OBJECTIVE: Mapping or matching the items in a clinical outcome assessment (COA) to concepts that define a condition is a common method for evaluating a COA\'s concept coverage. The purpose of this research was to address the lack of formal guidance for conducting this task by developing a framework for best practices in COA concept mapping and applying it to a case study.
METHODS: To develop the framework, we examined the literature and created a draft set of best practices which was then reviewed by experienced researchers through focus groups before being finalized. To conduct the case study, we extracted data from a systematic review of knee osteoarthritis (KO) symptoms and impacts and used the framework to map relevant concepts to items in the SF-36v2® Health Survey (SF-36v2).
RESULTS: The framework guides researchers in defining the purpose of and data sources for the mapping, establishing guiding principles and decision-making thresholds, and conducting the mapping exercise. The results of the case study demonstrate the usefulness of the framework in identifying 27/36 items (75%) in the SF-36v2 that addressed concepts that define KO.
CONCLUSIONS: This case study illustrates how the framework for best practices in COA concept mapping may be used, highlighting how establishing clear concept definitions and guiding principles and following a structured process throughout can help produce consistent, reliable, and reproducible results. The results from this rigorous approach can provide valuable evidence to support decisions about the appropriateness of a COA for the intended patient population.
In health-related quality of life research, mapping items in a clinical outcome assessment (COA) to concepts that define a health condition is one way to evaluate an instrument’s content validity (or, how well the instrument addresses the concepts it intends to measure). Without formal guidance on how to do this mapping, researchers can be inconsistent. This article describes the development of a framework for best practices in COA concept mapping. Informed by the literature and input from researchers with expertise in COA development and evaluation, the final framework guides researchers through the mapping process from start to finish, from helping to define the purpose of the task and identify the data sources, to establishing guiding principles and decision-making thresholds, conducting the mapping, and displaying the results. A case study—in which items from the SF-36v2® Health Survey were mapped to concepts from a systematic review of knee osteoarthritis symptoms and impacts—shows the framework in action, demonstrating how following the best practices can lead to consistent results that can support the evaluation of an instrument’s content validity.

BACKGROUND: Total Knee Arthroplasty (TKA) is frequently performed for advanced osteoarthritis, with patient-reported outcome measures (PROMs) traditionally reporting on efficacy. These subjective evaluations, although useful, may inaccurately reflect post-TKA activity levels. With technological advancements, smart implantable devices (SIDs) offer objective, real-time gait metrics, potentially providing a more accurate postoperative recovery assessment. This study compares these objective metrics with PROMs to evaluate TKA success more effectively.
METHODS: We conducted a retrospective cohort study with 88 participants undergoing TKA using a SID. Eligible patients were aged 18 years or older and had advanced osteoarthritis. We excluded those who had bilateral TKAs, joint infections, or neuromuscular disease. The SID system collected daily gait metrics, including step count, distance traveled, walking speed, stride length, cadence, and functional knee range of motion. The PROMs, including Knee Injury and Osteoarthritis Outcome Score-Joint Replacement, Veterans Rand 12 Physical Component Summary, and Veterans Rand 12 Mental Component Summary, were analyzed against SID gait metrics. Among the 88 patients, 80 provided continuous data over 12 weeks.
RESULTS: All gait metrics, except stride length, significantly increased at the 12-week point (P < .05). The PROMs also significantly improved postoperatively (P < .05). Initial low positive correlations between 12-week PROMs and SID metrics decreased after adjusting for demographic variables, leaving only weak correlations between the Veterans Rand 12 Physical Component Summary and Knee Injury and Osteoarthritis Outcome Score-Joint Replacement with functional knee range of motion (r = 0.389, P = .002; r = 0.311, P = .014, respectively), and Veterans Rand 12 Mental Component Summary with step count (r = 0.406, P = .001) and distance traveled (r = 0.376, P = .003).
CONCLUSIONS: This study indicates that both PROMs and SID gait metrics show significant improvements post-TKA, though they correlate weakly with each other, suggesting a possible discrepancy between perceived recovery and actual functional improvement. The SID gait metrics might provide a valuable addition to traditional PROMs by offering an objective representation of physical capabilities unaffected by patient compliance or subjective perceptions of recovery. Further research is needed to validate these findings in larger populations and to explore whether integrating SID metrics can enhance long-term functional outcomes.

Introduction: Adult polyglucosan body disease (APBD) has long been regarded as the adult-onset form of glycogen storage disease type IV (GSD IV) and is caused by biallelic pathogenic variants in GBE1. Advances in the understanding of the natural history of APBD published in recent years have led to the use of discrete descriptors (\"typical\" versus \"atypical\") based on adherence to traditional symptomatology and homozygosity for the p.Y329S variant. Although these general descriptors are helpful in summarizing common findings and symptoms in APBD, they are inherently limited and may affect disease recognition in diverse populations. Methods: This case series includes three American patients (cases 1-3) and four Brazilian patients (cases 4-7) diagnosed with APBD. Patient-reported outcome (PRO) measures were employed to evaluate pain, fatigue, and quality of life in cases 1-3. Results: We describe the clinical course and diagnostic odyssey of seven cases of APBD that challenge the utility and efficacy of discrete descriptors. Cases 1-3 are compound heterozygotes that harbor the previously identified deep intronic variant in GBE1 and presented with \"typical\" APBD phenotypically, despite lacking two copies of the pathogenic p.Y329S variant. Patient-reported outcome measures in these three cases revealed the moderate levels of pain and fatigue as well as an impacted quality of life. Cases 4-7 have unique genotypic profiles and emphasize the growing recognition of presentations of APBD in diverse populations with broad neurological manifestations. Conclusion: Collectively, these cases underscore the understanding of APBD as a spectrum disorder existing on the GSD IV phenotypic continuum. We draw attention to the pitfalls of commonly used genetic testing methods when diagnosing APBD and highlight the utility of patient-reported outcome questionnaires in managing this disease.

UNASSIGNED: Osteochondroma of the scapula constitutes only 3-5% of all osteochondromas; osteochondroma on dorsal aspect of scapula is a rare entity. Diagnosis is almost always clinicoradiologically. Additional computed tomography scan and magnetic resonance imaging may be required for osteochondroma of flat bones such as scapula. Indications for surgery include pain, deformity, dysfunction, neural or vascular compromise, failure of conservative management, or in clinical settings with the high suspicion of malignant transformation and occasionally cosmesis. Outcome of a surgery should be assessed by Patient-Reported Outcome Measures (PROMs) which appraises what \"matters to the patient.\"
UNASSIGNED: A 10-year-old boy presented to us with painless swelling over the right upper back since 3 years of age and discomfort over the area while sleeping on his back for 6 months. Diagnosis confirmed it to be a pedunculated osteochondroma arising from the dorsal scapula. Here, we report the diagnosis, treatment, and successful Patient-Reported Outcome using QuickDASH© score for an osteochondroma of dorsal scapula using CARE© case reporting guidelines.
UNASSIGNED: We report a rare site of osteochondroma, review the relevant literature, and also stress upon the necessity of analyzing PROMs after surgical treatment of benign tumors of bone which would enable us to evaluate the result of surgery on symptoms, functioning, and health-related quality of life from the patient\'s perspective.

BACKGROUND: Atopic dermatitis (AD) is a chronic, relapsing-remitting illness. In moderate-to-severe instances, recommendations urge patient-centered systemic therapy. Existing standards lack long-term treatment success requirements. A treat-to-target methodology was proposed for systemic therapy patients that requires global improvements to prompt decisions about treatment.
METHODS: We conducted an observational study between May 2021 and June 2022 in three Ecuadorian patients with severe AD who were treated with dupilumab to assess the clinical evolution and behavior of the subdomains evaluated by clinimetric tools.
RESULTS: Patients A and C satisfied disease-domain response criteria to dupilumab at 12 and 24 weeks, but B did not complete the algorithm objectives. Nonetheless, patient A improved AD severity, itching, bleeding, desquamation, sleep, daily activities, mood, emotions, sexual troubles, clothing, and sports subdomains. Patient B experienced reduced symptomatology, AD aggravation, daily activities impact, and work/study impairment. Patient C improved from severe to mild desquamation, itching, exudate, lichenification, and rough/dry skin. Sleep, shame, and study subdomains improved the most.
CONCLUSIONS: We provide a new operational construct for analyzing current patient-reported outcome measures (PROMs) and clinician-reported outcome measures (CROMs) based on subdomains to widen our understanding of the state of disease activity and make clinical decisions when the treat-to-target strategy is not attained.

UNASSIGNED: Most patients undergoing total knee arthroplasty (TKA) experience favorable outcomes. Some patients, however, experience prolonged post-operative knee pain and tenderness at the joint line. This has been attributed in some cases to soft tissue impingement due to pseudomeniscus. The purpose of this study is to evaluate patient outcomes for arthroscopic excision of pseudomeniscus for persistent knee pain after TKA and to perform a review of literature for pseudomeniscus after knee arthroplasty.
UNASSIGNED: A retrospective analysis of patients undergoing arthroscopy to remove soft tissue causing persistent knee pain after previous TKA was performed. Outcome scores were asked at three time points: prior to knee arthroplasty, prior to arthroscopy, and post-arthroscopy at final follow-up. Subjective satisfaction with surgery and willingness to repeat procedures were also recorded. A two-tailed distribution paired t-test was used to determine statistical significance (p < 0.05). We also performed a review of the literature for pseudomeniscus complications for comparison.
UNASSIGNED: Nine out of eleven patients were considered eligible to be assessed for post-operative satisfaction. Seven out of nine patients reported complete satisfaction and eight participants stated they would undergo the procedures again if they have to. Among 9 included patients, five patients have successfully completed the clinical score evaluation survey. The mean age of the patients with clinical score evaluation at the time of TKA was 61 years (range, 53-72) and the time between TKA and undergoing arthroscopy was 8 months (range, 5-13). The average follow-up period for patient-determined assessment after the arthroscopic procedure was 71 months (range, 16-115). All 5 patients reported improved Oxford Knee Scores (p=0.017), Western Ontario and McMaster Universities Osteoarthritis Index (p=0.023), and pain scores (p=0.018) comparing pre-arthroscopy to post-arthroscopy at follow-up.
UNASSIGNED: Arthroscopic removal of pseudomeniscus after TKA provides promising results in patients who experience persistent knee joint line pain and tenderness. Considering our results and review of literature, we recommend including pseudomeniscus in the differential for post arthroplasty knee pain.
UNASSIGNED: Case series with a review of literature.

OBJECTIVE: Proponents of disease-specific patient-reported outcome measurements (PROMs) often argue disease-agnostic measures do not adequately capture their patient population\'s experience. Patient-Reported Outcomes Measurement Information System (PROMIS) provides a disease-agnostic domain set that may adequately cover many diseases. This study seeks to investigate whether PROMIS\'s quality of life domain coverage can span patient-reported outcomes (PROs) elicited from patients across unrelated diseases.
METHODS: The Food and Drug Administration Voice of the Patient reports were an initiative to elevate patient voices regarding their condition and associated treatments. Two reviewers extracted patient-reported health-related (quality of life) domains from the reports and categorized them into PROMIS domains or non-PROMIS domains. Domain coverage was summarized for each report. Any extracted PROs not covered by PROMIS domains were placed in an \"other\" category and analyzed for common themes.
RESULTS: Across 26 reports, PROMIS covered 216 of 374 (70%) of the reports\' PRO domains. The heritable bleeding disorders report had the highest coverage (82%). Human immunodeficiency virus had the lowest coverage (50%). The most common PROMIS domain, \"ability to participate in social roles,\" appeared in 25 reports (96%). The most common domains not included in PROMIS were stigma, sensitivities, and sensory deficits as evident in 19 (73%), 18 (69%), and 18 reports (69%), respectively. If the top 3 unincluded domains were amended into PROMIS, the total domain coverage would increase to 84%.
CONCLUSIONS: PRO domains elicited in the Food and Drug Administration Voice of the Patient reports were widely captured by PROMIS, suggesting domains patients experience contain enough overlap to be recorded by appropriate PROMIS domains. PROMIS could increase its coverage by adding domains.

OBJECTIVE: The clinical benefits of total knee arthroplasty (TKA) are well defined, but little attention has been paid to the cross-cultural variation. The objective of this study was to compare case mix and outcomes following TKA in Swiss and Scottish patients.
METHODS: Data from local registries at a Swiss and a Scottish orthopaedic hospital were extracted to evaluate: (A) age, sex, body mass index (BMI), self-reported health status (EQ-5D), and joint awareness (Forgotten Joint Score-12 (FJS-12)) at pre-surgery, (B) improvement in EQ-5D and FJS-12 scores from pre-surgery to 1 year, and (C) patient satisfaction at 1 year.
RESULTS: Data from 2075 Swiss and 994 Scottish TKA patients were available from the local registries. Swiss and Scottish patients differed in age (69.3 vs 68.8 years, p = 0.046), sex ratio (62.9% vs 56.9% women, p = 0.002) and BMI (29.6 vs 30.9, p < 0.001). At pre-surgery, FJS-12 scores were comparable (Swiss 12.1 vs Scottish 10.9, n.s.), but EQ-5D scores were better in Swiss patients (0.52 vs 0.40, p < 0.001). Post-operative improvement was greater in Switzerland for the FJS-12 (+ 55.1 vs + 32.2, p < 0.001), but not for the EQ-5D (+ 0.31 vs + 0.29, n.s.). The satisfaction rate was similar in both groups (88.3% vs 89.6%, n.s.).
CONCLUSIONS: Subtle cross-cultural variation was evident in TKA case-mix factors between the two countries. Satisfaction and improvement in health status were similar, while improvement in joint-specific outcome was notably greater in Switzerland. Understanding cross-cultural variability of the outcome has important implications when interpreting study and registry data from other countries and when counselling a patient in daily practice.
METHODS: Retrospective cohort, Level III.

This case study describes the use of patient-reported outcome (PRO) in conjunction with psychosocial treatment for an adolescent oncology patient presenting with comorbid mental health difficulties. A nine-session evidence-based cognitive-behavioral treatment was implemented and PROs were used to document progress. Results demonstrated an overall pattern of increase in health-related quality of life and decrease in mood and anxiety symptoms as treatment progressed, with increases occurring after disease recurrence. This case study demonstrates the importance and utility of PROs. Implications for psychosocial treatment of adolescent and young adult patients and directions for future research are discussed.

OBJECTIVE: To report normative responses to the HAGOS questionnaire for Australian football players and to determine whether any of the HAGOS questionnaire sub scales can differentiate players with and without groin pain.
METHODS: Case-control.
METHODS: Clinical setting.
METHODS: Professional (n = 66) and semi-professional (n = 9) Australian football (AF) players with current groin pain (n = 16) and controls (n = 57) without current groin pain.
METHODS: The HAGOS subscales were compared between players with and without groin pain using the Wilcoxon rank-sum test with effect sizes (ES) calculated. Floor and ceiling effects were examined. A post-hoc factor analysis was undertaken.
RESULTS: Participants with current groin pain showed lower Physical Function of Daily Living (PFDL) and Physical Function in Sport and Recreation (PFSR) subscale scores (p < 0.05, ES: 0.77 and 0.90 respectively). Any groin pain (current and/or historical) lowered the Pain and Quality of Life (QOL) subscale scores (p < 0.05, ES: 0.38 and 0.72 respectively). Factor analysis showed 8 significant factors with one main factor identified representing items describing forceful activities (Eigenvalue = 18.02, Proportion = 0.49).
CONCLUSIONS: The HAGOS can distinguish AF players with current groin pain in the PFDL and PFSR subscales but not in the other four subscales. Any current or historical groin pain lowers scores on the QOL and Pain sub scales.
METHODS: Aetiology, Individual Case-Control Study, Level 3b.