scholarly journals 2016 American College of Rheumatology/European League Against Rheumatism criteria for minimal, moderate, and major clinical response in adult dermatomyositis and polymyositis

2017 ◽  
Vol 76 (5) ◽  
pp. 792-801 ◽  
Author(s):  
Rohit Aggarwal ◽  
Lisa G Rider ◽  
Nicolino Ruperto ◽  
Nastaran Bayat ◽  
Brian Erman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Conjoint Analysis ◽  
Relative Weight ◽  
Nominal Group ◽  
Response Criteria ◽  
Analysis Model ◽  
Major Improvement ◽  
Core Set ◽  
Improvement Score ◽  
Cent Change

To develop response criteria for adult dermatomyositis (DM) and polymyositis (PM). Expert surveys, logistic regression, and conjoint analysis were used to develop 287 definitions using core set measures. Myositis experts rated greater improvement among multiple pairwise scenarios in conjoint analysis surveys, where different levels of improvement in 2 core set measures were presented. The PAPRIKA (Potentially All Pairwise Rankings of All Possible Alternatives) method determined the relative weights of core set measures and conjoint analysis definitions. The performance characteristics of the definitions were evaluated on patient profiles using expert consensus (gold standard) and were validated using data from a clinical trial. The nominal group technique was used to reach consensus. Consensus was reached for a conjoint analysis-based continuous model using absolute per cent change in core set measures (physician, patient, and extramuscular global activity, muscle strength, Health Assessment Questionnaire, and muscle enzyme levels). A total improvement score (range 0–100), determined by summing scores for each core set measure, was based on improvement in and relative weight of each core set measure. Thresholds for minimal, moderate, and major improvement were ≥20, ≥40, and ≥60 points in the total improvement score. The same criteria were chosen for juvenile DM, with different improvement thresholds. Sensitivity and specificity in DM/PM patient cohorts were 85% and 92%, 90% and 96%, and 92% and 98% for minimal, moderate, and major improvement, respectively. Definitions were validated in the clinical trial analysis for differentiating the physician rating of improvement (p<0.001). The response criteria for adult DM/PM consisted of the conjoint analysis model based on absolute per cent change in 6 core set measures, with thresholds for minimal, moderate, and major improvement.

scholarly journals 2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Juvenile Dermatomyositis

2017 ◽  
Vol 76 (5) ◽  
pp. 782-791 ◽  
Author(s):  
Lisa G Rider ◽  
Rohit Aggarwal ◽  
Angela Pistorio ◽  
Nastaran Bayat ◽  
Brian Erman ◽  
...  
Keyword(s):  
Conjoint Analysis ◽  
Juvenile Dermatomyositis ◽  
Face Validity ◽  
Nominal Group ◽  
Response Criteria ◽  
Major Improvement ◽  
Moderate Improvement ◽  
Core Set ◽  
Improvement Score ◽  
Cent Change

To develop response criteria for juvenile dermatomyositis (DM). We analysed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial. At a consensus conference, experts considered 14 top candidate criteria based on their performance characteristics and clinical face validity, using nominal group technique. Consensus was reached for a conjoint analysis–based continuous model with a total improvement score of 0–100, using absolute per cent change in core set measures of minimal (≥30), moderate (≥45), and major (≥70) improvement. The same criteria were chosen for adult DM/polymyositis, with differing thresholds for improvement. The sensitivity and specificity were 89% and 91–98% for minimal improvement, 92–94% and 94–99% for moderate improvement, and 91–98% and 85–86% for major improvement, respectively, in juvenile DM patient cohorts using the IMACS and PRINTO core set measures. These criteria were validated in the PRINTO trial for differentiating between treatment arms for minimal and moderate improvement (p=0.009–0.057) and in the RIM trial for significantly differentiating the physician's rating for improvement (p<0.006). The response criteria for juvenile DM consisted of a conjoint analysis–based model using a continuous improvement score based on absolute per cent change in core set measures, with thresholds for minimal, moderate, and major improvement.

scholarly journals Canadian Recommendations for Clinical Trials of Pharmacologic Interventions in Rheumatoid Arthritis: Inclusion Criteria and Study Design: Table 1.

2011 ◽  
Vol 38 (10) ◽  
pp. 2095-2104 ◽  
Author(s):  
JACOB KARSH ◽  
EDWARD C. KEYSTONE ◽  
BOULOS HARAOUI ◽  
J. CARTER THORNE ◽  
JANET E. POPE ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Disease Activity ◽  
Study Design ◽  
New Drugs ◽  
Nominal Group ◽  
Consensus Method ◽  
Inclusion Criteria ◽  
Design Features

Objective.Current clinical trial designs for pharmacologic interventions in rheumatoid arthritis (RA) do not reflect the innovations in RA diagnosis, treatment, and care in countries where new drugs are most often used. The objective of this project was to recommend revised entry criteria and other study design features for RA clinical trials.Methods.Recommendations were developed using a modified nominal group consensus method. Canadian Rheumatology Research Consortium (CRRC) members were polled to rank the greatest challenges to clinical trial recruitment in their practices. Initial recommendations were developed by an expert panel of rheumatology trialists and other experts. A scoping study methodology was then used to examine the evidence available to support or refute each initial recommendation. The potential influence of CRRC recommendations on primary outcomes in future trials was examined. Recommendations were finalized using a consensus process.Results.Recommendations for clinical trial inclusion criteria addressed measures of disease activity [Disease Activity Score 28 using erythrocyte sedimentation rate (DAS28-ESR) > 3.2 PLUS ≥ 3 tender joints using 28-joint count (TJC28) PLUS ≥ 3 swollen joint (SJC28) OR C-reactive protein (CRP) or ESR > upper limit of normal PLUS ≥ 3 TJC28 PLUS ≥ 3 SJC28], functional classification, disease classification and duration, and concomitant RA treatments. Additional recommendations regarding study design addressed rescue strategies and longterm extension.Conclusion.There is an urgent need to modify clinical trial inclusion criteria and other study design features to better reflect the current characteristics of people living with RA in the countries where the new drugs will be used.

Clinical outcomes

2016 ◽  
Author(s):  
Astrid van Tubergen ◽  
Robert Landewé
Keyword(s):  
Clinical Practice ◽  
Medical Care ◽  
Clinical Outcomes ◽  
International Society ◽  
Axial Spondyloarthritis ◽  
Response Criteria ◽  
The Core ◽  
Core Set ◽  
Core Sets ◽  
Chronic Course

In general, axial spondyloarthritis (axSpA) follows a chronic course, requiring regular medical care and monitoring. The outcome of axSpA may vary substantially due to heterogenic presentation. For both research and clinical practice, it is important to have relevant, reliable, validated instruments for measuring outcome, to evaluate patients in a standardized way and capture all disease aspects. The Assessment in SpondyloArthritis international Society has developed core sets and instruments to measure these domains, and recommends only the most important domains being measured with best available methods. This chapter provides an overview of the most important outcomes in axSpA and most commonly used instruments to measure these. Additional measures frequently used but not (yet) included in the core set are addressed, and several sets of response criteria applied in axSpA research described. This chapter also provides guidance in which setting (research versus practice) and with which frequency these measures can be used.

Pegylated Interferon Alpha-2a in 75 Patients with Myeloproliferative Neoplasms: A Single Center Experience

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 2818-2818 ◽  
Author(s):  
Krisstina L. Gowin ◽  
Tania Jain ◽  
Heidi E. Kosiorek ◽  
John Camoriano ◽  
Raoul Tibes ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Complete Remission ◽  
Interferon Alpha ◽  
Research Funding ◽  
Progressive Disease ◽  
Pegylated Interferon ◽  
Response Criteria ◽  
Hematologic Toxicity ◽  
Minor Response ◽  
Pegylated Interferon Alpha

Abstract Background: Pegylated interferon alpha-2a (Peg INF2a) has been demonstrated to be active therapy for high-risk essential thrombocythemia (ET), polycythemia vera (PV), and early myelofibrosis (MF). We retrospectively analyzed the outcomes of Peg INF2a therapy in myeloproliferative (MPN) patients treated outside the constraints of a clinical trial. Methods: Charts were analyzed for demographic and clinical data. Toxicity to therapy was assessed per CTCAE 3.0 criteria. Therapeutic responses for ET and PV were calculated by the revised ELN/IWG-MRT criteria including complete remission (CR), partial resmission (PR), no response (NR), or progressive disease (PD). Responses in MF were calculated by EUNMET: complete response (CR), major response (MR), moderate response (MoR), minor response (MiR) and NRand the revised IWG-MRT/ELN criteria: CR, PR, clinical improvement (CI), stable disease (SD) or PD. Results: Patients: 75 patients were identified overall. There were 36 PV patients (48%), 20 ET patients (26.7%), and 19 MF patients (25.6%). Thirteen MF patients were post-PV/ET MF. The median age at diagnosis was 51.5 yrs (range 28.8-75.1). JAK2 V617 mutation was present in 53 patients (70.7%). Median baseline hemoglobin (g/dL), leukocyte (x10 9), and platelet count (x109) for PV was 13.6, 8.6, 369, for ET was 12.5, 6.8, 517, and for MF was 11.4, 8.0, and 420, respectively. DIPSS risk category for the 19 MF patients: Low in 6 (31.5%) patients, Intermediate-1 in 3 (15.7%), Intermediate-2 in 8 (42%), and High in 2 (10.5%) patients. The majority of patients (82.2%) had received at least one prior cytoreductive therapy for their disease. Therapy: Median starting dose of Peg INF2a was 45 micrograms/week (range 45-90). The median peak dose was 90 micrograms/week (range 45-270). The median tolerated dose was 60 micrograms/week (range 5.6-180). The median duration of treatment was 24 months (range 3.6-85). Toxicity: Overall the Peg INF2a was well tolerated. Hematological toxicity included: leukopenia at grade 1 in 13 patients (17.3%), grade 2 in 5 patients (6.7%), and grade 3 in 1 patient (1.3%), anemia at grade 1 in 10 patients (13.3%) and grade 2 in 1 patient (1.3%), thrombocytopenia at grade 1 in 13 patients (17.3%) and grade 2 in 1 patient (1.3%). The most common non-hematologic toxicity included: fatigue at grade 1 in 14 patients (18.7%) and grade 2 in 4 patients (5.3%), transaminitis at grade 1 in 6 patients (8%) and grade 2 in 3 patients (4%), myalgias at grade 1 in 4 patients (5.3%). Response: See Table #1 PV/ET: 56 patients were evaluated by ELN/IWG-MRT criteria overall: A complete remission (CR) was seen in 8 patients (14.3%), a partial remission (PR) in 18 patients (32.1%), either a CR or PR in 18 patients (32.1%) when histologic remissions were unable to be documented due to lack of restaging bone marrow examination, no response in 11 patients (19.6%), and progressive disease in 1 patient (1.8%). Of the 12 patients receiving at least1 phlebotomy per month, 10 patients (83.3%) became phlebotomy independent with therapy. Of the 20 ET patients, 12 patients (60%) had platelet normalization (<400 x 109). MF: 19 patients were evaluated by IWG-ELN criteria: a PR was seen in 2 patients (10.5%), CI in 4 patients (21.1%), SD in 12 patients (63.2%), and PD in 1 patient (5.3%). Utilizing EUNMET critieria: 1 patient (5.3%) with a CR, 5 patients (26.3%) with a MR, 3 patients (15.8%) with MoR, 5 patients (26.3%) with MiR, and 5 patients (26.3%) with NR. Table 1. Peg INF2a Patient Responses RESPONSE Disease IWG-ELN response criteria: (Patient #, %) (Barosi, Blood 2013, Tefferi, Blood 2013) EUMET response criteria: (Patient #, %) (Barosi, Blood 2005) PV: N=36 CR: 3 pts (8.3%) PR: 14 pts (38.8%) CR/PR: 14 pts (38.8%) NR: 5 pts (13.8%) PD: 0 NA ET: N=20 CR: 5 pts (25%) PR: 4 pts (20%) CR/PR: 4 pts (20%) NR: 6 pts (30%) PD: 1 (5%) NA MF: N=19 IWG-ELN: PR: 2 pts (10.5%) CI: 4 pts (21.1%) SD: 12 pts (63.2%) PD: 1 pt (5.3%) EUNMET: CR: 1 pt (5.3%) MR: 5 pts (26.3%) MoR: 3 pts (15.8%) MiR: 5 pts (26.3%) NR: 5 pts (26.3%) NA: Not applicable Conclusions: Peg INF2a is active and well-tolerated when administered outside of the support of a clinical trial. Given the majority of patients had previously failed cytoreductive therapy these results substantiate prior reports of efficacy of Peg INF2a in MPNs. Disclosures Mesa: Pfizer: Research Funding; Gilead: Research Funding; Promedior: Research Funding; Novartis Pharmaceuticals Corporation: Consultancy; CTI Biopharma: Research Funding; Incyte Corporation: Research Funding; NS Pharma: Research Funding; Genentech: Research Funding.

scholarly journals Evaluation of RANO response criteria compared to clinician evaluation in WHO grade III anaplastic astrocytoma: implications for clinical trial reporting and patterns of failure

2015 ◽  
Vol 122 (1) ◽  
pp. 197-203 ◽  
Author(s):  
Tomas Kazda ◽  
John G. Hardie ◽  
Deanna H. Pafundi ◽  
Timothy J. Kaufmann ◽  
Debra H. Brinkmann ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Anaplastic Astrocytoma ◽  
Response Criteria ◽  
Patterns Of Failure ◽  
Who Grade ◽  
Who Grade Iii ◽  
Grade Iii

Prioritizing Pharmaceutical Activities

2012 ◽  
Vol 26 (4) ◽  
pp. 367-375 ◽  
Author(s):  
Sophie Renet ◽  
Cynthia Tanguay ◽  
Kevin Hall ◽  
Jean-François Bussières
Keyword(s):  
Health Outcomes ◽  
Hospital Pharmacy ◽  
Relative Weight ◽  
Primary Objective ◽  
Conclusive Evidence ◽  
Low Level ◽  
Simulated Environment ◽  
Core Set ◽  
Wide Range ◽  
Pharmacy Leaders

Objectives: The primary objective was to examine the consistency of prioritization decisions made by pharmacy residents in a simulated environment where the available resources are constrained. Secondary objectives were to rank the factors that influenced their prioritization and to compare the residents’ results with those of Canadian pharmacy leaders. Methods: We have developed a prioritization exercise that aims at evaluating how pharmaceutical activities are prioritized. The simulation was conducted with hospital pharmacy residents in 2 Quebec universities in 2011. Results: Residents covered a similar number of activities in the prioritization simulation (mean 27 of 32). Teams tended to favor a broad range of services delivered less comprehensively. Participants ranked “perception of the favorable impact of the activity on health outcomes” higher than “conclusive evidence available to support the decisions.” The relative weight attributed per domain was similar between pharmacy residents and pharmacy leaders, but their ranking of factors that influenced their decisions was different. Conclusions: Pharmacy residents opted to provide a wide range of services, but at a low level of comprehensiveness. The high variation between each team’s coverage per activity in this simulation supports the observation that pharmacy residents do not agree on a core set of pharmaceutical activities that should be prioritized.

scholarly journals Consensus recommendations for the uniform reporting of clinical trials: report of the International Myeloma Workshop Consensus Panel 1

Blood ◽  
2011 ◽  
Vol 117 (18) ◽  
pp. 4691-4695 ◽  
Author(s):  
S. Vincent Rajkumar ◽  
Jean-Luc Harousseau ◽  
Brian Durie ◽  
Kenneth C. Anderson ◽  
Meletios Dimopoulos ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Response Criteria ◽  
Current Response ◽  
Consensus Panel ◽  
Consensus Recommendations ◽  
Clinical Trial Results

Abstract It is essential that there be consistency in the conduct, analysis, and reporting of clinical trial results in myeloma. The goal of the International Myeloma Workshop Consensus Panel 1 was to develop a set of guidelines for the uniform reporting of clinical trial results in myeloma. This paper provides a summary of the current response criteria in myeloma, detailed definitions for patient populations, lines of therapy, and specific endpoints. We propose that future clinical trials in myeloma follow the guidelines for reporting results proposed in this manuscript.

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