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Abstract Background Tofacitinib is an oral, small-molecule JAK inhibitor for the treatment of ulcerative colitis (UC). Using a novel electronic reporting tool, we aimed to prospectively describe the onset of tofacitinib efficacy during induction therapy in a real-world study. Methods Patient-reported outcome data (PROs) including the simple clinical colitis activity index (SCCAI), PRO Measurement Identification Systems (PROMIS) measures, and adverse events were collected daily for the first 14 days and at day 28 and 56. Paired t tests and P for trend were utilized to compare changes in SCCAI over time. Bivariate analyses and logistic regression models were performed to describe response (SCCAI <5) and remission (SCCAI ≤2) by clinical factors. Results Of all included patients (n = 96), 67% had failed ≥2 biologics, and 61.5% were on concomitant steroids. Starting at day 3, PROs showed significant and persistent decline of the mean SCCAI (−1.1, P < 000.1) including significantly lower SCCAI subscores for stool frequency (−0.3; P < .003), bleeding (−0.3; P < .0002) and urgency (−0.2; P < .001). Steroid-free remission at day 14, 28, and 56 was achieved in 25%, 30.2%, and 29.2% of patients, respectively. Neither prior biologics nor endoscopic severity were independently predictive of response or remission in multivariate models. Numeric improvements in all PROMIS measures (anxiety, depression, social satisfaction) were seen through day 56. Rates of discontinuation due to adverse events were low. Conclusions In this prospective real-world study, tofacitinib resulted in a rapid and persistent improvement in UC disease activity PROs. The safety findings were consistent with the established safety profile of tofacitinib.

Collectively, studies from medical and surgical intensive care units (ICU) suggest that long-term outcomes are poor for patients who have spent significant time in an ICU. We sought to identify determinants of post-intensive care physical and mental health outcomes 6–12 months after injury. Adult trauma patients [ISS ≥9] admitted to one of three Level-1 trauma centers were interviewed 6–12 months post-injury to evaluate patient-reported outcomes. Patients requiring ICU admission ​≥ ​3 days (“ICU patients”) were compared with those who did not require ICU admission (“non-ICU patients”). Multivariable regression models were built to identify factors associated with poor outcomes among ICU survivors. 2407 patients were followed [598 (25%) ICU and 1809 (75%) non-ICU patients]. Among ICU patients, 506 (85%) reported physical or mental health symptoms. Of them, 265 (52%) had physical symptoms only, 15 (3%) had mental symptoms only, and 226 (45%) had both physical and mental symptoms. In adjusted analyses, compared to non-ICU patients, ICU patients were more likely to have new limitations for ADLs (OR ​= ​1.57; 95% CI ​= ​1.21, 2.03), and worse SF-12 mental (mean Δ ​= ​−1.43; 95% CI ​= ​−2.79, −0.09) and physical scores (mean Δ ​= ​−2.61; 95% CI ​= ​−3.93, −1.28). Age, female sex, Black race, lower education level, polytrauma, ventilator use, history of psychiatric illness, and delirium during ICU stay were associated with poor outcomes in the ICU-admitted group. Physical impairment and mental health symptoms following ICU stay are highly prevalent among injury survivors. Modifiable ICU-specific factors such as early liberation from ventilator support and prevention of delirium are potential targets for intervention. [Display omitted] •Delirium during an intensive care unit (ICU) stay is linked with long-term physical impairment in injury survivors who spent three or more days in the ICU.•The use of ventilators in the ICU is another factor associated with long-term physical impairment and mental health symptoms in these patients.•Delirium and ventilator use are potentially modifiable, suggesting opportunities for improving patient outcomes.•This knowledge can inform the development of intervention programs specifically targeting delirium and ventilator use to mitigate long-term impairments.

This article presents the current state of patient-reported outcome measures and explains new opportunities for leveraging the recent adoption of electronic health records to expand the application of patient-reported outcomes in both clinical care and comparative effectiveness research. Historic developments of patient-reported outcome, electronic health record, and comparative effectiveness research are analyzed in two dimensions: patient centeredness and digitization. We pose the question, “What needs to be standardized around the collection of patient-reported outcomes in electronic health records for comparative effectiveness research?” We identified three converging trends: the progression of patient-reported outcomes toward greater patient centeredness and electronic adaptation; the evolution of electronic health records into personalized and fully digitized solutions; and the shift toward patient-oriented comparative effectiveness research. Related to this convergence, we propose an architecture for patient-reported outcome standardization that could serve as a first step toward a more comprehensive integration of patient-reported outcomes with electronic health record for both practice and research. The science of patient-reported outcome measurement has matured sufficiently to be integrated routinely into electronic health records and other electronic health solutions to collect data on an ongoing basis for clinical care and comparative effectiveness research. Further efforts and ideally coordinated efforts from various stakeholders are needed to refine the details of the proposed framework for standardization.

Background We assessed the effectiveness, safety and patient-reported outcomes (PROs) of dimethyl fumarate (DMF) in real-world clinical practice in patients with multiple sclerosis (PwMS) from Argentina. Methods We conducted a multicenter ambispective cohort study in Argentina between September 2020 and March 2023. Changes in annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, magnetic resonance imaging (MRI), no evidence of disease activity (NEDA), PROs (depression, anxiety, fatigue, burden of treatment and quality of life), and safety data were collected at clinical visits performed every 6 months for at least 24 months. Results We included 161 PwMS (64% female). DMF treatment was associated with a significant reduction in ARR from baseline after 24 months of treatment (from 0.87 to 0.23, p  < 0.001). Disability progression was observed in 27.9% vs. 9.3% pre- and post-DMF, and disability improvement was found in 13% of patients from baseline to month 24. MRI activity was significantly reduced compared with baseline. Fatigue, depression, and quality of life scores were significantly improved from baseline to 24 months. Flushing was the most frequent adverse event reported in 19.2%. No significant reduction was observed in the hospitalization rate pre- and post-DMF (19.8% vs. 5.6%, p  = 0.32). During follow-up, 135 (83%) patients were relapse-free, 110 (68.3%) were MRI free activity (Gad + lesion) and 108 (67%) reached NEDA. Conclusions DMF significantly reduced disease activity in PwMS from Argentina with a good safety profile in real-world settings. A significant impact on the quality of life during follow-up was found.

Objective To collect and assess the extant empirical literature assessing disease-specific health-related quality of life (HRQOL) in pediatric transplant recipients using the PedsQL 3.0 Transplant Module (PedsQL-TM) assessment. Study design A systematic search and review procedure was conducted of research reporting use and results of the PedsQL-TM with samples of pediatric heart, liver, kidney, and lung transplantation. Searches were conducted in nine scholarly databases and two additional sources to identify unpublished research. Multiple reviewers screened studies meeting inclusion criteria in accordance with PRISMA guidelines. Results A final sample of nine studies reported findings for the PedsQL-TM with pediatric organ transplant recipients. Most studies relied on either kidney or liver transplant recipients from single pediatric transplant centers. Factor validity of the PedsQL-TM and inter-rater reliability (IRR) between patients and parents have not been adequately determined. Internal consistency reliability was found as acceptable or excellent across multiple studies. PedsQL-TM scores were found to vary with other HRQOL issues, yet few studies examined their association with medication adherence or posttransplant health outcomes. Conclusions With the goal of enhancing and sustaining HRQOL in pediatric organ transplant recipients, the need for a psychometrically valid and reliable measure of transplant-specific HRQOL is apparent. Research on the PedsQL-TM supports the promise of this measure although future efforts should be taken to examine measurement issues such as factor validity and IRR. Assessing transplant-specific HRQOL in these patients is paramount for their care and appropriate decision-making by patients, families, and the transplant team.

This study aimed to explore the correlation of frailty status with disease characteristics and patient-reported outcomes (PROs) in patients with acute exacerbations of chronic obstructive pulmonary disease (AECOPD) and determine the sensitivity and specificity of modified COPD PRO scale (mCOPD-PRO) for detecting frailty. This cross-sectional study surveyed 315 inpatients with AECOPD from a tertiary hospital in China from August 2022 to June 2023. Patient frailty and PROs were assessed using the validated FRAIL scale and mCOPD-PRO, respectively. Spearman's was used to assess the relevance of lung disease indicators commonly used in clinical practice, and ordinal logistic regression analyses were used to identify the variables associated with frailty status. The validity of mCOPD-PRO in discriminating frail or non-frail individuals was determined using the receiver operating characteristic curve. The participants (N=302, mean age 72.4±9.1 years) were predominantly males (73.2%). Among them, 43 (14.3%) patients were not frail, whereas 123 (40.7%) and 136 (45.0%) patients were pre-frail and frail, respectively. The FRAIL scale was moderately correlated with the mCOPD-PRO scores (Spearman's rank correlation coefficient [Rs]=0.52, <0.01) for all dimensions (Rs=0.43-0.49, <0.01). Patients residing in rural areas (odds ratio [OR], 1.67; 95% confidence interval [95% CI], 1.01-2.76) and with higher mCOPD-PRO scores (OR, 4.78; 95% CI, 2.75-8.32) were more likely to be frail. Physically active patients (OR, 0.42; 95% CI, 0.21-0.84) were less likely to be frail. In addition, mCOPD-PRO had good discriminate validity for detecting frailty (area under the curve=0.78), with a sensitivity and specificity of 84.6% and 60.8%, respectively. The optimal probability threshold for mCOPD-PRO was ≥1.52 points. In patients with AECOPD, frailty is closely related to PROs and disease characteristics. Additionally, the mCOPD-PRO score can distinguish well between frail and non-frail patients. Our findings provide support for interventions targeting frail populations with AECOPD.

Purpose Familial chylomicronemia syndrome (FCS) is a rare genetic disorder characterized by high triglyceride levels, significant disease burden, and negative impacts on health-related quality of life. This project aimed to create a PROMIS-based patient-reported outcome measure that represents valid and important concerns for patients with FCS. Methods We reviewed the literature and data from a previous qualitative study of FCS to identify key FCS symptoms and impacts, which were mapped to PROMIS domains to create a pool of eligible items. Candidate items were reduced per expert feedback and patients with FCS completed cognitive interviews to confirm content validity and measure content. Results Literature and qualitative data review identified ten key symptoms and 12 key impacts of FCS, including abdominal pain, fatigue, difficulty thinking, and worry about pancreatitis attacks. We identified 96 items primarily from PROMIS, supplemented with items from the Quality of Life in Neurological Disorders™ (Neuro-QoL™) and the Functional Assessment of Chronic Illness Therapy (FACIT) measurement systems. This pool was reduced to 32 candidate items, which were assessed via cognitive interviews with eight participants with FCS. Cognitive interview results and additional expert feedback led to the removal of four items and finalization of the PROMIS Profile v1.0—familial chylomicronemia syndrome (FCS) 28. Conclusions The PROMIS Profile v1.0—familial chylomicronemia syndrome (FCS) 28 provides strong content validity for assessing quality of life among patients with FCS. The benefits of PROMIS, including norm-referenced mean values for each measure, will facilitate comparison of patients with FCS to other clinical populations.

Introduction Remote symptom monitoring (RSM) allows patients to electronically self-report symptoms to their healthcare team for individual management. Clinical trials have demonstrated overarching benefits; however, little is known regarding patient-perceived benefits and limitations of RSM programs used during patient care. Methods This prospective qualitative study from December 2021 to May 2023 included patients with cancer participating in standard-of-care RSM at the University of Alabama at Birmingham (UAB) in Birmingham, AL, and the Univeristy of South Alabama (USA) Health Mitchell Cancer Institute (MCI) in Mobile, AL. Semi-structured interviews focused on patient experiences with and perceptions surrounding RSM participation. Interviews occurred over the phone, via digital videoconference, or in person, at the convenience of the patient. Grounded theory was used to conduct content coding and identify recurring themes and exemplary quotes using NVivo. Results Forty patients (20 UAB, 20 MCI) were interviewed. Participants were predominately female (87.5%), aged 41-65 (50%), and married (57.5%). Data is consistent with local demographics, comprising mainly White (72.5%) and 27.5% Black individuals. Three main themes emerged regarding perceived benefits of RSM: (1) Facilitation of Proactive Management, identifying the patient’s needs and intervening earlier to alleviate symptom burden; (2) Promotion of Symptom Self-Management, providing patients autonomy in their cancer care; and (3) Improvement in Patient-Healthcare Provider Relationships, fostering genuine connections based on healthcare team’s responses. However, participants also noted Perceived Limitations of RSM; particularly when support of symptom management was unnecessary, ineffective, or felt impersonal. Conclusion This study focused on patient experiences when utilizing a RSM program while undergoing treatment for cancer and found benefits to its implementation that extended beyond symptom management. At the same time, patients noted drawbacks experienced during RSM, which can help with future tailoring of RSM programs. Patient perceptions should be regularly assessed and highlighted for successful and sustained implementation. Plain language summary Cancer and its treatment can cause patients to experience many forms of symptoms. This can have a major effect on their overall well-being: physically, mentally, and emotionally. Early symptom detection and management has been shown to significantly improve the quality of life of patients with cancer. Remote symptom monitoring (RSM) allow patients to report their symptoms to their healthcare team and receive personalized feedback and management from them, without having to leave the comfort of their home. Although previous studies have shown the overarching benefits of implementing RSM during care, little is known regarding how patients perceive its implementation. This research team wanted to gain a better understanding of perceptions patients had, whether good or bad, of RSM’s implementation while they were actively undergoing cancer care. A total of 40 patients were interviewed with patients commenting on RSM’s ability to facilitate proactive management, promote symptom self-management, and improve patient-healthcare provider relationships. Patients also had feelings that under certain circumstances, RSM did have some limitations. This study was able to help identify some of the perceived benefits and limitations to RSM implementation. This feedback will not only help to ensure the successful implementation of future RSM programs but will also let patients know that they are heard and that their feedback is pivotal.

BackgroundEating Assessment Tool 10 is a symptom survey and has several advantages over other existing questionnaires. The instrument has also proven to be useful in establishing initial dysphagia symptom severity and to aid in measuring treatment outcomes. Due to its wide applicability, the instrument has been translated into several languages. The aim of the present study was to translate and validate the Kannada version of Eating Assessment Tool 10.Method88 individuals with dysphagia and equal number of healthy individuals filled the Kannada version of Eating Assessment Tool. Internal consistency and test–retest reproducibility were used for reliability testing. Validity was also established by comparing the scores of dysphagia patients and healthy controls.ResultsThe overall Cronbach’s α for the tool was 0.9 indicating a good internal consistency. The internal consistency of each of the items was also high, and ranged 0.88 to 0.9. Twenty patients filled the questionnaire after a span of 48 h, and the ICC coefficient was found to be 0.89 indicating a high reliability. The control group has significantly lower scores for all scales when compared to the dysphagia group [t(174) = 78.41, p < 0.001].ConclusionThe present study demonstrates that the Kannada version of EAT 10 has good internal consistency, test retest reliability, and concurrent validity. The results of the study also reveal that it is a reliable and valid tool for screening dysphagia population.

Exit interviews with patients who completed the Phase 3 VIVID-1 mirikizumab clinical trial for moderately-to-severely active Crohn's disease explored the content validity of bowel urgency, stool frequency, and abdominal pain patient-reported outcome measures and perceptions of meaningful within-patient change and remission in these key Crohn's disease symptoms. Cognitive debriefing explored patient understanding of the bowel urgency numeric rating scale (Urgency NRS), Crohn's Disease Activity Index: Stool Frequency (CDAI-SF) and Abdominal Pain (CDAI-AP), and patient global rating/impression of severity/change (PGRS/PGIC). Perceptions of meaningful change and remission were explored qualitatively. Transcripts were analyzed using directed content and framework analysis. Interviewed participants (  = 62; mean age 44.8 years, 55% female, mean 12.0 years since Crohn's disease diagnosis) were from the United States (  = 29), Czech Republic (  = 10), Poland (  = 8), Germany (  = 7), Canada (  = 4), Australia (  = 3), and the United Kingdom (  = 1). Participants understood the Urgency NRS, CDAI-SF, and CDAI-AP and could use them to rate their bowel urgency, stool frequency, and abdominal pain. Participants considered these symptoms when responding to the PGRS/PGIC. Meaningful change was described as symptom relief resulting in the ability to live daily life without pain or fear/need of rushing to the toilet. Most participants agreed with a proposed remission definition of ≤3 type 6/7 bowel movements and None/Mild abdominal pain. The Urgency NRS, CDAI-SF, and CDAI-AP are content-valid patient-reported outcome measures in Crohn's disease. The PGRS/PGIC are conceptually related global assessments of bowel urgency, stool frequency, and abdominal pain. Patients considered reduction in these symptoms as meaningful and remission.