Patient-reported Outcomes Research Articles

Assessing the measurement properties of PROMIS Computer Adaptive Tests, short forms and legacy patient reported outcome measures in patients undergoing total hip arthroplasty.

The commonly used ('legacy') PROMs evaluating outcomes of total hip arthroplasty (THA), have several limitations regarding their measurement properties and interpretation of scores. One innovation in PROMs is the use of Computerized Adaptive Testing (CAT). The Patient-Reported Outcomes Measurement Information System (PROMIS®) is a validated system of CATs. The aim of this study was to assess the measurement properties of PROMIS and legacy instruments in patients undergoing THA. Patients in this multicenter study filled out a questionnaire twice, including Dutch-Flemish PROMIS v1.2 Physical Function (PROMIS-PF) and v1.1 Pain Interference (PROMIS-PI) CATs and short forms, PROMIS v1.0 Pain Intensity, and legacy PROMs (Hip disability and Osteoarthritis Outcome Score (HOOS), HOOS-Physical function Shortform (HOOS-PS), Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), Oxford Hip Score (OHS), and two numeric rating scales measuring pain). The reliability, measurement precision (Standard Error of Measurement (SEM)), smallest detectable change (SDC), and burden of PROMIS instruments were presented head-to-head to legacy PROMs. Furthermore, construct validity was assessed. 208 patients were included. All instruments had a sufficient test-retest reliability (range ICC: 0.83-0.96). The SEM of PROMIS CATs and short forms ranged from 1.8 to 2.2 T-score points, the SEM of legacy instruments 2.6-11.1. The SDC of PROMIS instruments ranged from 2.1 to 7.3 T-score points, the SDC of legacy instruments 7.2-30.9. The construct validity of PROMIS CAT and short forms were found sufficient, except for the PROMIS-PI short form. The burden of PROMIS CATs was smaller than PROMIS short forms (range 4.8-5.2 versus 8-20 items, respectively). The burden of legacy instruments measuring physical functioning ranged from 5 to 40 items. The PROMIS-PF is less burdensome, with high measurement precision, and almost no minimal or maximal scores, and an equal reliability compared to legacy instruments measuring physical functioning in patients undergoing THA. The PROMIS Pain Intensity 1a is comparable to the legacy pain instruments in terms of burden, reliability and SDC. Measuring the construct Pain Interference may not have additional value in this population because of its high correlation with instruments measuring physical functioning. The SDC values presented in this study can be used for individual patient monitoring.

Development and Validation of a Patient-Reported Outcome Measure of the Impact of Chronic Hand Eczema on Health-Related Quality of Life: the Hand Eczema Impact Scale (HEIS).

Chronic Hand Eczema (CHE) is an inflammatory skin disease that causes significant impact on health-related quality of life (HRQoL). The Hand Eczema Impact Scale (HEIS) is a new patient-reported outcome(PRO) measure designed to assess the impact of CHE on key domains of HRQoL. This study aimed to develop and evaluate content and psychometric validity of the HEIS. The HEIS was initially developed on the basis of a literature review and concept elicitation interviews. Qualitative cognitive debriefing interviews (n = 20) were conducted with patients with CHE to assess relevance and understanding of items, response options, and recall period. Psychometric properties (item performance, dimensionality, reliability, validity, responsiveness, and estimation of meaningful change thresholds) were then assessed using data (n = 258) from a phase 2b trial (NCT03683719). Cognitive debriefing confirmed all items were understood and relevant to patients. Inter-item correlations (all > 0.50) and confirmatory factor analysis (factor loadings ≥ 0.80) supported unidimensionality of the HEIS score, and mostly provided support for the HEIS Proximal Daily Activity Limitations (PDAL) score, with only one item loading below the prespecified threshold. Item properties and previous qualitative work supported retaining this item in the total score but removed from the HEIS PDAL domain. Internal consistency (Cronbach's alpha ≥ 0.89) and test-retest reliability (intra-class correlation coefficient ≥ 0.79) results were very strong. Strong correlations with concurrent measures (0.66-0.87) and significant differences between severity groups (p < 0.001) supported construct validity. Large effect sizes for mean change scores in participants that improved and significant differences between groups indicated ability to detect change. Anchor-based analyses supported within-individual responder definitions of ≥ 1.3 points for improvements in both HEIS score and HEIS PDAL score (covering three items) and of ≥ 1.5 points for HEIS embarrassment with the appearance of hands (Emb) score (covering two items). The 9-item HEIS is the first CHE-specific PROmeasure developed and validated according to regulatory guidance for assessment of the impact of CHE on key domains of HRQoL. This article provides evidence of strong content and psychometric validity and shows improvements of ≥ 1.3 points in HEIS score and HEIS PDAL score, and improvements of ≥ 1.5 points in HEIS Emb score represent clinically meaningful, important changes. NCT03683719.

Decision regret and long-term success rates after ventral buccal mucosa graft urethroplasty.

To characterise the long-term success rate of ventral onlay buccal mucosa graft urethroplasty (vBMG) in the management of bulbar urethral stricture disease (USD), assess patient-reported postoperative satisfaction and decision regret, and delineate clinical factors impacting patient-reported metrics. Patients with prior vBMG for bulbar USD, performed at Cleveland Clinic between 2003 and 2022, were contacted and brief structured interviews were performed. Stricture recurrence and need for secondary procedures, baseline demographics, and patient-reported outcome surveys were collected. The surveys included the Decision Regret Scale (DRS), the Urethral Stricture Symptom Impact Measure (USSIM) and the 10-item Patient-Reported Outcomes Measurement Information System Short Form, version 1.2 (PROMIS-10). Descriptive, univariate and multivariable analyses were performed for clinical outcomes and survey responses. A total of 104 patients recorded responses. The median patient age was 49 years and the median follow-up was 7.4 years at time of survey. The median graft length was 5 cm and 38% of patients underwent partial thickness augmented anastomotic urethroplasty. At time of follow-up, 10 patients underwent a secondary procedure. Moderate to severe regret on the DRS was found in 12% of patients, and greater regret was associated with recurrence. The mean physical and mental health PROMIS-10 Global Health T-scores were 52 and 53. The mean total USSIM score was 56. A significant correlation was found between USSIM and DRS scores, with higher DRS score and recurrence negatively impacting USSIM score. USSIM scoring across all domains was significantly worse in the moderate to severe DRS group. This study showed that vBMG for bulbar USD confers both high success rates and patient-reported satisfaction at extended follow-up, based on emerging and validated patient-reported outcome measures.

Impact of breast reconstruction on quality of life in women undergoing mastectomy for breast cancer: a systematic review and meta-analysis using the Breast-Q questionnaire

Introduction: Post-mastectomy breast reconstruction is crucial in breast cancer treatment; however, its impact on women’s quality of life (QOL) remains inadequately understood. The BREAST-Q questionnaire is used for assessing QOL related to breast surgery. Therefore, this study aimed to determine whether there are differences in QOL between women undergoing mastectomy with and without breast reconstruction using the BREAST-Q questionnaire. Methods: We conducted a systematic review and meta-analysis following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses 2020 guidelines and registered in PROSPERO. Articles in English, Portuguese, or Spanish were included, and studies on prophylactic mastectomy or breast-conserving surgery, duplicate studies, duplicate data, articles without original data (reviews, editorials, case reports, technical reports) and those without breast reconstruction were excluded. A comprehensive search was conducted across nine scientific databases. The defined search strategy was related to the terms standardized by the MeSH/PubMed controlled vocabularies, including Medical Subject Headings (MeSH), Embase subject headings (Emtree), and their synonyms. Statistical heterogeneity was assessed using the chi-squared and inconsistency tests. Statistical analyses were performed using the R software. A meta-analysis was performed for each BREAST-Q domain when data from multiple studies were available. Results: Of 1266 studies identified, three with 611 participants were included in the meta-analysis. Of these participants, 248 underwent mastectomy without reconstruction and 363 underwent post-mastectomy breast reconstruction. The meta-analysis revealed significantly higher scores in the domains “satisfaction with the breast,” “physical well-being of the chest,” “psychosocial well-being,” and “physical well-being” in women who underwent breast reconstruction compared with those who did not. Conclusion: This study demonstrates a positive association between breast reconstruction and enhanced QOL in women undergoing breast cancer treatment and mastectomy. Patient-reported outcome measures using questionnaires, such as Breast-Q, can elucidate satisfaction levels with the reconstruction, which will likely improve continuously with advances in modern plastic surgery.

Lifestyle changes and patient-reported outcomes over five years in a sample of people with multiple sclerosis after a single multimodal intensive lifestyle education workshop.

Modifiable lifestyle risk factors for progression of multiple sclerosis (MS) have been increasingly studied. This study employed a single-group design involving a one-off intensive live-in educational workshop on lifestyle modification for people with MS. We aimed to examine changes in a range of clinical and lifestyle variables and quality of life, self-efficacy, physical impact of MS and disability from baseline to 3- and 5-years post-intervention. 95 participants completed the baseline survey. Data included lifestyle risk factors of diet quality, meat and dairy consumption, omega 3 and vitamin D supplementation, physical activity, stress reducing activities, and smoking status, and use of disease-modifying therapies (DMTs). Patient-reported outcomes included health-related quality of life, self-efficacy, physical impact of MS and disability. Generalised estimating equation models were used to account for within-participant correlation over time. Sixty participants (63.2%) provided data at 3- and 5-years. Significant improvements in diet quality, omega 3 supplementation, and non-smoking were seen at both timepoints. Use of DMTs and disability remained unchanged. Mental (8.8- and 6.9-point) and physical (10.5- and 7.3-point) quality of life, and self-efficacy (2.4- and 1.9-point) improved significantly at 3- and 5-years, respectively. Physical impact of MS reduced from baseline to 3-years (-3.7 points) with a trend towards reduction at 5-years (-2.9 points; p = 0.079). Education on lifestyle modification can lead to lifestyle modification and short and long-term improvements in mental and physical wellbeing outcomes. Results suggest potential value in lifestyle modification as an adjunctive component to standard therapy for MS.

Patient-reported outcomes, and perceptions and knowledge about recurrence in women with hormone receptor-positive breast cancer.

Over half of hormone receptor-positive (HR+) breast cancer recurrences occur >5years from diagnosis, however, little is known about well-being or breast cancer risk perceptions and knowledge in long-term HR+ breast cancer survivors. From 1/2021 to 1/2022, we surveyed patients with a history of stage II/III, HR+ breast cancer, ≥5years from diagnosis, without recurrence about concerns and perceptions related to their diagnosis and recurrence risk, physical and emotional health, knowledge, and risk reduction. Logistic regression identified factors associated with overestimation of 5-10year distant recurrence risk. Among 166 women, median age at diagnosis was 51, 2.4% were Black and 1.2% Hispanic; 19.3% did not have a college degree. Median time from diagnosis was 10years (range: 5-23). Median PROMIS anxiety (53; range: 37-73), physical (51, range: 32-68), and mental (51, range: 25-68) scores were similar to population norms (score of 50). 40% of women estimated metastatic recurrence risk to be ≥20% 5-10years post-treatment; patients without a college degree were more likely to overestimate this risk (multivariable prevalence odds ratio: 3.69, 95% confidence interval: 1.49, 9.18). Only 17% correctly indicated HR+ breast cancer as having a higher risk of recurrence after 5years; over one-third inaccurately responded that alcohol in moderation decreases recurrence risk. While physical and emotional health were comparable to the general population, many survivors harbored inaccurate risk perceptions and knowledge. The association between lower educational attainment and risk overestimation underscores the importance of attention to literacy and numeracy when developing interventions to improve risk communication.

Wearables for the Bladder: Stakeholder Perspectives on Moving Multiple Sclerosis Bladder Dysfunction Interventions Into the 21st Century.

Bladder dysfunction (BD) is common in people with multiple sclerosis (MS) and can reduce participation in daily life. Detecting BD early allows for effective prevention-focused treatments such as pelvic floor physical therapy. Pairing neurotechnology with patient-reported outcomes to remotely measure BD could significantly improve monitoring and treatment of BD. Therefore, we describe the process and findings of stakeholder engagement from a human-centered design process to assemble a wearables for the bladder (WeB) kit. Four people with MS with varying BD severity, and 5 MS clinical/research experts had 4 virtual meetings. Commercially available bladder tools were graded for ability to evaluate, monitor, or treat BD. The Health Information Technology Usability Evaluation Scale (utility, usability, feasibility) was used for evaluation. Scoring was performed individually and as a group. Of the 11 devices, 5 obtained mean scores of greater than 6 of 10 for likability, usability, and device utility. The 2 highest scoring (9/10) devices were selected for the pilot. One device measures bladder urine levels, reporting the number/frequency of voids/leaks; the other guides pelvic floor exercises by pairing games on an app with biofeedback from intravaginal sensors. We uncovered critical differences in experts' and patients' appreciation of the tools, and the collaborative engagement led to substantial revisions of initial tool scores. This process underscores the critical role of stakeholder engagement in the selection of digital tools, especially in sensitive domains like pelvic function. Ongoing clinical validation of the selected tools will yield a validated, user-friendly WeB kit that is able to fill gaps in our ability to evaluate BD treatments in people with MS, ultimately reducing the impact of BD on quality of life.

Percutaneous External Fixation of Redistributed Orbital Fat in Transcutaneous Lower Eyelid Blepharoplasty.

While percutaneous external fixation has gained popularity in transconjunctival blepharoplasty due to its simplicity and minimal incision, internal fixation remains the standard approach in transcutaneous blepharoplasty. This study aims to assess the safety and effectiveness of percutaneous external fixation specifically in transcutaneous fat repositioning blepharoplasty. From May 2022 to December 2023, a consecutive cohort of 97 patients underwent this surgical technique. Building upon the principles of transconjunctival fat repositioning, the procedure involved a minimal incision targeting the muscle layer, a conservative release of the tear trough ligament, and the repositioning of fat secured with percutaneous external fixation. Surgical outcomes were evaluated using the Barton grading system and FACE-Q scales for patient-reported outcomes. Following a mean follow-up of 7.2 months (ranging from 6 to 17 months), a resolution of tear trough deformities and eyelid bags was achieved in over 92% of cases. Notably, significant improvements (P < 0.05) were observed in lower eyelid aesthetics as measured by the FACE-Q scale, with a concomitant enhancement in patients' social confidence (P < 0.05). Overall, patients expressed satisfaction with their decision to undergo the procedure (71.0±19.1). Complications necessitating revision surgery included isolated cases of hematoma evacuation, fat injection, excision of excess fat, and skin excision. External fixation in transcutaneous blepharoplasty is technically less complex and minimally invasive, effectively securing transposed fat pedicles safely, presenting a viable alternative to conventional fixation techniques.

Identifying Clusters of Curatively-Treated Esophageal Cancer Patients Using Patient-Reported Outcome Measures at Three Months from Discharge: A Secondary Analysis from a Longitudinal Single Center Study.

This study aims to identify meaningful clusters based on Patient-Reported Outcome Measures (PROMs) in curatively-treated esophageal cancer patients at three months post-discharge. This secondary analysis of a longitudinal single-center study included 46 esophageal cancer patients who underwent curative surgery. Patients were selected based on their completion of PROMs surveys at three months post-discharge, were aged 18 years or older, and had undergone surgical resection (esophagectomy) with or without neoadjuvant chemotherapy and/or radiotherapy. The analysis utilized t-distributed Stochastic Neighbor Embedding (t-SNE) for dimensionality reduction and hierarchical clustering to analyze PROMs data collected three months post-discharge. Clustering was performed on physical, emotional, cognitive, and social functioning variables, symptom burden, and health literacy. Three distinct clusters were identified: Cluster 1 (n = 24) with higher functioning and moderate symptoms, Cluster 2 (n = 14) with moderate functioning, higher symptoms, and lower health literacy, and Cluster 3 (n = 8) with the highest functioning, lowest symptoms, and highest health literacy. Significant differences between squamous cell carcinoma and adenocarcinoma subtypes were observed across several PROMs domains, including critical health literacy, general health status/quality of life, nausea and vomiting, and insomnia. These clusters provide an exploratory framework for tailoring post-operative interventions to enhance patient recovery, which necessitates further confirmatory investigations, including outcomes such as complications and mortality, in the analysis. This study fills a research gap by demonstrating the utility of PROMs in identifying distinct recovery patterns in esophageal cancer patients post-surgery. The findings support the use of PROMs to guide personalized post-operative care, potentially improving patient outcomes and quality of life. Further research is needed to validate these findings in larger, diverse populations.

Enhancing Clinicians' Use of Electronic Patient-Reported Outcome Measures in Outpatient Care: Mixed Methods Study.

Despite the increasing use of patient-reported outcome measures (PROMs) for collecting self-reported data among hospital outpatients, clinicians' use of these data remains suboptimal. Insight into this issue and strategies to enhance the use of PROMs are critical but limited. This study aimed to examine clinicians' use of PROM data for value-based outpatient consultations and identify efforts to enhance their use of PROMs in a Dutch university hospital. First, we aimed to investigate clinicians' use of outpatients' PROM data in 2023, focusing on adoption, implementation, and maintenance. Second, we aimed to develop insights into the organizational-level strategies implemented to enhance clinicians' use of PROM data from 2020 to 2023. This included understanding the underlying rationales for these strategies and identifying strategies that appeared to be missing to address barriers or leverage facilitators. Third, we aimed to explore the key factors driving and constraining clinicians' use of PROMs in 2023. We integrated data from 4 sources: 1-year performance data on clinicians' use of PROMs (n=70 subdepartments), internal hospital documents from a central support team (n=56), a survey among clinicians (n=47), and interviews with individuals contributing to the organizational-level implementation of PROMs (n=20). The Reach, Effectiveness, Adoption, Implementation, and Maintenance framework was used to analyze clinicians' adoption, implementation, and maintenance of PROMs. Strategies were analyzed using the Expert Recommendations for Implementing Change taxonomy, and results were structured around the constructs of capability, opportunity, and motivation. On average, around 2023, clinicians accessed PROM data for approximately 3 of 20 (14%) patients during their outpatient consultation, despite numerous strategies to improve this practice. We identified issues in adoption, implementation, and maintenance. The hospital's strategies, shaped organically and pragmatically, were related to 27 (37%) out of 73 Expert Recommendations for Implementing Change strategies. These strategies focused on enhancing clinicians' capability, opportunity, and motivation. We found shortcomings in the quality of execution and completeness of strategies in relation to addressing all barriers and leveraging facilitators. We identified variations in the factors influencing the use of PROMs among frequent PROM users, occasional users, and nonusers. Challenges to effective facilitation were apparent, with certain desired strategies being unfeasible or impeded. Enhancing clinicians' use of PROMs has remained challenging despite various strategies aimed at improving their capability, opportunity, and motivation. The use of PROMs may require more substantial changes than initially expected, necessitating a shift in clinicians' professional attitudes and practices. Hospitals can facilitate rather than manage clinicians' genuine use of PROMs. They must prioritize efforts to engage clinicians with PROMs for value-based outpatient care. Specific attention to their professionalization may be warranted. Tailored strategies, designed to address within-group differences in clinicians' needs and motivation, hold promise for future efforts.

The Importance of Patient-Reported Outcome Measures (PROMs) in Oncological Vulvoperineal Defect Reconstruction: A Systematic Review.

Patient-reported outcome measures (PROMs) have gained increased importance in assessing outcomes after reconstructive surgery. This also applies to the reconstruction of vulvoperineal defects after resection of gynecological or colorectal cancers in women. The objective of this study is to analyze the current state of PROM tool use within this patient population. By systematic literature searches in Embase, Medline, and Web of Science, English-language studies published after 1980, including randomized controlled trials, cohort studies, and case series reporting on vulvoperineal defect reconstruction, which were included if they also analyzed quality of life (QoL) and/or PROMs. The PROM tools used by each study were extracted, analyzed, and compared. The primary search yielded 2576 abstracts, of which 395 articles were retrieved in full text. Of these, 50 reported on vulvoperineal defect reconstruction, among which 27 studies analyzing QoL were found. Of those, 17 met the inclusion criteria for this systematic review. After full-text screening, 14 different PROM tools and 5 individual, non-standardized questionnaires were identified. Only 22% of studies used a validated PROM tool. Far too few studies currently use PROM tools to assess outcomes in oncological vulvoperineal defect reconstruction. Less than half of the used PROMs are validated. No PROM was designed to specifically measure QoL in this patient population. The standardized implementation of a validated PROM tool in the clinical treatment of this patient population is an essential step to improve outcomes, enable the comparison of research, and support evidence-based treatment approaches.

Dorsal and Volar Approaches for Proximal Interphalangeal Joint Replacement: Comparing Outcomes Through Systematic Review and Meta-Analysis.

The 2 primary surgical approaches for proximal interphalangeal joint (PIPJ) arthroplasty, dorsal or volar, have been extensively described in the literature. However, the ongoing debate regarding which approach offers superior results or is associated with fewer complications persists. This systematic review aims to compare the outcomes of PIPJ arthroplasty between the dorsal and volar approaches. A comprehensive search of multiple databases was conducted, and studies meeting predetermined criteria were included. Data extraction, assessment of bias risk, and statistical analysis were performed to compare treatment modalities. Outcome measures included range of motion (ROM), patient-reported outcome measures (PROMs), revisions, and reported complications. Among 368 screened articles, 5 studies involving 302 patients (310 implants) were eligible for final review. No significant differences were observed between the 2 approaches regarding postoperative ROM (mean difference [MD] 2.24; 95% confidence interval [CI] -3.83, 8.32; P = .47) and PROMs (standardized mean difference [SMD] 0.18; 95% CI -0.12, 0.48; P = .25). Complication rates, including revision/fusion, persistent pain, stiffness, infection, and dislocation, did not significantly differ between the approaches. Notably, dorsal approach was associated with higher risk of swan-neck deformity (9 out of 82 implants), while no such cases were reported in the volar approach (0 out of 101 implants). Despite limitations and heterogeneity in the literature, both dorsal and volar approaches for PIPJ arthroplasty appear to yield equivalent outcomes for patients. II, therapeutic.

Comparing Innervated and Non-Innervated Glabrous Skin Flaps for Volar Digital Defects: Insights from Patient-Reported Outcomes.

Volar soft tissue defects in digits necessitate reconstructions that restore appearance sensation and minimize complications. This study compares innervated toe pulp (TP) and non-innervated medialis pedis (MP) flaps for reconstructing such defects, focusing on objective and subjective outcomes. Between 1998 and 2017, 101 free glabrous skin flap reconstructions were performed at our institution for volar digital soft tissue injuries, comprising 75 TP and 26 MP flaps. Follow-up assessments included the Michigan Hand Outcomes Questionnaire, Disabilities of the Arm, Shoulder and Hand Questionnaire, Foot and Ankle Disability Index, and sensory testing (static and moving two-point discrimination, Semmes-Weinstein monofilament test). Data analysis utilized the Mann-Whitney U test and Pearson correlation coefficients. The study cohort included 29 participants (15 TP, 14 MP) with an average follow-up of 106 months. Functional and sensory outcomes revealed no significant differences between TP and MP flaps. Both flap types achieved satisfactory function and sensibility, with no statistically significant distinctions in patient-reported outcomes. Subjective complaints were similarly distributed across both groups, with a few reports of cold intolerance and discomfort at the donor site in the TP group. TP and MP flaps provide adequate coverage and sensory outcomes for volar digital defects without significant differences between innervated and non-innervated flap transfers. The flap choice should be tailored to individual patient needs and defect characteristics, emphasizing the importance of patient-centered decision-making in reconstructive surgery. Further research is required to explore the long-term outcomes of these reconstruction methods, especially for larger defects.

Development and validation of the NCC-BC-A scale to assess patient-reported outcomes for breast cancer patients in China.

The commonly used international patient-reported outcome scales for breast cancer were developed before the advent of multiple targeted therapies and immunotherapies, rendering them potentially insufficient for current clinical practices. Therefore, it is necessary to develop a specific patient-reported outcome scale tailored for breast cancer patients in China to optimize the management model for these patients. A comprehensive literature search was performed in the PubMed, Embase, Wanfang, and CNKI databases to extract dimensions and items for a potential patient-reported outcome scale. The Delphi method was used to modify, add, subtract, and adjust the language of items until the experts reached a consensus on the first draft. This draft was further refined using a cognitive test and a presurvey. The optimized scale was used for a formal survey, and the items were further analyzed and screened using metrics such as the coefficient of variation, correlation coefficient, internal item consistency, factor analysis, reliability, and validity. A total of 10,954 articles were analyzed, and 237 were used to create a pool of 277 patient-reported outcome items. Through two rounds of Delphi expert consultation, the experts' authority coefficients were 0.739 and 0.826. After a cognitive test, several items were adjusted to enhance understanding. Further adjustments were made following a presurvey of 200 advanced breast cancer patients, resulting in a 38-item patient-reported outcomes scale, termed NCC-BC-A. In the national formal survey, 588 advanced breast cancer patients participated. Principal component analysis showed good consistency among the items and sufficient difference between the dimensions. The results were normally distributed with good variation. The Cronbach's α coefficient of the scale was 0.925 and the test-retest reliability was 0.9041. The NCC-BC-A scale has high validity and reliability. It comprehensively considered the characteristics of systemic treatment for breast cancer, and the specific context within China. Its implementation may help clinicians to pay more attention to quality of life of breast cancer patients and to optimize the system for managing this condition.

Concurrent validity of the Single Assessment Numerical Evaluation and hip-specific patient-reported outcome measures.

The Single Assessment Numerical Evalution (SANE) score is a pragmatic alternative to longer patient-reported outcome measures (PROMs). The purpose of this study was to investigate the concurrent validity of the SANE and hip-specific PROMs in a generalized population of patients with hip pain at a single timepoint upon initial visit with an orthopaedic surgeon who is a hip preservation specialist. We hypothesized that SANE would have a strong correlation with the 12-question International Hip Outcome Tool (iHOT)-12, the Hip Outcome Score (HOS), and the Hip disability and Osteoarthritis Outcome Score (HOOS), providing evidence for concurrent validity of the SANE and hip-specific outcome measures in patients with hip pain. This study was a cross-sectional retrospective database analysis at a single timepoint. Data were collected from 2,782 patients at initial evaluation with a hip preservation specialist using the iHOT-12, HOS, HOOS, and SANE. Outcome scores were retrospectively analyzed using Pearson correlation coefficients. Mean raw scores were iHOT-12 67.01 (SD 29.52), HOS 58.42 (SD 26.26), HOOS 86.85 (SD 32.94), and SANE 49.60 (SD 27.92). SANE was moderately correlated with the iHOT-12 (r = -0.4; 95% CI -0.35 to -0.44; p < 0.001), HOS (r = 0.57; 95% CI 0.53 to 0.60; p < 0.001), and HOOS (r = -0.55; 95% CI -0.51 to -0.58; p < 0.001). The iHOT-12 and HOOS were recorded as a lower score, indicating better function, which accounts for the negative r values. This study was the first to investigate the relationship between the SANE and the iHOT-12, HOS, and HOOS in a population of patients with hip pain at the initial evaluation with an orthopaedic surgeon, and found moderate correlation between SANE and the iHOT-12, HOS, and HOOS. The SANE may be a pragmatic alternative for clinical benchmarking in a general population of patients with hip pain. The construct validity of the SANE should be questioned compared to legacy measures whose content validity has been more rigorously investigated.

Using a Patient-Reported Outcome Measure to Assess Physical, Psychosocial, and Existential Issues in COPD.

Background: Chronic obstructive pulmonary disease (COPD) is marked by severe physical symptoms, impaired quality of life, and high psychological distress. Despite its impact, the identification of not only physical but also psychosocial and existential issues in the clinic lags behind that of other patient groups. Methods: This study aimed to assess physical, psychosocial, and existential issues among patients with COPD using a patient-reported outcome measure for general palliative care (the 'PRO-Pall') in a Danish outpatient clinic. We included 115 adults with COPD who completed the PRO-Pall either electronically or in the clinic. Sociodemographic and illness-related data were retrieved from their electronic health records. Results: We found that shortness of breath, tiredness, and difficulty walking were predominant physical issues. Worry about change in social roles was the most frequently reported psychosocial issue, while existential issues were reported by approximately one in ten patients. Most patients (44.5%) felt able to share their feelings with family or friends, and a majority (62.2%) felt their illness-related issues were addressed satisfactorily. Females expressed a greater need for rest and males more frequently reported intimacy issues. Higher COPD-impact on life measured by the COPD Assessment Test was associated with lower ratings on the quality-of-life item of the PRO-Pall independent of age, gender, lung function, and smoking status. Conclusions: Patients reported issues in physical, psychosocial, and existential dimensions of the PRO-Pall. The PRO-Pall shows potential as a broader alternative to measures that focus mainly on physical issues.

Validation of 11 added items of the outpatient version of the Utrecht Symptom Diary in patients receiving chemotherapy or targeted therapy

IntroductionThe Utrecht Symptom Diary (USD) is a validated Dutch patient-reported outcome measurement (PROM) tool - based on the Edmonton Symptom Assessment System - to assess and monitor symptoms in cancer patients. The USD contains 11 items concerning frequently occurring symptoms in cancer patients (pain, sleeping problems, dry mouth, dysphagia, lack of appetite, abnormal stool, nausea, shortness of breath, fatigue, anxiety and depressed mood) and an item on overall well-being. For the outpatient USD 11 items concerning frequently occurring signs and symptoms in patients receiving chemotherapy and/or targeted therapy were added to the USD: taste alteration, oral pain, weight loss, diarrhoea, hair changes, skin problems, nail problems, eye problems, tingling, concentration problems and problems with sexuality. This current study aimed to evaluate the 11 added items on this treatment specific outpatient USD in cancer patients receiving intravenous chemotherapy and/or targeted therapy.MethodsObservational longitudinal retrospective cohort study including all adult outpatients with cancer receiving intravenous chemotherapy and/or targeted therapy in an academic hospital in the Netherlands who completed at least one outpatient USD as part of routine care (2012–2021). Relevance, comprehensiveness as well as criterion and construct validity were assessed.Results1733 patients who completed ≥ 1 outpatient USD during intravenous chemotherapy and/or targeted therapy were included for analysis. Relevance as well as comprehensiveness of the items on the outpatient USD in this patient population was shown. Criterion validation was demonstrated for all added items of the outpatient USD – except for the item on oral pain. An additional analysis showed that mouth problems were detected with both outpatient USD items oral pain and dry mouth. Construct validity was demonstrated for the items hair changes and skin and nail problems. Construct validity on eye problems was not tested due to the low number of paired outpatient USDs.ConclusionsThe treatment specific outpatient USD is a validated PROM in outpatients with cancer receiving intravenous chemotherapy and/or targeted therapy. Considering its validity in this broad group of patients, we think the treatment-specific outpatient USD is widely applicable. In addition to providing tailored supportive symptom care, the USD-data can be used to increase knowledge about symptom burden in daily practice in this population.

The value of assessing deep disease healing by probe-based confocal laser endomicroscopy and histology for long-term prognosis of ulcerative colitis.

The benefits of deep disease healing need evaluation by long-term clinical research in different populations. Confocal laser endomicroscopy (CLE) is a superior method for evaluating deep disease healing. This prospective study enrolled ulcerative colitis (UC) patients in clinical remission who underwent colonoscopy, CLE, and histological assessment. Patients were monitored for relapse by patient-reported outcomes and colonoscopy evaluation of mucosal healing. The ability of different methods of mucosal healing to predict long-term disease recurrence was assessed using Kaplan-Meier estimation and Cox proportional hazard regression. Forty-two patients in clinical remission were assessed by colonoscopy. Those with Mayo endoscopic subscores (MES)≤1 were enrolled. The 48-month recurrence rates in present healing group, assessed by CLE (colonic barrier assessment and ENHANCE index) and by histological examination (Geboes scale), were 20.0%, 26.7%, and 11.1%, respectively, and were significantly lower than absent healing group (P<0.05). Univariate Cox proportional risk regression analysis in absent of healing disease, determined by the ENHANCE index and Geboes scale, indicated an increased risk of recurrent events, with hazard ratios (HR) of 3.87 (95% CI: 1.18, 12.62) and 8.20 (95% CI: 1.06, 63.30), respectively. Multivariate Cox proportional hazard regression analysis adjusted for the extent of inflammation (E3 or not) showed a significant difference only for the ENHANCE index, with an HR of 3.53 (95% CI: 1.03, 12.10), P=0.045. Deep disease healing has a lower recurrence rate. The colonic barrier healing assessment, ENHANCE index, and histological Geboes scale have superior long-term prognostic value for UC patients.

3D-printed custom ankle braces for people with Charcot-Marie-Tooth disease: A pilot study.

Charcot-Marie-Tooth disease (CMT) is a neurodegenerative condition resulting in footdrop, ankle instability and impaired balance and gait. This study aimed to determine (1) whether 3D-printed custom ankle braces improve function and balance in people with CMT and (2) whether this is an acceptable device for use in this population. A within-subject comparison pragmatic/pilot study was undertaken. Ten people with CMT (mean [SD] age 48 [14] years, 60% male) were fitted with 3D-printed ankle braces. Following a 4-week wear-in period, walking and balance tests and patient-reported outcomes were assessed in two experimental conditions: (i) usual shoes and (ii) usual shoes with 3D-printed custom ankle braces. Differences in outcome measures between experimental conditions were analysed using linear mixed models. Comfort, aesthetics and overall satisfaction of the brace were assessed via 100-mm visual analogue scale (VAS). Adverse events and tripping/falls associated with the brace during the wear-in period were also recorded by participants using daily diaries. A significant improvement was seen during single-leg balance with eyes open (p=0.026, Cohen's d=0.55) and a significant reduction in foot pain (p=0.045, Cohen's d=0.82), with use of the ankle brace. Mean (SD) 100mm VAS scores were 62.7mm (17.9) for overall comfort and 73.9mm (21.2) for overall satisfaction. Subjective data from the daily dairies showed that one participant found the brace too firm around the ankle due to loss of soft tissue mass and two participants found it challenging to don and doff the brace due to loss of hand dexterity. This pilot study suggests that a 3D-printed custom ankle brace may improve balance and reduce foot pain in people with CMT; however, larger-scale trials are needed to further explore the impact of this brace on function and balance. Further customisation of the brace may also be required to improve acceptability for some people.

The Microneedle Revolution: Transforming Vitiligo Treatment Landscapes-A Comprehensive Review of the Literature.

Vitiligo is characterized by the emergence of depigmented patches on the skin, posing significant therapeutic challenges. Microneedling, a novel treatment strategy, capitalizes on its potential to invigorate skin regeneration and facilitate melanocyte migration. This review critically evaluates the efficacy, safety, and patient satisfaction associated with microneedling for vitiligo treatment, juxtaposing it with traditional therapies to elucidate its viability as either a standalone or complementary treatment modality. A comprehensive literature review was conducted across major databases, including PubMed and Cochrane Library, using keywords like "microneedling" and "vitiligo," encompassing studies up to January 2024. This review focused on microneedling's effectiveness, safety, and patient satisfaction, including both solo and combined treatments. Data synthesis emphasized repigmentation rates, side effects, and patient feedback. An exhaustive review of studies pertaining to microneedling for vitiligo, especially focusing on outcomes when administered alone or in conjunction with agents such as tacrolimus and 5-fluorouracil, was undertaken. Key evaluation metrics included repigmentation success rates, adverse effects, and patient-reported outcomes. Microneedling demonstrates a favourable safety profile, with mild and manageable side effects such as pain, erythema, and temporary hyperpigmentation. Severe adverse events are rare, further supporting its safety as a treatment option for vitiligo. The evidence amassed suggests that microneedling significantly augments repigmentation rates, particularly in synergy with topical treatments. It is generally well-tolerated, manifests minimal adverse effects, and has garnered high patient satisfaction levels. Nonetheless, the absence of standardized treatment protocols and paucity of long-term efficacy data underscore the imperative for further empirical inquiry. Microneedling emerges as a propitious treatment alternative for vitiligo, demonstrating both safety and efficacy, notably when used in tandem with topical agents. Future investigations should aim at protocol standardization and long-term efficacy evaluation to firmly establish its therapeutic stature within vitiligo management paradigms.