Patient Related Outcome Measures最新文献

This systematic review aims to explore the utilization of patient-reported measures (PRMs) in primary care, focusing on healthcare providers' use of PRM data and factors influencing its effectiveness. Following the PRISMA 2020 guidelines, we registered the review in PROSPERO (CRD420251030695) and screened 2465 records, ultimately including eight studies. Data extraction and quality appraisal were conducted using a structured approach and the Mixed Methods Appraisal Tool (MMAT). Findings suggest that effective PRM data use depends on integration into electronic health records (EHRs) and clinical workflows, with barriers including technical limitations and organizational culture. PRM data can support clinical decision-making, shared decision-making, and communication and support professional empowerment and resource optimization. However, evidence quality was moderate, and conclusions should be interpreted with caution due to the small number of studies. Differences in health systems and study heterogeneity further constrain generalizability. This review highlights the need for seamless EHR integration, streamlined instruments, and active professional engagement to optimize PRM implementation, while identifying a critical research gap and calling for future studies on cost-effectiveness and equity impacts.

Background: Patient Acceptable Symptom State (PASS) describes the highest level of symptom beyond which patients consider themselves well. PASS can be used to establish what patients' view as an acceptable outcome after surgery. The Oxford Elbow Score (OES) is one of the most used elbow-specific patient-reported outcome measure (PROM), but a PASS-value has not yet been established for the OES after elbow arthroplasty. The primary purpose of this study was to determine the PASS for the OES after primary elbow arthroplasty due to acute distal humeral fracture.

Methods: This is a retrospective multi-center cohort study. All nationwide patients treated with elbow arthroplasty due to acute distal humeral fracture from January 1, 2008, until December 1, 2021, were invited to participate. Data were collected retrospectively using electronic health records. Study participants answered the OES, and a PASS-anchor question was used to assess if patients were in an acceptable state at follow-up. Logistic modelling was used to determine the PASS-value, defined as the value of OES needed to achieve an acceptable postoperative result with at least 95% probability. A PASS-value was estimated for the total population, for patients with <5 years of follow-up, and patients with ≥5 years of follow-up.

Results: The OES and PASS-anchor was answered by 159 (62%) patients. Median OES was 41 (0-48) in the total population. One hundred and thirty-nine patients (87%) reported an acceptable result. The 95% PASS-value for the OES was 33 (95% CI 29.6-38.5) in the total population.

Conclusion: The PASS-value for the OES after elbow arthroplasty due to acute distal humeral fracture was 33 for the total population and similar for patients with short-term and long-term follow-up. We recommend using the PASS-value to aid in interpretation of clinical trials and registry-based studies as well as identifying clinical failures in registries.

Purpose: User satisfaction and ease of use of continuous glucose monitoring (CGM) systems are key factors in patients' device acceptance. CGM user satisfaction is often assessed through questionnaires, but item selection varies widely across studies. The aim of this study was to design, develop and validate a Questionnaire for User Satisfaction Standardized for CGM performance studies (QUSS-CGM).

Methods: Selection of attributes and design of questionnaire items was based on a systematic literature search of publications on CGM performance evaluation studies. Content and response process validation of a draft-questionnaire was performed by experts (n=9) and people with diabetes (n=10), respectively. The resulting German pre-QUSS-CGM questionnaire underwent validation in two CGM performance studies ("pilot" studies) performed between June and August of 2024, via a pooled psychometric evaluation (exploratory factor analysis (EFA) and reliability) of n=126 questionnaires from these studies, followed by bidirectional translation to English.

Results: Two hundred and five items on user satisfaction in CGM performance studies were identified by systematic literature search and classified into six attributes according to their content. Items were summarized in a 25-item draft-questionnaire on a 5-point Likert scale. Content and face validity were considered acceptable with a scale-level content validity index (S-CVI/Ave) of 0.90 and a scale-level face validity index (S-FVI/Ave) of 0.93, both based on the average method. EFA revealed a two-factor structure for the final QUSS-CGM questionnaire summarized to 11 items, demonstrating high internal consistency (Cronbach's α of 0.84).

Conclusion: The QUSS-CGM was designed, developed, and validated as a reliable and standardized tool to measure user satisfaction in CGM performance evaluation studies.

Background: Emotional reactions to treatment could affect treatment adherence and treatment outcomes, and it is therefore important to assess the emotional impact of treatment and consider this impact in clinical decision-making. The Emotional Impact of Diabetes Treatment Questionnaire - Status version (EIDTQ-Status) was developed based on qualitative research with patients as the first patient-reported outcome measure to assess both the positive and negative emotional impacts of type 2 diabetes (T2D) and its treatment. This study assessed the psychometric properties of the EIDTQ-Status.

Methods: Participants with T2D treated with a range of medications were recruited from eight clinical sites in the United States. Analysis of the EIDTQ-Status focused on item performance, subscale identification (including exploratory factor analysis), development of a scoring algorithm, test-retest reliability (intraclass correlation coefficients in a third of the participants who were randomized to attend a second visit), internal consistency reliability (Cronbach's alpha), and construct validity (via comparisons to previously validated generic and diabetes-specific instruments).

Results: The sample included 250 participants (mean age = 59.7 years old; 54.4% female). Based on item performance and exploratory factor analysis, 14 items were retained and grouped into three subscales: (1) positive emotions, (2) negative emotions, and (3) sense of control over diabetes, eating, and weight, as well as a total score. The EIDTQ-Status demonstrated good internal consistency reliability (Cronbach's alphas of the three subscales and total score: 0.92, 0.88, 0.85, 0.77). Test-retest reliability was acceptable with no significant differences between administrations 7+2 days apart among stable participants (n=37; intraclass correlation coefficients: 0.85, 0.67, 0.62, and 0.88). Construct validity was supported via significant correlations with validated instruments (P<0.0001). The EIDTQ-Status distinguished among participants who differed in reports of emotional well-being. Exploratory analysis suggests the EIDTQ-Status may differentiate between treatments. Compared with injectable semaglutide (n=47), tirzepatide-treated participants (n=58) reported a significantly greater sense of control over diabetes, eating, and weight (P=0.025).

Conclusion: The EIDTQ-Status had strong factor structure with three subscales and a total score that demonstrated good reliability and validity. This questionnaire may be useful in clinical trials and observational research assessing the emotional impact of treatment for T2D.

Purpose: The primary objective of this study is to assess the feasibility of administering an online meditation program compared to usual care to glaucoma patients to improve mental health outcomes such as quality of life, depression, anxiety, and sleep quality. The secondary objective of this study is to improve mental health outcomes among glaucoma patients.

Patients and methods: This is a study protocol for a 12-week randomized control trial employing a mixed methods design. Glaucoma patients will be recruited from the Ivey Eye Institute in St. Joseph's Hospital Center. Eligible patients will include glaucoma patients of at least 75 years of age. Consented participants will be randomized to an online meditation group or usual care group in blocks of 4, or 2 per arm. Patients assigned to the online meditation group will undergo guided meditation sessions led by a meditation instructor via Microsoft Teams. Patients randomized to the usual care arm will undergo their usual glaucoma treatment. All study questionnaires including feasibility metrics, SF-12, CES-D, HADS-A and PSQI will be administered to both treatment groups at week 1, week 3, week 6, and week 12. Following study conclusion, feasibility metrics will be sent to participants in the intervention arm. In addition, patients randomized to the usual care arm will be given the opportunity to enroll in the meditation program after the 12 week study period, but no data will be collected.

Discussion and conclusion: To the best of the authors' knowledge, this study is the first of its kind to assess the feasibility of administering an online meditation intervention to glaucoma patients. If the findings from this study demonstrate positive results in favor of the treatment, this will be used to justify larger clinical trials and eventually, the integration of online meditation into the standard of care for glaucoma patients.

Purpose: Iron deficiency anemia (IDA) affects approximately 5 million people in the United States (US), significantly impacting quality of life (QoL) and overall health. Iron stores can be replenished through oral iron or, if ineffective/poorly tolerated, through intravenous iron (IVI) therapy. Despite IVI effectiveness, patients do delay/miss infusion appointments, leading to incomplete treatment response. Here we evaluated multiple-dose IVI therapy logistics and adherence from the patient perspective.

Patients and methods: US adult patients (≥18 years) with confirmed IDA who were currently prescribed an IVI treatment course were asked to respond to a 46-question online survey in early 2023. Questions covered patient demographics, appointment logistics, IVI infusion experience, impact of infusion on daily activities, reason(s) for missing any doses, and considerations for improving adherence.

Results: A total of 323 patients completed the survey, 193 of whom were prescribed ≥2 IVI infusions per month. Of these patients, 71/193 (36.8%) reported missing at least one dose, despite its effectiveness at improving symptoms. Most patients (122/193 [64.3%]) agreed that multiple-dose IVI therapy adversely impacted their QoL by reducing their productivity (63.4%) and attendance at important events (64.8%), with 80.3% reporting having to schedule their life around therapy. A third of patients (73/193 [38.1%]) were unsatisfied with their infusion frequency, with most (163/193 [84.5%]) agreeing that fewer IVI infusions would improve adherence, and most also (174/193 [90.1%]) preferring a single-dose option if available.

Conclusion: Time spent arranging and receiving multiple IVI infusions per month can interfere with patients' daily activities, leaving them dissatisfied with their IVI treatment experience. In this study, single-dose IVI infusions were a preferred option for patients with IDA, which may improve adherence to the complete course of treatment and offer improved QoL. Therefore, patient preference, convenience, and satisfaction should be considered and discussed when determining the type of IVI treatment.

Purpose: Myopia is a global public health issue, estimated to affect 50% of the world's population by 2050. Evidence suggests that reducing myopia by one diopter decreases the risk of myopia-related retinal complications by 40%. Despite the availability of several myopia control methods, their clinical adoption remains limited. To improve their uptake, enhancing parental awareness of myopia control is crucial. This study explores parental awareness of myopia-related complications, control interventions and their perspectives on myopia management in France and the UK.

Patient and methods: A structured survey was conducted among 200 self-selected parents of children with myopia (100 France, 100 UK), via the TOM app, an online platform for tracking medication adherence. The survey assessed parental understanding of myopia complications, awareness of control strategies, perspectives on management, and financial concerns.

Results: 74% of French and 88% of the UK parents reported being aware of at least one myopia-related long-term complication, with cataract the most recognized in France (66%) and glaucoma in the UK (76%). 50% of French and 43% of UK parents were unsure or unaware that myopia progression could be slowed. UK parents reported higher adoption rates of myopia control methods, including orthokeratology (47% France, 68% UK), atropine (46% France, 63% UK), and red-light therapy (44% France, 67% UK). Financial burden was a significant concern, with 59% of French and 52% of UK parents feeling strained by costs, particularly for glasses, contact lenses, and specialized treatments. Parents of children with faster myopia progression (>-1 dioptres/year) were more likely to report financial stress (p<0.001).

Conclusion: While most parents are aware of myopia complications, notable gaps exist in understanding control options. UK parents adopted control methods more frequently than French parents. Financial burden remains a significant concern, emphasizing the need for enhanced parental education and affordable access to myopia control.

Purpose: The Emotional Well-Being Questionnaire (EWQ) assesses a broad spectrum of mental health conditions and related symptoms-depression, anxiety, asthenia, and insomnia-highly relevant in type 2 diabetes (T2D), where emotional distress can impair adherence and outcomes. This pilot cross-sectional study provides preliminary validation evidence for the EWQ six-factor model in a T2D cohort using confirmatory factor analysis (CFA), evaluating reliability and convergent validity for mental-health screening.

Patients and methods: A sample of 240 adults (T2D n = 122; control n = 118) with T2D completed the EWQ and nine-item Patient Health Questionnaire (PHQ-9). Confirmatory factor analysis (CFA) was conducted to assess the six-factor model fit, and convergent validity was evaluated through analysis of EWQ scores with PHQ-9 scores.

Results: Confirmatory factor analysis indicated acceptable-borderline fit for a pilot sample (CFI = 0.886; TLI = 0.883; RMSEA = 0.071), providing preliminary support for the six-factor structure. The EWQ demonstrated reliability in the T2D group (Cronbach's α = 0.79). Convergent validity was supported by a significant positive correlation with PHQ-9 scores (r = 0.652, p < 0.001), confirming the EWQ's capacity to assess depressive symptoms in this population.

Conclusion: The EWQ's six-factor structure showed preliminary adequacy in adults with T2D. While internal consistency and convergent validity with the PHQ-9 were supportive, overall model fit indices were moderate; therefore, findings should be interpreted with caution and replicated in larger, more diverse samples before clinical implementation. Future studies should focus on cross-cultural validation, measurement invariance, and longitudinal assessment to refine its clinical utility.

Background: Residual low back pain (LBP) is frequently reported after percutaneous kyphoplasty (PKP) for osteoporotic vertebral fractures (OVFs), yet its underlying mechanisms remain unclear. Paravertebral muscles (PVMs) degeneration, particularly fat infiltration and atrophy may contribute to persistent postoperative pain.

Objective: To evaluate the association between PVMs degeneration and residual LBP after PKP and identify imaging-based predictors for risk stratification.

Methods: This retrospective cohort study included 213 patients (mean age 70.88 ± 8.58 years; 82.2% female) with single-level OVFs who underwent PKP between January 2021 and June 2023. Patients with multiple-level fractures, chronic LBP, neurological deficits, prior spinal surgery, incomplete imaging, or inadequate follow-up were excluded. Fat infiltration percentage (FI%) and cross-sectional area of the multifidus (MF), erector spinae (ES), and psoas major (PS) were measured at the L4 level using transverse T2-weighted MRI. Residual LBP was defined as postoperative VAS ≥3.5 at 12-month follow-up. Logistic regression and ROC analyses were conducted and appropriate univariate tests (t-test or Mann-Whitney U-test) were performed.

Results: Residual LBP occurred in 13.6% of patients and was associated with higher VBQ scores (3.14 ± 0.38 vs 2.57 ± 0.25, P=0.001), greater postoperative kyphosis (16.03 ± 6.69° vs 6.70 ± 4.80°, P=0.001), increased FI% of ES/MF (57.28 ± 5.63% vs 43.40 ± 14.93%, P=0.001), reduced PS area (10.74 ± 4.23 cm² vs 16.15 ± 3.71 cm², P=0.001), and concentrated cement distribution (11.5% vs 73.6%, P=0.001). Independent predictors included elevated VBQ (OR=85.2, 95% CI 7.006-1036.458), kyphosis (OR=1.14, 95% CI 1.017-1.276), FI% of ES/MF (OR=1.082, 95% CI 1.008-1.160), and PS area (OR=0.509, 95% CI 0.285-0.910). ROC analysis identified FI% ≥49.78% and PS area ≤11.937 cm² as optimal cutoffs.

Conclusion: Preoperative magnetic resonance imaging assessment of paravertebral muscle may help identify patients at risk for residual low back pain after kyphoplasty. Incorporating preoperative imaging and postoperative physical therapy referral may improve patient outcomes.

Background: Stroke outcomes are often measured through objective scales, which may neglect subtle cognitive changes and fail to capture patients' subjective experiences of recovery and quality of life. This study aimed to examine the interrelations among self-perceived burden, loneliness, and rumination in stroke survivors through the patient-reported outcomes and to provide theoretical insights and intervention strategies for improving psychological well-being and quality of life in stroke patients.

Methodology: Data from 1024 stroke patients who aged 18 years and above were prospectively collected in September 2022 in Zhengzhou, China. PROs included Self-Perceived Burden Scale, UCLA-Loneliness Scale, and Event-Related Rumination Inventory. Statistical methods employed included correlation analysis and mediation effect analysis.

Results: A total of 1024 participants completed this survey (90.9%), with 56.2% males and a mean age of 62.22 (SD = 13.60) years. Approximately 84.28% of stroke patients felt moderate loneliness. Self-perceived burden was positively correlated with rumination (r = 0.516, 95% CI [0.460, 0.574]) and loneliness (r = 0.370, 95% CI [0.307, 0.431]). Rumination was also positively associated with loneliness (r = 0.493, 95% CI [0.443, 0.541]). Both intrusive and deliberate rumination served as mediators in the relationship between SPB and loneliness (b = 0.119, 55.09%, b = 0.031, 14.35%, respectively).

Conclusion: Intrusive and deliberate rumination mediated the relationship between self-perceived burden and loneliness in stroke patients. Rumination in stroke patients should be emphasized as a modifiable factor to reduce loneliness and improve quality of life.