scholarly journals Observational Comparative Effectiveness Studies of Drug Therapies

2012 ◽  
Vol 172 (18) ◽  
pp. 1412 ◽  
Author(s):  
James S. Floyd ◽  
Bruce M. Psaty
Keyword(s):  
Comparative Effectiveness ◽  
Effectiveness Studies

THU0620-HPR MEASUREMENT OF LOW DISEASE ACTIVITY USING THE CLINICAL DISEASE ACTIVITY INDEX (CDAI) VERSUS THE DISEASE ACTIVITY SCORE 28 (DAS 28): IMPACT OF INFLAMMATION MARKERS ON THE COMPARATIVE EFFECTIVENESS OF BIOLOGICS FOR THE TREATMENT OF RHEUMATOID ARTHRITIS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 553.1-553
Author(s):  
K. Janke ◽  
K. Biester ◽  
D. Krause ◽  
B. Richter ◽  
C. Schürmann ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Disease Activity ◽  
Comparative Effectiveness ◽  
Inflammatory Markers ◽  
Activity Index ◽  
Disease Activity Index ◽  
Signs And Symptoms ◽  
Acute Phase Reactants ◽  
Das 28 ◽  
Effectiveness Studies

Background:Biologics for the treatment of rheumatoid arthritis (RA) have different modes of action to target auto-inflammatory processes causing the signs and symptoms of the disease. Different biologics may thus have different effects on inflammatory markers. For instance, previous studies have shown that the interleukin-6-inhibitor tocilizumab (TOC) decreases the level of acute phase reactants (APRs) [1]. Such direct effects on inflammatory markers may lead to an overestimation of clinical response if disease activity is measured via scores including inflammatory markers, such as the Disease Activity Score 28 (DAS 28). The detected changes in disease activity may not adequately reflect the clinical improvement of signs and symptoms.Objectives:In our study, we compared biologics with each other using two different disease activity scores: the DAS 28 including APRs and the clinical disease activity index (CDAI) excluding APRs. The aim of this study was to assess whether the use of the two different scores affects comparative effectiveness studies on biologics for the treatment of RA.Methods:We compared results on the comparative effectiveness of biologics using the corresponding thresholds for low disease activity (LDA) for the DAS 28 (< 3.2) and the CDAI (≤ 10). We performed two separate network meta-analyses (NMAs) after a thorough step-by-step evaluation of the similarity, homogeneity and consistency assumptions of the patient populations and the study data.Our study formed part of a systematic review (including NMAs) that was largely based on clinical study reports and re-analyses of LDA using individual patient data provided by sponsors for studies conducted up to 2017. Thus, the analyses include hitherto unknown data on LDA analysed by means of the CDAI, especially data from older studies. An extensive comparison of DAS 28 and CDAI in different patient populations was possible.Results:For all analysed patient populations, comparisons of TOC versus other biologics yielded remarkable results: advantages for TOC were found in NMAs using the DAS 28, which were not confirmed in NMAs using the CDAI. For methotrexate (MTX)-naïve patients, using the DAS 28, TOC showed a greater benefit than abatacept (ABA), certolizumab pegol (CZP), and etanercept (ETA), which was not confirmed by the CDAI. In contrast, TOC showed less benefit than adalimumab (ADA) and ETA. For patients after MTX failure and using the DAS 28, TOC showed a greater benefit than ABA, ADA, anakinra (ANA), ETA, golimumab (GOL), and infliximab (INF). With the exception of ANA, these advantages were not confirmed by the CDAI. Similar differences between DAS 28 and CDAI were shown in patients treated with biologics in monotherapy or after failure of biologics.Conclusion:In comparative effectiveness studies of biologics, the assessment of LDA using the DAS 28 instead of the CDAI leads to a consistent overestimation of the benefit of TOC in all patient populations, regardless of pre-treatment or combined therapy with MTX. The inclusion of APRs in disease activity scores may thus introduce bias. A score excluding inflammatory markers should therefore be used to ensure valid results.References:[1]Smolen JS, Aletaha D. Interleukin-6 receptor inhibition with tocilizumab and attainment of disease remission in rheumatoid arthritis: the role of acute-phase reactants. Arthritis Rheum 2011; 63(1): 43-52.Disclosure of Interests:Kirsten Janke: None declared, Katharina Biester: None declared, Dietmar Krause Grant/research support from: Pfizer and AbbVie (Abbott), Bernd Richter: None declared, Christoph Schürmann: None declared, Katharina Hirsch: None declared, Beate Wieseler: None declared

scholarly journals CL3 UNDERSTANDING THE PATIENT VOICE IN COMPARATIVE EFFECTIVENESS STUDIES: THE ROLE OF BENCHMARK COMPARISONS

2019 ◽  
Vol 22 ◽  
pp. S35
Author(s):  
R. Rendas-Baum ◽  
B. Laird ◽  
K. Rychlec ◽  
J.E. Brown ◽  
M. Bayliss
Keyword(s):  
Comparative Effectiveness ◽  
Patient Voice ◽  
Effectiveness Studies

Pilot Study of the Juvenile Dermatomyositis Consensus Treatment Plans: A CARRA Registry Study

2020 ◽  
Vol 48 (1) ◽  
pp. 114-122 ◽  
Author(s):  
Kuan Liu ◽  
George Tomlinson ◽  
Ann M. Reed ◽  
Adam M. Huber ◽  
Olli Saarela ◽  
...  
Keyword(s):  
Missing Data ◽  
Pilot Study ◽  
Comparative Effectiveness ◽  
Juvenile Dermatomyositis ◽  
Confounding By Indication ◽  
Childhood Arthritis ◽  
Moderate Improvement ◽  
Treatment Plans ◽  
Effectiveness Studies ◽  
New Onset

Objectives.To determine the feasibility of comparing the Childhood Arthritis and Rheumatology ResearchAlliance (CARRA) consensus treatment plans (CTP) in treating moderate new-onset juvenile dermatomyositis (JDM) using the CARRA registry, and to establish appropriate analytic methods to control for confounding by indication and missing data.Methods.A pilot cohort of 39 patients with JDM from the CARRA registry was studied. Patients were assigned by the treating physician, considering patient/family preferences, to 1 of 3 CTP: methotrexate (MTX) and prednisone (MP); intravenous (IV) methylprednisolone, MTX, and prednisone (MMP); or IV methylprednisolone, MTX, prednisone, and IV immunoglobulin (MMPI). The primary outcome was the proportion of patients achieving moderate improvement at 6 months under each CTP. Statistical methods including multiple imputation and inverse probability of treatment weighting were used to handle missing data and confounding by indication.Results.Patients received MP (n = 13), MMP (n = 18) and MMPI (n = 8). Patients in all CTP had significant improvement in disease activity. Of the 36 patients who remained in our pilot study at 6 months, 16 (44%) of them successfully achieved moderate improvement at 6 months (6/13, 46% for MP; 7/15, 47% for MMP; 3/8, 38% for MMPI). After correcting for confounding, there were no statistically significant pairwise differences between the CTP (P = 0.328–0.88).Conclusion.We gained valuable experience and insight from our pilot study that can be used to guide the design and analysis of comparative effectiveness studies using the CARRA registry CTP approach. Our analytical methods can be adopted for future comparative effectiveness studies and applied to other rare disease observational studies.

Comparative effectiveness research in spine surgery

2012 ◽  
Vol 33 (1) ◽  
pp. E2 ◽  
Author(s):  
Kalil G. Abdullah ◽  
Edward C. Benzel ◽  
Thomas E. Mroz
Keyword(s):  
Cost Effectiveness ◽  
Spine Surgery ◽  
Comparative Effectiveness Research ◽  
Spinal Surgery ◽  
Comparative Effectiveness ◽  
Effectiveness Research ◽  
Effectiveness Studies

Comparative effectiveness research (CER) has impending significance for the field of spine surgery. This article outlines the rationale for comparative effectiveness research and reviews recommended priorities of spinal surgery emphasis. It also examines recent key studies of CER in the spine surgery literature and associated cost-effectiveness studies. It concludes with a discussion of the direction of CER in the spine surgery community.

scholarly journals The role of matching when adjusting for baseline differences in the outcome variable of comparative effectiveness studies

2015 ◽  
Vol 4 (4) ◽  
pp. 341-349 ◽  
Author(s):  
Carlos G Grijalva ◽  
Christianne L Roumie ◽  
Harvey J Murff ◽  
Adriana M Hung ◽  
Cole Beck ◽  
...  
Keyword(s):  
Comparative Effectiveness ◽  
Outcome Variable ◽  
Effectiveness Studies
Sign in / Sign up

Export Citation Format

Share Document