William Benjamin Nowell

Objective: To evaluate rheumatoid arthritis (RA) symptoms and disease activity among patients initiating new treatment, we examined clinical, patient‐reported outcome (PRO), and wearable biosensor data, and we explored longitudinal associations among these three data types. Methods: Patients with RA with moderate‐to‐high disease activity (Clinical Disease Activity Index (CDAI) >10) and prescribed upadacitinib or adalimumab were enrolled (March 2021–October 2022) during in‐office visits at 28 community rheumatology clinics in the United States and were observed for ≥12 weeks. At‐home data collection included disease activity and symptom measures from the Patient‐Reported Outcomes Measurement Information System (PROMIS) using the PatientSpot app. Biosensor activity data were collected with a wrist‐worn Fitbit device. Changes over time and associations among data types were analyzed with Pearson R correlations and linear regression models. Results: Of 299 patients recruited, 204 (68%) started upadacitinib or adalimumab within 30 days; 150 (50%) provided clinical and PRO data, and of the main study population, 95 patients (63%) provided biosensor data. Mean CDAI improved 13 units and PROMIS Pain Interference, Fatigue, and Sleep Disturbance scores improved by 4 to 5 units from baseline to follow‐up. Activity improved for patients considered sedentary at baseline. Moderate correlations between CDAI and PROs ranged from 0.38 to 0.56. Linear regression model showed that changes in PROs explained 52% of the variability in change in CDAI. Conclusion: Treatment response is observed across diverse data types and tracking RA disease activity outside of the clinic for patients initiating new treatments may be useful in evaluating disease status and optimal changes in therapy, even in the absence of physician‐derived measures.

Objective: People with rheumatoid arthritis (RA) are at increased risk of serious infection, but less is known about nonserious infections. Our prospective cohort study evaluated associations between medications for RA and the risk of nonserious infections. Methods: We remotely recruited adults with RA in a community rheumatology practice‐based research network. Participants joined the ArthritisPower Registry (now PatientSpot) and completed a baseline and up to six monthly follow‐up surveys. Using data from consecutive monthly surveys, we assessed associations between medication use at the prior survey and infection report at the subsequent survey, adjusting for confounders. Results: We recruited 351 people with RA (mean age 60, 84% female) who reported 439 infections (330 infections per 100 patient‐years). Associations between medication use and infection were assessed among 1,075 qualifying observations with 289 (27%) total infections and 146 (14%) infections with health care encounters or antibiotic use. Compared to those receiving conventional synthetic disease‐modifying antirheumatic drugs who were biologic or JAK inhibitor (JAKi) naïve, current biologic or JAKi use was not associated with either infection outcome. Infections were numerically more common with glucocorticoids ≥10 mg/day (odds ratio 1.94, 95% confidence interval 0.89–4.24). Season, previous infection, poorer function, and rural residence were significantly associated with one or both infection outcomes. Conclusion: Biologics and JAKi were not associated with greater risk for nonserious infections compared to conventional therapies. Given that nonserious infection risk may be due more to exposures and general health status, future studies should assess whether the common practice of interrupting medications in people with infection improves or worsens outcomes.

Objective: The objective of this study was to develop and evaluate a new patient‐facing tool to facilitate patient self‐examination of joints as a complement to telehealth during the management of rheumatoid arthritis (RA). Methods: Using a community‐based rheumatology network, we evaluated the efficacy of an in‐app cartoon‐based joint examination module that guides patients through self‐conducted physical examinations by comparing patient‐derived joint counts with those recorded by physicians during in‐office visits. A total of 100 patients were enrolled from outpatient rheumatology clinics. Scatter plots and regression models were used to analyze the data. Results: Overall, rheumatologists recorded fewer tender and swollen joints than patients with RA. The correlation between patient and provider assessments was higher for the tender joint count (r = 0.5, P < 0.0001) than for the swollen joint count (r = 0.33, P < 0.001). In terms of disease activity, patients’ joint counts led to composite disease activity scores that were accurate in predicting low disease activity (LDA) or remission but were likely to overestimate active disease compared to physicians’ assessments, with a positive predictive value of a patient assessing that they were in LDA at 95% with specificity of 94%, whereas the negative predictive value was only 50% with a sensitivity of 54%. Conclusion: As remote therapeutic monitoring becomes increasingly used in health care, this study has relevance for the future expansion and application of telehealth procedures in rheumatology, particularly in providing insights regarding the types of patient training that might be required and the choice of patients in whom RA disease activity remote monitoring will have the highest utility.

Introduction This study aimed to explore the experience of living with amyotrophic lateral sclerosis (ALS) and to develop a conceptual model for this rare disease. Methods Concept elicitation interviews were conducted (January–September 2024) with people living with ALS (PLwALS; n = 31), caregivers (n = 20), and clinicians (n = 10). Qualitative data were analyzed separately to develop a conceptualization of the experience of living with ALS. Concept saturation was assessed every 5–6 interviews, and a conceptual model was developed. Results The mean age of PLwALS was 42.4 years (standard deviation [SD] 11.5), 81% were female, 84% were white, and 23% had SOD1-ALS. The mean time since diagnosis was 4.6 years (SD 4.2); mean normed Rasch Overall ALS Disability Scale score was 76 (SD 17.16). Signs, symptoms, and functions reported during PLwALS interviews included neuromuscular, bulbar, speech, neurocognitive (e.g., memory issues), and a range of physical functioning issues (e.g., motor coordination). PLwALS also reported impacts on a range of activities and psychosocial interactions (e.g., eating, depressed mood, and relationships), alongside management strategies they employed. Interviews with caregivers and clinicians supported findings from the PLwALS interviews. Caregivers also identified signs such as drooling/excess salivation, and impacts related to ALS management (e.g., need for writing aids). Clinicians additionally considered loss of speech and neurocognitive signs (e.g., behavior/personality change) as ALS clinical manifestations. Concept saturation was reached, and a consolidated, comprehensive conceptual model was developed. Conclusion This research provides a holistic understanding of the experience of living with ALS and is the first conceptual model based on in-depth concept elicitation interviews. The findings highlight the range of signs, symptoms, and impacts that PLwALS experience, emphasizing its serious humanistic impact and high unmet need, and will help to guide patient-centric evaluation of clinical outcome assessments in future ALS studies.

Objectives: Patients with RA are at an increased risk for serious infections, but less is known about more common, non-serious infections. We aimed to assess the frequency and characteristics of non-serious infections and their effect on medication interruptions, quality of life, and disease flares. Methods: We remotely recruited adults with RA from July 2022 to July 2023 through a community rheumatology practice–based research network. Participants joined the ArthritisPower Registry (now PatientSpot) and completed a baseline survey and up to six monthly follow-up surveys, focused on patient-reported outcomes (PROs), infections, medication interruptions, and disease flares. The impact of infections was evaluated by comparing survey measures in those with vs without an infection. Results: We recruited 351 patients with RA who contributed 1674 monthly observations. Patients reported 523 infections (31% of observations), most frequently upper respiratory infections. Although few infections led to emergency department visits or hospitalizations, infections frequently led to missed work (37%) or RA treatment interruptions (26%). RA treatment interruptions were more common in those with vs without health-care encounters or antibiotic use (38% vs 12%) and in more severe vs less severe infections (47% vs 20%). Infections were associated with worse scores for fatigue but not function, depression, or social participation. Frequency of disease flares was greater in those with an infection who interrupted RA treatment, especially in patients receiving tumor necrosis factor (TNF) inhibitors. Conclusion: Infections are common in patients with RA and frequently lead to medication interruptions. These infections are associated with greater fatigue and impaired work productivity and may contribute to more disease flares.

Amyotrophic lateral sclerosis (ALS) is a motor neuron disease that leads to significant morbidity and mortality, but with substantial variability in the rate of progression [1,2]. A common primary outcome measure used in ALS clinical studies is the ALSFRS-R. Change in this measure may also be used to stratify fast and slow progressors, typically with the assumption of linear decline. Reported limitations of the ALSFRS-R and the assumption of linear slope may hinder novel therapy development. Here we use two distinct populations from a clinical trial database (PRO-ACT) and a natural history registry (ALS TDI) to show that ALSFRS-R fine and gross motor subdomains are more sensitive to disease progression than total score and bulbar and respiratory subdomain scores. We also present a novel non-linear method for defining fast and slow progressors. Together these results may improve demonstration of outcomes in ALS interventional studies using the ALSFRS-R.

Objective: We sought to identify (1) what types of information US adults with rheumatic and musculoskeletal diseases (RMD) perceive as most important to know about their disease, and (2) what functions they would use in an RMD-specific smartphone app.Methods: Nominal groups with patients with RMD were conducted using online tools to generate a list of needed educational topics. Based on nominal group results, a survey with final educational items was administered online, along with questions about desired functions of a smartphone app for RMD and wearable use, to patients within a large community rheumatology practice–based research network and the PatientSpot registry. Chi-square tests and multivariate regression models were used to determine differences in priorities between groups of respondents with rheumatic inflammatory conditions (RICs) and osteoarthritis (OA), and possible associations.Results: At least 80% of respondents considered finding a rheumatologist, understanding tests and medications, and quickly recognizing and communicating symptoms to doctors as extremely important educational topics. The highest-ranked topic for both RIC and OA groups was “knowing when the medication is not working.” The app functions that most respondents considered useful were viewing laboratory results, recording symptoms to share with their rheumatology provider, and recording symptoms (eg, pain, fatigue) or disease flares for health tracking over time. Approximately one-third of respondents owned and regularly used a wearable activity tracker.Conclusion: People with RMD prioritized information about laboratory test results, medications, and disease and symptom monitoring, which can be used to create educational and digital tools that support patients during their disease journey.

Objective: We performed a scoping review of the relevant literature on home‐based telehealth in rheumatology to understand its appropriate application in rheumatology practice.Methods: We searched the Cochrane Library, PubMed, Web of Science, and scientific meeting abstracts to identify articles that specifically addressed telehealth suitability, barriers to telehealth, patient‐reported outcomes (PROs) collected in telehealth settings, and telehealth satisfaction. From the initial search of 4,882 studies, 23 reports were included. In addition, 10 abstracts were also eligible for analysis, resulting in a total of 33 articles: 2 randomized clinical trials, 9 prospective cohort studies, and 22 retrospective studies.Results: We found that triage appointments or predictive models could be helpful in selecting patients for telehealth and that telehealth interventions were appropriate for follow‐up of patients with systemic lupus erythematosus and inflammatory arthritis, but that conducting new patient visits over telehealth was not ideal. Barriers to telehealth include patient factors (age, technology access) and need for physician/process factors (eg, physical examinations). PROs collected in regular practice can be incorporated into telehealth. Several small, single‐center studies suggest that telehealth does not lead to negative outcomes compared with in‐person visits, and overall, patients report high patient satisfaction with telehealth. In several scenarios, home‐based telehealth was equivalent to in‐person visits with regard to patient outcomes and satisfaction.Conclusion: The widespread potential of telehealth to manage and deliver care for people with rheumatic disease is significant. As such, further research in the form of randomized controlled trials can help contribute to growing evidence that shapes telehealth implementation for patients with rheumatic diseases.