DOI: 10.6084/m9.figshare.29349536.v1
title: Real-world clinical outcomes of patients with moderate-to-severe rheumatoid arthritis initiating upadacitinib in the United Kingdom: Final analysis from a prospective observational cohort study (ENDEAVOUR)

description: Upadacitinib is recommended by National Institute for Health and Care Excellence in the UK in adults with moderate-to-severe rheumatoid arthritis (RA). This observational study assessed real-world clinical outcomes and patient-reported outcomes (PROs) in patients receiving upadacitinib for 6 months in the UK. Patients from 14 centres in whom the decision to initiate upadacitinib had already been made were enrolled. Baseline data were retrospectively collected from patient records. Clinician-reported data were collected at routine clinic visits 3 and 6 months after upadacitinib initiation. Patient-reported data were collected directly from patients using an app (electronic PROs, ePROs). The primary end-point was proportion of patients achieving clinical remission (DAS28 CRP &lt;2.6) after 6 months of upadacitinib. Data are available for 63 patients at all three datapoints and for 53 patients for the primary end-point. At 6 months, 40% (21/53) of patients achieved clinical remission and 21% (11/53) achieved low disease activity. Response was seen at 3 months for all efficacy end-points. ePROs allowed the capture of early patient-reported data which demonstrated clinically important improvements in pain and fatigue within 10 days and other PROs within 2 months. Improvements were also seen in metrics of activity, work and quality of life (QOL). Patients in ENDEAVOUR showed similar early effectiveness with upadacitinib to that observed in clinical trials. Use of ePROs demonstrated rapid onset of action and meaningful improvements in QOL providing a potential opportunity to reduce outpatient visits for early responders, thus reducing the burden on rheumatology services. The ENDEAVOUR study looked at the impact of treatment with upadacitinib in people with moderate-to-severe rheumatoid arthritis (RA) in the UK. The study enrolled people for whom the decision to start treatment with upadacitinib had already been made. It assessed the impact of upadacitinib at the start of treatment (using medical records) and at routine outpatient hospital visits, 3 and 6 months after the start of treatment (using information reported by their doctor). Doctors used the 28-joint Disease Activity Score (DAS28) which takes into consideration the number of tender and swollen joints, patient global assessment of RA and markers of inflammation to define response to treatment, with a score of &lt;2.6 indicating clinical remission and a score of 2.6-3.2 indicating low disease activity. People receiving upadacitinib showed a response to treatment at 3 months and at 6 months, 40% (21/53) of patients had achieved clinical remission and 21% (11/53) had achieved low disease activity. Other measures of effectiveness showed similar results. People with RA also used an app to log their own experience of the impact of upadacitinib on pain, tiredness (fatigue), overall health, disability, ability to work and quality of life (QOL). Information directly recorded by people with RA using the app showed clinically important improvements in fatigue within 7 days, pain within 10 days, overall health, mood and disability within 1 month and their ability to work and QOL within 3 months. The effectiveness results of ENDEAVOUR were similar to that seen in previous clinical trials of upadacitinib. Use of an app to collect patient experience showed rapid onset of action and meaningful improvements in symptoms and QOL with upadacitinib. This has the potential to reduce the burden on rheumatology services by reducing outpatient hospital visits for those people who respond quickly to treatment with upadacitinib.