Journal of Patient-Reported Outcomes最新文献

Background: The main objective was to evaluate the psychometric properties of the OutPatient Experience Questionnaire for Child and Adolescent Mental Health Services (OPEQ-CAMHS) among patients aged 16 and above, with a secondary objective of developing a parsimonious set of items and a short version of the instrument for this age group.

Methods: A national pilot study was conducted with adolescents from outpatient CAMHS in Norway, testing a new digital, continuous measurement approach using a measurement instrument developed through a comprehensive methodological framework. The study assessed missing data, ceiling effects, factor structure, internal consistency, discriminant validity, and construct validity. A shorter version was derived from psychometric results.

Results: The pilot study included responses from 555 (46.3%) patients. Low proportions of missing or "not applicable" responses were found for 18 of the 20 items, and all items were below the ceiling-effect criterion. Two scales emerged: "structure and process", and "outcome", both meeting alpha criteria. Each individual item demonstrated a stronger correlation with its expected scale than with any of the other scales. Construct validity was confirmed through associations with relevant variables expected to be associated with patient-reported experiences, including self-reported current state and well-being. The results supported a six-item short version.

Conclusions: Psychometric testing confirmed data quality, internal consistency, and construct validity of OPEQ-CAMHS. The short version addresses respondent burden concerns and is now ready for broad implementation in Norwegian CAMHS and potentially in similar healthcare settings worldwide.

Background: The Stool Frequency (SF) and Abdominal Pain (AP) items from the Crohn's Disease Activity Index are together referred to as the "Patient Reported Outcome" (PRO). The SF item measures the number of very soft/liquid stools and the AP item measures abdominal pain severity, which are common Crohn's disease (CD) symptoms that patients consider important to treat. This study evaluated the psychometric properties of both PRO items separately and estimated thresholds for clinical remission in moderately to severely active CD.

Methods: The measurement properties of the PRO items were analyzed using pooled data from VIVID-1 (NCT03926130), a Phase 3, randomized, placebo- and active-controlled study in adults with moderately to severely active CD. Analyses used weekly average scores of the SF and AP items at Weeks 0 (Baseline), 4, 12, and 52. Remission thresholds were estimated using the Patient Global Rating of Severity (PGRS) and Patient Global Impression of Change (PGIC) as primary anchors as well as qualitative evidence from exit interviews.

Results: Data from 1065 participants (mean age: 36.2 years [standard deviation: 13 years]) were analyzed. During the trial, scores improved for both PRO items. Both items demonstrated moderate-to-good test-retest reliability for participants defined as stable based on PGRS and PGIC. Most correlations of related assessments were moderate (0.30≤|ρ| <0.70) with SF and moderate-to-large (0.30≤|ρ| ≤0.90) with AP. By contrast, as anticipated, both items had weak correlations (|ρ| <0.30) with endoscopic and laboratory assessments. The PRO items could discriminate between groups of participants known to differ based on other assessments. The PRO items were able to detect change, as score changes in both items between Baseline and Weeks 12 and 52 differed significantly between most PGRS and PGIC categories. Anchor-based analyses combined with responses from the exit interviews suggested that an SF score of ≤ 3 and an AP score of ≤ 1 could together represent clinical remission.

Conclusion: These results support the reliability, construct-validity, and responsiveness of both PRO items in moderately to severely active CD and confirm previously suggested scores for both items that could represent clinical remission.

Trial registration: Clinicaltrials.gov, NCT03926130. Registered 23 April 2019, https://clinicaltrials.gov/study/NCT03926130 .

Background: The Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep Disturbance v1.0 item bank (27 items) measures sleep disturbances. Rather than the full item bank, an 8-item short form (PROMIS SD SF 8b) or computerized adaptive testing (CAT) can be used. This study compares the performance of the PROMIS SD SF 8b with two CAT algorithms in postmenopausal women.

Methods: This is a secondary analysis of data collected for the original psychometric testing of the PROMIS Sleep Disturbance item bank, in a sub-sample of women aged ≥55. A graded response model (GRM) was fitted for the item bank, then simulations evaluated the performance of CAT algorithms and the short form, in terms of root mean square error (RMSE) versus the latent trait estimate derived from the full bank. Two CAT algorithms were tested: CAT1 (stop once standard error <0.3 or 12 items administered) and CAT2 (stop once 8 items administered). Convergent and divergent hypotheses for validity were tested through correlations with the Pittsburgh Sleep Quality Index (PSQI) and Epworth Sleepiness Scale (ESS). Known-groups comparisons were made between those with and without self-reported sleep disorder.

Results: A sample of 337 women was analyzed. Unidimensionality and item-level fit to the GRM was supported; however, the local independence assumption was violated. The CAT1 algorithm showed 4.18 items on average, with a minor decrease in performance (higher RMSE value) compared to CAT2 or the PROMIS SD SF 8b. Administering 8 items adaptively (CAT2) compared to fixed (PROMIS SD SF 8b) performed similarly (RMSE difference = 0.001). Reliability exceeded 0.90 across most of the latent trait for all approaches. Correlations with the PSQI and ESS were largely as hypothesized, with minor differences in coefficient values between the approaches (all within 0.05). Women reporting a sleep disorder had greater sleep disturbance than those who did not (p < 0.001 for all).

Conclusions: The results of this study support using the PROMIS Sleep Disturbance item bank in postmenopausal women. The choice of PROMIS SD SF 8b versus CAT can largely be driven by practical reasons (respondent burden and operational complexity) rather than concerns of differential reliability and validity.

Background: Upper extremity physical function is an essential health domain in the rehabilitation care for patients with upper limb amputation or absence. The PROMIS-9 UE Physical Function short form is a recently established instrument designed for individuals with upper limb amputation or absence. The instrument's responsiveness to changes after receiving a prosthesis has not been investigated. The current study aimed to evaluate the ability of the PROMIS-9 UE to detect changes in bimanual (two-handed) functional capacity after patients' receipt of a prosthesis.

Methodology: A retrospective chart review was conducted on the longitudinal PROMIS-9 UE outcome measure scores completed between April 2016 and February 2024. Participants included individuals with an outcome collected before and after prosthesis receipt.

Results: The final sample size included 124 individuals (91 male, 33 female; 43.4 ± 15.0 years old, 34.4 ± 103.0 months since amputation, and 62.9% injury etiology). Analyses found significant improvement across all patients in the PROMIS-9 UE scores from baseline to post prosthesis intervention (baseline: 25.3 ± 8.6, post: 29.5 ± 9.6; p < 0.001). The PROMIS-9 UE demonstrated a moderate responsiveness (Standardized Response Mean = 0.6) to prosthetic intervention. This was a directional change consistent with subjective patient reports of increased functionality. Furthermore, findings from the linear mixed model demonstrated significant responsiveness for the PROMIS-9 UE instrument to detect post-intervention changes after controlling for potential confounding effects (p < 0.001).

Conclusions: These findings suggest the PROMIS-9 UE Physical Function instrument demonstrates moderate clinical utility in capturing patient progress following upper limb prosthesis intervention.

Purpose: Diabetes Mellitus (DM) management is increasingly focusing on patient-centered care, making patient-reported experience measures (PREMs) critical for understanding the subjective aspects of diabetes treatment and self-management. These measures differ based on cultural contexts and individual perspectives, leading different countries to the development of country-specific tools to assess care quality from the patient's viewpoint. This review aimed to identify available instruments for assessing patient-reported experiences in individuals with diabetes and examine the different domains, items, and the validity and reliability of these instruments.

Methods: Following PRISMA-ScR guidelines, databases including PubMed, Embase, CINAHL, Cochrane, and Scopus were searched for English-language articles without year limitations. This scoping review focused on PREMs that evaluate the quality of diabetes care among adolescent and adult patients with type 1 and type 2 DM. Studies that used patient expectation questionnaires, involved individuals not receiving care, or focused on patient-reported outcomes rather than experiences were excluded.

Results: Eight articles from six countries representing different healthcare settings were included, mostly from developed countries. A variety of methodologies were used to develop these PREM instruments, with unique domains and items. Content analysis revealed five commonly measured domains: (1) care planning, (2) patient education, (3) professionalism, (4) quality of care, and (5) hospital care and transition, reflecting diverse patient experiences across healthcare services.

Conclusions: This scoping review identifies a limited number of tools for evaluating PREMs in diabetes care, highlighting variability in their development and domain coverage. Five core domains are proposed across different settings, with an emphasis on culturally adapted measures to enhance the accuracy of patient experience capture in diverse populations.

Background: Peripheral artery disease (PAD) negatively affects walking performance, health-related quality of life (HRQoL) and mental health. Exercise training is recommended as a first-line treatment for PAD, with potential impact on all these outcomes, but the optimal program design is not completely ascertained. The aim of this study was to compare arm-ergometry (AEx) and treadmill supervised exercise training (TEx) on HRQoL and mental health in patients with PAD.

Methods: This was an ancillary study of the ARMEX trial, a single-center, single-blinded, parallel group, randomized clinical trial, enrolling symptomatic PAD patients referred to a cardiovascular rehabilitation program (CRP). Participants were randomized (1:1) to a 12-week AEx or TEx, along with the core components of a CRP (nutritional and psychological support). Participants completed the short form 36 Health Survey and the Hospital Anxiety and Depression scale before and after the intervention. Differences between groups in the change from baseline to the end of the study were analyzed using ANCOVA, adjusted for baseline values, or the Mann-Whitney U test.

Results: Fifty-six patients (66 ± 8.4 years; 87.5% male) were included: AEx (n = 28) and TEx (n = 28). Physical functioning, role-physical, bodily-pain, general health, mental health and physical component summary (PCS) significantly improved in AEx group. In the TEx group, physical functioning, role-physical, bodily-pain, vitality, social functioning, role-emotional and PCS significantly improved. Role-physical and role-emotional improved more in TEx, with no between-group differences in the other domains. Changes in PCS were significantly associated with changes in walking distances. Hospital Anxiety and Depression scale scores improved in both groups, without between-group differences. This improvement was associated with self-reported walking distance.

Conclusion: Both exercise protocols improved HRQoL and mental health in patients with symptomatic PAD, highlighting exercise-based programs as important treatment strategies for this population.

Trial registration number: ISRCTN54908548 (retrospectively registered).

Background: Ankle fracture patients are a heterogenous group with differences in age, sex, fracture morphology, and treatment provided. With the increased focus on patient-centered treatment, patient-reported outcome measures (PROMs) are increasingly adopted by clinicians to facilitate best clinical practice. The Manchester Oxford Foot Questionnaire (MOXFQ) has demonstrated good measurement properties when used in patients with foot or ankle disease. The PROM has three domains: (1) Pain; (2) Walking/Standing; and (3) Social Interaction. One study found sufficient content validity for the Pain and Walking/Standing domains when used in the evaluation of ankle fracture patients. Another validation study demonstrated acceptable structural validity and reliability for the MOXFQ in ankle fracture patients 12 weeks after injury. The aim of this study is to assess the structural validity and reliability of the Norwegian version of the MOXFQ in the context of an ankle fracture patients one year after surgery and provide patient acceptable symptom state (PASS) estimates.

Methods: A pragmatic cross-sectional study design was used to collect the one-year MOXFQ follow-up data from patients surgically treated for an ankle fracture in the period 2017 to 2020 at (Stavanger University Hospital). The structural validity and internal consistency were assessed using confirmatory factor analysis. A separate test-retest study including patients at least one year since ankle surgery was used in the assessment of reliability and measurement error.

Results: A confirmatory factor analysis of the three-factor model of the MOXFQ had a good model fit (TLI 0.94; CFI 0.95; RMSEA 0.094; SRMR 0.039). However, the measurement model demonstrated poor discriminant validity of the three factors. A unidimensional model of the 16 items had worse model fit, while a second-order factor model demonstrated strong factor loadings for a second-order factor. A bi-factor model also revealed a strong general factor but also unique variance in the Pain and Social Interaction domain. The domains had good internal consistency (McDonald's omega 0.80 to 0.95) and test-retest reliability (ICC 0.80 to 0.92). The standard errors of measurements for the three domains were between 6.5 and 7.5, and 5.5 for the MOXFQ-Index (scale 0 to 100). PASS estimates for the (sub)scales were: Pain 45; Walking/Standing 39; Social Interaction 19; and MOXFQ-Index 34.

Conclusion: The MOXFQ with three domains demonstrated sufficient structural validity and reliability when used in the evaluation of a one-year postoperative ankle fracture population. Reporting the scores of the Pain and Walking/Standing domains was best supported.

Background: Multiple diseases, such as Adolescent Idiopathic Scoliosis (AIS), present at adolescent age and the impact on quality of life (QoL) prolongs into adulthood. For the EQ-5D, a commonly used instrument to measure QoL, the current guideline is ambiguous whether the youth or adult version is to be preferred at adolescent age. To assess which is most suitable, this study tested for equivalence along predefined criteria of the youth (EQ-5D-5L) and adult (EQ-5D-Y-5L) version in an adolescent population receiving bracing therapy for AIS.

Methodology: 107 adolescents were recruited from 4 scoliosis centers in the Netherlands between March 2022 and January 2023; they completed both EQ-5D's and the SRS-22r (scoliosis-specific questionnaire). The following criteria were evaluated using the individual and sum of domains (level-sum-score (LSS)). Our primary criterion for non-equivalence of the EQ-5D's was less than excellent (≤ 0.9) intra-individual agreement using Intraclass Correlation Coefficient (ICC) analysis for LSS and weighted (quadratic) kappa for domains. Secondary criteria were differences in ceiling using McNemar test; a different number of quantified hypotheses for construct validity achieved using the SRS-22r as comparator; differences in test-retest reliability by comparing ICC/kappa values using a Z-test.

Results: Adolescents had a mean age of 14 years (range 12-18), and 78% were female. Ceiling was mostly comparable between EQ-5D's, ranging from 78 to 81% for mobility and self-care, 52-54% for usual activities, and 31-36% for pain/discomfort. The EQ-5D-5L showed more ceiling (57%) compared to the EQ-5D-Y-5L (41%) on anxiety/depression (p = 0.006). Agreement between the EQ-5D's did not meet our criterion for the LSS (ICC 0.79 (95% confidence interval 0.70-0.85)), and decreased further at the domain-level. Both EQ-5D's achieved 5/7 validity hypotheses. Test-retest reliability was slightly better for EQ-5D-5L LSS (ICC 0.76 (0.64-0.84)) compared to EQ-5D-Y-5L LSS (ICC 0.69 (0.55-0.79)), although this was statistically insignificant (p = 0.284). This pattern was similar for most domains.

Conclusions: The EQ-5D versions showed insufficient agreement, and cannot be considered fully equivalent. While they were similar in terms of validity and test-retest reliability, differences in score distribution were present. Taken together, we advise using the EQ-5D-5L to monitor the QoL in adolescent patients with AIS, as it avoids switching instruments and thus data discontinuities. Future studies should verify these findings in different patient groups and the general population.

Background: Rising number of children with complex medical conditions necessitate regular healthcare quality evaluation to achieve optimal outcomes. To address the need for a periodic and quick assessment of quality of care in serious childhood illnesses, we developed a short version of previously validated 45-item PaRental Experience with care for Children with serIOUS illnesses (PRECIOUS) measure.

Methodology: PRECIOUS was administered by parents of children living with serious illnesses at two time-points (baseline and two weeks) in an online survey. PRECIOUS Short-Form (PRECIOUS-SF) items were derived from the full PRECIOUS measure, which comprises five scales, using an exploratory factor analysis and best subset regression. The measurement properties of PRECIOUS-SF scales were assessed using the concurrent validity using Pearson correlation (r) with the PRECIOUS scales, internal consistency (Cronbach's α) within each scale, convergent validity with overall QoC rating, and test-retest reliability (intraclass correlation coefficient, ICC) between baseline and two-week responses.

Results: PRECIOUS-SF included 10 items across four scales - (1) access to financial and medical resources (2), collaborative and goal-concordant care (3), caregiver support and respectful care and (4) reduction of caregiving stressors. A fifth and optional scale was suggested for hospitalization-specific processes. PRECIOUS-SF scales correlated strongly with corresponding PRECIOUS scales (r = 0.91 to 0.98) and demonstrated satisfactory internal consistency (α = 0.77 to 0.91) and test-retest reliability (ICCs > 0.70).

Conclusions: PRECIOUS-SF demonstrated internal consistency, convergent validity, test-retest reliability, and concurrent validity with PRECIOUS. PRECIOUS-SF offers a practical tool for routine quality of care assessment in pediatric serious illnesses for promoting timely service evaluation and quality improvement.

Background: Chronic rhinosinusitis (inclusive of subtypes with nasal polyps [CRSwNP], without nasal polyps [CRSsNP], and allergic fungal rhinosinusitis [AFRS]) causes inflammation of the nose mucosa and paranasal sinuses. Unfortunately, evidence supporting use of clinical outcome assessments (COAs) in regulated clinical trials to assess key measurement concepts of these conditions is limited.

Objective: To identify key disease-related symptoms and impacts, potential outcomes of interest for new treatments, and COAs available to measure those outcomes among adult and adolescent individuals living with CRSwNP, CRSsNP, and AFRS.

Methods: Literature, clinical trial, and product label reviews were conducted to identify symptoms, impacts, and COAs used to assess CRSwNP, CRSsNP, and AFRS patient experiences in clinical trials. The disease related concepts identified in the literature were mapped to selected COAs to determine conceptual coverage of each COA.

Results: Twenty-five articles, twenty-five clinical trial records, and four product labels were included in the review. Across conditions, nasal obstruction, nasal discharge, and altered smell were the most frequently identified symptoms. The most frequently identified impacts of CRSwNP and CRSsNP were on emotional functioning and sleep, and adopting new behaviors for AFRS. Findings for key symptoms and impacts in adolescents were limited. More than 20 COAs used in these conditions were identified, and 14 COAs (e.g., Sinonasal Outcome Test [SNOT-22]) were evaluated for conceptual coverage of the concepts identified in the literature.

Conclusion: Results specify several symptom and impact outcomes, that if improved, would reflect treatment benefit for patients living with CRSwNP, CRSsNP, and/or AFRS. Several COAs demonstrated coverage of key measurement concepts and warrant further evaluation for use in clinical trials.