BACKGROUND: Systemic sclerosis (SSc) is an autoimmune, connective tissue disease characterized by vasculopathy and fibrosis of the skin and internal organs. METHODS: We randomized 15 participants with early diffuse cutaneous SSc to tofacitinib 5 mg twice a day or matching placebo in a phase I/II double-blind, placebo-controlled trial. The primary outcome measure was safety and tolerability at or before week 24. To understand the changes in gene expression associated with tofacitinib treatment in each skin cell population, we compared single-cell gene expression in punch skin biopsies obtained at baseline and 6 weeks following the initiation of treatment. RESULTS: Tofacitinib was well tolerated; no participants experienced grade 3 or higher adverse events before or at week 24. Trends in efficacy outcome measures favored tofacitnib. Baseline gene expression in fibroblast and keratinocyte subpopulations indicated IFN-activated gene expression. Tofacitinib inhibited IFN-regulated gene expression in SFRP2/DPP4 fibroblasts (progenitors of myofibroblasts) and in MYOC/CCL19, representing adventitial fibroblasts ( P < 0.05), as well as in the basal and keratinized layers of the epidermis. Gene expression in macrophages and DCs indicated inhibition of STAT3 by tofacitinib ( P < 0.05). No clinically meaningful inhibition of T cells and endothelial cells in the skin tissue was observed. CONCLUSION: These results indicate that mesenchymal and epithelial cells of a target organ in SSc, not the infiltrating lymphocytes, may be the primary focus for therapeutic effects of a Janus kinase inhibitor. TRIAL REGISTRATION: ClinicalTrials.gov NCT03274076. FUNDING: Pfizer, NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) R01 AR070470, NIH/NIAMS K24 AR063120, Taubman Medical Research Institute and NIH P30 AR075043, and NIH/NIAMS K01 AR072129.

Remote patient monitoring (RPM) increased exponentially during the COVID-19 pandemic. RPM programs commonly incorporate tools to capture and transmit health-relevant data from the home to the clinical space to augment the clinical decision-making process of health care providers. Given the potential to improve patient health outcomes, health care systems around the world are actively engaged in fashioning, implementing, and exploring the outcomes of various RPM program models. However, new challenges to health care systems include increasing RPM program enrollment, optimizing condition-specific RPM programs to best address the needs of specific patient groups, integrating new RPM-derived data streams into existing IT infrastructure, overcoming limited availability of desired remote monitoring technologies, and quantifying the health outcomes produced by RPM use. Herein, we identify stakeholders for RPM in the United States, summarize the landscape of RPM tools available for chronic disease management, discuss the current regulatory environment, delve into the benefits and challenges of integrating these tools into clinical practice, summarize aspects of coverage and reimbursement, and examine the knowledge and policy gaps regarding sustained use of RPM in clinical practice, along with associated opportunities.

Objective: Gastrointestinal (GI) involvement is common in patients with systemic sclerosis (SSc; scleroderma). The Patient‐Reported Outcomes Measurement Information System (PROMIS) GI symptom item bank captures upper and lower GI symptoms (reflux, disrupted swallowing, nausea/vomiting, belly pain, gas/bloating/flatulence, diarrhea, constipation, and fecal incontinence). The objective of this study was to evaluate the construct validity of the PROMIS GI bank in SSc.Methods: A total of 167 patients with SSc were administered the PROMIS GI bank and the University of California, Los Angeles, Scleroderma Clinical Trials Consortium Gastrointestinal Scale (GIT 2.0) instrument. GIT 2.0 is a multi‐item instrument that measures SSc‐associated GI symptoms. Product‐moment correlations and a multitrait–multimethod analysis of the PROMIS GI scales with the GIT 2.0 symptom scales were used to evaluate convergent and discriminant validity.Results: Patients with SSc GI involvement had PROMIS GI scale scores 0.2–0.7 SD worse than the US general population. Correlations among scales measuring the same domains for the PROMIS GI and GIT 2.0 measures were large, ranging from 0.61 to 0.87 (average r = 0.77). The average correlation between different symptom scales was 0.22, supporting discriminant validity.Conclusion: This study provides support for the construct validity of the PROMIS GI scales in SSc. Future research is needed to assess the responsiveness to change of these scales in patients with SSc.

Interstitial lung disease (ILD) is the leading cause of mortality in patients with systemic sclerosis (SSc). Risk of developing progressive ILD is highest among patients with diffuse cutaneous dise...