OBJECTIVE: As the importance of the patient\'s perspective on treatment outcome is becoming increasingly clear, the availability of patient-reported outcome measures (PROMs) has grown accordingly. There remains insufficient information regarding the quality of PROMs in patients with soft-tissue sarcomas (STSs). The objectives of this systematic review were (1) to identify all PROMs used in STS patients and (2) to critically appraise the methodological quality of these PROMs.
METHODS: Literature searches were performed in MEDLINE and Embase on April 22, 2024. PROMs were identified by including all studies that evaluate (an aspect of) health-related quality of life in STS patients by using a PROM. Second, studies that assessed measurement properties of the PROMs utilized in STS patients were included. Quality of PROMs was evaluated by performing a COSMIN analysis.
RESULTS: In 59 studies, 39 PROMs were identified, with the Toronto Extremity Salvage Score (TESS) being the most frequently utilized. Three studies evaluated methodological quality of PROMs in the STS population. Measurement properties of the TESS, Quick Disability of the Arm, Shoulder and Hand (QuickDASH) and European Organization for Research and Treatment for Cancer Quality of Life Questionnaire (EORTC-QLQ-C30) were reported. None of the PROMs utilized in the STS population can be recommended for use based on the current evidence and COSMIN analysis.
CONCLUSIONS: To ensure collection of reliable outcomes, PROMs require methodological evaluation prior to utilization in the STS population. Research should prioritize on determining relevant content and subsequently selecting the most suitable PROM for assessment.

UNASSIGNED: Chronic cough, a cough lasting >8 weeks, includes refractory chronic cough (RCC) and unexplained chronic cough (UCC). Patient-reported outcome (PRO) measures are needed to better understand chronic cough impacts that matter most to patients. The 19-item Leicester Cough Questionnaire (LCQ), an existing PRO measure of chronic cough, assesses impacts of cough across physical, psychological, and social domains. However, the content validity of the LCQ evaluating these concepts in patients with RCC/UCC had not been established.
UNASSIGNED: To evaluate the content validity of the LCQ in patients with RCC/UCC.
UNASSIGNED: A cross-sectional, qualitative interview study.
UNASSIGNED: First, previously completed qualitative interview results in adults with RCC/UCC (N = 30) were evaluated and mapped to LCQ concepts. Next, a clinical cough expert reviewed each LCQ item and assessed the salience of its concepts for patients with RCC/UCC. Finally, semistructured interviews-including both concept elicitation and cognitive debriefing-were conducted in adults with RCC/UCC (N = 20) to elicit a comprehensive set of participant experiences and to assess the appropriateness of using the LCQ in this population.
UNASSIGNED: Concepts reported in the past and present qualitative interviews were included across all LCQ items, and most impacts reported to be the \"most bothersome\" were assessed in the LCQ. In the current study, all participants indicated that reduced cough frequency would be an important treatment target. During cognitive debriefing, each LCQ item was endorsed by ⩾70% of participants. Additionally, participants were generally able to understand, recall, and select a response for each LCQ item. All participants and the clinical expert indicated that the LCQ was appropriate and assessed the impacts most relevant to patients with RCC/UCC.
UNASSIGNED: Our findings support the content validity of the LCQ and demonstrate that this measure is fit-for-purpose and includes important cough impacts in adults with RCC/UCC.

UNASSIGNED: Loss of mobility is common in persons with multiple sclerosis (PwMS), but little is known about this impairment from the patient\'s perspective.
UNASSIGNED: The aim is to model longitudinal variation in a mobility patient-reported outcome (PRO) and compare trajectories to those observed for Timed 25-Foot Walk (T25FW) in a retrospective cohort.
UNASSIGNED: Latent-class growth analysis was applied to 47,508 measures of Performance Scales© Mobility PRO (PS-Mobility) over ~4 years for 8524 PwMS. For 7347 PwMS, there were 41,988 T25FW measures during this period. Repeated measures correlation and concordance of trajectory assignment were evaluated.
UNASSIGNED: At the group level, PS-Mobility and T25FW linearly worsened and repeated-measures correlation was moderate. Eight latent classes with varying shapes that worsened described PS-Mobility variation, compared to six latent classes for T25FW that differed by intercept. The agreement between PS-Mobility and T25FW cluster assignment was modest. A higher proportion of individuals who were Black/African American, older, Medicaid beneficiaries, living in deprived neighborhoods, had longer disease duration, had progressive disease, and ever smokers were assigned to more impaired clusters.
UNASSIGNED: Cross-sectionally, PS-Mobility and T25FW were highly correlated, but longitudinally correlation was modest to moderate, underscoring the importance of considering both objective and subjective perspectives in evaluating mobility changes in PwMS.

BACKGROUND: Parkinson\'s disease represents a burdensome condition with complex manifestations. A licensed, standardized paper-based questionnaire is completed by both patients and physicians to monitor the progression and state of the disease. However, integrating the obtained scores into digital systems still poses a challenge.
METHODS: Paper-based handwriting is intuitive and an efficient mode of human-computer interaction. Accordingly, we transformed a consumer-grade tablet into a device where an exact digital copy of the disease-specific questionnaire can be filled with the supplied pen. Utilizing a small convolutional neural network directly on the device and trained on MNIST data, we translated the handwritten digits to appropriate LOINC codes and made them accessible through a FHIR-compatible HTTP interface.
RESULTS: When evaluating the usability from a patient-centric point of view, the System Usability Score revealed an excellent rating (SUS = 83.01) from the participants. However, we identified some challenges associated with the magnetic pen and the flat design of the device.
CONCLUSIONS: In setups where certified medical devices are not required, consumer hardware can be used to map handwritten digits of patients to appropriate medical standards without manual intervention through healthcare professionals.

Incorporating the patient\'s perspective into the entire product life cycle of medical device development is paramount for ensuring patient-centric evaluation. By prioritizing patient-centric evaluation, medical device developers can better address patient needs and enhance the quality and effectiveness of health care solutions. Patient-reported outcomes (PROs), patient preference information (PPI), and qualitative inquiry are methodologies to incorporate and amplify the patient\'s voice. In nephrology, unlike in other clinical domains, the utilization of PROs, PPI, and qualitative inquiry in medical device development has been notably sparse. Consequently, a glaring absence of patient involvement in the development of devices leaves the impact of these devices on patient well-being and functionality largely unexplored. Many forward-thinking programs as well as Food and Drug Administration guidance on the use of PROs and PPI are effectively bringing PROs into nephrology device development. Many resources exist to help researchers select high-quality PROs. There are unique considerations for using PROs and PPI to support regulatory decision-making, including fit-for-purpose, concepts of interest, context of use, and least burdensome selection. The rapid evolution of patient-centric initiatives in nephrology will serve to ensure that medical devices meet the needs of people with kidney disease and improve the quality of care.

BACKGROUND: Computerized adaptive testing (CAT) is an effective way to reduce time, repetitious redundancy, and respond burden, and has been used to measure outcomes in many diseases. This study aimed to develop and validate a comprehensive disease-specific CAT for chronic obstructive pulmonary disease (COPD) patient-reported outcome measurement.
METHODS: The discrimination and difficulty of the items from the modified patient-reported outcome scale for COPD (mCOPD-PRO) were analyzed using item response theory. Then the initial item, item selection method, ability estimation method, and stopping criteria were further set based on Concerto platform to form the CAT. Finally, the reliability and validity were validated.
RESULTS: The item discrimination ranged from 1.05 to 2.71, and the item difficulty ranged from - 3.08 to 3.65. The measurement reliability of the CAT ranged from 0.910 to 0.922 using random method, while that ranged from 0.910 to 0.924 using maximum Fisher information (MFI) method. The content validity was good. The correlation coefficient between theta of the CAT and COPD assessment test and modified Medical Research Council dyspnea scale scores using random method was 0.628 and 0.540 (P < 0.001; P < 0.001) respectively, while that using MFI method was 0.347 and 0.328 (P = 0.007; P = 0.010) respectively. About 11 items (reducing by 59.3%) on average were tested using random method, while about seven items (reducing by 74.1%) on average using MFI method. The correlation coefficient between theta of the CAT and mCOPD-PRO total scores using random method was 0.919 (P < 0.001), while that using MFI method was 0.760 (P < 0.001).
CONCLUSIONS: The comprehensive disease-specific CAT for COPD patient-reported outcome measurement is well developed with good psychometric properties, which can provide an efficient, accurate, and user-friendly measurement for patient-reported outcome of COPD.

BACKGROUND: Physical activity (PA) measured by accelerometry is proposed as a novel trial endpoint for heart failure (HF). However, standardised methods and associations with established markers are lacking. This study aimed to examine PA measurements and accelerometer repeatability in patients with HF and age- and sex-matched controls, and study correlations with established prognostic HF markers, body composition, and quality of life (QoL).
METHODS: Accelerometry was performed in 105 patients with HF with left ventricular ejection fraction (LVEF) ≤ 40% and in 46 controls. Participants also underwent dual X-ray absorptiometry, cardiopulmonary exercise testing, a six-minute walking test (6MWT), echocardiography, and NT-proBNP measurement, and completed a QoL questionnaire.
RESULTS: Average acceleration was markedly reduced in patients with HF compared with healthy controls (16.1 ± 4.8 mg vs 27.2 ± 8.5 mg, p < 0.001). Healthy controls spent a median daily 56 min (IQR 41-96 min) in moderate-to-vigorous PA (MVPA), whereas HF patients spent only 12 min (IQR 6-24) in MVPA. In HF patients, average acceleration correlated moderately with 6MWT (R = 0.41, p < 0.001) and maximal oxygen uptake (peak VO2) (R = 0.36, p < 0.001) but not with NT-proBNP, LVEF, or QoL. Patients in NYHA class II showed a higher average acceleration than patients in NYHA III (16.6 ± 4.9 mg vs 14.0 ± 3.6 mg, p = 0.01).
CONCLUSIONS: Daily PA was severely reduced in patients with HF compared with healthy controls. In HF patients, we found moderate correlations of accelerometer measurements with markers of physical capacity but not with LVEF or NT-proBNP.
BACKGROUND: NCT05063955. Registered 01 June 2021-retrospectively registered.

BACKGROUND: Differences in patient-reported outcome measures (PROMs) between manual total knee arthroplasty (mTKA) and robotic-assisted TKA (rTKA) have not been adequately assessed. We compared the minimal clinically important difference for improvement (MCID-I) and Worsening (MCID-W) between mTKA and rTKA patients.
METHODS: Patients who underwent primary TKA (874 mTKA, 439 rTKA) with complete pre-operative and one-year post-operative PROMs were retrospectively identified using a multi-hospital joint arthroplasty registry. Patient-Reported Outcomes Measurement Information System Physical Function Short Form 10a (PROMIS PF-10a), PROMIS Global - Physical, or Knee Injury and Osteoarthritis Outcome Score-Physical Function Short Form (KOOS-PS) were collected. The MCID-I, MCID-W, and \"no significant change\" rates were calculated using distribution-based methods. Propensity-score matching was performed to control for confounding.
RESULTS: Similar 90-day pulmonary embolism (P = 0.26), deep venous thrombosis (P = 0.67), and emergency department visit (P = 0.35) rates were found. The 90-day readmission rate for mTKA was 1.7 and 3.4% for rTKA (P = 0.08), and overall revision rates were 2.2% for mTKA and 0.7% for rTKA (P = 0.07). Revision-free survival was 99% at one and two years for both groups (P = 0.65 and P = 0.43, respectively). There were no differences in the proportion of patients achieving MCID-I or MCID-W for PROMIS PF-10a, PROMIS Global - Physical, or KOOS-PS. The MCID-I for PROMIS PF-10a was achieved in 65.5 and 62.2% of patients who had mTKA and rTKA, respectively (P = 0.32).
CONCLUSIONS: Our study demonstrated similar complication rates, and MCID-I and MCID-W attainment rates between mTKA and rTKA patients. Future studies should assess MCID attainment rates in the long term and in larger cohorts, comparing mTKA and rTKA.

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: There is a lack of information on return to sport and patient-reported outcome measures (PROMs) in amateur athletes after isolated spine injuries.
METHODS: A single-center cohort study in amateur athletes aged 18 to 60 with isolated spine injuries; clinical data collection and follow-up via telephone interview and standardized PROMs (Short-Form 36, Oswestry and Neck Disability Index, Tampa Scale of Kinesiophobia, Hospital Anxiety and Depression Scale, Pain Visual Analog Scale). Bivariate analyses of potential influencing factors on PROMs were conducted using the Wilcoxon Signed-Rank Test. p-values < 0.05 were considered statistically significant.
RESULTS: Out of the 80 included participants, 78% (n = 62) were active in sport at follow-up. PROMs were slightly worse than those described for the age-adjusted general population. There were consistent associations of better PROMs with having reached the subjective preinjury level of performance in sport, while injury severity and surgical or conservative therapy did not show consistent associations with PROMs.
CONCLUSIONS: Most amateur athletes resume their sports activity after a spine injury. Better outcomes are associated with individuals\' resumption of sport and subjective level of performance, while injury severity and surgical or conservative therapy do not show consistent associations with PROMs, highlighting the importance of patient education, rehabilitation, and encouragement.