and 1678 respondents were valid for inclusion. Avoiding weekly injection pain was associated with a significant utility gain of 0.030 (95% CI 0.026-0.035, p<0.001) in Survey A and 0.044 (95% CI 0.038-0.051, p<0.001) in Survey B. Additionally, less complex injection devices and lower injection frequencies had a significant impact in both Survey A (0.020, 95% CI 0.016-0.025, p<0.001; 0.009, 95% CI 0.005-0.014, p<0.001) and Survey B (0.008, 95% CI 0.002-0.014, p=0.006; 0.009, 95% CI 0.003-0.014, p=0.003).

Conclusion: Several aspects are associated with a significant impact on utilities for people with GHD and potential caregivers. Treatment options without injection pain, a time-consuming and complex injection process and daily injections are expected to result in higher health-related quality of life. These results may inform future economic evaluations and treatment choices.

Purpose: Being a young carer of a parent with dementia can be challenging, with many carers undertaking various practical and caring tasks. The weekend course Time to be young? gathers young carers, aiming to support them to cope with their challenges in everyday life. The aim of this study was to explore their role as a caregiver and the experienced impact of the course on their strategies of coping in their everyday life.

Participants and methods: The study had a qualitative descriptive design inspired by Lindseth and Norberg's phenomenological hermeneutical method, using individual semi-structured interviews for data collection. The participants were recruited from former participants of the course Time to be young?, and the final sample included eight participants.

Results: Through the analysis, four main themes were identified: 1) Help to accept the situation, 2) A sense of community, 3) The need for information and 4) The need to live one's own life. The study found that attendance at Time to be young? for young adults having a parent with dementia affected their coping strategies in their situation as a young care.

Conclusion: The study demonstrated the importance of courses like Time to be young?, and need for a meeting place, tailored information about dementia, and an opportunity to share and reflect upon their experiences as a young carer.

Introduction: Polytrauma remains a major global health challenge, with rapid intervention being critical for survival, especially during the "Golden Hour". This study examines the impact of Helicopter Emergency Medical Services (HEMS) on procedural care during the transfer of polytraumatized patients to urban hospitals in Romania.

Methods: A retrospective cohort study was conducted at the County Emergency Hospital "St. Ap. Andrei" in Galați, covering January 2020 to October 2021. The study analyzed data from 89 patients transported by the Romania's Mobile Emergency Service for Resuscitation and Extrication (SMURD) Galați air unit. Key parameters included demographics, injury mechanism, vital statistics, and prehospital interventions. Statistical analyses were performed using SPSS, with significance set at p < 0.05.

Results: Out of 89 patients (mean age 21.6 years, 80.3% male), trauma causes were primarily traffic accidents (34.8%) and falls (33.7%). A Glasgow Coma Scale (GCS) score ≤8 was noted in 28.1% of cases, with head trauma observed in 51.6% of patients. HEMS interventions frequently involved oxygen therapy (89.5%) and patient stabilization maneuvers, leading to a mortality rate of 6.7%. Notably, helicopter transport enabled efficient transfer and improved survival outcomes in this cohort.

Discussion: HEMS demonstrated benefits in reducing intervention times and enhancing prehospital care quality for polytrauma patients, especially in hard-to-reach areas. The study aligns with global data on HEMS's role in trauma systems, underlining the importance of multidisciplinary collaboration and rapid transport.

Conclusion: HEMS plays a crucial role in improving survival rates for severely injured patients through timely interventions and specialized care. Further research comparing HEMS and ground services could refine trauma management protocols in similar settings.

Introduction: To develop and psychometrically validate the Self-management Questionnaire for Patients with Lower Extremity Arterial Disease Patients Who Underwent Endovascular Revascularization (LESQ).

Methods: We developed the LESQ and validated it in a Chinese population. A three-round cross-sectional descriptive survey in six hospitals in China, involving samples of 271, 269, and 623 participants, respectively. The surveys were conducted between February 2021 to March 2022.

Results: The final version of the LESQ, with 22 items, was divided into three domains using exploratory factor analysis: medical management, rehabilitation exercise management, and daily life management. The questionnaire had good internal consistency reliability, with a Cronbach's α of 0.953 and good retest reliability, with the coefficients of 0.917, respectively. The content validity of the LESQ was 0.939. The three domains of the questionnaire were confirmed by confirmatory factor analysis. The optimal cut-off points were 52 and 70, respectively, using latent profile analysis.

Discussion: The LESQ is a new self-report questionnaire for measuring self-management ability with good reliability and validity through validation.

Purpose: The prevalence of chronic kidney disease (CKD) is increasing and CKD often goes undiagnosed and untreated until its later stages when irreversible damage has occurred. Patients with CKD have been reported to have lower quality of life than the general population, but the patient-reported outcome data on CKD patients in Finland are limited.

Patients and methods: The primary outcome of this structural, multiple-choice survey study was to assess life satisfaction and experienced health status in Finnish patients with CKD. The results were presented as numbers (n) and percentages (%). The secondary outcome was to identify patient groups using the K-means clustering method based on preselected response variables and to assess the associated background factors.

Results: In total, 558 patients with CKD responded to the electronic survey. Of the 395 patients who completed the whole survey, 39.7% reported their health status as good, pretty good, or excellent, and 59.9% were fairly or very satisfied with their life. Two clusters of patients could be identified based on their health status and life satisfaction: patients with (1) poorer or (2) better well-being. Patients with poorer well-being were more likely to have at least 3 comorbidities (66.8% vs 44.3%) and lack follow-up visits entirely (10.5% vs 1.9%), compared with patients with better well-being. The patients with poorer well-being were less often knowledgeable about the disease, its causes (35.5% vs 48.1%), and its care (30.7% vs 20.3%) than patients with better well-being, and they showed weaker adherence to lifestyle interventions such as following dietary instructions (30.3% vs 40.5%).

Conclusion: Screening for CKD to enable early diagnosis, early commitment to treatment, and empowering the patient by providing education are key for improvement of health and life satisfaction in patients with CKD. Therefore, resources should be allocated to these measures of action.

Background: Satisfaction with care is a concept quantified through diverse measurement tools. However, studies have indicated that measuring satisfaction is challenging due to the construct's multidimensional expression. Thus, obtaining valid results requires careful consideration of the construct's nature and measurement methods.

Purpose: The primary aim of this study was to examine how studies involving cancer caregivers have addressed the dimensionality of the construct when using satisfaction with care as an outcome, and whether this is reflected in the score reporting practices. We chose to investigate this by conducting a scoping review of the measurement tool Family Satisfaction with End-of-Life Care Scale (FAMCARE Scale), where scores can be reported as the mean of overall score, subscale scores and single-item scores.

Methods: This scoping review consisted of systematic searches using Medline, CINAHL, Embase, PsycInfo, Cochrane Library, and Epistemonikos. Two researchers used the Rayyan Qatar Computing Research Institute system to perform a blinded screening process. We extracted information on study design, purpose, evaluating of structural validity, variations in the type of scores reported, and justification for choosing the type(s) of scores that were analyzed.

Results: Twenty-three studies were included in the review, and their designs and reporting practices of score type varied substantially. Five studies reported analyses to test the scale's structural validity. Ten studies provided a justification for their choice of reporting method. The most common reporting practice found was using mean of overall scores, present in 20 of the included studies. Twelve studies reported mean of subscale scores, and ten reported single-item mean scores.

Conclusion: We found substantial variability in score reporting practices, highlighting the need for a more in-depth understanding and reflection on the multidimensional nature of caregiver satisfaction.

Purpose: Quantifying patient-perceived benefits and disadvantages of treatments in a real-world setting is increasingly important in healthcare decision-making. The Patient's Qualitative Assessment of Treatment (PQAT) assesses patient-perceived benefits and disadvantages of treatment, and associated trade-offs potentially influencing patients' willingness to continue treatment. It has then been modified to capture patients' perceived magnitude of benefits and disadvantages of treatment quantitatively, as well as qualitatively (PQATv2). However, the PQAT and the PQATv2 were designed for use and validated in a clinical trial setting. The objective of this study was to adapt and test the content validity of a version of the PQATv2 for use in real-world settings (PQAT-RW).

Patients and methods: The PQATv2 was adapted for use in real-world settings (PQAT-RW), and its content was validated in 16 patients with varied chronic medical conditions and medication regimens via semi-structured qualitative interviews.

Results: All participants reported that the PQAT-RW was "easy to understand". The majority (n = 11/16) reported that the items covered all important aspects of their treatment experience, and that no items needed to be removed or added to the instrument. Analysis of free-text responses identified eight global concepts considered by participants when evaluating the benefits and disadvantages of treatment: treatment effectiveness, side effects and method of administration were most frequently considered (as both benefits and disadvantages), followed by frequency of administration, financial considerations, storage, packaging and drug preparation.

Conclusion: The results of this study support the content validity of the PQAT-RW. They also demonstrate that using qualitative responses to contextualize quantitative responses provides unique insight into diverse and individualized patient-perceived benefits and disadvantages, and their relative importance, in real-world settings.

Introduction: Many research activities have focused on SARS-CoV-2 infection and subsequent COVID-19 respiratory illness during the pandemic. However, significant racial inequities emerged months after the COVID-19 pandemic began. The similarity between racial/ ethnic disparities in COVID-19 and those for other diseases raised awareness about the context for risk exposure and healthcare access. The purpose of this study is to examine social and structural determinants of health among COVID-19 survivors, carepartners, and the perspectives of healthcare stakeholders who experienced disruption during the early pandemic.

Material and methods: A purposive sample of interviews (n=9) and focus groups (n=10) were used to collect data regarding knowledge of barriers to effective COVID-19 risk mitigation, recovery, and chronic disease self-management. This included nurses, physicians, COVID-19 survivors and their carepartners, public health, and community leaders connected with the healthcare systems in rural counties of South Carolina.

Results: Five major themes were identified across the subgroups. The themes: The COVID-19 Illness Trajectory Added Major Health Challenges and Stressors, Access to Care Is Lacking, Support is Needed for COVID-19 Survivors and Care Partners, Support Must be Distributed Equitably, and Racism and Structural Issues Affect Stress reflect the strengths, opportunities, and inequities perceived within these groups.

Conclusion: This research is the first qualitative study focused on COVID-19 survivor-carepartner dyads that consider the intersectionality of race/ ethnicity, geography, and health that is known to occur when engaging healthcare systems. The themes illustrate the need for infectious disease prevention at all socioecological levels: structural/ systemic, community, organizational/ institutional, interpersonal, and individual.