scholarly journals Sensitivity to change of the Health Assessment Questionnaire (HAQ) and other clinical and health status measures in rheumatoid arthritis results of short-term clinical trials and observational studies versus long-term observational studies

1992 ◽  
Vol 5 (3) ◽  
pp. 130-136 ◽  
Author(s):  
Donna J. Hawley ◽  
Frederick Wolfe
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trials ◽  
Observational Studies ◽  
Health Assessment Questionnaire ◽  
Health Assessment ◽  
Sensitivity To Change ◽  
Short Term ◽  
Health Status Measures ◽  
Assessment Questionnaire

scholarly journals SAT0125 ASSOCIATION BETWEEN CHANGE IN HEALTH ASSESSMENT QUESTIONNAIRE DISABILITY INDEX AND TREATMENT RESPONSE IN PATIENTS WITH RHEUMATOID ARTHRITIS IN TOCILIZUMAB CLINICAL TRIALS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 998.2-998
Author(s):  
S. Unizony ◽  
J. Dang ◽  
J. Han ◽  
M. Michalska ◽  
J. H. Best
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trials ◽  
Disease Activity ◽  
Physical Function ◽  
Health Assessment Questionnaire ◽  
Health Assessment ◽  
Phase Iii ◽  
Link Type ◽  
Assessment Questionnaire ◽  
Questionnaire Disability

Background:The efficacy and safety of intravenous (IV) and subcutaneous (SC) tocilizumab (TCZ) in combination with conventional synthetic disease-modifying antirheumatic drugs (csDMARDs) and as monotherapy in patients with rheumatoid arthritis (RA) has been demonstrated in large clinical trials and real-world data studies. The Health Assessment Questionnaire Disability Index (HAQ-DI) is commonly used to assess physical function in patients with RA. While HAQ-DI outcomes at Week 24 in TCZ clinical trials have been reported, outcomes at Week 12 and results stratified by treatment response categories at Weeks 12 and 24 have not been previously described.Objectives:To report the association between change in HAQ-DI from baseline to Weeks 12 and 24 and Disease Activity Score in 28 joints (DAS28) response categories in patients who received TCZ or comparators in TCZ clinical trials.Methods:Data from patients with active RA who received TCZ or a comparator from 6 Phase III or IV TCZ-IV studies (OPTION [NCT00106548], RADIATE [NCT00106522], TOWARD [NCT00106574] LITHE [NCT00109408], ACT-RAY [NCT00810199] and ADACTA [NCT01119859]) and 1 Phase III TCZ-SC study (BREVACTA [NCT01232569]) were analyzed. Mean change in HAQ-DI score at Weeks 12 and 24 was assessed in patients stratified by DAS28 disease activity level (DAS28 < 2.6 [remission], DAS28 ≥ 2.6 to ≤ 3.2 [low disease activity; LDA], DAS28 > 3.2 to ≤ 5.1 [moderate disease activity; MDA], DAS28 > 5.1 [high disease activity; HDA] at Weeks 12 and 24. The adjusted least squares mean (LSM) change from baseline was estimated using a mixed model with repeated measures, including region (North America vs non-North America), RA duration (> 2 years vs ≤ 2 years), baseline HAQ-DI and DAS28, treatment, visit, visit by treatment and visit by baseline HAQ-DI.Results:Data from 5051 patients were included. Across all studies, the mean duration of RA ranged from 6.3 to 12.6 years. At baseline, patients had severe RA with a mean DAS28 ≥ 6.3; baseline HAQ-DI was ≥ 1.5. At Week 12, patients who achieved remission or LDA had greater improvements in HAQ-DI than those in MDA or HDA (Figure 1). Results were similar at Week 24 (Figure 2). Among patients who received TCZ and achieved remission or LDA, mean improvement in HAQ-DI was ≥ 0.65 and ≥ 0.44, respectively, at Week 12 (Figure 1) and ≥ 0.48 and ≥ 0.43 at Week 24 (Figure 2). Mean changes in HAQ-DI were similar between patients who received TCZ-IV in combination with MTX or as monotherapy (ACT-RAY) and in those who received TCZ-IV or ADA as monotherapy (ADACTA).Conclusion:Patients with long-standing, severe RA who received IV or SC TCZ as monotherapy or in combination with csDMARDs had improvement in physical function and disease activity at Week 12 that was maintained at Week 24. Overall, across all the trials, response to treatment was associated with improvement in physical function.Acknowledgments :This study was sponsored by Genentech, Inc. Support for third-party writing assistance, furnished by Health Interactions, Inc, was provided by Genentech, Inc.Disclosure of Interests: :Sebastian Unizony Grant/research support from: Genentech, Inc., Joseph Dang Shareholder of: Genentech, Inc., Employee of: Genentech, Inc., Jian Han Shareholder of: Genentech, Inc., Employee of: Genentech, Inc., Margaret Michalska Shareholder of: Genentech, Inc., Employee of: Genentech, Inc., Jennie H. Best Shareholder of: Genentech, Inc., Employee of: Genentech, Inc.

scholarly journals AB0140 A HIGH-CONFIDENCE DEFINITION OF THERAPEUTIC RESPONSE IN RHEUMATOID ARTHRITIS USING A MONTE CARLO SIMULATION APPROACH

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 1097.2-1098
Author(s):  
V. Strand ◽  
S. Cohen ◽  
L. Zhang ◽  
T. Mellors ◽  
A. Jones ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Monte Carlo ◽  
Disease Activity ◽  
Health Assessment Questionnaire ◽  
Health Assessment ◽  
High Confidence ◽  
Response Status ◽  
Definition Of ◽  
Fidelity Score ◽  
Assessment Questionnaire

Background:Therapy choice and therapy change depend on the ability to accurately assess patients’ disease activity. The clinical assessments used to evaluate treatment response in rheumatoid arthritis have inherent variability, normally considered as measurement error, intra-observer variability or within subject variability. Each contribute to variability in deriving response status as defined by composite measures such as the ACR or EULAR criteria, particularly when a one-time observed measurement lies near the boundary defining response or non-response. To select an optimal therapeutic strategy in the burgeoning age of precision medicine in rheumatology, achieve the lowest disease activity and maximize long-term health outcomes for each patient, improved treatment response definitions are needed.Objectives:Develop a high-confidence definition of treatment response and non-response in rheumatoid arthritis that exceeds the expected variability of subcomponents in the composite response criteria.Methods:A Monte Carlo simulation approach was used to assess ACR50 and EULAR response outcomes in 100 rheumatoid arthritis patients who had been treated for 6 months with a TNF inhibitor therapy. Monte Carlo simulations were run with 2000 iterations implemented with measurement variability derived for each clinical assessment: tender joint count, swollen joint count, Health Assessment Questionnaire disability index (HAQ-DI), patient pain assessment, patient global assessment, physician global assessment, serum C-reactive protein level (CRP) and disease activity score 28-joint count with CRP.1-3 Each iteration of the Monte Carlo simulation generated one outcome with a value of 0 or 1 indicating non-responder or responder, respectively.Results:A fidelity score, calculated separately for ACR50 and EULAR response, was defined as an aggregated score from 2000 iterations reported as a fraction that ranges from 0 to 1. The fidelity score depicted a spectrum of response covering strong non-responders, inconclusive statuses and strong responders. A fidelity score around 0.5 typified a response status with extreme variability and inconclusive clinical response to treatment. High-fidelity scores were defined as >0.7 or <0.3 for responders and non-responders, respectively, meaning that the simulated clinical response status label among all simulations agreed at least 70% of the time. High-confidence true responders were considered as those patients with high-fidelity outcomes in both ACR50 and EULAR outcomes.Conclusion:A definition of response to treatment should exceed the expected variability of the clinical assessments used in the composite measure of therapeutic response. By defining high-confidence responders and non-responders, the true impact of therapeutic efficacy can be determined, thus forging a path to development of better treatment options and advanced precision medicine tools in rheumatoid arthritis.References:[1]Cheung, P. P., Gossec, L., Mak, A. & March, L. Reliability of joint count assessment in rheumatoid arthritis: a systematic literature review. Semin Arthritis Rheum43, 721-729, doi:10.1016/j.semarthrit.2013.11.003 (2014).[2]Uhlig, T., Kvien, T. K. & Pincus, T. Test-retest reliability of disease activity core set measures and indices in rheumatoid arthritis. Ann Rheum Dis68, 972-975, doi:10.1136/ard.2008.097345 (2009).[3]Maska, L., Anderson, J. & Michaud, K. Measures of functional status and quality of life in rheumatoid arthritis: Health Assessment Questionnaire Disability Index (HAQ), Modified Health Assessment Questionnaire (MHAQ), Multidimensional Health Assessment Questionnaire (MDHAQ), Health Assessment Questionnaire II (HAQ-II), Improved Health Assessment Questionnaire (Improved HAQ), and Rheumatoid Arthritis Quality of Life (RAQoL). Arthritis Care Res (Hoboken) 63 Suppl 11, S4-13, doi:10.1002/acr.20620 (2011).Disclosure of Interests:Vibeke Strand Consultant of: Abbvie, Amgen, Arena, BMS, Boehringer Ingelheim, Celltrion, Galapagos, Genentech/Roche, Gilead, GSK, Ichnos, Inmedix, Janssen, Kiniksa, Lilly, Merck, Novartis, Pfizer, Regeneron, Samsung, Sandoz, Sanofi, Setpoint, UCB, Stanley Cohen: None declared, Lixia Zhang Shareholder of: Scipher Medicine Corporation, Employee of: Scipher Medicine Corporation, Ted Mellors Shareholder of: Scipher Medicine Corporation, Employee of: Scipher Medicine Corporation, Alex Jones Shareholder of: Scipher Medicine Corporation, Employee of: Scipher Medicine Corporation, Johanna Withers Shareholder of: Scipher Medicine Corporation, Employee of: Scipher Medicine Corporation, Viatcheslav Akmaev Shareholder of: Scipher Medicine Corporation, Employee of: Scipher Medicine Corporation

scholarly journals THU0106 PREDICTORS OF FLARE IN RHEUMATOID ARTHRITIS PATIENTS WITH LOW DISEASE ACTIVITY

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 267.1-267
Author(s):  
K. W. Moon
Keyword(s):  
Rheumatoid Arthritis ◽  
Multivariate Analysis ◽  
Observational Study ◽  
Disease Activity ◽  
Health Assessment Questionnaire ◽  
Health Assessment ◽  
Low Disease Activity ◽  
Erythrocyte Sedimentation ◽  
Euroqol 5D ◽  
Assessment Questionnaire

Background:Low disease activity (LDA) in patients with rheumatoid arthritis (RA) are usually recognized as stable state. In according to most guidelines for RA, monotherapy of disease modifying anti-rheumatic drug (DAMRD) was recommended for RA patents with LDA. But some of patients with LDA suffer from flare in their disease course. Until now, we don’t have enough data on factors that can predict flare in RA patients with LDA.Objectives:The aim of this study is to evaluate predictor of flare in RA patient with LDA from long-term (3 year) cohort data.Methods:Korean observational study network for arthritis (KORONA) registry is a nationwide Korean RA specific cohort registry that collecting data annually from 5,376 RA patients in 23 centers across South Korea. We include the data from 1, 801 RA patients with LDA (28 –joint disease activity score (DAS 28) < 3.2 at enrollment) who had consecutive data of DAS28 for 3 years. Flare was defined as an increase in DAS28 compared with baseline of >1.2 or >0.6 if concurrent DAS28 ≥3.2. Cox regression analysis was used to identify baseline predictors of flare.Results:Among 1,801 RA patients, 673 patients (37.4%) experienced flare in 3 years. When we compare the baseline characteristics of both flare and non-flare group, more women and more non-adherent patients for medication were observed in flare group. Flare group had longer disease duration, lower EuroQol 5D score, higher health assessment questionnaire (HAQ) score, and higher erythrocyte sedimentation rate (ESR) than non-flare group at baseline. In multivariate analysis, physician’s VAS, HAQ score, ESR, and poor adherence for medication were significant predictors of flare (Table 1).Table 1.Multivariate analysis of prediction of flare with baseline variablesMeasureHazard ratio95% Confidence IntervalP-valueFemale1.1300.906-1.4090.280Age0.9960.988-1.0050.414Physician’s VAS1.0081.002-1.013<0.01Pain VAS1.0020.998-1.0060.34EQ5D0.9520.534-1.6960.87HAQ1.4071.109-1.786<0.01ESR1.0081.002-1.014<0.01Poor adherence1.2721.047-1.545<0.05VAS: Visual Analogue Scale; EQ5D: EuroQol 5D; HAQ: Health Assessment Questionnaire; ESR: Erythrocyte Sedimentation RateConclusion:RA patient who have risk factors for flare, even though their disease activity was low, require more proactive treatment.References:[1]Bechman K, Tweehuysen L, Garrood T, Scott DL, Cope AP, Galloway JB, et al. Flares in Rheumatoid Arthritis Patients with Low Disease Activity: Predictability and Association with Worse Clinical Outcomes. J Rheumatol. 2018;45(11):1515-21.[2]Singh JA, Saag KG, Bridges SL, Jr., Akl EA, Bannuru RR, Sullivan MC, et al. 2015 American College of Rheumatology Guideline for the Treatment of Rheumatoid Arthritis. Arthritis Rheumatol. 2016;68(1):1-26.[3]Sung YK, Cho SK, Choi CB, Park SY, Shim J, Ahn JK, et al. Korean Observational Study Network for Arthritis (KORONA): establishment of a prospective multicenter cohort for rheumatoid arthritis in South Korea. Semin Arthritis Rheum. 2012;41(6):745-51.Disclosure of Interests:None declared

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