Journal Article

POS1243 ASSOCIATION OF SJÖGREN’S DISEASE ACTIVITY (ClinESSDAI) AND SYMPTOM BURDEN (ESSPRI) WITH PATIENT-REPORTED OUTCOME MEASURES

Villasis-Keever, A., Hughes, M., Sbarigia, U., Alemao, E., Barton, V., Gottenberg, J. E., Massey, N., Weatherby, S., Zazzetti, F., Borsi, A., Dhatt, H., Sheahan, A., Noel, W.

2024

Overview

Background:Clinical EULAR Sjögren’s syndrome disease activity index (clinESSDAI) and EULAR Sjögren’s syndrome patient-reported index (ESSPRI) are commonly utilized clinical trial endpoints, reflecting disease activity and patient-reported symptomatology, respectively. The relationship between the thresholds for disease activity as measured by these clinical outcome assessments (COAs) reflecting physician and patient perspectives respectively, and their correlation with other patient-reported outcome measures (PROMs) remains to be elucidated.Objectives:This study aimed to evaluate the relationship between COAs and PROMs in Sjögren’s disease.Methods:Data were drawn from the Adelphi Real World Sjögren’s (SjD) Disease Specific Programme™, a cross-sectional survey, with retrospective data collection, of rheumatologists and their next six consecutively consulting patients with SjD in France, Germany, Italy, Spain, and the United States in June – October 2018. Rheumatologists reported on patients’ clinical characteristics, including subjective disease severity, and systemic involvement for the calculation of a clinESSDAI proxy score. Patients voluntarily completed questionnaires including components for ESSPRI proxy score calculations and other PROMs including EQ-5D-3L (US tariff) (0-1; MCID = 0.04) [1], EuroQoL Visual Analogue Scale (EQ-VAS), Functional Assessment of Chronic Illness Therapy – Fatigue Scale (FACIT-F) (0-52; MCID = 3-6 points) [2], and work productivity and activity impairment questionnaire (WPAI). Associations between clinESSDAI and ESSPRI, and PROM scores and subjective disease severity were examined through univariate linear and logistic regression modelling. Mean differences in PROM scores were determined by two-sample t-tests and compared with published values for minimal clinically important differences (MCID) [1,2]. Statistical significance was p<0.05 for all tests, and the published thresholds utilized for the purposes of this study were as follows: clinESSDAI (low <5, moderate 5-13, high >13); ESSPRI (<5, ≥5).Results:Rheumatologists (n=319) reported data on 1,879 SjD patients. All PROMs were significantly associated with both clinESSDAI and ESSPRI (Table 1). Mean EQ-5D-3L utility scores were 0.79, 0.74, and 0.66 for low, moderate, and high clinESSDAI respectively; and 0.82 and 0.64 for ESSPRI <5 and ≥5, respectively. Mean FACIT-F scores were 33.96, 30.76, and 29.10 for low, moderate, and high clinESSDAI respectively; and 36.19 and 24.62 for ESSPRI <5 and ≥5, respectively. All mean differences within clinESSDAI and ESSPRI groups were statistically significant.Conclusion:ClinESSDAI and ESSPRI are significantly associated with PROMs, suggesting a relationship with systemic disease activity and patient-reported symptomatology. ESSPRI’s greater incremental association with other PROMs suggests that patient experience focuses more on non-systemic aspects of disease, implying ESSPRI may be more closely associated with other PROMs. However, difference in COAs and their scales (clinESSDAI: 0-123; ESSPRI: 0-10) may impact the magnitude of the observed association. Existing clinESSDAI disease activity thresholds are generally consistent with MCIDs in low-moderate disease activity; however, mean differences beyond MCIDs at the moderate-high threshold suggest a broad range in outcome scores. Similarly, difference in PROM means beyond the MCID suggests that ESSPRI scores <5 and ≥5 may be too broadly grouped to comprehensively reflect differences in patient outcomes. These findings suggest that both clinESSDAI and ESSPRI require contextualised interpretation in clinical trial results and indicate that current published thresholds understate the difference in EQ-5D-3L and FACIT-F experienced by patients.REFERENCES:[1] Luo N, et al. Medical Care. 2010; 48(4):365-371.[2] Nordin Å, et al. BMC Med Res Methodol. 2016;16:62.Acknowledgements:Medical writing support was provided by Panita Maturavongsadit, PhD, of Lumanity Communications Inc., and was funded by Janssen Global Services, LLC.Disclosure of Interests:Jacques-Eric Gottenberg AbbVie, Janssen, BMS, UCB, MSD, Novartis, Pfizer, Gilead, Galapagos, Lilly, Sanofi, Nicola Massey Adelphi Real World, Megan Hughes Adelphi Real World, Victoria Barton Adelphi Real World, Sarah Weatherby Adelphi Real World, Federico Zazzetti May own shares/stock options of Johnson & Johnson, Johson & Johnson Innovative Medicine, Andras Borsi May own shares/stock options of Johnson & Johnson, Johnson & Johnson Innovative Medicine, Wim Noel May own shares/stock options of Johnson & Johnson, Johson & Johnson Innovative Medicine, Evo Alemao May own shares/stock options of Johnson & Johnson, Johson & Johnson Innovative Medicine, Harman Dhatt May own shares/stock options of Johnson & Johnson, Johnson & Johnson Innovative Medicine, Angelina Villasis-Keever May own shares/stock options of Johnson & Johnson, Johnson & Johnson Innovative Medicine, Anna Sheahan May own shares/stock options of Johnson & Johnson, Johnson & Johnson Innovative Medicine, Urbano Sbarigia May own shares/stock options of Johnson & Johnson, Johnson & Johnson Innovative Medicine.

• Publisher: BMJ Publishing Group Ltd and European League Against Rheumatism,Elsevier B.V,Elsevier Limited
• Subject: Clinical outcomes / Clinical trials / Data collection / Medicine / Observational studies/ registry / Patient Reported Outcome Measures / Patients / Quality of life / Scientific Abstracts / Sjogren's syndrome / Statistical analysis / Stock options