Abstract Background/Aims ENDEAVOUR was an observational study which assessed clinical and patient-reported outcomes (PROs) in patients with moderate-to-severe rheumatoid arthritis (RA) receiving upadacitinib in 14 UK centres. Primary results have been published, with response seen at 3 months for all efficacy endpoints. Electronic PROs (ePROs) collected directly from patients using an app demonstrated clinically important improvements in fatigue within 7 days and pain within 10 days, with other ePROs improving within 2 months. This analysis was conducted to assess whether clinician review of ePROs might offer a practical option to reduce NHS Rheumatology outpatient appointments. Methods Patient-reported data were collected in real time directly from patients using an app with clinician-reported data collected at routine clinic visits 3 and 6 months after upadacitinib initiation. Post-hoc statistical analyses were conducted to determine whether ePROs correlated with Physician Global Assessment of Disease Activity (PGA) at 3 months. Results Data were available for 63 patients at baseline with 58 having PGA at 3 months. Mean (standard deviation) PGA fell from 6.4 (2.1) at baseline to 3.8 (2.6) at 3 months. Patient persistence with the app to record ePROs was high; 78.8% of patients were persistent app users defined as recording data on at least 75% of available opportunities. Data for ePROs and PGA at 3 months were recorded for 39 patients for pain Visual Analogue Score (VAS) and 38 for 5-level EuroQol-5 dimensions questionnaire (EQ-5D-5L), fatigue VAS, Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F), Health Assessment Questionnaire Disability Index (HAQ-DI), HAQ-DI pain VAS and Patient Health Questionnaire (PHQ-2). There were significant correlations between ePROs (EQ-5D-5L, FACIT-F, fatigue VAS, HAQ-DI, HAQ-DI pain VAS, pain VAS, PHQ-2) and PGA at 3 months. EQ-5D, HAQ-DI, HAQ-DI pain VAS and VAS pain were most closely related to PGA with correlation coefficients (r) of -0.7237 (95% CI -0.8473 to -0.5256), 0.6638 (0.4368 to 0.8113), 0.7091 (0.5036 to 0.8387) and 0.6943 (0.4851 to 0.8284), respectively, p 0.0001 for all. This work is promising, although limited by small patient numbers since 3-month data was only available for both ePROs and PGA for 38-39 patients. Conclusion Upadacitinib is efficacious as recorded by both ePROs and clinicians. This analysis demonstrated the utility of using an app to record real time PRO data in patients with RA. Patient persistence with the app was excellent, together with significant correlations between ePROs and physician-reported data, providing a viable alternative to collecting multiple PRO data in clinic. Clinician review of ePRO data could allow Patient Initiated Follow-up (PIFU) for selected patients rather than a routine out-patient clinic appointment at 3 months, helping to reduce the burden on Rheumatology services by reducing the number of appointments. Disclosure J. Galloway: Honoraria; Has received honoraria or speaker fees or advisory board work from AbbVie, Alfasigma, Lilly, Pfizer, UCB, Takeda and Janssen. Grants/research support; Has received research grants from AbbVie, GSK and Pfizer. M. Bukhari: Honoraria; Has received honoraria for speaking and attended advisory boards with Bristol-Myers Squib, UCB celltech, Roche/Chugai, Pfizer, Abbvie, Merck, Mennarini, Sanofi-aventis, Eli-Lilly, Janssen, Amgen, Novartis, Galapagos, Gilead and Theramex, Has received honoraria from educational groups revalidaid and TREG consultants. Other; Has been sponsored to attend regional, national and international meetings by UCB celltech, Roche/Chugai, Pfizer, Abbvie, Merck, Mennarini, Janssen, Bristol-Myers Squib, Novartis and Eli-lilly. N. Goodson: None. C. Hansell: Corporate appointments; Is an employee of AbbVie. Shareholder/stock ownership; May hold AbbVie stock or stock options. T. Niblock: Corporate appointments; Is an employee of AbbVie. Shareholder/stock ownership; May hold AbbVie stock or stock options.
Galloway et al. (Wed,) studied this question.