scholarly journals OP0118 EFFECT OF WITHDRAWING ETANERCEPT OR METHOTREXATE ON PATIENT-REPORTED OUTCOMES IN RHEUMATOID ARTHRITIS PATIENTS IN REMISSION ON COMBINATION THERAPY: RESULTS FROM THE SEAM-RA TRIAL

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 66.1-66
Author(s):  
J. Curtis ◽  
E. Karis ◽  
V. Bykerk ◽  
G. Kricorian ◽  
P. Yen ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Joint Pain ◽  
Patient Reported Outcomes ◽  
Sustained Remission ◽  
Research Support ◽  
Treatment Groups ◽  
Sf 36 ◽  
Patient Reported ◽  
The Mean ◽  
Domain Scores

Background:Limited studies have assessed the effect of withdrawal of either methotrexate (MTX) or etanercept (ETN) on patient-reported outcomes (PROs) in rheumatoid arthritis (RA).Objectives:To evaluate the baseline and change in PROs following withdrawal of MTX or ETN in RA patients with sustained remission receiving combination ETN+MTX.Methods:Adult patients with RA on ETN+MTX and in remission (SDAI ≤3.3) for ≥12 months (including a 24-week, open-label, run-in period) were randomized to a 48-week double-blind period to receive ETN 50 mg weekly (N=101), oral MTX 10-25 mg weekly (N=101) or continue ETN+MTX (N=51). The primary endpoint was maintenance of SDAI remission without disease worsening (DW) at week 48 between ETN and MTX groups. Patients who experienced SDAI >11 at any time after randomization, or SDAI >3.3 and ≤11 during 2 consecutive or on 3 non-consecutive visits were considered to have DW and resumed ETN+MTX. PROs assessed were patient global assessment of disease activity (PtGA, 0-100 mm), patient joint pain (PtJP, 0-100 mm), Health Assessment Questionnaire-Disability Index (HAQ-DI), and the 36-item short-form health survey (SF-36) component and domain scores. A 2-sample t-test was used to compare the treatment differences between groups. A subgroup analysis for patients with DW was also performed (DW analysis set) and compared PROs between ETN vs MTX arms (ETN+MTX not shown given the small sample size).Results:Of the 253 patients randomized, 121 (47.8%) experienced DW and were included in the DW analysis set. Baseline demographics were generally balanced between the 3 treatment groups. Most patients were women (76.3%), White (87.0%), and with a mean age of 55.6 years. The mean (SD) MTX dose was 16.3 (4.69) mg and the mean (SD) duration of RA was 10.3 (7.8) years. At week 48, a significantly greater proportion of patients on ETN vs MTX monotherapy maintained SDAI remission (49.5% vs 28.7%; P=0.004) after therapy withdrawal. In the overall population, PtGA and PtJP scores were very low at baseline (PtGA–MTX: 4.4, ETN: 4.5, ETN+MTX: 3.5; PtJP–MTX: 4.9, ETN: 5.5, ETN+MTX: 3.5) and showed some worsening over the study period in all treatment groups, with a mean change at week 48 ranging from 5.0 to 10.0 units for PtGA and 3.7 to 8.1 units for PtJP. Patients on ETN had less worsening, with a nominally significant treatment difference observed between ETN and MTX monotherapy groups for PtGA at almost all timepoints, and for PtJP at weeks 12 and 36 (Figure). Mean HAQ-DI (MTX: 0.32; ETN: 0.26; ETN+MTX: 0.28) and SF-36 scores (physical component [PCS]–MTX: 52.1, ETN: 52.7, ETN+MTX: 52.3; mental component [MCS]–MTX: 55.5, ETN: 55.8, ETN+MTX: 57.1) at baseline show that patients had low disability and excellent health-related quality of life compared with normative values for the general non-RA population. HAQ-DI scores were well maintained at weeks 24 and 48 (change from baseline at week 48–MTX: 0.14; ETN: 0.15; ETN+MTX: 0.21). The SF-36 PCS, MCS, and domain scores decreased minimally from baseline with treatment differences that were not nominally significant between groups. Among patients with DW during the study, those on ETN showed less PtGA and PtJP worsening from baseline than those on MTX at weeks 12, 36, and 48 (Figure). Other PROs (HAQ-DI [change from baseline at week 24–ETN: 0.34; MTX: 0.21; at week 48–ETN: 0.15; MTX: 0.15], SF-36 PCS, MCS, and domain scores) showed a similar degree of worsening in both the MTX and ETN arms.Conclusion:In patients with sustained SDAI remission on ETN+MTX, mental and physical health as measured by SF-36 was comparable with that of the non-RA population. Withdrawal of ETN (MTX monotherapy) resulted in a greater worsening of PtGA and PtJP than withdrawal of MTX (ETN monotherapy), and patients on ETN monotherapy restored these scores close to baseline towards the end of the treatment period. These findings demonstrate that ETN monotherapy has a greater effect on maintaining overall patient assessment of disease and joint pain compared with MTX monotherapy.Disclosure of Interests:Jeffrey Curtis Speakers bureau: AbbVie, BMS, Gilead, Lilly, Novartis, Sanofi, Scipher, Amgen, Corrona, Janssen, Myriad, and Pfizer, Consultant of: AbbVie, BMS, Gilead, Lilly, Novartis, Sanofi, Scipher, Amgen, Corrona, Janssen, Myriad, and Pfizer, Grant/research support from: AbbVie, BMS, Gilead, Lilly, Novartis, Sanofi, Scipher, Amgen, Corrona, Janssen, Myriad, and Pfizer, Elaine Karis Shareholder of: Amgen Inc., Employee of: Amgen Inc., Vivian Bykerk Speakers bureau: Amgen, BMS, Gilead, Pfizer, Sanofi-Genzyme/Regeneron, Scipher Medicine, and UCB., Consultant of: Amgen, BMS, Gilead, Pfizer, Sanofi-Genzyme/Regeneron, Scipher Medicine, and UCB., Grant/research support from: Amgen and Novartis, Greg Kricorian Shareholder of: Amgen Inc., Employee of: Amgen Inc., Priscilla Yen Shareholder of: Amgen Inc., Employee of: Amgen Inc., Paul Emery Speakers bureau: AbbVie, BMS, Celltrion, Gilead, Lilly, MSD, Novartis, Pfizer, Roche, Samsung, Sandoz, and UCB., Consultant of: AbbVie, BMS, Celltrion, Gilead, Lilly, MSD, Novartis, Pfizer, Roche, Samsung, Sandoz, and UCB., Paul Haraoui Speakers bureau: AbbVie, Celgene, Janssen, Pfizer, and UCB., Consultant of: AbbVie, Amgen, BMS, Celgene, Eli Lilly, Janssen, Merck, Pfizer, Roche, Sandoz, Sanofi-Genzyme, and UCB., Grant/research support from: Roche, AbbVie, Amgen, Merck, and Pfizer, David Collier Shareholder of: Amgen Inc., Employee of: Amgen Inc., Brad Stolshek Shareholder of: Amgen Inc., Employee of: Amgen Inc.

scholarly journals Patient-reported outcomes from a phase 3 study of baricitinib versus placebo or adalimumab in rheumatoid arthritis: secondary analyses from the RA-BEAM study

2017 ◽  
Vol 76 (11) ◽  
pp. 1853-1861 ◽  
Author(s):  
Edward C Keystone ◽  
Peter C Taylor ◽  
Yoshiya Tanaka ◽  
Carol Gaich ◽  
Amy M DeLozier ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Joint Pain ◽  
Patient Reported Outcomes ◽  
Physical Component Score ◽  
Study Endpoint ◽  
Primary Study ◽  
Inadequate Response ◽  
Phase 3 ◽  
Sf 36 ◽  
Patient Reported

BackgroundTo assess the effect of baricitinib on patient-reported outcomes (PROs) in patients with active rheumatoid arthritis and an inadequate response to methotrexate (MTX).MethodsIn this double-blind phase 3 study, patients were randomised 3:3:2 to placebo (n=488), baricitinib 4 mg once daily (n=487), or adalimumab 40 mg biweekly (n=330) with background MTX. PROs included the SF-36, EuroQol 5-D (EQ-5D) index scores and visual analogue scale, Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), Health Assessment Questionnaire-Disability Index (HAQ-DI), Patient’s Global Assessment of Disease Activity (PtGA), patient’s assessment of pain and Work Productivity and Activity Impairment Questionnaire-Rheumatoid Arthritis (WPAI-RA), and measures collected in electronic patient daily diaries: duration and severity of morning joint stiffness (MJS), Worst Ttiredness and Worst Joint Pain. The primary study endpoint was at week 12. Treatment comparisons were assessed with logistic regression for categorical measures or analysis of covariance for continuous variables.ResultsCompared with placebo and adalimumab, baricitinib showed statistically significant improvements (p≤0.05) in HAQ-DI, PtGA, pain, FACIT-F, SF-36 physical component score, EQ-5D index scores and WPAI-RA daily activity at week 12. Improvements were maintained for measures assessed to week 52. Statistically significant improvement in patient diary measures (MJS duration and severity), worst tiredness and worst joint pain were observed for baricitinib versus placebo and adalimumab at week 12 (p≤0.05).ConclusionsBaricitinib provided significantly greater improvement in most PROs compared with placebo and adalimumab, including physical function MJS, pain, fatigue and quality of life. Improvement was maintained to the end of the study (week 52).Trial registrationNCT01710358.

scholarly journals SAT0121 PAIN AND OTHER PATIENT-REPORTED OUTCOMES IN PATIENTS WITH RHEUMATOID ARTHRITIS WHO DID OR DID NOT ACHIEVE TREATMENT RESPONSE BASED ON IMPROVEMENT IN SWOLLEN JOINTS IN TOCILIZUMAB CLINICAL TRIALS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 994.2-995
Author(s):  
A. Sebba ◽  
J. Han ◽  
S. Mohan
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trials ◽  
Pain Score ◽  
Patient Reported Outcomes ◽  
Component Score ◽  
Link Type ◽  
Pain Scores ◽  
Sf 36 ◽  
Patient Reported ◽  
Nonresponder Group

Background:Significant improvements in pain and other patient-reported outcomes (PROs) have been shown in large clinical trials in patients with rheumatoid arthritis (RA) who receive tocilizumab (TCZ) compared with placebo (PBO). Recent data suggest that pain in RA may be noninflammatory as well as inflammatory, and improvement in pain scores and other PROs may be seen in patients who do not respond to treatment based on disease activity measures that evaluate inflammation.Objectives:To assess changes in pain scores and other PROs in patients with RA who did or did not achieve ≥ 20% improvement in SJC in TCZ clinical trials.Methods:Data from patients with active RA who received intravenous TCZ 8 mg/kg + MTX or PBO + MTX in 3 Phase III studies (OPTION [NCT00106548], TOWARD [NCT00106574] and LITHE [NCT00109408]) were included. All patients had moderate to severe RA with an inadequate response or intolerance of MTX (OPTION, LITHE) or conventional synthetic disease-modifying antirheumatic drugs (csDMARDs; TOWARD). Changes in pain (visual analog scale [VAS], 0-100 mm), Health Assessment Questionnaire Disability Index (HAQ-DI, 0-3), 36-Item Short Form Survey (SF-36) physical component score (PCS) and mental component score (MCS; 0-50) and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score (0-52) from baseline to Week 24 were evaluated. Results were compared between patients receiving TCZ + MTX and those receiving PBO + MTX in both patients who achieved ≥ 20% improvement in SJC (responders) and those who did not (nonresponders). The changes from baseline were analyzed using a mixed model with repeated measures, including the following covariates and interactions: treatment, visit, baseline of endpoint, region, baseline DAS28 and interactions of visit with treatment and baseline of endpoint.Results:Data from 1254 responders (TCZ + MTX, n = 831; PBO + MTX, n = 423) and 620 nonresponders (TCZ + MTX, n = 225; PBO + MTX, n = 395) were included. Patients receiving TCZ + MTX had significantly greater improvement in pain scores and HAQ-DI compared with PBO + MTX in the responder group (–27.19 vs –16.77 and –0.55 vs –0.34, respectively;P< 0.0001 for both) and nonresponder group (–9.59 vs 2.53 and –0.20 vs 0.01;P< 0.0001 for both) at Week 24 (Figure 1). Similar results were seen at Week 16 in the nonresponder group (–11.06 vs –2.38 and –0.23 vs –0.04;P< 0.0001 for both) prior to initiation of rescue treatment. At Week 24 in the responder group, patients receiving TCZ + MTX had significantly greater improvements compared with PBO + MTX in SF-36 PCS and MCS (9.16 vs 5.71 and 6.55 vs 3.79, respectively;P< 0.0001 for both) (Figure 2) and FACIT-Fatigue (8.39 vs 5.11;P< 0.0001). In the nonresponder group, patients receiving TCZ + MTX had significantly greater improvements compared with PBO + MTX in SF-36 PCS at Week 16 (3.81 vs 1.65;P= 0.0006) and Week 24 (4.42 vs 1.01;P< 0.0001) (Figure 2) and FACIT-Fatigue at Week 16 (3.82 vs 1.32;P= 0.0039) and Week 24 (3.90 vs 1.40;P= 0.0111).Conclusion:Patients with RA who received TCZ + MTX had significantly greater improvements in pain score and other PROs than those who received PBO + MTX regardless of whether they achieved ≥ 20% improvement in SJC. Clinical outcome at Week 24 correlated well with PROs, with a relatively larger improvement in pain score and other PROs in the responder group than in the nonresponder group; relative to PBO + MTX, these improvements appear numerically similar in the responder and nonresponder groups with consistently smaller difference between the groups in TCZ-treated arms. The consistent effect of TCZ on PROs in both responder and nonresponder groups warrants further study on the impact of TCZ on sources of pain independent of that caused by joint inflammation.Figure: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:Anthony Sebba Consultant of: Genentech, Gilead, Lilly, Regeneron Pharmaceuticals Inc., Sanofi, Speakers bureau: Lilly, Roche, Sanofi, Jian Han Shareholder of: Genentech, Inc., Employee of: Genentech, Inc., Shalini Mohan Shareholder of: Genentech, Inc., Employee of: Genentech, Inc.

scholarly journals POS0919 BIMEKIZUMAB SHOWS SUSTAINED LONG-TERM IMPROVEMENTS IN PATIENT-REPORTED OUTCOMES AND QUALITY OF LIFE IN ANKYLOSING SPONDYLITIS: 3-YEAR RESULTS FROM A PHASE 2B STUDY

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 720.2-721
Author(s):  
X. Baraliakos ◽  
M. Dougados ◽  
K. Gaffney ◽  
R. Sengupta ◽  
M. Magrey ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Ankylosing Spondylitis ◽  
Patient Reported Outcomes ◽  
Morning Stiffness ◽  
Spinal Pain ◽  
Research Support ◽  
Sf 36 ◽  
Patient Reported ◽  
Full Analysis

Background:Bimekizumab (BKZ), a monoclonal antibody that selectively inhibits interleukin (IL)-17A and IL-17F, has demonstrated clinical efficacy and safety in patients with ankylosing spondylitis (AS) treated over a period up to 96 weeks.1,2Objectives:To report 3-year interim patient-reported outcomes (PROs) in patients with active AS treated with BKZ in a phase 2b dose-ranging study (BE AGILE; NCT02963506) and its open-label extension (OLE; NCT03355573).Methods:BE AGILE study design has been described previously.1 Patients treated with BKZ 160 mg or 320 mg every 4 weeks (Q4W) at Week 48 in BE AGILE were eligible for OLE entry. All OLE patients received BKZ 160 mg Q4W. Outcome measures are reported for the OLE full analysis set (patients who entered the OLE and had ≥1 dose of BKZ and ≥1 valid efficacy variable measurement in the OLE), and include: BASDAI, BASDAI50 responder rate, BASFI, fatigue (BASDAI Q1), morning stiffness (mean of BASDAI Q5 + 6), total spinal pain (numeric rating scale [NRS]), SF-36 PCS and MCS, and ASQoL. Missing data were imputed using multiple imputation (MI; based on the missing at random assumption) for continuous variables and non-responder imputation (NRI) for dichotomous variables.Results:262/303 (86%) patients randomised at BE AGILE study baseline (BL) completed Week 48 on BKZ 160 mg or 320 mg, of whom 255/262 (97%) entered the OLE (full analysis set: 254). From baseline to Week 48 in BE AGILE, BKZ-treated patients showed clinically relevant improvements in disease activity (BASDAI, BASDAI50), physical function (BASFI), fatigue, morning stiffness, spinal pain, and quality of life (SF-36 PCS and MCS, ASQoL) (Figure 1). Group-level improvements in all reported continuous efficacy measures exceeded published minimally important difference (MID), minimum clinically important improvement (MCII), and/or minimum clinically important difference (MCID) thresholds (Figure 1).3,4 Efficacy in all reported outcome measures was maintained or continued to improve from Week 48 to Week 144 or 156 (Figure 1).Conclusion:BKZ treatment was associated with sustained and consistent efficacy in patients with active AS over 3 years, including patient-reported disease activity, physical function, fatigue, morning stiffness, spinal pain, and quality of life.References:[1]van der Heijde D. Ann Rheum Dis 2020;79:595–604.[2]Baraliakos X. Arthritis Rheumatol 2020;72 (suppl 10).[3]Ogdie A. Arthritis Care Res 2020;72 (S10):47–71.[4]Maruish ME. User’s manual for the SF-36v2 Health Survey (3rd ed). 2011; Lincoln, RI: QualityMetric Incorporated.Acknowledgements:This study was funded by UCB Pharma. Editorial services were provided by Costello Medical.Disclosure of Interests:Xenofon Baraliakos Speakers bureau: AbbVie, BMS, Chugai, Eli Lilly, Galapagos, Gilead, MSD, Novartis, Pfizer, UCB Pharma, Paid instructor for: AbbVie, BMS, Chugai, Eli Lilly, Galapagos, Gilead, MSD, Novartis, Pfizer, UCB Pharma, Consultant of: AbbVie, BMS, Chugai, Eli Lilly, Galapagos, Gilead, MSD, Novartis, Pfizer, UCB Pharma, Maxime Dougados Consultant of: AbbVie, Eli Lilly, Novartis, Pfizer and UCB Pharma, Grant/research support from: AbbVie, Eli Lilly, Novartis, Pfizer and UCB Pharma, Karl Gaffney Speakers bureau: AbbVie, Eli Lilly, Novartis, UCB Pharma, Consultant of: AbbVie, Eli Lilly, Novartis, UCB Pharma, Grant/research support from: AbbVie, Gilead, Eli Lilly, Novartis, UCB Pharma, Raj Sengupta Speakers bureau: AbbVie, Biogen, Celgene, MSD, Novartis, UCB Pharma, Consultant of: AbbVie, Biogen, Celgene, Eli Lilly, MSD, Novartis, UCB Pharma, Grant/research support from: AbbVie, Celgene, UCB Pharma, Marina Magrey Consultant of: AbbVie, Eli Lilly, Novartis, Pfizer, UCB Pharma, Grant/research support from: AbbVie, UCB Pharma, Natasha de Peyrecave Employee of: UCB Pharma, Marga Oortgiesen Employee of: UCB Pharma, Thomas Vaux Employee of: UCB Pharma, Carmen Fleurinck Employee of: UCB Pharma, Valerie Ciaravino Employee of: UCB Pharma, Atul Deodhar Speakers bureau: Janssen, Novartis, Pfizer, Consultant of: AbbVie, Amgen, BMS, Boehringer Ingelheim, Celgene, Eli Lilly, Gilead, GSK, Janssen, Novartis, Pfizer, UCB Pharma, Grant/research support from: AbbVie, Eli Lilly, GSK, Novartis, Pfizer, UCB Pharma

scholarly journals FRI0020 CLINICAL TREATMENT RESPONSE STILL DOES NOT MATCH PATIENT REPORTED IMPROVEMENT, EVEN IN EARLY RHEUMATOID ARTHRITIS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 582.1-582
Author(s):  
S. Pazmino ◽  
A. Lovik ◽  
A. Boonen ◽  
D. De Cock ◽  
V. Stouten ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Disease Activity ◽  
Early Rheumatoid Arthritis ◽  
Health Assessment ◽  
Sustained Remission ◽  
Research Support ◽  
Early Ra ◽  
Severity Scores ◽  
Patient Reported ◽  
Over Time

Background:Commonly used disease activity scores in rheumatoid arthritis (RA) include one patient reported outcome (PRO) -the patient’s global health assessment (PGA). Exploratory factor analysis (EFA) was performed on data from the 2 year Care in early Rheumatoid Arthritis (CareRA) trial to explain the evolution of disease burden extracting 3 factors.1Objectives:To assess the evolution and relative responsiveness over time of clinical, laboratory and patient assessments included in composite scores, together with other PROs like pain, fatigue and functionality in patients with early RA (≤1 year) treated to target (T2T) within the CareRA trial.Methods:DMARD naïve patients with early RA (n=379) were included, randomized to remission induction with COBRA-like treatment schemes (n=332) or MTX monotherapy (n=47) and T2T.Components of disease activity scores (swollen/tender joint count (S/TJC), C-reactive protein (CRP) or erythrocyte sedimentation rate (ESR), and physician (PhGH) or patient (PGA) global health assessment), pain and fatigue (both on 0-100 scale) and HAQ were recorded at every visit.Missing data was handled with multiple imputation (n=15). Clustering was removed with multiple outputation (n=1000), then each of the 15 000 datasets was analyzed by EFA with principal component extraction and oblimin rotation. The analyses were combined after re-ordering the factors by maximizing factor congruence. The 3 extracted factors and their individual components (with their loadings) were: 1. Patient containing PGA (0.87), pain (0.86), fatigue (0.90) and HAQ (0.5) 2.Clinical with SJC (0.92), TJC (0.89) and PhGH (0.76) and 3.Laboratory with CRP(0.87) and ESR (0.78).1(Pazmino, ACR 2019 abstract, Table 3)Afterwards, variables were first normalized to a 0-1 scale, then multiplied -weighted- by the factor loadings previously obtained.1For each Patient, Clinical and Laboratory severity score, the weighted variables belonging to each score were summed together and then re-scaled to 0-1 (higher values suggest more burden).The percentage (%) improvement from baseline to week 104 and the area under the curve (AUC) across time points were calculated per factor.Differences in % improvement and AUC were compared between patients not achieving and achieving early and sustained (week 16 to 104) disease activity score remission (DAS28CRP <2.6) with ANOVA. Bonferroni correction was used for multiple testing.Results:Severity scores of Patient, Clinical and Laboratory factors improved rapidly over time (Figure 1). In patients achieving sustained remission (n=122), Patient, Clinical and Laboratory scores improved 56%, 90% and 27% respectively. In patients not achieving sustained remission (n=257) the improvement was 32%, 78% and 9% respectively (p<0.001 only for clinical improvement).Patients in CareRA who achieved sustained remission had an AUC of 15.1, 3.4 and 4.7 in Patient, Clinical and Laboratory scores respectively, compared to 32.3, 10.0, and 7.2 in participants not achieving sustained remission (p<0.001 for all comparisons).Conclusion:Patient, Clinical and Laboratory severity scores improved rapidly over time in patients achieving rapid and sustained disease control. However, overall, Patient burden seemed not to improve to the same extent as Clinical burden. Patient’s unmet needs in terms of pain, fatigue, functionality and overall well-being should thus be given more attention, even in patients in sustained remission.References:[1]Pazmino S,et al.Including Pain, Fatigue and Functionality Regularly in the Assessment of Patients with Early Rheumatoid Arthritis Separately Adds to the Evaluation of Disease Status [abstract]. ACR. 2019.Disclosure of Interests:Sofia Pazmino: None declared, Anikó Lovik: None declared, Annelies Boonen Grant/research support from: AbbVie, Consultant of: Galapagos, Lilly (all paid to the department), Diederik De Cock: None declared, Veerle Stouten: None declared, Johan Joly: None declared, Delphine Bertrand: None declared, Rene Westhovens Grant/research support from: Celltrion Inc, Galapagos, Gilead, Consultant of: Celltrion Inc, Galapagos, Gilead, Speakers bureau: Celltrion Inc, Galapagos, Gilead, Patrick Verschueren Grant/research support from: Pfizer unrestricted chair of early RA research, Speakers bureau: various companies

scholarly journals Patient-reported outcomes for tofacitinib with and without methotrexate, or adalimumab with methotrexate, in rheumatoid arthritis: a phase IIIB/IV trial

RMD Open ◽  
2019 ◽  
Vol 5 (2) ◽  
pp. e001040 ◽  
Author(s):  
Vibeke Strand ◽  
Eduardo Mysler ◽  
Robert J Moots ◽  
Gene V Wallenstein ◽  
Ryan DeMasi ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Patient Reported Outcomes ◽  
Short Form ◽  
Controlled Trial ◽  
Inadequate Response ◽  
Link Type ◽  
Combination Treatments ◽  
Sf 36 ◽  
Patient Reported ◽  
Phase Iiib

ObjectiveTo provide the first direct comparison of patient-reported outcomes (PROs) following treatment with tofacitinib monotherapy versus tofacitinib or adalimumab (ADA) in combination with methotrexate (MTX) in patients with rheumatoid arthritis (RA) with inadequate response to MTX (MTX-IR).MethodsORAL Strategy (NCT02187055), a phase IIIB/IV, head-to-head, randomised controlled trial, assessed non-inferiority between tofacitinib 5 mg two times per day monotherapy, tofacitinib 5 mg two times per day+MTX and ADA 40 mg every other week+MTX. PROs assessed included the following: Patient Global Assessment of disease activity (PtGA), Pain, Health Assessment Questionnaire-Disability Index, Functional Assessment of Chronic Illness Therapy-Fatigue and 36-Item Short-Form Health Survey (SF-36) summary and domain scores.ResultsSubstantial improvements from baseline were reported across all PROs in all treatment arms, which, in the majority, met or exceeded minimum clinically important differences. Compared with tofacitinib monotherapy, tofacitinib+MTX combination treatment conferred significantly greater improvements in PtGA, Pain and SF-36 physical component summary scores at month 6. Statistically or numerically greater improvements were often, but not uniformly, reported for combination treatments compared with tofacitinib monotherapy at other time points.ConclusionTreatment with tofacitinib+MTX, ADA+MTX and tofacitinib monotherapy resulted in clinically meaningful improvements in PROs in MTX-IR patients with RA. These were comparatively greater with combination treatments versus tofacitinib monotherapy, although differences between treatment arms were small, limiting our ability to confer clinical meaning.Trial registration numberNCT02187055.

Changes in quality of life in relation to disease activity in systemic lupus erythematosus: post-hoc analysis of the BLISS-52 Trial

Lupus ◽  
2019 ◽  
Vol 28 (14) ◽  
pp. 1628-1639 ◽  
Author(s):  
M Jolly ◽  
N Annapureddy ◽  
L Arnaud ◽  
H Devilliers
Keyword(s):  
Disease Activity ◽  
Lupus Erythematosus ◽  
Patient Reported Outcomes ◽  
Patient Reported Outcome ◽  
Sf 36 ◽  
Patient Reported ◽  
Outcome Scores ◽  
Activity Measures ◽  
Flare Index ◽  
The Mean

Objectives To quantify changes in generic patient-reported outcomes against clinically meaningful, disease activity measures in systemic lupus erythematosus (SLE). Methods Using BLISS-52 trial data (867 SLE patients), we estimated the mean difference in change of patient-reported outcome scores (Medical Outcomes Study SF-36 and FACIT-fatigue) in relation to disease activity (SELENA-SLEDAI, SELENA-SLEDAI flare index, SLE responder index and British Isles Lupus Assessment Group (BILAG)), considering all study visits by the mean of multivariate mixed models. Predefined disease activity criteria were used to define for improvement and worsening. Results Mean changes in physical component summary/mental component summary and FACIT-fatigue in response to changes in SELENA-SLEDAI and SELENA-SLEDAI flare index were significantly lower than 2.5. New SELENA-SLEDAI flare index flare led to a significant change in all patient-reported outcome scores, except role emotional. Mean improvement in patient-reported outcomes with achievement of SLE responder index ranged between +6.2 (physical function) and +11.3 (bodily pain) for SF-36 domains, + 3.4 and +3.3 for mental component summary and physical component summary, and was +4.2 for FACIT-fatigue. When considering disease activity changes by organ system, changes in BILAG (constitutional) was independently associated with significant changes in FACIT-fatigue and all SF-36 domains (except physical function), changes in BILAG (musculoskeletal and hematological) were independently associated with significant changes in patient-reported outcome scores, except for role emotional (musculoskeletal) and general health/mental health (hematological). Mean changes in every SF-36 domain varied (and was >5) with SLE responder index attainment. Conclusions Knowledge of changes in patient-reported outcomes, against clinically meaningful changes in SLE disease activity measures, is crucial for designing of clinical trials, interpretation of results and shared decision-making for patient care.

scholarly journals POS0445 PHYSICIAN AND PATIENT REPORTED EFFECTIVENESS OUTCOMES ARE SIMILAR IN TOFACITINIB AND TNF INHIBITORS IN RHEUMATOID ARTHRITIS PATIENTS: DATA FROM A RHEUMATOID ARTHRITIS REGISTRY IN CANADA

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 452.1-452
Author(s):  
M. Movahedi ◽  
A. Cesta ◽  
X. Li ◽  
E. Keystone ◽  
C. Bombardier
Keyword(s):  
Rheumatoid Arthritis ◽  
Real World ◽  
Treatment Initiation ◽  
Activity Index ◽  
Disease Activity Index ◽  
Research Support ◽  
Real World Data ◽  
Treatment Groups ◽  
Patient Reported ◽  
Anxiety Depression

Background:Tofacitinib (TOFA) is an oral, small molecule drug used for rheumatoid arthritis (RA) treatment as an alternative option to biologic disease modifying antirheumatic drugs (bDMARDs) including tumor necrosis factor inhibitors (TNFi).Objectives:We aimed to evaluate physician and patient reported effectivness outcomes in TNFi compared to TOFA, using real-world data from the Ontario Best Practices Research Initiative (OBRI).Methods:RA patients enrolled in the OBRI initiating their TOFA or TNFi (Adalimumab, Certolizumab, Etanercept, Golimumab, Infliximab, and Biosimilars) between 1st June 2014 (TOFA approval date in Canada) and 31st Dec 2019 were included. Patients were required to have physician and patient reported effectivness outcomes data available at treatment initiation and 6-month (± 2 months) follow-up. These included clinical disease activity index (CDAI), rheumatoid arthritis disease activity index (RADAI), HAQ-DI, sleep problem, and anxiety/depression scores. Multiple imputation (Imputation Chained Equation, N=20) was used to deal with missing data for covaraites at treatment initiation. To deal with confounding by indication, we estimated propensity scores for covariates with an absolute standard difference greater than 0.1 between the two treatment groups.Results:A total of 419 patients were included. Of those, 226 were initiating a TNFi and 193 TOFA, and had a mean (SD) disease duration of 8.0 (8.7) and 12.6 (9.6) years, respectively. In the TNFi group, 81.9% were female and mean age (SD) at treatment initiation was 56.6 (13.4) years. In the TOFA group, 85% were female and mean (SD) age at treatment initiation was 60.3 (11.2) years. The TNFi group was less likely to have prior biologic use (21.7%) compared to the TOFA group (67.9%). At treatment initiation, physical function measured by HAQ-DI was significantly lower in TNFi compared to the TOFA group (1.2 vs.1.4).The rate of CDAI LDA/remission at 6 months was 36.7% and 33.2% in TNFi and TOFA group, respectively. The generalized linear mixed models (GLMM) adjusting for propensity score quantile, showed that there was no significant difference in CDAI LDA/remission (ORs: 0.85, 95% CI: 0.51, 1.43), RADAI (coefficient: 0.48, 95% CI: -0.18, 1.14), HAQ-DI (coefficient: -0.01, 95% CI: -0.18, 0.16), sleep problems (coefficient: -0.25, 95% CI: -0.95, 0.45), and anxiety/depression scores (coefficient: 0.12, 95% CI: -0.35, 0.58) between the two treatment groups (TOFA used as reference).Conclusion:In this real-world data study, we found that, physician and patient reported effectivness outcomes are similar in the TNFi and TOFA groups 6 months after treatment initiation in patients with RA.Disclosure of Interests:Mohammad Movahedi: None declared, Angela Cesta: None declared, Xiuying Li: None declared, Edward Keystone Grant/research support from: Amgen, Merck, Pfizer Pharmaceuticals, PuraPharm. Speaker Honoraria Agreements: AbbVie, Amgen, Bristol-Myers Squibb Company, Celltrion, Myriad Autoimmune, F. Hoffmann-La Roche Inc, Gilead, Janssen Inc, Lilly Pharmaceuticals, Merck, Pfizer Pharmaceuticals, Sandoz, Sanofi-Genzyme, Samsung Bioepsis. Consulting Agreements/Advisory Board Membership: AbbVie, Amgen, Bristol-Myers Squibb Company, Celltrion, Myriad Autoimmune, F. Hoffmann-La Roche Inc, Gilead, Janssen Inc, Lilly Pharmaceuticals, Merck, Pfizer Pharmaceuticals, Sandoz, Sanofi-Genzyme, Samsung Bioepsis, Claire Bombardier Grant/research support from: OBRI was funded by peer reviewed grants from CIHR (Canadian Institute for Health Research), Ontario Ministry of Health and Long-Term Care (MOHLTC), Canadian Arthritis Network (CAN) and unrestricted grants from: Abbvie, Amgen, Aurora, Bristol-Meyers Squibb, Celgene, Hospira, Janssen, Lilly, Medexus, Merck, Novartis, Pfizer, Roche, Sanofi, & UCB. Dr. Bombardier held a Canada Research Chair in Knowledge Transfer for Musculoskeletal Care and a Pfizer Research Chair in Rheumatology

scholarly journals Nonoperative Management of Femoroacetabular Impingement: Clinical Outcomes at 5-Years--A Prospective Study (143)

2021 ◽  
Vol 9 (10_suppl5) ◽  
pp. 2325967121S0028
Author(s):  
Andrew Zogby ◽  
James Bomar ◽  
Kristina Johnson ◽  
Kelly Randich ◽  
Vidyadhar Upasani ◽  
...  
Keyword(s):  
Femoroacetabular Impingement ◽  
Patient Reported Outcomes ◽  
Operative Management ◽  
Activity Level ◽  
Treatment Groups ◽  
Patient Reported ◽  
The Mean ◽  
Non Operative Management ◽  
Fai Syndrome

Objectives: Mid-term and long-term outcomes data on non-operative management of femoroacetabular impingement (FAI) syndrome remains sparse despite expanding research on the topic. Our purpose is to present 5-year outcomes data utilizing a non-operative protocol on a consecutive series of patients with FAI syndrome. Methods: Between 2013 and 2016, patients were prospectively recruited in a non-operative FAI study. The protocol consisted of an initial trial of rest, physical therapy, and activity modification. Patients who remained symptomatic were offered an intra-articular steroid injection. Patients with recurrent symptoms were offered arthroscopic treatment. Patient-reported outcomes including the modified Harris Hip Score (mHHS) and Non-arthritic Hip Score (NAHS) were collected 1-, 2-, and 5-years after enrollment. We present the 5-year data. Statistical analysis was performed to determine outcomes based on FAI type and treatment. Results: 133 hips in 100 patients were enrolled. Sixty-seven hips in 50 patients were available for 5-year follow up. At enrollment, the mean mHHS and NAHS were 69.6±13.1 and 76.3±14.7 respectively. In total, 73% of the cohort was managed non-operatively. Of the 11 patients requiring surgery, six (55%) converted to surgery within one year of enrollment, 4 (36%) converted to surgery between one and 2 years, and one patient converted to surgery between 2 and 5 years. At final follow up, the mean mHHS and NAHS were 89.6±10.7 and 88.0±12.1 respectively. At 1-year follow up, only the activity modification group made a significant increase in mHHS and NAHS (p<0.03), by two year follow up, all three treatment groups had made statistically significant improvements in mHHS and NAHS (p<0.05), by 5-years follow up, the activity modification group and the scope group had maintained their statistically significant improvement in mHHS and NAHS (p<0.03). There was no significant difference in mHHS or NAHS between treatment groups at 5-year follow-up (p>0.4)(Table 1), and no difference in proportion of hips meeting the MCID for mHHS based on treatment course (p=0.961). There was no difference in mHHS or NAHS between FAI types at any time point (p>0.06)(Table 2), or in the proportion of hips that met MCID among FAI types (p=0.511). 72% of patients returned to the same or similar sport/activity level, and there was no difference in the proportion of patients that returned to sports/activities among treatment type (p=0.095) or FAI type (p=0.273). Conclusions: Non-operative management of FAI syndrome is effective in a majority of adolescent patients, with robust improvements in patient-reported-outcomes persisting at 5-year follow-up.

scholarly journals Treatment response and several patient-reported outcomes are early determinants of future self-efficacy in rheumatoid arthritis

2021 ◽  
Vol 23 (1) ◽  
Author(s):  
Doumen Michaël ◽  
De Cock Diederik ◽  
Pazmino Sofia ◽  
Bertrand Delphine ◽  
Joly Johan ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Treatment Response ◽  
Self Efficacy ◽  
Global Assessment ◽  
Patient Reported Outcomes ◽  
Self Management ◽  
Remission Induction ◽  
Early Ra ◽  
Sf 36 ◽  
Patient Reported

Abstract Background Self-efficacy, or patients’ confidence in their ability to control disease and its consequences, was recently prioritised in EULAR recommendations for inflammatory arthritis self-management strategies. However, it remains unclear which factors influence self-efficacy in early rheumatoid arthritis (RA). Methods Data were analysed from the 2-year RCT Care in early RA (CareRA), which studied remission-induction treatment regimens for early RA. Participants completed the Arthritis Self-Efficacy Scale (ASES), Short-Form 36 (SF-36), Revised Illness Perception Questionnaire (IPQ-R), Utrecht Coping List (UCL), RAQoL and Health Assessment Questionnaire (HAQ). Depending on time to first remission (DAS28-CRP < 2.6) and persistence of remission, treatment response was defined as persistent response, secondary failure, delayed response, late response or non-response. The association between ASES scores and clinical/psychosocial factors was explored with Spearman correlation and multivariate linear mixed models. Baseline predictors of week 104 ASES were identified with exploratory linear regression followed by multiple regression of significant predictors adjusted for DAS28-CRP, HAQ, treatment arm, treatment response, cumulative CRP/SJC28 and demographic/serologic confounders. Results All 379 patients had a recent diagnosis of RA and were DMARD-naïve at study initiation. Most patients were women (69%) and RF/ACPA-positive (66%), and the mean (SD) age was 52 (13) years. For all tested outcome measures, better perceived health correlated with higher self-efficacy. While patient-reported factors (HAQ, SF-36, RAQoL, IPQ-R, pain, fatigue and patient’s global assessment) showed moderate/strong correlations with ASES scores, correlations with physician-reported factors (physician’s global assessment, SJC28), TJC28 and DAS28-CRP were weak. Only more favourable outcomes on patient-reported factors and DAS28-CRP were associated with higher ASES scores at each time point. An earlier, persistent treatment response predicted higher ASES scores at both weeks 52 and 104. Significant baseline predictors of week 104 ASES included HAQ; SF-36 mental component score, vitality, mental health and role emotional; IPQ-R illness coherence, treatment control, emotional representations and consequences; UCL Passive reacting; and the RAQoL. Conclusions Patient-reported outcomes and treatment response were early determinants of long-term self-efficacy in an early RA trial. These results provide further relevance for the window of opportunity in an early treat-to-target strategy and could help to timely identify patients who might benefit from self-management interventions. Trial registration EudraCT 2008-007225-39

PROMs and PREMs in Dutch integrated head and neck cancer care: Measurements and evaluation.

2017 ◽  
Vol 35 (8_suppl) ◽  
pp. 209-209
Author(s):  
Lydia Francisca Jacoba van Overveld ◽  
Robert P. Takes ◽  
Jozé C.C. Braspenning ◽  
Ludi E Smeele ◽  
Matthias A.W. Merkx ◽  
...  
Keyword(s):  
Head And Neck Cancer ◽  
Head And Neck ◽  
Neck Cancer ◽  
Patient Reported Outcomes ◽  
Patient Centred Care ◽  
Treatment Groups ◽  
Patient Reported ◽  
Eortc Qlq ◽  
Domain Scores

209 Background: Providing patient-centred care is an essential component of high quality integrated care. Nowadays, patient reported outcomes and experiences are increasingly used to measure quality of care. As part of a quality registration, the Dutch Head and Neck Audit, patient reported outcomes (PROs) and experiences (PREs) of patients with head and neck cancer (HNC) are measured with questionnaires and evaluated to increase patient-centred care. Methods: Patientswere recruited from nine hospitals participating in the DHNA. Validated questionnaires were distributed at baseline, 3, 6 and 12 months follow-up. Included PROMs were EQ-5D-3L, EORTC QLQ-C30 and EORTC QLQ-H&N35. Included PREMs were Consumer Quality index for Oncologic care (CQO) and Radiotherapeutic care (CQR). CQO and CQR have similar domains with different questions. With descriptive analysis, ANOVA and mixed model analysis, differences over time and between treatment groups were analyzed. Results: Questionnaires were filled in by 238 patients. Pain decreased significantly at 6 and 12 months follow-up and dry mouth increased significantly at 3, 6 and 12 months follow-up compared to baseline. Sticky Salvia, problems with social eating and sense problems increased at 3 and 6 months follow-up, but were similar to the baseline score at 12 months follow-up. Pain and sticky saliva differed between radiotherapy and chemoradiotherapy or surgery and radiotherapy respectively (p ≤ 0.05). Regarding the CQO domain scores, all treatment groups differ significantly from each other (p ≤ 0.05). This was not regarding the CQR domain scores. Except for the domain Organisation, no differences between the CQO and CQR domain scores were found. Recognizing the emotional side of HNC (care) and intensifying guidance after the treatment period needs improvement. Conclusions: This study gives clues to improve healthcare according the experiences of the patient and we can predict more carefully the outcomes of the patients with different treatment types. PROMs according to the ICHOM criteria and PREMs are promising for measuring and improving quality and personalization of HNC care. However, the usage of two PREMs had no added value to evaluate and address points of improvement.

Sign in / Sign up

Export Citation Format

Share Document