Psychometric analysis of the modified COVID-19 Yorkshire Rehabilitation Scale (C19-YRSm) in a prospective multicentre study.

BACKGROUND: Long COVID (LC) is a novel multisystem clinical syndrome affecting millions of individuals worldwide. The modified COVID-19 Yorkshire Rehabilitation Scale (C19-YRSm) is a condition-specific patient-reported outcome measure designed for assessment and monitoring of people with LC. OBJECTIVES: To evaluate the psychometric properties of the C19-YRSm in a prospective sample of people with LC. METHODS: 1314 patients attending 10 UK specialist LC clinics completed C19-YRSm and EuroQol 5D-5L (EQ-5D-5L) longitudinally. Scale characteristics were derived for C19-YRSm subscales (Symptom Severity (SS), Functional Disability (FD) and Overall Health (OH)) and internal consistency (Cronbach's alpha). Convergent validity was assessed using the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale. Known groups validity was assessed for the Other Symptoms subscale as tertiles, as well as by hospitalisation and intensive care admission. Responsiveness and test-retest reliability was evaluated for C19-YRSm subscales and EQ-5D-5L. The minimal important difference (MID) and minimal clinically important difference (MCID) were estimated. Confirmatory factor analysis was applied to determine the instrument's two-factor structure. RESULTS: C19-YRSm demonstrated good scale characteristic properties. Item-total correlations were between 0.37 and 0.65 (for SS and FD), with good internal reliability (Cronbach's alphas>0.8). Item correlations between subscales ranged between 0.46 and 0.72. Convergent validity with FACIT was good (-0.46 to -0.62). The three subscales discriminated between different levels of symptom burden (p<0.001) and between patients admitted to hospital and intensive care. There was moderate responsiveness for the three subscales ranging from 0.22 (OH) to 0.50 (SS) which was greater than for the EQ-5D-5L. Test-retest reliability was good for both SS 0.86 and FD 0.78. MID was 2 for SS, 2 for FD and 1 for OH; MCID was 4 for both the SS and FD. The factor analysis supported the two-factor SS and FD structure. CONCLUSIONS: The C19-YRSm is a condition-specific, reliable, valid and responsive patient-reported outcome measure for LC.

Implementation of an integrated primary care prevention and management program for chronic low back pain (LBP): patient-reported outcomes and predictors of pain interference after six months.

BACKGROUND: Integrated primary care programs for patients living with chronic pain which are accessible, interdisciplinary, and patient-centered are needed for preventing chronicity and improving outcomes. Evaluation of the implementation and impact of such programs supports further development of primary care chronic pain management. This study examined patient-reported outcomes among individuals with low back pain (LBP) receiving care in a novel interdisciplinary primary care program. METHODS: Patients were referred by primary care physicians in four regions of Quebec, Canada, and eligible patients received an evidence-based interdisciplinary pain management program over a six-month period. Patients were screened for risk of chronicity. Patient-reported outcome measures of pain interference and intensity, physical function, depression, and anxiety were evaluated at regular intervals over the six-month follow-up. A multilevel regression analysis was performed to evaluate the association between patient characteristics at baseline, including risk of chronicity, and change in pain outcomes. RESULTS: Four hundred and sixty-four individuals (mean age 55.4y, 63% female) completed the program. The majority (≥ 60%) experienced a clinically meaningful improvement in pain intensity and interference at six months. Patients with moderate (71%) or high risk (81%) of chronicity showed greater improvement in pain interference than those with low risk (51%). Significant predictors of improvement in pain interference included a higher risk of chronicity, younger age, female sex, and lower baseline disability. CONCLUSION: The outcomes of this novel LBP program will inform wider implementation considerations by identifying key components for further effectiveness, sustainability, and scale-up of the program.

BACK-to-MOVE: Machine learning and computer vision model automating clinical classification of non-specific low back pain for personalised management.

BACKGROUND: Low back pain (LBP) is a major global disability contributor with profound health and socio-economic implications. The predominant form is non-specific LBP (NSLBP), lacking treatable pathology. Active physical interventions tailored to individual needs and capabilities are crucial for its management. However, the intricate nature of NSLBP and complexity of clinical classification systems necessitating extensive clinical training, hinder customised treatment access. Recent advancements in machine learning and computer vision demonstrate promise in characterising NSLBP altered movement patters through wearable sensors and optical motion capture. This study aimed to develop and evaluate a machine learning model (i.e., 'BACK-to-MOVE') for NSLBP classification trained with expert clinical classification, spinal motion data from a standard video alongside patient-reported outcome measures (PROMs). METHODS: Synchronised video and three-dimensional (3D) motion data was collected during forward spinal flexion from 83 NSLBP patients. Two physiotherapists independently classified them as motor control impairment (MCI) or movement impairment (MI), with conflicts resolved by a third expert. The Convolutional Neural Networks (CNNs) architecture, HigherHRNet, was chosen for effective pose estimation from video data. The model was validated against 3D motion data (subset of 62) and trained on the freely available MS-COCO dataset for feature extraction. The Back-to-Move classifier underwent fine-tuning through feed-forward neural networks using labelled examples from the training dataset. Evaluation utilised 5-fold cross-validation to assess accuracy, specificity, sensitivity, and F1 measure. RESULTS: Pose estimation's Mean Square Error of 0.35 degrees against 3D motion data demonstrated strong criterion validity. Back-to-Move proficiently differentiated MI and MCI classes, yielding 93.98% accuracy, 96.49% sensitivity (MI detection), 88.46% specificity (MCI detection), and an F1 measure of .957. Incorporating PROMs curtailed classifier performance (accuracy: 68.67%, sensitivity: 91.23%, specificity: 18.52%, F1: .800). CONCLUSION: This study is the first to demonstrate automated clinical classification of NSLBP using computer vision and machine learning with standard video data, achieving accuracy comparable to expert consensus. Automated classification of NSLBP based on altered movement patters video-recorded during routine clinical examination could expedite personalised NSLBP rehabilitation management, circumventing existing healthcare constraints. This advancement holds significant promise for patients and healthcare services alike.

Application of Patient-Reported Outcome Measurements in Adult Tumor Clinical Trials in China: Cross-Sectional Study.

BACKGROUND: International health policies and researchers have emphasized the value of evaluating patient-reported outcomes (PROs) in clinical studies. However, the characteristics of PROs in adult tumor clinical trials in China remain insufficiently elucidated. OBJECTIVE: This study aims to assess the application and characteristics of PRO instruments as primary or secondary outcomes in adult randomized clinical trials related to tumors in China. METHODS: This cross-sectional study identified tumor-focused randomized clinical trials conducted in China between January 1, 2010, and June 30, 2022. The ClinicalTrials.gov database and the Chinese Clinical Trial Registry were selected as the databases. Trials were classified into four groups based on the use of PRO instruments: (1) trials listing PRO instruments as primary outcomes, (2) trials listing PRO instruments as secondary outcomes, (3) trials listing PRO instruments as coprimary outcomes, and (4) trials without any mention of PRO instruments. Pertinent data, including study phase, settings, geographic regions, centers, participant demographics (age and sex), funding sources, intervention types, target diseases, and the names of PRO instruments, were extracted from these trials. The target diseases involved in the trials were grouped according to the American Joint Committee on Cancer Staging Manual, 8th Edition. RESULTS: Among the 6445 trials examined, 2390 (37.08%) incorporated PRO instruments as part of their outcomes. Within this subset, 26.82% (641/2390) listed PRO instruments as primary outcomes, 52.72% (1260/2390) as secondary outcomes, and 20.46% (489/2390) as coprimary outcomes. Among the 2,155,306 participants included in these trials, PRO instruments were used to collect data from 613,648 (28.47%) patients as primary or secondary outcomes and from 74,287 (3.45%) patients as coprimary outcomes. The most common conditions explicitly using specified PRO instruments included thorax tumors (217/1280, 16.95%), breast tumors (176/1280, 13.75%), and lower gastrointestinal tract tumors (173/1280, 13.52%). Frequently used PRO instruments included the European Organisation for Research and Treatment of Cancer Quality of Life Core Questionnaire-30, the visual analog scale, the numeric rating scale, the Traditional Chinese Medicine Symptom Scale, and the Pittsburgh Sleep Quality Index. CONCLUSIONS: Over recent years, the incorporation of PROs has demonstrated an upward trajectory in adult randomized clinical trials on tumors in China. Nonetheless, the infrequent measurement of the patient's voice remains noteworthy. Disease-specific PRO instruments should be more effectively incorporated into various tumor disease categories in clinical trials, and there is room for improvement in the inclusion of PRO instruments as clinical trial end points.

Correlation between disease activity and patient-reported health-related quality of life in rheumatoid arthritis: a cross-sectional study.

OBJECTIVE: We aimed to provide a comprehensive assessment of health-related quality of life (HRQoL) in patients with rheumatoid arthritis (RA) of different activities and to evaluate the correlation between clinical activity measures and HRQoL instruments. This research also analysed the extent to which different aspects of HRQoL (physical, psychological and social) were affected. DESIGN: Cross-sectional, observational, non-interventional study. SETTING: The study was conducted at the Department of Rheumatology and Immunology, Qilu Hospital, Shandong University. METHODS: From December 2019 to October 2020, a total of 340 RA patients participated in the survey using convenient sampling. Three generic instruments, EQ-5D-5L,SF-12 and the AQoL-4D, as well as an RA-specific instrument,the Stanford Health Assessment Questionnaire Disability Index (HAQ-DI), were administered to assess patients' HRQoL. The Disease Activity Score 28-Erythrocyte Sedimentation Rate (DAS28-ESR) was used by doctors to measure patients' clinical activity. Multivariable linear regression was used to compare patients' HRQoL across different levels of activity. Spearman's correlation was used to assess the correlation between doctor-reported clinical activity and HRQoL. RESULTS: A total of 314 patients with RA participated in this study. The mean score of HAQ-DI was 0.87 (SD: 0.91). Using patients in the clinical remission group as a reference, patients in the moderate and high disease activity groups showed significantly reduced health state utility values and HRQoL scores (all p<0.05). On the contrary, there was an increase in HAQ-DI scores, indicating more impairment (p<0.05). All instruments included in the study tended to differentiate disease activity based on multiple criteria, with scores showing a moderate to strong correlation with RA activity (|rs|=0.50 to 0.65). Among them, the disease-specific instrument had the highest correlation. CONCLUSIONS: RA can have considerable impairment on patients' HRQoL, both in terms of physical and psychosocial functioning. Given the strong correlation between clinical activity and HRQoL scores, and the fact that HRQoL can be an important clinical supplement. The EQ-5D-5L is probably the most appropriate generic measurement instrument for measuring HRQoL in RA patients.

Rapid and sustained improvements in itch and quality of life with upadacitinib plus topical corticosteroids in adults and adolescents with atopic dermatitis: 52-week outcomes from the phase 3 AD Up study.

Purpose: Atopic dermatitis (AD) adversely impacts quality of life (QoL). We evaluated the effect of upadacitinib, an oral selective Janus kinase inhibitor approved for moderate-to-severe AD, plus topical corticosteroids (+TCS) on patient-reported outcomes (PROs) over 52 weeks.Materials and methods: In the phase 3 AD Up study (NCT03568318), adults and adolescents with moderate-to-severe AD were randomized 1:1:1 to once-daily upadacitinib 15 mg, 30 mg, or placebo + TCS. Itch, skin pain/symptoms, sleep, QoL, daily activities, emotional state, mental health, and patient impressions of disease severity/improvement/treatment satisfaction were assessed.Results: This analysis included 901 patients. Within 1-2 weeks, PRO improvements were greater with both upadacitinib doses than with placebo (p <.05). Improvements increased through weeks 4-8; rates were generally maintained through week 52. At week 52, the proportion of patients with clinically meaningful improvements in itch (Worst Pruritus Numerical Rating Scale improvement ≥4), skin pain (AD Symptom Scale Skin Pain improvement ≥4), sleep (AD Impact Scale [ADerm-IS] Sleep improvement ≥12), daily activities (ADerm-IS Daily Activities improvement ≥14), and emotional state (ADerm-IS Emotional State improvement ≥11) ranged from 62.1%-77.7% with upadacitinib 15 mg + TCS and 71.3%-83.6% with upadacitinib 30 mg + TCS.Conclusions: Upadacitinib + TCS results in rapid, sustained improvements in burdensome AD symptoms and QoL.

Content validity of SarQoL, a quality of life questionnaire specific to sarcopenia.

BACKGROUND: The Sarcopenia & Quality of Life (SarQoL) questionnaire is a patient-reported outcome measure designed for assessing health-related quality of life in individuals with sarcopenia. Despite its wide acceptance in the scientific literature, its content validity has only been partially demonstrated so far. AIMS: To enhance the evidence supporting the content validity of the SarQoL questionnaire. METHODS: Following COSMIN methodology, semi-structured interviews were conducted with 17 Belgian older adults who met the EWGSOP2 criteria for the diagnosis of sarcopenia and 11 experts in sarcopenia, with clinical or research background. Comprehensiveness, relevance and comprehensibility of SarQoL content were assessed through individual transcripts and were qualitatively analyzed thematically according to the seven dimensions of SarQoL. RESULTS: The majority of the concepts elicited during the semi-structured interviews fitted within existing SarQoL dimensions. Importantly, the different domains of SarQoL were consensually considered as relevant by patients and experts. Some new emergent concepts were identified by the participants. While many of them could be considered as enrichments of existing dimensions or sub-concepts, other new concepts (i.e. self-fulfilment, acceptance of the reduced condition, adaptation/use of strategies, depression) may highlight two potential dimensions not covered by SarQoL, i.e. patient empowerment and depression. Cognitive interviews also highlighted that SarQoL items and instructions were clear and comprehensible. CONCLUSIONS: SarQoL, in its current form, demonstrates good evidence of content validity for assessing health-related quality of life in patients with sarcopenia. We do not recommend adding new items or dimensions to SarQoL. Instead, for researchers or clinicians who aim to specifically address self-empowerment or depression of sarcopenic populations, we suggest completing the assessment of quality of life by concurrently using additional validated scales of patient empowerment or depression.

Development and validation of clinical implementation methods for patient-reported outcomes in Japanese multi-center palliative care units.

BACKGROUND: Patient-Reported Outcomes (PROs) are recommended for use in clinical oncology. However, they are not routinely used in professional palliative care practices in Japan. The reasons include both patient and healthcare provider factors and the implementation of PROs. This study aimed to develop and validate clinical implementation methods for PROs in Japanese palliative care units. METHODS: The Consolidated Framework for Implementation Research (CFIR) was conducted with four palliative care units in Japan. The study was conducted in six steps: unit assessment, development and implementation of a PRO implementation plan, PRO post-implementation survey and analysis of its utilization, a review of the PRO implementation process, creation of a PRO implementation method in a palliative care unit, and use and verification of the implementation method. Steps 1-5 were the development phase, and step 6 was the verification phase. RESULTS: Interviews were conducted with healthcare providers prior to PRO implementation. Intervention characteristics, patient needs in the palliative care unit, and factors related to the organization were identified as barriers. The implementation plan was developed, and the core members were selected. The implementation procedures were created in the above mentioned steps. PROs were used in the palliative care units. The same was true in the validation phase. CONCLUSIONS: This study guided PROs in specialized palliative care unit in a clinical setting. The method was developed and validated for the implementation of PROs in the palliative care unit. In the PRO implementation process, it was important to assess the unit, address the barriers to implementation, and reduce the burden on healthcare providers. Furthermore, healthcare providers had to be supported by the champion, a person responsible for the implementation of PROs in the palliative care unit.

Developing and validating multi-omics prediction models for late patient-reported dysphagia in head and neck radiotherapy.

Background and purpose. To investigate models developed using radiomic and dosiomic (multi-omics) features from planning and treatment imaging for late patient-reported dysphagia in head and neck radiotherapy.Materials and methods. Training (n = 64) and testing (n = 23) cohorts of head and neck cancer patients treated with curative intent chemo-radiotherapy with a follow-up time greater than 12 months were retrospectively examined. Patients completed the MD Anderson Dysphagia Inventory and a composite score ≤60 was interpreted as patient-reported dysphagia. A chart review collected baseline dysphagia and clinical factors. Multi-omic features were extracted from planning and last synthetic CT images using the pharyngeal constrictor muscle contours as a region of interest. Late patient-reported dysphagia models were developed using a random forest backbone, with feature selection and up-sampling methods to account for the imbalanced data. Models were developed and validated for multi-omic feature combinations for both timepoints.Results. A clinical and radiomic feature model developed using the planning CT achieved good performance (validation: sensitivity = 80 ± 27% / balanced accuracy = 71 ± 23%, testing: sensitivity = 80 ± 10% / balanced accuracy = 73 ± 11%). The synthetic CT models did not show improvement over the plan CT multi-omics models, with poor reliability of the radiomic features on these images. Dosiomic features extracted from the synthetic CT showed promise in predicting late patient-reported dysphagia.Conclusion. Multi-omics models can predict late patient-reported dysphagia in head and neck radiotherapy patients. Synthetic CT dosiomic features show promise in developing successful models to account for changes in delivered dose distribution. Multi-center or prospective studies are required prior to clinical implementation of these models.

Targeted Combined Endpoint Improvement in Patient and Disease Domains in Atopic Dermatitis: A Treat-to-Target Analysis of Adults with Moderate-to-Severe Atopic Dermatitis Treated with Upadacitinib.

A treat-to-target approach was recently developed to guide systemic treatment for adults with atopic dermatitis (AD). Recommendations outlined criteria for a 3-month initial acceptable treatment target and a 6-month optimal target, evaluated using global assessment of patient-reported disease severity, as well as Eczema Area and Severity Index, itch assessed on an 11-point numerical rating scale, Dermatology Life Quality Index, or Patient-Oriented Eczema Measure. Achievement of these targets with once-daily upadacitinib (15 mg and 30 mg) monotherapy was evaluated using integrated adult data from the Measure Up 1 and 2 phase 3 studies. Among the 852 patients treated with upadacitinib 15 mg or 30 mg, the 3-month initial acceptable target was achieved by >80%, >78%, and ≥87% of patients, and the 6-month optimal target was achieved by ≥53%, >61%, and >73% of patients at weeks 2, 16, and 52, respectively. Achievement of all 6 individual criteria for each of the target goals also increased over time. These findings suggest that upadacitinib 15 mg and 30 mg may help improve standards of care in patients with moderate-to-severe AD by achieving 6-month target goals at 16 weeks and as early as 2 weeks for most patients.

Patient-reported outcomes during accelerating the en-masse retraction of the upper anterior teeth using low-intensity electrical stimulation: a randomized controlled trial.

BACKGROUND: Low-intensity electrical stimulation (LIES) is considered a relatively recent technology that has received little attention in orthodontics as a method of acceleration. This study aimed to evaluate patient-reported outcome measures when LIES is used to accelerate the en-masse retraction of the upper anterior teeth. MATERIALS AND METHODS: The sample consisted of 40 patients (8 males, 32 females; mean age 21.1 ± 2.3 years), with Class II division I malocclusion who required extraction of the first premolars to retract upper anterior teeth. They were randomly assigned to the LIES group (n = 20) and the conventional en-masse retraction group (CER; n = 20). Patient responses regarding pain, discomfort, burning sensation, swelling, chewing difficulty, speech difficulty, and painkillers' consumption were recorded at these nine assessment times: 24 h (T1), 3 days (T2), and 7 days (T3) after force application, then in the second month after 24 h (T4), 3 days (T5), and 7 days (T6) of force re-activation, and finally after 24 h (T7), 3 days (T8), and 7 days (T9) of force re-activation in the third month. RESULTS: The mean values of pain perception were smaller in the LIES group than those in the CER group at all assessment times with no statistically significant differences between the two groups except during the second and third months (T5, T6, T8, and T9; P < 0.005). However, discomfort mean values were greater in the LIES group with significant differences compared to CER group during the first week of the follow-up only (T1, T2, and T3; P < 0.005). Burning sensation levels were very mild in the LIES group, with significant differences between the two groups at T1 and T2 only (P < 0.001). Speech difficulty was significantly greater in the LIES group compared to CER group at all studied times (P < 0.001). High levels of satisfaction and acceptance were reported in both groups, without any significant difference. CONCLUSION: Both the LIES-based acceleration of en-masse retraction of upper anterior teeth and the conventional retraction were accompanied by mild to moderate pain, discomfort, and chewing difficulty on the first day of retraction. These sensations gradually decreased and almost disappeared over a week after force application or re-activation. TRIAL REGISTRATION: ClinicalTrials.gov, ClinicalTrials.gov, NCT05920525. Registered 17 June 2023 - retrospectively registered, http://clinicaltrials.gov/study/NCT05920525?term=NCT05920525&rank=1 .

Longitudinal assessment of real-world patient adherence: a 12-month electronic patient-reported outcomes follow-up of women with early breast cancer undergoing treatment.

BACKGROUND: Electronic patient-reported outcomes (ePROs) assess patients' health status and quality of life, improving patient care and treatment effects, yet little is known about their use and adherence in routine patient care. AIMS: We evaluated the adherence of invasive breast cancer and ductal carcinoma in situ (DCIS) patients to ePROs follow-up and whether specific patient characteristics are related to longitudinal non-adherence. METHODS: Since November 2016, the Breast Center at Charité - Universitätsmedizin Berlin has implemented an ongoing prospective PRO routine program, requiring patients to complete ePROs assessments and consent to email-based follow-up in the first 12 months after therapy starts. Frequencies and summary statistics are presented. Multiple logistic regression models were performed to determine an association between patient characteristics and non-adherence. RESULTS: Out of 578 patients, 239 patients (41.3%, 95%CI: 37.3-45.5%) completed baseline assessment and all five ePROs follow-up during the first 12 months after therapy. On average, above 70% of those patients responded to the ePROs follow-up assessment. Adherence to the ePROs follow-up was higher during the COVID-19 pandemic than in the time periods before (47.4% (111/234) vs. 33.6% (71/211)). Factors associated with longitudinal non-adherence were younger age, a higher number of comorbidities, no chemotherapy, and a low physical functioning score in the EORTC QLQ-C30 at baseline. CONCLUSIONS: The study reveals moderate adherence to 12-month ePROs follow-up assessments in invasive early breast cancer and DCIS patients, with response rates ranging from 60 to 80%. Emphasizing the benefits for young patients and those with high disease burdens might further increase adherence.

Patients with more complex ankle fractures are associated with poorer patient-reported outcome: an observational study of 11,733 patients from the Swedish Fracture Register.

BACKGROUND AND PURPOSE: Patient-reported outcome measures (PROMs) following ankle fractures, including all fracture types, have not been reported. It is therefore unclear whether fracture morphology correlates with outcome. We aimed to analyze PROMs in patients with an ankle fracture in relation to the Arbeitsgemeinschaft für Osteosynthesefragen/Orthopaedic Trauma Association (AO/OTA) fracture classification using population-based register data from the Swedish Fracture Register (SFR). METHODS: All patients aged ≥ 18 years with an ankle fracture (AO/OTA 44A1-C3) registered in the SFR between 2012 and 2019 were retrieved from the register. Patients with completed PROM questionnaires (Short Musculoskeletal Function Assessment and EuroQol-Visual Analogue Scale) on both day 0 (pre-trauma) and 1-year post-trauma were included. The difference in PROMs between day 0 and 1 year was calculated for each patient (delta value) and mean delta values were calculated at group level, based on the AO/OTA fracture classification. RESULTS: 11,733 patients with 11,751 fractures with complete PROMs were included. According to the AO/OTA classification, 21% were A fractures, 67% were B fractures and 12% were C fractures. All groups of patients, regardless of fracture class (A1-C3), displayed an impairment in PROMs after 1 year compared with day 0. Type C fractures displayed a larger impairment in PROMs at group level than type B, which in turn had a greater impairment than type A. The same pattern was seen in groups 3, 2, and 1 for A and B fractures. CONCLUSION: We found that the AO/OTA classification is prognostic, where more complex fractures were associated with poorer PROMs.

Listening to patients, for the patients: The COVAD Study-Vision, organizational structure, and challenges.

BACKGROUND: The pandemic presented unique challenges for individuals with autoimmune and rheumatic diseases (AIRDs) due to their underlying condition, the effects of immunosuppressive treatments, and increased vaccine hesitancy. OBJECTIVES: The COVID-19 vaccination in autoimmune diseases (COVAD) study, a series of ongoing, patient self-reported surveys were conceived with the vision of being a unique tool to gather patient perspectives on AIRDs. It involved a multinational, multicenter collaborative effort amidst a global lockdown. METHODS: Leveraging social media as a research tool, COVAD collected data using validated patient-reported outcomes (PROs). The study, comprising a core team, steering committee, and global collaborators, facilitated data collection and analysis. A pilot-tested, validated survey, featuring questions regarding COVID-19 infection, vaccination and outcomes, patient demographics, and PROs was circulated to patients with AIRDs and healthy controls (HCs). DISCUSSION: We present the challenges encountered during this international collaborative project, including coordination, data management, funding constraints, language barriers, and authorship concerns, while highlighting the measures taken to address them. CONCLUSION: Collaborative virtual models offer a dynamic new frontier in medical research and are vital to studying rare diseases. The COVAD study demonstrates the potential of online platforms for conducting large-scale, patient-focused research and underscores the importance of integrating patient perspective into clinical care. Care of patients is our central motivation, and it is essential to recognize their voices as equal stakeholders and valued partners in the study of the conditions that affect them.

Patient- and Clinician-Reported Outcomes for Tirbanibulin in Actinic Keratosis in Clinical Practice Across the United States (PROAK).

BACKGROUND: The Patient-Reported Outcomes in Actinic Keratosis (PROAK) study evaluated patient- and clinician-reported outcomes (PRO; ClinRO) during 24 weeks of follow-up among adult patients with actinic keratosis (AK) on the face or scalp who were administered tirbanibulin 1% ointment in real-world community practices in the United States.  Methods: Quality of life (QoL) was assessed by Skindex-16 at week (W) 8. Additionally, effectiveness (Investigator Global Assessment [IGA]), PRO and ClinRO (Treatment Satisfaction Questionnaire for Medication and Expert Panel Questionnaire), safety, and tolerability were assessed at W8 and W24. RESULTS: The safety population included 300 patients; the full analysis set included 290 patients (278 patients at W24). At W8, a statistically significant difference (P<0.03) was observed for Skindex-16 domains in all assessed subgroups. Clinicians and patients reported high global satisfaction (mean [SD] scores of 74.9 [23.9] and 72.0 [24.6], respectively) at W24. Overall skin appearance improved from baseline to W24 (83.6% clinicians; 78.5% patients). IGA success (IGA score of 0-1) was achieved by 71.9% of patients at W24 with a similar % at W8 (73.8%) suggesting a stable effectiveness over time. About 5% of patients reported at least one adverse event, 4% reported at least one serious adverse event and no patients reported serious adverse drug reactions. At W8, the most frequently reported local skin reactions were mild/moderate erythema (47.6%) and flaking/scaling (49.6%). CONCLUSIONS: Treatment with tirbanibulin demonstrated effectiveness in the management of AK lesions and a favorable safety and tolerability profile. Furthermore, QoL was improved as early as W8, and both patients and clinicians reported high levels of treatment satisfaction, independently of patients' characteristics. J Drugs Dermatol. 2024;23(5):338-346. doi:10.36849/JDD.8264.

Mapping the Evidence on the Impact of mHealth Interventions on Patient-Reported Outcomes in Patients With Breast Cancer: A Systematic Review.

PURPOSE: To comprehensively synthesize the existing evidence concerning mHealth interventions for patients with breast cancer (BC). DESIGN: On July 30, 2023, we searched PubMed, PsycINFO, and Google Scholar for articles using the following inclusion criteria: evaluation of mHealth interventions in patients with cancer, at least 30 participants with BC, randomized control trials or prospective pre-post studies, determinants of health (patient-reported outcomes [PROs] and quality of life [QoL]) as primary outcomes, interventions lasting at least 8 weeks, publication after January 2015. Publications were excluded if they evaluated telehealth or used web-based software for desktop devices only. The quality of the included studies was analyzed with the Cochrane Collaboration Risk of Bias Tool and the Methodological Index for Non-Randomized Studies. RESULTS: We included 30 studies (20 focused on BC), encompassing 5,691 patients with cancer (median 113, IQR, 135.5). Among these, 3,606 had BC (median 99, IQR, 75). All studies contained multiple interventions, including physical activity, tailored information for self-management of the disease, and symptom tracker. Interventions showed better results on self-efficacy (3/3), QoL (10/14), and physical activity (5/7). Lifestyle programs (3/3), expert consulting (4/4), and tailored information (10/11) yielded the best results. Apps with interactive support had a higher rate of positive findings, while interventions targeted to survivors showed worse results. mHealth tools were not available to the public in most of the studies (17/30). CONCLUSION: mHealth interventions yielded heterogeneous results on different outcomes. Identifying lack of evidence on clinical scenarios (eg, patients undergoing systemic therapy other than chemotherapy) could aid in refining strategic planning for forthcoming research endeavors within this field.

Total knee arthroplasty following lateral closing-wedge high tibial osteotomy versus primary total knee arthroplasty: a propensity score matching study.

BACKGROUND: The disparity in patient-reported outcomes between total knee arthroplasty (TKA) following high tibial osteotomy (HTO) and primary TKA has yet to be fully comprehended. This study aims to compare the patient-reported outcomes, radiological parameters and complication rates between TKA following HTO and primary TKA. METHODS: Sixty-five patients who underwent TKA following lateral closing-wedge HTO were compared to a matched group of primary TKA at postoperative 6-months and 1-year. Between-group confounders of age, gender, smoking status, Body Mass index, preoperative Numeric Rating Scale (NRS) pain in rest, Knee injury and Osteoarthritis Outcome Score-Physical function Shortform (KOOS-PS), EuroQol five-dimensional (EQ-5D) overall health score, and Oxford Knee Score (OKS) were balanced by propensity score matching. Patient-reported outcome measures were NRS pain in rest, KOOS-PS, EQ-5D overall health score, and OKS. Radiological parameters were femorotibial angle, medial proximal tibial angle, anatomical lateral distal femoral angle, posterior tibial slope, and patellar height assessed by Insall-Salvati ratio. The complication rates of TKA were compared between the two groups. The HTO survival time, the choice of staple removal before or during TKA in patients who underwent TKA following HTO patients, and the rate of patellar resurfacing were assessed. The p value < 0.0125 indicates statistical significance after Bonferroni correction. RESULTS: After propensity score matching, no significant between-group differences in the patient-reported outcome measures, radiographical parameters and complication rates were found (p > 0.0125). In the TKA following HTO group, with an average HTO survival time of 8.7 years, staples were removed before TKA in 46 patients (71%) and during TKA in 19 patients, and 11 cases (17%) had patella resurfacing. In the primary TKA group, 15 cases (23%) had patella resurfacing. CONCLUSION: The short-term assessment of TKA following HTO indicates outcomes similar to primary TKA. A previous HTO does not impact the early results of subsequent TKA, suggesting that the previous HTO has minimal influence on TKA outcomes. LEVEL OF EVIDENCE: III, cohort study.

Patient reported outcomes and patient experiences of immune checkpoint modulators for advanced or recurrent melanoma: a mixed methods study.

PURPOSE: Little is known about late and long-term patient-reported outcomes (PROs) of immune checkpoint modulators (ICMs) outside clinical trials. We conducted a cross-sectional, mixed-methods study to describe long-term PROs among advanced melanoma patients who began standard of care treatment with ICMs at least 1 year previously. METHODS: All participants completed the Functional Assessment of Cancer Therapy-Immune Checkpoint Modulator (FACT-ICM), assessing 46 immune-related side effects on a 5-point Likert scale, and a subset completed individual interviews. Descriptive statistics were computed for quantitative data and applied thematic analysis was used to examine qualitative data. RESULTS: Participants (N = 80) had a mean age of 67 years, and the majority were male (66%), non-Hispanic White (96%), and college graduates (61%). Single-agent nivolumab was the most common first (47%) and current/recent ICM (64%). On the FACT-ICM, 98% of participants reported at least one side effect, and 78% reported moderate or severe side effects. The most common moderate or severe side effects were aching joints (43%) and fatigue (38%). In interviews (n = 20), we identified five themes regarding patients' longer-term experiences after ICMs: lasting fatigue or decline in functioning, minimal side effects, manageable thyroid and pituitary dysfunction, skin conditions can be difficult to manage, and treating the cancer is worth the side effects. CONCLUSIONS: Nearly all patients reported side effects of ICMs at least 1 year after starting treatment. Our findings suggest that ICM side effect screening and management-especially for aching joints and fatigue-are indicated during long-term care of people living with advanced melanoma.

Missingness mechanisms and generalizability of patient reported outcome measures in colorectal cancer survivors - assessing the reasonableness of the "missing completely at random" assumption.

BACKGROUND: Patient-Reported Outcome Measures (PROM) provide important information, however, missing PROM data threaten the interpretability and generalizability of findings by introducing potential bias. This study aims to provide insight into missingness mechanisms and inform future researchers on generalizability and possible methodological solutions to overcome missing PROM data problems during data collection and statistical analyses. METHODS: We identified 10,236 colorectal cancer survivors (CRCs) above 18y, diagnosed between 2014 and 2018 through the Danish Clinical Registries. We invited a random 20% (2,097) to participate in a national survey in May 2023. We distributed reminder e-mails at day 10 and day 20, and compared Initial Responders (response day 0-9), Subsequent Responders (response day 10-28) and Non-responders (no response after 28 days) in demographic and cancer-related characteristics and PROM-scores using linear regression. RESULTS: Of the 2,097 CRCs, 1,188 responded (57%). Of these, 142 (7%) were excluded leaving 1,955 eligible CRCs. 628 (32%) were categorized as initial responders, 418 (21%) as subsequent responders, and 909 (47%) as non-responders. Differences in demographic and cancer-related characteristics between the three groups were minor and PROM-scores only marginally differed between initial and subsequent responders. CONCLUSION: In this study of long-term colorectal cancer survivors, we showed that initial responders, subsequent responders, and non-responders exhibit comparable demographic and cancer-related characteristics. Among respondents, Patient-Reported Outcome Measures were also similar, indicating generalizability. Assuming Patient-Reported Outcome Measures of subsequent responders represent answers by the non-responders (would they be available), it may be reasonable to judge the missingness mechanism as Missing Completely At Random.