Search

Background: Incisional hernia (IH) is common and recurrence rates remain high. Although the goal of treatment should be to improve quality of life, studies addressing this aspect are notably absent. We aimed to evaluate the long-term recurrence rate of open mesh repair of IH, investigate the burden of persisting discomfort, explore patient satisfaction, and identify risk factors for negative outcomes. Methods: A single-center, retrospective study was conducted on all open mesh repairs of IH performed between January 2002 and October 2013. Clinical data were gathered from medical records and operative reports, while patient-reported outcome measures (PROMs) were obtained through telephone interviews. Risk factors for recurrence were assessed by survival analysis. PROMs were analyzed across patient subgroups by clinical and demographic variables. Results: This study included 271 patients undergoing medical record review, with 136 patients completing the telephone interview. Recurrence rates at 2, 5, 10, and 15 years were 6%, 8%, 11%, and 12%, respectively. Risk factors for recurrence were obesity and an estimated mesh-defect overlap <7 cm. Bridged repair posed no increased risk. For PROMs, the median follow-up time was 13.6 years after which 78.7% of the patients reported no discomfort, and 89.0% were satisfied with their surgery. Patients <65 years and females experienced more long-term discomfort. Conclusions: Recurrence rates were higher in obese patients and when the estimated mesh-defect overlap was <7 cm, but not in bridged repairs. Young patients and females are at increased risk for long-term discomfort. High satisfaction levels were reported.

BACKGROUND AND PURPOSE: Since patients with primary brain tumor are expected to become long-term survivors, the prevention of long-term treatment-induced side effects is particularly important. This study aimed to explore whether symptom experience and symptom distress change over five years in adults with primary brain tumors treated with proton therapy. An additional aim was to explore whether symptom experience and symptom distress correlate. MATERIALS AND METHODS: The study had a longitudinal observational design. Adult (≥18 years) patients (n = 170) with primary brain tumors treated with proton therapy were followed over five years. Symptom experience and symptom distress were evaluated using the patient-reported Radiotherapy-Related Symptom Assessment Scale. Data from baseline, 1, 12, and 60 months were analyzed using non-parametric tests. RESULTS: Of the 170 patients, the levels of symptoms and symptom distress were low. Fatigue increased at 1 (p=0.005) and 12 months (p=0.025) and was the most frequent symptom from baseline to 60 months' follow-up. Cognitive impairment increased at 12 (p=0.027) and 60 months (p<0.001) and was the most distressing symptom at 60 months' follow-up. There were significant, moderate to strong, correlations at all time points between symptom experience and symptom distress of fatigue, insomnia, pain, dyspnea, cognitive impairment, worry, anxiety, nausea, sadness, constipation, and skin reactions. CONCLUSION: Symptom experience and symptom distress changed in intensity over time with cognitive impairment as the most distressing symptom at 60 months. Future research should focus on identifying effective interventions aimed at alleviating these symptoms and reducing symptom distress for this vulnerable group of patients.

Background Quality of life (QoL) is a key outcome measure in healthcare. However, the heterogeneity in its definitions presents challenges in the objective evaluation of improvement. Universal questionnaires, tailored for a broad demographic group, inadequately represent the unique experiences of intensive care unit (ICU) survivors, including a lack of ability to discriminate issues related to QoL from issues that do not. Methods Using a 218-item, 13-domain provisional questionnaire, we assessed 395 adult ICU survivors, with a minimum 72-h stay at one of three Swedish university hospital ICUs, at 6 months to three years post-discharge. Their responses were compared to those of 195 controls, matched for age and sex and randomly recruited from the Swedish Population Registry. By multi-group exploratory factor analysis, we compared dimensionality in QoL perceptions between the two groups, emphasising patterns of correlation to 13 domain-specific QoL questions. Model fit was assessed using information criteria. Internal consistency reliability for each scale was determined using McDonald’s omega or Cronbach’s alpha. All analyses were conducted using Mplus, applying full information maximum likelihood to handle missing data. Results All domains except Cognition had a subset of questions correlating to the domain-specific QoL question in at least the ICU survivor group. The similarity between the two groups varied, with Physical health, Sexual health and Gastrointestinal (GI) functions mainly correlating the same issues to QoL in the two groups. In contrast, Fatigue, Pain, Mental health, activities of daily living, Sleep, Sensory functions and Work life showed considerable differences. In all, about one-fourth of the issues correlated to QoL in the ICU survivor group and about one-tenth of the issues in the control group. Conclusions We found most issues experienced by ICU survivors to be unrelated to quality of life. Our findings indicate that the consequences of post-IC

BACKGROUND: There are few studies providing a more comprehensive picture of advanced hybrid closed-loop (AHCL) systems in clinical practice. The aim was to evaluate the effects of the AHCL systems, Tandem® t: slim X2™ with Control IQ™, and MiniMed™ 780G, on glucose control, safety, treatment satisfaction, and practical barriers for individuals with type 1 diabetes. METHOD: One hundred forty-two randomly selected adults with type 1 diabetes at six diabetes outpatient clinics in Sweden at any time treated with either the Tandem Control IQ (TCIQ) or the MiniMed 780G system were included. Glycated hemoglobin A1c (HbA1c) and glucose metrics were evaluated. Treatment satisfaction and practical barriers were examined via questionnaires. RESULTS: Mean age was 42 years, median follow-up was 1.7 years, 58 (40.8%) were females, 65% used the TCIQ system. Glycated hemoglobin A1c was reduced by 0.6% (6.8 mmol/mol; 95% confidence interval [CI] = 0.5-0.8% [5.3-8.2 mmol/mol]; P < .001), from 7.3% to 6.7% (57-50 mmol/mol). Time in range (TIR) increased with 14.5% from 57.0% to 71.5% (95% CI = 12.2%-16.9%; P < .001). Time below range (TBR) (<70 mg/dL, <3.9 mmol/L) decreased from 3.8% to 1.6% (P < .001). The standard deviation of glucose values was reduced from 61 to 51 mg/dL (3.4-2.9 mmol/L, P < .001) and the coefficient of variation from 35% to 33% (P < .001). Treatment satisfaction increased, score 14.8 on the Diabetes Treatment Satisfaction Questionnaire (DTSQ) (change version ranging from -18 to 18, P < .001). Four severe hypoglycemia events were detected and no cases of ketoacidosis. Skin problems were experienced by 32.4% of the study population. CONCLUSIONS: Advanced hybrid closed-loop systems improve glucose control with a reasonable safety profile and high treatment satisfaction. Skin problems are common adverse events., This study was financed by grants from the Swedish state under the agreement between the Swedish government and the country councils, the ALF agreement, and the Region of Västra Götaland.

Objective: Assessment of posttraumatic hypothalamic–pituitary dysfunctions is expected to be the most relevant assessment to offer patients with severe intracranial affection. In this study, we aim to investigate the prevalence of hypopituitarism in patients with severe acquired traumatic brain injury (TBI) compared with nontraumatic brain injury (NTBI) and to relate pituitary insufficiency to functional and patient-reported outcomes. Design: This is a prospective study. Methods: We included patients admitted for inpatient neurorehabilitation after severe TBI (N = 42) and NTBI (N = 18). The patients underwent a pituitary function assessment at a mean of 2.4 years after the injury. Functional outcome was assessed by using Functional Independence Measure and Glasgow Outcome Scale-Extended (both 1 year after discharge from neurorehabilitation) and patient-reported outcome was assessed by using Multiple Fatigue Inventory-20 and EQ-5D-3L. Results: Hypopituitarism was reported in 10/42 (24%) patients with TBI and 7/18 (39%) patients with NTBI (P = .23). Insufficiencies affected 1 axis in 14/17 (82%) patients (13 hypogonadotropic hypogonadism and 1 growth hormone [GH] deficiency) and 2 axes in 3/17 (18%) patients (1 hypogonadotropic hypogonadism and GH deficiency, and 2 hypogonadotropic hypogonadism and arginin vasopressin deficiency). None had central hypoadrenalism or central hypothyroidism. In patients with both TBI and NTBI, pituitary status was unrelated to functioning and ability scores at 1 year and to patient-reported outcome scores at a mean of 2.4 years after the injury. Conclusion: Patients with severe acquired brain injury may develop long-term hypothalamus–pituitary insufficiency, with an equal occurrence in patients with TBI and NTBI. In both types of patients, mainly isolated deficiencies, most commonly affecting the gonadal axis, were seen. Insufficiencies were unrelated to functional outcomes and patient-reported outcomes, probably reflecting the comple

Background: Patient-reported outcome (PRO) data may help us better understand the life of breast cancer patients. We have previously collected PRO data in a national Danish breast cancer study in patients undergoing adjuvant chemotherapy. The aim of the present post-hoc explorative study is to apply Machine Learning (ML) algorithms using permutation importance to explore how specific PRO symptoms influence nonadherence to six cycles of planned adjuvant chemotherapy in breast cancer patients. Methods: We here investigate ePRO-data from the 347 patients. The ePRO presented 42 PROCTCAE questions on 25 symptoms. Patients completed the ePRO before each cycle of chemotherapy. Number of patients with completion of the scheduled six cycles of chemotherapy were registered. Two ML models were applied. One aimed at discovering the individual relative importance of the different questions in the dataset while the second aimed at discovering the relationships between the questions. Permutation importance was used. Results: Out of 347 patients 238 patients remained in the final dataset, 15 patients dropped out. Two symptoms: aching joints and numbness/tingling, were the most important for dropout in the final dataset, each with an importance value of about 0.04. Model’s average ROC-AUC-score being 0.706. In the second model a low performance score made the results very unreliable. Conclusion: In conclusion, this explorative data analysis using ML methodologies in an ePRO dataset from a population of women with breast cancer treated with adjuvant chemotherapy unravels that the symptoms aching joints and numbness/tingling could be important for drop out of planned adjuvant chemotherapy.

Received: 04 July 2023; Accepted: 18 January 2024; Published: 08 February 2024, Background: Presbyopia is an age-related condition that causes a decreased ability to focus on nearby objects. Multifocal contact lenses are commonly used to address this issue. However, there seems to be a notable dissatisfaction among multifocal contact lens wearers. The absence of a reliable instrument to measure the patient's perspective, despite the widespread use of this method, highlights the need for further research in this area. Objective: The objective of this study is to develop an item-bank integrating all domains necessary to assess the patient's perspective on multifocal contact lens performance, offering a comprehensive measure. The item-bank will ensure a high level of content validity, be self-administered, and will initially be available in Spanish. The aim of this tool is to serve as a valuable resource for research and optometric clinics, facilitating the follow-up of patients with presbyopia who wear multifocal contact lenses or those who are newly starting to use them. Methodology: The MCL-PRO item bank, followed a systematic and step-wise inductive approach to gather information, following the recommendations outlined in the COSMIN guidelines and similar studies. The process involved the following steps: (1) Literature review and relevant existing items identification (2) Social media review, (3) Semi-structured focus groups, (4) performing qualitative analysis, (5) refining and revising the items, and (6) generating the content of the item bank. Results: A total of 575 items were included in the item-bank hosted under 8 different domains that were found to be important for presbyopic population: visual symptoms (213), activity limitation (111), ocular symptoms (135), convenience (36), emotional well-being (33), general symptoms (16), cognitive issues (21) and economic issues (10). Conclusion: The item-bank created has followed standardised methodology for its development and encloses all the aspects for MCL performance evaluation from patien, Depto. de Optometría y Visión, Fac. de Óptica y Optometría, TRUE, pub

Purpose: Lenvatinib and pembrolizumab (LEN+PEMBRO) demonstrated clinically meaningful and statistically significant improvements in efficacy versus treatment of physician's choice (TPC) in patients with advanced endometrial cancer (aEC) in the phase 3 Study 309/KEYNOTE-775. Health-related quality-of-life (HRQoL) is reported. Patients and Methods: Patients were randomly assigned to receive LEN+PEMBRO (n = 411; LEN 20 mg/day; PEMBRO 200 mg Q3W) or TPC (n = 416; doxorubicin 60 mg/m2 Q3W or paclitaxel 80 mg/m2 [weekly, 3 weeks on/1 week off]). Impact of treatment on HRQoL assessed by the global health status/quality of life (GHS/QoL) score of the European Organisation for Research and Treatment of Cancer Quality-of-Life Questionnaire (EORTC QLQ-C30) was a secondary objective; other scales of the Quality-of-Life Questionnaire (QLQ-C30), EORTC QLQ-Endometrial, 24 questions (EORTC QLQ-EN24), and EuroQoL 5 dimensions, 5 levels (EQ-5D-5L) were exploratory objectives. HRQoL was assessed on day 1 of each cycle. Completion/compliance, change from baseline, time to first and definitive deterioration were assessed. No multiplicity adjustments were applied for HRQoL endpoints. Results: The latest timepoint at which the predefined rates of completion (≥60%) and compliance (≥80%) were met was week 12. HRQoL at week 12 between treatment groups was generally similar. Time to first deterioration symptom scales favoured LEN+PEMBRO for QLQ-C30 dyspnoea, and QLQ-EN24 for poor body image, tingling/numbness, and hair loss; and TPC was favoured for QLQ-C30 pain, appetite loss, and diarrhoea, and QLQ-EN24 muscular pain. While the QLQ-C30 physical functional scale favoured TPC, other functional scales were generally similar between arms. Time to definitive deterioration favoured LEN+PEMBRO on most scales. Conclusion: HRQoL data from Study 309/KEYNOTE-775, with previously published efficacy and safety results, indicate that LEN+PEMBRO has an overall favourable benefit/risk profile versus TPC fo

BackgroundAlthough dietary adjustments are recommended frequently for bowel symptoms, evidence of diet's impact on bowel function is lacking. The aim was to develop a patient-reported outcome instrument, for children with and without Hirschsprung's disease (HD), to explore experiences of dietary effects on bowel function.MethodsChildren with and without HD and their parents participated. Questionnaire items regarding the impact of diet on bowel function originated from focus group discussions. Specific food items, reported in the literature or in focus groups to cause bowel functional effects, were listed requesting each item's effect size and effect type. Content validity was tested within two separate semistructured interviews. A pilot test was performed. Assessing comprehension, relevance and wording clarity structurally, revisions were made accordingly. Children's bowel function was assessed through the validated Rintala Bowel Function Score.ResultsA total of 13 children with and without HD, median age 7 (range 2-15) years, and 18 parents participated in the validation. Each question's relevance had been ranked highly early in the validation process but most questions needed refining for improving clarity and comprehension. Wordings regarding bowel symptoms and emotions connected to food in particular were perceived to be sensitive and complex. Specifically wording regarding some bowel symptoms (gases, pain) and parental stress emotions (guilt, ambivalence) were, consistent with participants' opinions, subjected to multiple step revisions. Following the validation process, which included two semistructure interviews with different participants and then a pilot test with a third cohort, a full track overview of changes and rewording made in all steps of the validation process was presented. The final questionnaire then comprised 13 questions assessing foods' significance for bowel function, emotions, social impact and 90 specific food items' possible effects and

Objective: The aim of the study is to identify and prioritize early intervention (EI) stakeholders' perspectives of supports and barriers to implementing the Young Children's Participation and Environment Measure (YC-PEM), an electronic patient-reported outcome (e-PRO) tool, for scaling its implementation across multiple local and state EI programs. Methods: An explanatory sequential (quan > QUAL) mixed-methods study was conducted with EI families (n = 6), service coordinators (n = 9), and program leadership (n = 7). Semi-structured interviews and focus groups were used to share select quantitative pragmatic trial results (e.g., percentages for perceived helpfulness of implementation strategies) and elicit stakeholder perspectives to contextualize these results. Three study staff deductively coded transcripts to constructs in the Consolidated Framework for Implementation Research (CFIR). Data within CFIR constructs were inductively analyzed to generate themes that were rated by national early childhood advisors for their relevance to longer term implementation. Results: All three stakeholder groups (i.e., families, service coordinators, program leadership) identified thematic supports and barriers across multiple constructs within each of four CFIR domains: (1) Six themes for intervention characteristics, (2) Six themes for process, (3) three themes for inner setting, and (4) four themes for outer setting. For example, all stakeholder groups described the value of the YC-PEM e-PRO in forging connections and eliciting meaningful information about family priorities for efficient service plan development (intervention characteristics). Stakeholders prioritized reaching families with diverse linguistic preferences and user navigation needs, further tailoring its interface with automated data capture and exchange processes (process); and fostering a positive implementation climate (inner setting). Service coordinators and program leadership further articulated the val

BACKGROUND: Free-text, verbatim replies in the words of people with Parkinsons disease (PD) have the potential to provide unvarnished information about their feelings and experiences. Challenges of processing such data on a large scale are a barrier to analyzing verbatim data collection in large cohorts. OBJECTIVE: To develop a method for curating responses from the Parkinsons Disease Patient Report of Problems (PD-PROP), open-ended questions that asks people with PD to report their most bothersome problems and associated functional consequences. METHODS: Human curation, natural language processing, and machine learning were used to develop an algorithm to convert verbatim responses to classified symptoms. Nine curators including clinicians, people with PD, and a non-clinician PD expert classified a sample of responses as reporting each symptom or not. Responses to the PD-PROP were collected within the Fox Insight cohort study. RESULTS: Approximately 3,500 PD-PROP responses were curated by a human team. Subsequently, approximately 1,500 responses were used in the validation phase; median age of respondents was 67 years, 55% were men and median years since PD diagnosis was 3 years. 168,260 verbatim responses were classified by machine. Accuracy of machine classification was 95% on a held-out test set. 65 symptoms were grouped into 14 domains. The most frequently reported symptoms at first report were tremor (by 46% of respondents), gait and balance problems (>39%), and pain/discomfort (33%). CONCLUSION: A human-in-the-loop method of curation provides both accuracy and efficiency, permitting a clinically useful analysis of large datasets of verbatim reports about the problems that bother PD patients.

PurposeEfficient analytical methods are necessary to make reproducible inferences on single-item longitudinal ordinal patient-reported outcome (PRO) data. A thorough simulation study was performed to compare the performance of the semiparametric probabilistic index models (PIM) with a longitudinal analysis using parametric cumulative logit mixed models (CLMM).MethodsIn the setting of a control and intervention arm, we compared the power of the PIM and CLMM to detect differences in PRO adverse event (AE) between these groups using several existing and novel summary scores of PROs. For each scenario, PRO data were simulated using copula multinomial models. Comparisons were also exemplified using clinical trial data.ResultsOn average, CLMM provided substantially greater power than the PIM to detect differences in PRO-AEs between the groups when the baseline-adjusted method was used, and a small advantage in power when using the baseline symptom as a covariate.ConclusionAlthough the CLMM showed the best performance among analytical methods, it relies on assumptions difficult to verify and that might not be fulfilled in the real world, therefore our recommendation is the use of PIM models with baseline symptom as a covariate.

BackgroundThe Tenosynovial giant cell tumor Observational Platform Project (TOPP) registry is an international prospective study that -previously described the impact of diffuse-type tenosynovial giant cell tumour (D-TGCT) on patient-reported outcomes (PROs) from a baseline snapshot. This analysis describes the impact of D-TGCT at 2-year follow-up based on treatment strategies.Material and methodsTOPP was conducted at 12 sites (EU: 10; US: 2). Captured PRO measurements assessed at baseline, 1-year, and 2-year follow-ups were Brief Pain Inventory (BPI), Pain Interference, BPI Pain Severity, Worst Pain, EQ-5D-5L, Worst Stiffness, and -Patient-Reported Outcomes Measurement Information System. Treatment interventions were no current/planned treatment (Off-Treatment) and systemic treatment/surgery (On-Treatment).ResultsA total of 176 patients (mean age: 43.5 years) were included in the full analysis set. For patients without active treatment strategy -(Off-Treatment) at baseline (n = 79), BPI Pain Interference (1.00 vs. 2.86) and BPI Pain Severity scores (1.50 vs. 3.00) were numerically favorable in patients remaining Off-Treatment compared with those who switched to an active treatment strategy at year 1. From 1-year to 2-year -follow-ups, patients who remained Off-Treatment had better BPI Pain Interference (0.57 vs. 2.57) and Worst Pain (2.0 vs. 4.5) scores compared with patients who switched to an alternative treatment strategy. In addition, EQ-5D VAS scores (80.0 vs. 65.0) were higher in patients who remained -Off-Treatment between 1-year and 2-year follow-ups compared with patients who changed treatment strategy. For patients receiving systemic treatment at baseline, numerically favorable scores were seen in patients remaining on systemic therapy at 1-year follow-up: BPI Pain Interference (2.79 vs. 5.93), BPI Pain Severity (3.63 vs. 6.38), Worst Pain (4.5 vs. 7.5), and Worst Stiffness (4.0 vs. 7.5). From 1-year to 2-year follow-up, EQ-5D VAS scores (77.5 vs. 65.0) w

Patient-reported outcomes (PROs) are valuable for shared decision making and research. Patient-reported outcome measures (PROMs) are questionnaires used to measure PROs, such as health-related quality of life (HRQL). Although core outcome sets for trials and clinical practice have been developed separately, they, as well as other initiatives, recommend different PROs and PROMs. In research and clinical practice, different PROMs are used (some generic, some disease-specific), which measure many different things. This is a threat to the validity of research and clinical findings in the field of diabetes. In this narrative review, we aim to provide recommendations for the selection of relevant PROs and psychometrically sound PROMs for people with diabetes for use in clinical practice and research. Based on a general conceptual framework of PROs, we suggest that relevant PROs to measure in people with diabetes are: disease-specific symptoms (e.g. worries about hypoglycaemia and diabetes distress), general symptoms (e.g. fatigue and depression), functional status, general health perceptions and overall quality of life. Generic PROMs such as the 36-Item Short Form Health Survey (SF-36), WHO Disability Assessment Schedule (WHODAS 2.0), or Patient-Reported Outcomes Measurement Information System (PROMIS) measures could be considered to measure commonly relevant PROs, supplemented with disease-specific PROMs where needed. However, none of the existing diabetes-specific PROM scales has been sufficiently validated, although the Diabetes Symptom Self-Care Inventory (DSSCI) for measuring diabetes-specific symptoms and the Diabetes Distress Scale (DDS) and Problem Areas in Diabetes (PAID) for measuring distress showed sufficient content validity. Standardisation and use of relevant PROs and psychometrically sound PROMs can help inform people with diabetes about the expected course of disease and treatment, for shared decision making, to monitor outcomes and to improve healthcare.

There are many consequences of heart failure (HF), including symptoms, impaired health-related quality of life (HRQoL), and physical and social limitations (functional status). These have a substantial impact on patients' lives, yet are not routinely captured in clinical trials. Patient-reported outcomes (PROs) can quantify patients' experiences of their disease and its treatment. Steps can be taken to improve the use of PROs in HF trials, in regulatory and payer decisions, and in patient care. Importantly, PRO measures (PROMs) must be developed with involvement of patients, family members, and caregivers from diverse demographic groups and communities. PRO data collection should become more routine not only in clinical trials but also in clinical practice. This may be facilitated by the use of digital tools and interdisciplinary patient advocacy efforts. There is a need for standardization, not only of the PROM instruments, but also in procedures for analysis, interpretation and reporting PRO data. More work needs to be done to determine the degree of change that is important to patients and that is associated with increased risks of clinical events. This ‘minimal clinically important difference’ requires further research to determine thresholds for different PROMs, to assess consistency across trial populations, and to define standards for improvement that warrant regulatory and reimbursement approvals. PROs are a vital part of patient care and drug development, and more work should be done to ensure that these measures are both reflective of the patient experience and that they are more widely employed.

Introduction: 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. Conclusion: 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.

Introduction. La dystrophie musculaire oculopharyngée (DMOP) est une maladie neuromusculaire dont les symptômes incluent une dysphagie progressive. Le Dysphagimètre a été développé dans un contexte de préparation aux essais thérapeutiques en DMOP en lien avec l’absence d’outils de type autorapporté spécifique à cette population pour évaluer la dysphagie. Méthodes. Les étapes de la Food and Drug Administration (FDA) sur le développement d’outils de type autorapporté ont été suivies. La Phase 1 comprenait une recension systématique de la littérature et une consultation d’experts pour identifier les symptômes clés. La Classification Internationale du Fonctionnement, du Handicap et de la Santé (CIF) a été utilisée comme cadre conceptuel. La Phase 2 comprenait des groupes de discussion et des questionnaires en ligne auprès de personnes atteintes et une séance de travail avec des experts, lesquels ont permis de générer les items du questionnaire. Des entrevues cognitives ont également été conduites en français et en anglais pour évaluer la clarté de l’outil et son exhaustivité. À la Phase 3, une réduction des items et une documentation des qualités métrologiques ont été réalisées en utilisant le modèle de Rasch et la théorie classique des tests. Résultats. Le Dysphagimètre consiste en deux parties. La partie A évalue la capacité à avaler des aliments de différentes textures (21 items) et des liquides (3 items). L’échelle à 21 items s’ajuste au modèle de Rasch (Index de séparation des personnes = 0,95) et l’échelle des liquides a montré une excellente cohérence interne (Alpha de Cronbach = 0,90). La partie B du Dysphagimètre comprend six items qui évaluent l’impact de la dysphagie dans le contexte des repas et deux items qui évaluent l’inconfort avec la salive épaisse (Alpha de Cronbach = 0,91 et 0,79). Des preuves de validité de construit ont été documentées. Conclusion. Le Dysphagimètre est un outil pouvant être utilisé pour mesurer la gravité de la dysphagie et ses impa, Introduction. Oculopharyngeal muscular dystrophy (OPMD) is a neuromuscular disease with progressive dysphagia. The Dysphagiameter was developed in the context of clinical trial preparation for OPMD, as there was no patient-reported outcome measure (PROM) specific to OPMD to assess dysphagia. Methods. The guidance of the Food and Drug Administration (FDA) on PROMs development was followed. Phase1 included a systematic literature review and an expert consultation to identify the key symptoms. The International Classification of Functioning, Disability and Health (ICF) was used as a conceptual framework. Phase 2 included focus groups and online surveys with people affected by OPMD and a work session with experts, which led to the generation of the items. Cognitive interviews were conducted in French and English to assess the clarity of the PROM and its comprehensiveness. In phase 3, item reduction and psychometric assessment were done using Rasch analysis and classical test theory (CTT). Results. The Dysphagiameter consists of two parts. Part A assesses capacity to swallow foods of various textures (21 items) and liquids (3 items). The 21-item scale was confirmed with a Rasch analysis (Person separation index = 0.95) and the liquid scale showed excellent internal consistency (Cronbach Alpha = 0.90). Part B of the Dysphagiameter includes six items assessing the impact on mealtime and two items assessing discomfort related to thick saliva (Cronbach Alpha = 0.91 and 0.79). Evidence of construct validity was documented. Conclusion. The Dysphagiameter is a PROM that can be used to assess the severity of dysphagia and its impact on people affected in OPMD, specifically in the context of clinical trials. The documentation of its psychometric properties is to be continued.

Background: Electronic Patient-Reported Outcomes (ePROs) have potential to improve health outcomes and healthcare. The development of health-technology applications, such as ePROs, should include the potential users and be theoretically grounded. Swedish Youth Health Clinics (YHCs) offer primarily sexual and psychological healthcare for young people aged 12 to 25 years old. Young people in healthcare settings are considered a vulnerable group. The development of a collection of Patient-Reported Outcomes (PROs) in an Electronic Health Report Form (eHRF) for identifying health and health-related problems in young people, was preceded by a qualitative interview study, exploring young people’s views on using an eHRF at YHCs and which questions about health an eHRF should contain. The aim of the current study was to develop and evaluate the usability of an eHRF prototype for identifying health and health-related problems in young people visiting YHCs. Methods: This study used a participatory design. During the development, an expert panel consisting of eight researchers and one Information Technology worker, participated. A wide literature search was performed to find PROs to construct an eHRF prototype to cover health areas. A mixed methods usability evaluation included 14 participants (young people, healthcare professionals, and an expert panel). Results: The development resulted in an eHRF prototype, containing ten reliable and valid health questionnaires addressing mental-, physical-, and sexual health and social support, a self-efficacy question, and background questions, in total 74 items. The interviews in the usability evaluation resulted in three categories describing the usability of the eHRF: ‘Captures the overall health of young people but needs clarification’, ‘Fun, easy, and optional and will keep young people’s interest’, and ‘Potential contribution to improve the health consultation’. The quantitative results support the usability of the eHRF for YHCs. Co

Patients treated for vulvar carcinoma may experience losses in mobility and physical activity. In this study, we assess the prevalence and severity of mobility problems using patient-reported outcomes of three questionnaires: EQ-5D-5L to estimate QoL and perceived health; SQUASH to estimate habitual physical activity; and a problem-specific questionnaire on bicycling. Patients treated for vulvar carcinoma between 2018 and 2021 were recruited, and 84 (62.7%) responded. The mean age was 68 ± 12 years (mean ± standard deviation). Self-reported QoL and perceived health were 0.832 ± 0.224 and 75.6 ± 20.0, respectively. Dutch physical activity guidelines were met by 34.2% of participants. Compared to baseline values, the times spent walking, bicycling, and participating in sports were all reduced. During bicycling, patients experienced moderate or severe pain in the skin of the vulva (24.5%), pain in the sit bones (23.2%), chafing (25.5%), or itching (8.9%). Overall, 40.3% experienced moderate or severe bicycling problems or could not bicycle, 34.9% felt that their vulva impeded bicycling, and 57.1% wished to make more or longer bicycling journeys. To conclude, vulvar carcinoma and its treatment reduce self-reported health, mobility, and physical activity. This motivates us to investigate ways to reduce discomfort during physical activities, and help women regain their mobility and self-reliance., Medical Instruments & Bio-Inspired Technology

Background: Sesamoid position change after distal chevron osteotomy for moderate to severe hallux valgus is not well known in the literature. The objective of this study was to determine whether the sesamoid position changed over 2 years after distal chevron osteotomy for moderate to severe hallux valgus. Methods: Ninety-seven patients who underwent distal chevron osteotomy for moderate to severe hallux valgus were prospectively assessed for 2 years. There were 91 females, and the mean age was 54.9 (SD 10.9) years. The Self-Reported Foot and Ankle Score (SEFAS) was used for functional assessment. Foot pain was assessed by a 0-10 visual analog scale (VAS), and patient satisfaction by a 5-point Likert scale. Radiologically, hallux valgus angle (HVA), first-second intermetatarsal angle (IMA), distal metatarsal articular angle (DMAA), metatarsal head displacement, and sesamoid position by the AOFAS criteria were analyzed. Patients were classified according to the medial sesamoid position on the first postoperative weightbearing radiograph (1º-WB-Xray) into the reduced sesamoid group (66 patients) and nonreduced sesamoid group (31 patients). Results: All patients were assessed preoperatively and postoperatively at 1 month and 2 years. On the 1º-WB-Xray, IMA was significantly lower in the reduced group (P = .038), but HVA (P = .063) and DMAA (P = .246) were not significantly different. At the final follow-up, no patients in the reduced group had sesamoid position change from 1º-WB-Xray, whereas 8 (25.8%) patients in the nonreduced group had change of their sesamoid positions. The SEFAS was not significantly different between groups preoperatively (P = .386) or at 1 month postoperation (P = .064). The final SEFAS, VAS pain, and satisfaction scores were significantly better in the reduced group, although the clinical significance of these changes remains unknown. Conclusion: A nonreduced position of the sesamoids obtained in surgery can cause their increased malposition ove

Aims Systematic use of patient-reported outcomes (PROs) have the potential to improve quality of care and reduce costs of health care services. We aimed to describe whether PROs in patients diagnosed with heart disease are directly associated with health care costs. Methods and results A national cross-sectional survey including PROs at discharge from a heart centre with 1-year follow-up using data from national registers. We included patients with either ischaemic heart disease (IHD), arrhythmia, heart failure (HF), or valvular heart disease (VHD). The Hospital Anxiety and Depression Scale, the heart-specific quality of life, the EuroQol five-dimensional questionnaire, and the Edmonton Symptom Assessment Scale were used. The economic analysis was based on direct costs including primary, secondary health care, and medical treatment. Patient-reported outcomes were available from 13 463 eligible patients out of 25.241 [IHD (n = 7179), arrhythmia (n = 4322), HF (n = 987), or VHD (n = 975)]. Mean annual total direct costs in all patients were euro23 228 (patients with IHD: euro19 479, patients with arrhythmia: euro21 076, patients with HF: euro34 747, patients with VDH: euro48 677). Hospitalizations contributed overall to the highest part of direct costs. For patients discharged with IHD or arrhythmia, symptoms of anxiety or depression, worst heart-specific quality of life or health status, and the highest symptom burden were associated with increased economic expenditure. We found no associations in patients with HF or VHD. Conclusion Patient-reported outcomes at discharge from a heart centre were associated with direct health care costs in patients with IHD and arrhythmia.

Introduction. L’ataxie récessive spastique de Charlevoix-Saguenay (ARSCS) est une maladie neurologique héréditaire se manifestant par des atteintes cérébelleuses (incoordination), neuropathiques (perte de force et de sensibilité en distal) et pyramidales (déficits moteurs). Les atteintes nuisent aussi à la participation sociale et à la réalisation des activités de tous les jours. Il n’y a pas de traitement curatif pour l’ARSCS, cependant, des essais thérapeutiques sont attendus dans un avenir proche. Afin de mesurer les effets des nouveaux traitements, il est essentiel d’inclure des outils permettant de mesurer la santé perçue par les personnes atteintes, que l’on nomme patient-reported outcome measure (PROM) ou outil autorapporté. Objectifs. 1) Développer un outil de type PROM mesurant les manifestations et impacts de la maladie pour les adultes atteints d’ARSCS ou d’une autre ataxie récessive. 2) Documenter sa validité de contenu, sa validité de construit (validité structurelle et discriminante) et son interprétabilité. Méthodologie. Un devis mixte séquentiel exploratoire a été utilisé, ainsi que le cadre conceptuel PROMIS. Pour concevoir l’outil, une recension des écrits, des entrevues semi-dirigées et des groupes de discussion auprès des personnes atteintes ont eu lieu. À la suite de l’analyse de contenu, les thèmes identifiés ont été formulés en question pour former les items. Une première version de l’outil a été soumise à une consultation d’experts et à des entrevues cognitives. La version préliminaire de l’outil a été mise en ligne afin d’être testée par les personnes atteintes d’ataxie récessive. La documentation des propriétés psychométriques a été réalisée en observant la distribution des résultats, par des analyses factorielles (exploratoire et confirmatoire) et par la méthode des groupes connus. Résultats. La collecte de données qualitative a permis d’obtenir la perception des personnes atteintes face à leur maladie afin de développer un outil centré su, Introduction. Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS) is a hereditary neurological disease manifested by cerebellar (incoordination), neuropathic (loss of strength and sensitivity distally) and pyramidal (motor deficits) impairments. The impairments also interfere with social participation and the performance of everyday activities. There is no cure for ARSACS, however, therapeutic trials are expected in the near future. To measure the effects of new treatments, it is essential to include tools to measure the perception of people with the disease, which we call patient-reported outcome measure (PROM) or self-reported tools. Objectives. 1) To develop a PROM measuring the manifestations and impacts of the disease for adults with ARSACS or another recessive ataxia. 2) To document its content validity, its construct validity (structural and discriminant validity) and its interpretability. Methodology. An exploratory sequential mixed design was used, as well as the PROMIS conceptual framework. To design the tool, a systematic literature review, semistructured interviews and focus groups with people with recessive ataxia was conducted. Following the content analysis, the identified themes were put into questions to form the items. A first version of the tool was submitted to expert consultation and cognitive interviews. The preliminary version of the tool has been put online to be tested by people with recessive ataxia. The documentation of the psychometric properties was carried out by observing the distribution of the results, by factorial analyzes (exploratory and confirmatory) and by the known groups method. Results. Qualitative data collection made it possible to obtain the perception of people affected regarding their disease to develop a tool centered on this perception. The PROM includes 38 items divided into three components: physical functions and activities, mental functions, and social functions. A total of 125 people compl

Objective: To investigate the association between sex, self-reported ankle function, pain intensity, kinesiophobia, and perceived ankle instability in athletes with chronic ankle instability (CAI). Design: Cross-Sectional Study. Setting: University. Participants: College club sports athletes with CAI (n = 42). Main outcome measures: Relationships with the Cumberland Ankle Instability Tool (CAIT) score and the Tampa Scale for Kinesiophobia-11 (TSK-11), the Foot and Ankle Ability Measure (FAAM), sex (0: male, 1: female), and ankle pain intensity by the Numeric Rating Scale were explored with multiple regression analysis. Results: The regression model explained 50.3% of the variance of the CAIT score (P < 0.001), and the TSK-11 score (B = -0.382, P = 0.002), the FAAM sports subscale score (B = 0.122, P = 0.038), and sex (B = -2.646, P = 0.031) were significant independent variables for the CAIT score (P < 0.001), while pain intensity was not significant (B = -0.182, P = 0.504). These results indicated that higher TSK-11 score, lower FAAM sports subscale score, and being female were related to lower CAIT score. Conclusions: Kinesiophobia related to perceived instability along with self-reported function and sex in athletes with CAI. Clinicians should assess the psychological aspects of athletes with CAI.

Objective The study aimed to reach a consensus on the most relevant patient-reported outcomes (PROs), the corresponding measures (PROMs), and measurement frequency during severe asthma patient follow-up. Methods Two Delphi rounds were conducted. The questionnaire was developed based on a systematic literature review, a focus group with patients, and a nominal group with experts. It assessed PROs’ relevance and the appropriateness (A) and feasibility (F) of PROMs using a Likert scale (1=totally agree; 9=totally disagree). The consensus was established when ≥75% of participants agreed (1-3) or disagreed (7-9). Results Sixty-three professionals (25 hospital pharmacists, 14 allergists, 13 pulmonologists, and 11 nurses) and 5 patients answered the Delphi questionnaire. A consensus was reached on all PROs regarding their relevance. Experts agreed on the use of ACT (A:95.24%; F:95.24%), mini AQLQ (A:93.65; F:79.37%), mMRC dyspnea scale (A:85.71%; F:85.71%), TAI (A:92.06%; F:85.71%), MMAS (A:75.40%; F:82%), and the dispensing register (A:96.83%; F:92.06%). Also considered suitable were: SNOT-22 (A:90.48%; F:73.80%), PSQI (A:82.54; F:63.90%), HADS (A:82.54; F:64%), WPAI (A:77.78%; F:49.20%), TSQM-9 (A:79.37; F:70.50%) and knowledge of asthma questionnaire (A:77%; F:68.80%); however, their use in clinical practice was considered unfeasible. Panelists also agreed on the appropriateness of EQ-5D, which was finally included despite being considered unfeasible (A: 84.13%; F:67.20%) in clinical practice. Agreement was reached on using ACT, TAI, mMRC, and a dispensing register every three months; mini-AQLQ and MMAS every six months; and EQ-5D every twelve months. Conclusion This consensus paves the way toward patient-centered care, promoting the development of strategies supporting routine assessment of PROs in severe asthma management.

Objectives Older patients are typically underrepresented in clinical trials despite representing a major proportion of the patient population. We aim to describe the feasibility of performing body composition measures, physical function measures, and patient-reported questionnaires within the first 24 hours of admission in a large sample of older acutely admitted medical patients. In addition, we aim to characterize patients with missing measurements. Design Secondary analyses of cross-sectional data from a cohort study. Setting and Participants A total of 1071 acutely admitted patients aged ≥65 years from the acute medical ward at Bispebjerg Hospital, were enrolled within the first 24 hours of hospitalization. Methods Body composition was investigated using direct segmental multifrequency bioelectrical impedance analyses (DSM-BIA) and physical function was assessed using hand grip strength (HGS) and the 30-second sit-to-stand test (STS). The orientation-memory-concentration test (OMC) was used to evaluate the prevalence of cognitive impairments within 24 hours of hospitalization, and the OMC in conjunction with the Strength, Assistance walking, Rise from a chair, Climb stairs, and Falls questionnaire (SARC-F) was used to assess the feasibility of patient-reported outcomes (PROs). Results Mean age was 78.8 ± 7.8 years (53.0% female). HGS was performed in 96.2% of the enrolled patients, whereas the PRO, 30-second STS, and DSM-BIA were performed in 91.2%, 69.2%, and 59.8% of patients, respectively. The main barrier for performing the 30-second STS and body composition measurements was an inability to mobilize the patient from the hospital bed. Conclusions and Implications The assessment of HGS and PROs show excellent feasibility in clinical research including older patients, even when the patients are enrolled and tested within 24 hours of an acute admission. Assessments of DSM-BIA and the 30-second, Objectives: Older patients are typically underrepresented in clinical trials despite representing a major proportion of the patient population. We aim to describe the feasibility of performing body composition measures, physical function measures, and patient-reported questionnaires within the first 24 hours of admission in a large sample of older acutely admitted medical patients. In addition, we aim to characterize patients with missing measurements. Design: Secondary analyses of cross-sectional data from a cohort study. Setting and Participants: A total of 1071 acutely admitted patients aged ≥65 years from the acute medical ward at Bispebjerg Hospital, were enrolled within the first 24 hours of hospitalization. Methods: Body composition was investigated using direct segmental multifrequency bioelectrical impedance analyses (DSM-BIA) and physical function was assessed using hand grip strength (HGS) and the 30-second sit-to-stand test (STS). The orientation-memory-concentration test (OMC) was used to evaluate the prevalence of cognitive impairments within 24 hours of hospitalization, and the OMC in conjunction with the Strength, Assistance walking, Rise from a chair, Climb stairs, and Falls questionnaire (SARC-F) was used to assess the feasibility of patient-reported outcomes (PROs). Results: Mean age was 78.8 ± 7.8 years (53.0% female). HGS was performed in 96.2% of the enrolled patients, whereas the PRO, 30-second STS, and DSM-BIA were performed in 91.2%, 69.2%, and 59.8% of patients, respectively. The main barrier for performing the 30-second STS and body composition measurements was an inability to mobilize the patient from the hospital bed. Conclusions and Implications: The assessment of HGS and PROs show excellent feasibility in clinical research including older patients, even when the patients are enrolled and tested within 24 hours of an acute admission. Assessments of DSM-BIA and the 30-second STS show good feasibility but are less feasible in immobile

[Objectives] This study aims to describe the distribution of anchor-based minimal important change (MIC) estimates in standard deviation (SD) units and examine if the robustness of such estimates depends on the specific SD used or on the methodological credibility of the anchor-based estimates. [Design and Setting] We included all anchor-based MIC estimates from studies published in MEDLINE and relevant literature databases upto October 2018. Each MIC was converted to SD units using baseline, endpoint, and change from baseline SDs. We performed a descriptive analysis of MICs in SD units and checked how the distribution would change if MICs with low methodological credibility were excluded from the analysis. [Results] We included 1, 009 MIC estimates from 182 studies. The medians and interquartile ranges of MICs in SD units were 0.43 (0.25 to 0.69), 0.42 (0.22 to 0.70), and 0.51 (0.28 to 0.78) for baseline, endpoint, and change SD units, respectively. Some MICs were extremely large or small. The distribution did not change significantly after excluding MICs estimated by less credible methods. [Conclusions] The size of the universally applicable MIC in SD units could not be determined. Anchor-based MICs in SD units were widely distributed, with more than half in the range of 0.2 to 0.8.

Variable disease progression confounds accurate prognosis in Fabry disease. Evidence supports the long-term benefit of early intervention with disease-specific therapy, but current guidelines recommend treatment initiation based on signs that may present too late to avoid irreversible organ damage. Findings from the 'PRoposing Early Disease Indicators for Clinical Tracking in Fabry Disease' (PREDICT-FD) initiative included expert consensus on 27 early indicators of disease progression in Fabry disease and on drivers of and barriers to treatment initiation in Fabry disease. Here, we compared the PREDICT-FD indicators with guidance from the European Fabry Working Group and various national guidelines to identify differences in signs supporting treatment initiation and how guidelines themselves might affect initiation. Finally, anonymized patient histories were reviewed by PREDICT-FD experts to determine whether PREDICT-FD indicators supported earlier treatment than existing guidance. Current guidelines generally aligned with PREDICT-FD on indicators of renal involvement, but most lacked specificity regarding cardiac indicators. The prognostic significance of neurological indicators such as white matter lesions (excluded by PREDICT-FD) was questioned in some guidelines and excluded from most. Some PREDICT-FD patient-reported signs (e.g., febrile crises) did not feature elsewhere. Key drivers of treatment initiation in PREDICT-FD were: (A) male sex, young age, and clinical findings (e.g., severe pain, organ involvement), (B) improving clinical outcomes and preventing disease progression, and (C) a family history of Fabry disease (especially if outcomes were severe). All guidelines aligned with (A) and several advocated therapy for asymptomatic male patients. There was scant evidence of (B) in current guidance: for example, no countries mandated ancillary symptomatic therapy, and no guidance advocated familial screening with (C) when diagnosis was confirmed. Barriers wer

Background: The purpose of this study is to analyze the potential influence of subclinical hypothyroidism (SCH) on improvement in patient-reported outcome measures following primary total knee arthroplasty. Methods: A prospective, comparative cohort study between 92 SCH and 90 euthyroid patients was performed. Patients were followed up to 5 postoperative years. Patient-reported outcome measure was assessed by the Western Ontario and McMaster Universities Osteoarthritis Index questionnaire. The Knee Society Scores were used for functional evaluation, and 5-point Likert scale for patient satisfaction. The Hospital Anxiety and Depression scale was also used. Results: All outcome scores significantly improved from preoperative to final follow-up in both groups (P = .001). There were no significant differences between groups in Knee Society Scores (P = .057) at the final follow-up, but Western Ontario and McMaster Universities Osteoarthritis Index scores were significantly lower in the SCH group (P = .015). Likewise, the patient satisfaction rate was significant lower in the SCH group (0.010). Conclusion: SCH patients have a slower functional recovery than euthyroid patients, and trended toward lower improvements in patient-reported scores. Depression was the most important negative factor. The findings of this study can provide the surgeon with an important information for better counseling the SCH patients.

The current revolution of digital health technology and machine learning offers enormous potential to improve patient care. Nevertheless, it is essential to recognize that dermatology requires an approach different from those of other specialties. For many dermatological conditions, there is a lack of standardized methodology for quantitatively tracking disease progression and treatment response (clinimetrics). Furthermore, dermatological diseases impact patients in complex ways, some of which can be measured only through patient reports (psychometrics). New tools using digital health technology (e.g., smartphone applications, wearable devices) can aid in capturing both clinimetric and psychometric variables over time. With these data, machine learning can inform efforts to improve health care by, for example, the identification of high-risk patient groups, optimization of treatment strategies, and prediction of disease outcomes. We use the term personalized, data-driven dermatology to refer to the use of comprehensive data to inform individual patient care and improve patient outcomes. In this paper, we provide a framework that includes data from multiple sources, leverages digital health technology, and uses machine learning. Although this framework is applicable broadly to dermatological conditions, we use the example of a serious inflammatory skin condition, chronic cutaneous graft-versus-host disease, to illustrate personalized, data-driven dermatology.

The present study reports on the development of a questionnaire that evaluates children's experiences of their physical classroom environment, activities and interactions. It also explores the psychometric properties of the questionnaire and how acoustical characteristics of the classroom, class size and student characteristics (age, sex and Swedish as a second language) influence the self-reported outcomes. After development, the questionnaire name should read Activities and Interactions in the Classroom were administered to 101 students (7.9–10.7 years old). Demographic information about the students was collected from parents. The psychometric properties including test–retest reliability are good but can be improved by reducing the number of items. Acoustical characteristics of the classrooms and student characteristics did not predict questionnaire outcomes. As the acoustical conditions in students’ classrooms were close to optimal, communication fostering support should be in focus. The present questionnaire can be used to assess young students’ experiences of their classroom environment and learning situations. © The Author(s) 2021.

Itopride, a mixed D2 antagonist and cholinesterase inhibitor, has prokinetic effects on gastric motility. The Leuven Postprandial Distress Scale is a validated patient-reported outcome instrument for functional dyspepsia (FD) postprandial distress syndrome (PDS). We aimed to use the LPDS to assess treatment outcome in PDS and PDS/EPS (epigastric pain syndrome). Patients with PDS, with or without non-predominant EPS symptoms, were enrolled in an 8-week double-blind placebo-controlled multi-center trial with itopride (100 mg t.i.d.). Patients completed LPDS diaries and questionnaires to assess treatment response. Mann-Whitney test and mixed models were used. One hundred patients (79% females, 39.1 ± 1.5 yo) were included. No significant difference was observed between treatment arms (p = 0.6). Compared to baseline, itopride treatment significantly improved the LPDS score (p = 0.001) and all individual symptoms (p < 0.0001). In the placebo arm, this was only the case for belching and epigastric pain (p < 0.05). In an exploratory analysis, outcomes in "pure" PDS (n = 45) and overlapping PDS/EPS (n = 55) patients were assessed and showed that the latter subgroup has the largest benefit with itopride compared to placebo (p = 0.03). Using the LPDS score in a pilot controlled trial in FD, itopride shows no therapeutic benefit over placebo after 8 weeks of treatment. In an exploratory post hoc analysis, itopride but not placebo was associated with improvement of symptoms compared to baseline, and this was most prominent in patients with overlapping PDS/EPS. The efficacy of itopride in this subgroup needs to be evaluated in a large study using the same outcome measure. (clinialtrials.org ref.: NCT04647955).

Aims: The aim of this cross-sectional study was to examine the prevalence of the multidimensional phenomenon of fatigue in adults with congenital heart disease. Background: Adults with congenital heart disease are a growing population, and patient-reported outcomes can provide valuable information about the patient's experience of living with CHD. Fatigue is a multidimensional phenomenon that can be described as an overwhelming feeling of exhaustion with a reduced capacity of mental and physical work. Fatigue can be observed clinically in adults with congenital heart disease, but the actual prevalence is unknown. Methods: Fatigue was assessed by the Multidimensional Fatigue Inventory which enables the respondent to report the presence of fatigue according to five dimensions: "general fatigue,""physical fatigue,""mental fatigue,""reduced motivation,"and "reduced activity."The questionnaire was sent to 463 patients in Lund and Umeå. Four groups with complex CHD and two groups with moderately complex CHD were included. The reliability (internal consistency) of the Multidimensional Fatigue Inventory was tested for all dimensions and groups of diagnosis. Results: The response rate was 56.6% (n= 262). In patients with complex CHD, 40.0-59.4% reported severe to very severe general fatigue, and patients with a single ventricle reported the highest prevalence (59.4%). Among patients with complex CHD, 29.2-40.0% reported severe to very severe mental fatigue. The Multidimensional Fatigue Inventory had a high reliability measured with Cronbach's alpha. Conclusions: The study findings show a relatively high prevalence of fatigue in adults with congenital heart disease, and general fatigue was the most prevalent. Further studies are needed regarding fatigue and its causes and consequences in adults with congenital heart disease. Relevance to clinical practice: The Multidimensional Fatigue Inventory proved to be an instrument with high reliability and low internal loss, which sugg

Apremilast is approved for the treatment of psoriasis and psoriatic arthritis (PsA). Real-world evidence on the efficacy and safety of apremilast in clinical practice is limited. We assessed the use of apremilast in patients with PsA in Belgium clinical practice. The multicentre, observational, prospective APOLO study enrolled patients with active PsA initiating apremilast in Belgium between April 2017 and December 2018. Primary outcome was PsA Response Criteria (PsARC) after 6 months of apremilast treatment. Secondary outcomes included PsA Impact of Disease 12 (PsAID12) and Health Assessment Questionnaire Disability Index (HAQ-DI). Disease-specific outcomes and patient-reported outcomes (PROs) were analysed for patients who received apremilast within 30 days prior to their study inclusion and completed at least 150 days of treatment (reference set [REF]). Of 107 patients enrolled in the study, 106 received at least one dose of apremilast and 69 were included in the REF. PsARC response was achieved by 43.5% of patients (30/69) in the REF at month 6; mean global and composite scores including 68-joint count for pain/tenderness (68-TJC) and 66-joint count for swelling (66-SJC) improved, and 27% and 42% of patients with 68-TJC and 66-SJC > 0 at baseline had complete joint count resolution, respectively. Mean global and composite PsAID12 and HAQ-DI scores decreased at 6 months, indicating improved quality of life. Apremilast was well tolerated and the reported adverse events were in line with the known safety profile. Results from the APOLO study indicate that treatment with apremilast in Belgian clinical practice improves the signs and symptoms of PsA as well as patient quality of life. CLINICALTRIALS. NCT03096990.

IntroductionHypoactive Sexual Desire Disorder (HSDD) / Female Sexual Interest/Arousal Disorder (FSIAD) impacts health-related quality of life (HRQoL) of women and their partners, yet existing measures fail to adequately capture relevant concepts (ie, what is essential to measure including symptoms/impacts) important to women with HSDD/FSIAD.ObjectivesTo identify HRQoL tools used to assess women with HSDD/FSIAD, and to evaluate their psychometric properties (ie, reliability, validity, and responsiveness).MethodsWe conducted searches in PubMed, Embase and PsychINFO from June 5, 1989 to September 30, 2020 for studies in women with HSDD/FSIAD and psychometric analyses (English only). Principles of the Preferred Reporting Items for Systematic reviews and Meta-Analyses, the COnsensus-based Standards for the selection of health Measurement INstruments Risk of Bias Checklist and other psychometric criteria were applied. Based on this search, 56 papers were evaluated including 15 randomized-controlled trials, 11 observational/single arm/open label studies, and 30 psychometric studies.ResultsOf the 18 measures identified, the Female Sexual Function Index (FSFI) and Female Sexual Distress Scale-Revised (FSDS-R) were included in most studies (> 50%). General HRQoL instruments were not used in any of the clinical trials; the SF-12, SF-36 and EQ-5D-5L were reported in two observational studies. No instruments achieved positive quality ratings across all psychometric criteria. The FSFI, FSDS-R, Sexual Event Diary (SED) and the Sexual Desire Relationship Distress Scale (SDRDS), were the only measures to receive a positive rating for content validity.ConclusionReliable and valid HRQoL measures that include sexual desire and distress are needed to provide a more systematic and comprehensive assessment of HRQoL and treatment benefits in women with HSDD/FSIAD. While inferences about HRQoL are limited due to the lack of uniformity in concepts assessed and limited psychometric evaluat

Purpose We investigated objective and patient-reported outcomes after resection arthroplasty or shortening oblique osteotomy (SOO) of the lesser metatarsals combined with arthrodesis of the first metatarsophalangeal (MTP) joint for severe rheumatoid forefoot deformities. Methods 17 feet from 14 women (mean age, 67.8 years) underwent resection arthroplasty of the lesser metatarsal heads (MTH resection group), while 13 feet from nine women and two men (mean age, 68.7 years) underwent SOO of the lesser metatarsals (MTH preservation group). Arthrodesis of the first MTP joint was performed in all cases. Mean follow-up in the MTH resection and preservation groups was 25.0 and 21.3 months, respectively. Preoperative and postoperative clinical evaluation included Japanese Society for Surgery of the Foot (JSSF) scale and self-administered foot evaluation questionnaire (SAFE-Q) scores. Results Mean total JSSF scale significantly improved from 53.4 to 76.4 in the MTH resection group (p < .001) and from 50.1 to 74.2 in the MTH preservation group (p = .002). Pain and pain-related and shoe-related SAFE-Q subscale scores significantly improved after surgery in both groups. In the MTH resection group, recurrence of painful callosities and claw toe deformity was observed in four and three feet, respectively. In the MTH preservation group, one patient experienced recurrence of painful callosities and one underwent revision surgery for IP joint dislocation. Conclusion Resection arthroplasty or SOO of the lesser metatarsals combined with arthrodesis of the first MTP joint achieved significant improvement with respect to pain relief, deformity correction, and footwear comfort.

Background: Patient-reported outcomes (PROs) are currently poorly integrated in the clinical evaluation of disease activity in patients with ANCA-associated vasculitis (AAV). Objectives: To assess the distribution of the Patient Global Assessment (PtGA) in patients with AAV in stable remission, and to identify correlates of PtGA; to assess the discordance between PtGA score and Physician Global Assessment (PhGA). Methods: Patients with a diagnosis of AAV in stable remission (BVAS=0) and with a Physician Global Assessment (PhGA)=0 were included. The following PROs on a 0-100 visual analogue scale (VAS) were assessed: PtGA, fatigue, pain, general health, sleep quality, and chronic damage according to the patient's opinion. The Cragg Hurdle model was used to assess the predictors of PtGA. Results: 65 patients were included, female 57%, mean age 61±12 years. Median disease duration 6 years (IQR 3-12). Vasculitis damage index (VDI) was 4.4±2.3. Despite having been classified as being in remission, PtGA was elevated in 37% of patients. The PtGA was not associated with older age, comorbidities, educational level, the type of AAV diagnosis, number of organ-systems involved, previous relapses, disease duration, nor higher VDI. Female sex was significantly associated with PtGA: 51% of female patients reported an elevated PtGA compared to 18% of males (p=0.009). PtGA significantly correlated with all the other assessed PROs. Pain and fatigue were the main determinants of an elevated PtGA. Conclusions: A significant proportion of patients with AAV considered to be in remission by the physician still declares to have persistent aspects of uncontrolled disease. PtGA is significantly influenced by pain and fatigue, which should receive more attention in the future assessment of patients with AAV.

Purpose: To evaluate the use, results, and reporting of patient-reported outcome measures specific to patients undergoing inguinal hernia repair. Methods: A systematic review was performed and reported according to the PRISMA 2020 statement. A protocol was registered at PROSPERO (CRD42021243468). Systematic searches were performed in PubMed and EMBASE. We only included randomized controlled trials that involved postoperative administration of a hernia-specific patient-reported outcome measure. Risk of bias was evaluated with the Cochrane risk of bias-tool 2.0. Results: Twenty trials and four different instruments were included: the Carolinas Comfort Scale (nine studies), Activities Assessment Scale (six studies), Inguinal Pain Questionnaire (seven studies), and Surgical Pain Scales (one study). Included trials used patient-reported outcome measures and compared either different surgical approaches (11 studies), types of mesh/fixation (seven studies), or types of anesthesia/analgesia (two studies). Results were reported using several different methods including means, medians, or proportions of either overall results, results from subscales, or results from single questionnaire items. Seven of the 20 included studies specified a patient-reported outcome measure as a primary outcome and provided clear reporting of sample size calculation. Conclusion: Reporting of results from patient-reported outcome measures in inguinal hernia research was characterized by heterogeneity. The results were reported using several different methods, which impedes proper evidence synthesis. Only half of the included studies applied a patient-reported outcome measure as primary outcome. Ultimately, the heterogeneity in outcome reporting is an important methodological problem obstructing the full utilization of patient-reported outcome measures in inguinal hernia research.

Background: No phase 3 studies have yet been conducted for patients with non-clear cell (CC) renal cell carcinoma (RCC) exclusively due to the rare occurrence of the disease and the heterogenicity in tumor morphology. Consequently, there is no evidence of the optimal treatment, and new approaches are needed. One approach is individualizing treatment based on the gene sequencing of tumor tissue. Additionally, recent studies involving the patient-reported outcomes (PROs) of patients treated for metastatic cancer have shown significant benefits for quality of life, median overall survival, and overall survival. The use of gene sequencing and PROs can be of great importance to patients with rare cancer types, including patients with non-CC RCC, and should be investigated in clinical trials, especially for cases where evidence based on phase 3 studies is difficult to obtain. Objective: We describe the INDIGO study, in which patients, based on gene analyses, will be allocated into 4 treatment arms containing 14 treatments and use electronic PROs. We aim to improve the treatment of patients with non-CC RCC. The end points in the study will be the overall response rate (complete and partial) in the total patient population, which will be based on the RECIST (Response Evaluation Criteria in Solid Tumors) version 1.1 criteria, and the time to treatment failure. Methods: INDIGO is a prospective phase 2 trial, and 30 patients will be enrolled. The patients will receive systemic treatment based on genetic analyses of their tumor tissue. All patients will receive electronic questionnaires in a dedicated app-a questionnaire regarding symptoms and side effects and another regarding health-related quality of life. Depending on the treatment regimen, the patients will be seen by a medical doctor every third, fourth, or sixth week, and the effect of the systemic treatment will be evaluated every 6 weeks via a computed tomography scan. The study has been approved by the Danish Medic

Background: Real-world data from electronic health records (EHRs) represent a wealth of information for studying the benefits and risks of medical treatment. However, they are limited in scope and should be complemented by information from the patient perspective. Objective: The aim of this study is to develop an innovative research infrastructure that combines information from EHRs with patient experiences reported in questionnaires to monitor the risks and benefits of medical treatment. Methods: We focused on the treatment of overactive bladder (OAB) in general practice as a use case. To develop the Benefit, Risk, and Impact of Medication Monitor (BRIMM) infrastructure, we first performed a requirement analysis. BRIMM’s starting point is routinely recorded general practice EHR data that are sent to the Dutch Nivel Primary Care Database weekly. Patients with OAB were flagged weekly on the basis of diagnoses and prescriptions. They were invited subsequently for participation by their general practitioner (GP), via a trusted third party. Patients received a series of questionnaires on disease status, pharmacological and nonpharmacological treatments, adverse drug reactions, drug adherence, and quality of life. The questionnaires and a dedicated feedback portal were developed in collaboration with a patient association for pelvic-related diseases, Bekkenbodem4All. Participating patients and GPs received feedback. An expert meeting was organized to assess the strengths, weaknesses, opportunities, and threats of the new research infrastructure. Results: The BRIMM infrastructure was developed and implemented. In the Nivel Primary Care Database, 2933 patients with OAB from 27 general practices were flagged. GPs selected 1636 (55.78%) patients who were eligible for the study, of whom 295 (18.0% of eligible patients) completed the first questionnaire. A total of 288 (97.6%) patients consented to the linkage of their questionnaire d

Background: Partial nephrectomy (PN) is the gold standard for the treatment of stage cT1 renal cell carcinoma (RCC). However, the increasing incidence of RCC in the elderly population calls for alternative minimally invasive treatments to reduce the negative effects on patients’ health-related quality of life (HRQoL) and subsequent healthy life expectancy. Objective: To assess and compare short-term HRQoL and self-reported health status after PN and percutaneous cryoablation (PCA) of patients treated for RCC stage cT1. Design, setting, and participants: Patients who underwent PN or PCA between 2019 and 2021 for RCC stage cT1 at two university hospitals in Denmark were assessed. The exclusion criteria included insufficient understanding of the Danish language, dementia, metastatic RCC, conversion to nephrectomy, and salvage procedures. Intervention: PN and PCA. Outcome measurements and statistical analysis: The European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire was distributed before treatment, and 14 and 90 d postoperatively. A linear mixed-effect model was used to analyze the changes from baseline to follow-up between PN and PCA treatment. Results and limitations: The cohort included 165 patients (PN: 79; PCA: 86). The completion rate was 96–98%. Patients receiving PCA were significantly older (median 69.1 vs 62.1 yr) and had lower scores on physical (p < 0.001) and role functioning (p = 0.009) than PN. A statistically significant change from baseline to 14 d was found for several HRQoL scales, which favored PCA over PN. However, the observed change was no longer significant at 90-d follow-up. Limitations include sample size and confounding by indication. Conclusions: This study found a significant difference between baseline and 14-d follow-up in several HRQoL and symptoms scales, favoring PCA over PN. However, no significant differences were observed in any HRQoL scales between PN and PCA of RCC stage cT1 from basel

Study Design.This was a dual-center study over an eight-year period on patients undergoing single level fusion surgery with either posterior- (PLIF) or transforaminal lumbar interbody fusion (TLIF). We analyzed prospectively collected pre- and postoperative data from the national Danish surgical spine database (DaneSpine).Objective.The aim of this study was to compare clinical and patient-reported outcome (PRO) 2 years after TLIF or PLIF in patients with symptomatic lumbar mechanical disc degeneration.Summary of Background Data.PLIF and TLIF are well-described techniques for treating lumbar mechanical disc degeneration but whether the theoretical differences between the two techniques translate to different clinical outcomes is unknown.Methods.The primary outcome was Oswestry Disability Index (ODI) score at 2-year follow-up. Secondary outcome measures were scores on the European Quality of Life-5 Dimensions (EQ-5D) and visual analog scale (VAS) and the rate of intraoperative complications. To minimize baseline differences between the groups, propensity-score matching was employed in a 1:1 fashion, balancing the groups on preoperative factors including age, sex, back and leg pain, ODI, EQ-5D, and previous spine surgery.Result.The matched cohort included 211 patients in each cohort. There was no significant difference between the groups in the mean score on the ODI at two years (PLIF: 33 ± 20 vs. TLIF: 35 ± 20, P = 0.328). We found no statistically significant differences in EQ-5D score (0.54 ± 0.35 vs. 0.51 ± 0.34, P = 0.327), VAS score for back pain (47 ± 32 vs. 48 ± 29, P = 0.570) or leg pain (42 ± 33 vs. 41 ± 32, P = 0.936) between the PLIF and TLIF groups, respectively, at 2-year follow-up. Dural tears occurred in 9.5% in the PLIF group and 1.9% in the TLIF group (P = 0.002) corresponding to a relative risk of 5.0 (95% CI 1.7-14.4).Conclusion.We found no significant difference in PRO at 2-year follow-up between PLIF and TLIF for the treatment of lumbar disc de

BACKGROUND: The impact of obesity on patient-reported outcome (PRO) after total knee arthroplasty (TKA) surgery has demonstrated varying results. We evaluated knee pain, Activity in Daily Life function (ADL), and satisfaction after TKA surgery in patients with and without prior bariatric surgery (BS). METHODS: Scandinavian Obesity Surgery Registry (SOReg) and the Swedish Knee Arthroplasty Register (SKAR) were used to identify patients operated on with primary TKA for osteoarthritis (OA) between 2009 and 2019 that had a BS within 2 years before the TKA (BS group). These patients were compared to patients with TKA without prior BS (no BS group). The patients filled in the Knee injury and Osteoarthritis Outcome Score (KOOS) preoperatively and one year postoperatively as well as satisfaction with the surgery one year postoperatively. Multiple linear regression analysis was used to evaluate 1-year postoperative KOOS pain and ADL function between the 2 groups. Adjustments were made for sex, age, and preoperative KOOS pain and ADL function respectively. RESULTS: Forty-four patients were included in the BS group and 3,525 patients in the no BS group. We found no statistically or clinically significant difference in one-year postoperative KOOS pain and ADL function between the BS group and the no BS group. The majority of the patients in both groups were classified as satisfied or very satisfied one year postoperatively to the TKA. CONCLUSIONS: Our results indicate that patients without BS prior to the TKA gain similar 1-year outcome in pain, ADL function and satisfaction as patients with prior BS., Funding agency:Örebro University

Monitoring the performance and functional status of baseball pitchers’ upper extremity is important in maintaining the athlete’s health and performance. This study validated a Dutch translation of the original English Kerlan-Jobe Orthopaedic Clinic (KJOC) against the previously validated Disabilities of the Arm, Shoulder and Hand (DASH) and Western Ontario Shoulder Instability Index (WOSI) questionnaires in a group of talented juvenile Dutch baseball pitchers. Three times, from 2014–2016, 107 pitchers completed the Dutch KJOC, DASH and WOSI questionnaires. Participants’ questionnaire scores were analysed for the whole group and the symptomatic player subgroup separately. Internal consistency, construct validity and ceiling and floor effects were examined. Cronbach’s alpha was consistently above 0.8 for the three time periods for the whole group, and ranged between 0.62 and 0.86 for the symptomatic subgroup. Spearman’s rank correlation coefficients ranged from 0.47 to 0.67 for the whole group and 0.32 to 0.99 for the symptomatic subgroup. No floor effects were observed in the scores of the KJOC and only a ceiling effect for the whole group (15.2%) at one time period. The Dutch version of the KJOC has shown acceptable internal consistency and construct validity and can be used to assess overhead athletes’ shoulder and elbow functionality., Biomechatronics & Human-Machine Control

Background: Skin reaction is a common side-effect of radiotherapy and often only assessed as clinician-reported outcome (CRO). The aim was to examine and compare patient-reported outcome (PRO) of skin reactions with CRO for signs of acute skin reactions for patients with primary brain tumour receiving proton beam radiotherapy (PBT). A further aim was to explore patients' experiences of the skin reactions. Methods: Acute skin reactions were assessed one week after start of treatment, mid-treatment and end of treatment among 253 patients with primary brain tumour who underwent PBT. PRO skin reactions were assessed with the RSAS and CRO according to the RTOG scale. Fleiss' kappa was performed to measure the inter-rater agreement of the assessments of skin reactions. Results: The results showed a discrepancy between PRO and CRO acute skin reactions. Radiation dose was associated with increased skin reactions, but no correlations were seen for age, gender, education, occupation, other treatment or smoking. There was a poor agreement between patients and clinicians (κ = -0.016) one week after the start of PBT, poor (κ = -0.045) to (κ = 0.396) moderate agreement at mid treatment and poor (κ = -0.010) to (κ = 0.296) moderate agreement at end of treatment. Generally, patients' symptom distress toward skin reactions was low at all time points. Conclusion: The poor agreement between PRO and CRO shows that the patient needs to be involved in assessments of skin reactions for a more complete understanding of skin reactions due to PBT. This may also improve patient experience regarding involvement in their own care.