[en] The goal of this document is to update the original OARSI recommendations specifically for the design, conduct, and reporting of clinical trials that target symptom or structure modification among individuals with knee osteoarthritis (OA). To develop recommendations for the design, conduct, and reporting of clinical trials for knee OA we initially drafted recommendations through an iterative process. Members of the working group included representatives from industry and academia. After the working group members reviewed a final draft, they scored the appropriateness for recommendations. After the members voted we calculated the median score among the nine members of the working group who completed the score. The document includes 25 recommendations regarding randomization, blocking and stratification, blinding, enhancing accuracy of patient-reported outcomes (PRO), selecting a study population and index knee, describing interventions, patient-reported and physical performance measures, structural outcome measures, biochemical biomarkers, and reporting recommendations. In summary, the working group identified 25 recommendations that represent the current best practices regarding clinical trials that target symptom or structure modification among individuals with knee OA. These updated recommendations incorporate novel technologies (e.g., magnetic resonance imaging (MRI)) and strategies to address the heterogeneity of knee OA.
Disciplines :
Rheumatology
Author, co-author :
McAlindon, Timothy E.
Driban, J. B.
Henrotin, Yves ; Université de Liège > Département des sciences de la motricité > Unité de recherche sur l'os et le cartilage (U.R.O.C.)
Hunter, D. J.
Jiang, G. L.
Skou, S. T.
Wang, S.
Schnitzer, T.
Language :
English
Title :
OARSI Clinical Trials Recommendations: Design, conduct, and reporting of clinical trials for knee osteoarthritis
Altman R., Brandt K., Hochberg M., Moskowitz R., Bellamy N., Bloch D.A., et al. Design and conduct of clinical trials in patients with osteoarthritis: recommendations from a task force of the Osteoarthritis Research Society. Results from a workshop. Osteoarthritis Cartilage 1996, 4:217-243.
Chan A.W., Altman D.G. Epidemiology and reporting of randomised trials published in PubMed journals. Lancet 2005, 365:1159-1162.
Moher D., Hopewell S., Schulz K.F., Montori V., Gotzsche P.C., Devereaux P.J., et al. CONSORT 2010 explanation and elaboration: updated guidelines for reporting parallel group randomised trials. BMJ 2010, 340:c869.
Jones B., Jarvis P., Lewis J.A., Ebbutt A.F. Trials to assess equivalence: the importance of rigorous methods. BMJ 1996, 313:36-39.
Schulz K.F., Chalmers I., Hayes R.J., Altman D.G. Empirical evidence of bias. Dimensions of methodological quality associated with estimates of treatment effects in controlled trials. JAMA 1995, 273:408-412.
Lachin J.M. Properties of simple randomization in clinical trials. Control Clin Trials 1988, 9:312-326.
Peto R., Pike M.C., Armitage P., Breslow N.E., Cox D.R., Howard S.V., et al. Design and analysis of randomized clinical trials requiring prolonged observation of each patient. I. Introduction and design. Br J Cancer 1976, 34:585-612.
Enas G.G., Enas N.H., Spradlin C.T., Wilson M.G., Wiltse C.G. Baseline comparability in clinical trials: prevention of "Poststudy Anxiety". Drug Inf J 1990, 24:541-548.
McAlindon T.E., Bannuru R.R., Sullivan M.C., Arden N.K., Berenbaum F., Bierma-Zeinstra S.M., et al. OARSI guidelines for the non-surgical management of knee osteoarthritis. Osteoarthritis Cartilage 2014, 22:363-388.
Schulz K.F., Grimes D.A. Blinding in randomised trials: hiding who got what. Lancet 2002, 359:696-700.
Wood L., Egger M., Gluud L.L., Schulz K.F., Juni P., Altman D.G., et al. Empirical evidence of bias in treatment effect estimates in controlled trials with different interventions and outcomes: meta-epidemiological study. BMJ 2008, 336:601-605.
Hróbjartsson A., Emanuelsson F., Skou Thomsen A.S., Hilden J., Brorson S. Bias due to lack of patient blinding in clinical trials. A systematic review of trials randomizing patients to blind and nonblind sub-studies. Int J Epidemiol 2014, 43:1272-1283.
Devereaux P.J., Manns B.J., Ghali W.A., Quan H., Lacchetti C., Montori V.M., et al. Physician interpretations and textbook definitions of blinding terminology in randomized controlled trials. JAMA 2001, 285:2000-2003.
Haahr M.T., Hrobjartsson A. Who is blinded in randomized clinical trials? A study of 200 trials and a survey of authors. Clin Trials 2006, 3:360-365.
The Cochrane Collaboration's Tool for Assessing Risk of Bias. (Accessed March 2015). http://ohg.cochrane.org/sites/ohg.cochrane.org/files/uploads/Risk%20of%20bias%20assessment%20tool.pdf.
Kam-Hansen S., Jakubowski M., Kelley J.M., Kirsch I., Hoaglin D.C., Kaptchuk T.J., et al. Altered placebo and drug labeling changes the outcome of episodic migraine attacks. Sci Transl Med 2014, 6:218ra5.
Wise R.A., Bartlett S.J., Brown E.D., Castro M., Cohen R., Holbrook J.T., et al. Randomized trial of the effect of drug presentation on asthma outcomes: the American Lung Association Asthma Clinical Research Centers. JAllergy Clin Immunol 2009, 124:436-444. 444e1-8.
Bannuru R.R., Schmid C.H., Sullivan M.C., Kent D.M., Wong J.B., McAlindon T.E. Differential response of placebo treatments in osteoarthritis trials: a systematic review and network meta-analysis. Osteoarthritis Cartilage 2014, 22(Supplement):S24-S25.
Levine J.D., Gordon N.C. Influence of the method of drug administration on analgesic response. Nature 1984, 312:755-756.
Buckalew L.W., Coffield K.E. An investigation of drug expectancy as a function of capsule color and size and preparation form. JClin Psychopharmacol 1982, 2:245-248.
Trijau S., Avouac J., Escalas C., Gossec L., Dougados M. Influence of flare design on symptomatic efficacy of non-steroidal anti-inflammatory drugs in osteoarthritis: a meta-analysis of randomized placebo-controlled trials. Osteoarthritis Cartilage 2010, 18:1012-1018.
Dworkin R.H., Turk D.C., Peirce-Sandner S., He H., McDermott M.P., Hochberg M.C., et al. Meta-analysis of assay sensitivity and study features in clinical trials of pharmacologic treatments for osteoarthritis pain. Arthritis Rheumatol 2014, 66:3327-3336.
Harris R.E., Williams D.A., McLean S.A., Sen A., Hufford M., Gendreau R.M., et al. Characterization and consequences of pain variability in individuals with fibromyalgia. Arthritis Rheum 2005, 52:3670-3674.
Farrar J.T., Troxel A.B., Haynes K., Gilron I., Kerns R.D., Katz N.P., et al. Effect of variability in the 7-day baseline pain diary on the assay sensitivity of neuropathic pain randomized clinical trials: an ACTTION study. Pain 2014, 155:1622-1631.
Riddle D.L., Stratford P.W. Unilateral vs bilateral symptomatic knee osteoarthritis: associations between pain intensity and function. Rheumatology (Oxford) 2013, 52:2229-2237.
Kahn T.L., Soheili A.C., Schwarzkopf R. Poor WOMAC scores in contralateral knee negatively impact TKA outcomes: data from the osteoarthritis initiative. JArthroplasty 2014, 29:1580-1585.
Lane N.E., Brandt K., Hawker G., Peeva E., Schreyer E., Tsuji W., et al. OARSI-FDA initiative: defining the disease state of osteoarthritis. Osteoarthritis Cartilage 2011, 19:478-482.
Gewandter J.S., Dworkin R.H., Turk D.C., McDermott M.P., Baron R., Gastonguay M.R., et al. Research designs for proof-of-concept chronic pain clinical trials: IMMPACT recommendations. Pain 2014, 155:1683-1695.
Dworkin R.H., Turk D.C., Peirce-Sandner S., Baron R., Bellamy N., Burke L.B., et al. Research design considerations for confirmatory chronic pain clinical trials: IMMPACT recommendations. Pain 2010, 149:177-193.
Dworkin R.H., Turk D.C., Peirce-Sandner S., Burke L.B., Farrar J.T., Gilron I., et al. Considerations for improving assay sensitivity in chronic pain clinical trials: IMMPACT recommendations. Pain 2012, 153:1148-1158.
Wolfe F., Hawley D.J., Peloso P.M., Wilson K., Anderson J. Back pain in osteoarthritis of the knee. Arthritis Care Res 1996, 9:376-383.
Oteo-Alvaro A., Ruiz-Iban M.A., Miguens X., Stern A., Villoria J., Sanchez-Magro I. High prevalence of neuropathic pain features in patients with knee osteoarthritis: a cross-sectional study. Pain Pract 2014.
Hochman J.R., Gagliese L., Davis A.M., Hawker G.A. Neuropathic pain symptoms in a community knee OA cohort. Osteoarthritis Cartilage 2011, 19:647-654.
Wasan A.D., Kaptchuk T.J., Davar G., Jamison R.N. The association between psychopathology and placebo analgesia in patients with discogenic low back pain. Pain Med 2006, 7:217-228.
Sullivan M.J., Thorn B., Haythornthwaite J.A., Keefe F., Martin M., Bradley L.A., et al. Theoretical perspectives on the relation between catastrophizing and pain. Clin J Pain 2001, 17:52-64.
Wolfe F. Determinants of WOMAC function, pain and stiffness scores: evidence for the role of low back pain, symptom counts, fatigue and depression in osteoarthritis, rheumatoid arthritis and fibromyalgia. Rheumatology (Oxford) 1999, 38:355-361.
European Medicines Agency Guideline on the Clinical Development of Medicinal Products Intended for the Treatment of Pain (Draft) 2013, (Accessed 24.07.14). http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/05/WC500143769.pdf.
United States Food and Drug Administration Guidance for Industry: Analgesic Indications: Developing Drug and Biological Products (Draft) 2014, (Accessed 24.07.14). http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM384691.pdf.
Suokas A.K., Walsh D.A., McWilliams D.F., Condon L., Moreton B., Wylde V., et al. Quantitative sensory testing in painful osteoarthritis: a systematic review and meta-analysis. Osteoarthritis Cartilage 2012, 20:1075-1085.
Mapp P.I., Walsh D.A. Mechanisms and targets of angiogenesis and nerve growth in osteoarthritis. Nat Rev Rheumatol 2012, 8:390-398.
Schaible H.G. Mechanisms of chronic pain in osteoarthritis. Curr Rheumatol Rep 2012, 14:549-556.
Kalunian K.C., Arendt-Nielsen L., Turkel C.C., DeGryse R., McAlindon T.E., Boon A.J., et al. Results from a single center, double-blind, randomized, placebo-controlled, parallel-group study of the efficacy and safety of intra-articular onabotulinumtoxina for osteoarthritis knee pain. Osteoarthritis Cartilage 2014, 22(Supplement):S192.
Arendt-Nielsen L., Jiang G.L., DeGryse R., Turkel C.C. Asingle center, double-blind, randomized, placebo-controlled, parallel-group study of the efficacy and safety of intra-articular onabotulinumtoxina as treatment for osteoarthritis knee pain: results from the experimental pain models. Osteoarthritis Cartilage 2014, 22(Supplement):S26.
Thakur M., Dickenson A.H., Baron R. Osteoarthritis pain: nociceptive or neuropathic?. Nat Rev Rheumatol 2014, 10:374-380.
Baron R., Forster M., Binder A. Subgrouping of patients with neuropathic pain according to pain-related sensory abnormalities: a first step to a stratified treatment approach. Lancet Neurol 2012, 11:999-1005.
Freynhagen R., Baron R., Gockel U., Tolle T.R. painDETECT: a new screening questionnaire to identify neuropathic components in patients with back pain. Curr Med Res Opin 2006, 22:1911-1920.
Bennett M.I., Smith B.H., Torrance N., Potter J. The S-LANSS score for identifying pain of predominantly neuropathic origin: validation for use in clinical and postal research. JPain 2005, 6:149-158.
Bouhassira D., Attal N., Fermanian J., Alchaar H., Gautron M., Masquelier E., et al. Development and validation of the neuropathic pain symptom inventory. Pain 2004, 108:248-257.
Bouhassira D., Attal N., Alchaar H., Boureau F., Brochet B., Bruxelle J., et al. Comparison of pain syndromes associated with nervous or somatic lesions and development of a new neuropathic pain diagnostic questionnaire (DN4). Pain 2005, 114:29-36.
Scholz J., Mannion R.J., Hord D.E., Griffin R.S., Rawal B., Zheng H., et al. Anovel tool for the assessment of pain: validation in low back pain. PLoS Med 2009, 6:e1000047.
Hawker G.A., Davis A.M., French M.R., Cibere J., Jordan J.M., March L., et al. Development and preliminary psychometric testing of a new OA pain measure - an OARSI/OMERACT initiative. Osteoarthritis Cartilage 2008, 16:409-414.
Schulz K.F., Altman D.G., Moher D. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. Int J Surg 2011, 9:672-677.
McAlindon T., LaValley M., Schneider E., Nuite M., Lee J.Y., Price L.L., et al. Effect of vitamin D supplementation on progression of knee pain and cartilage volume loss in patients with symptomatic osteoarthritis: a randomized controlled trial. JAMA 2013, 309:155-162.
The European Medicines Agency (EMEA) Reflection Paper on the Regulatory Guidance for the Use of Health-related Quality of Life (HRQL) Measures in the Evaluation of Medicinal Products 2005, (Accessed September 2014). http://www.ispor.org/workpaper/emea-hrql-guidance.pdf.
U.S.Food and Drug Administration Guidance for Industry - Patient-reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims 2009, (Accessed March 2015). http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf.
(FDA) FaDA Guidance for industry: patient report outcome measures: use in clinical medical product development to support labelling claims: draft guidance. Health Qual Life Outcomes 2006, 4.
Bellamy N., Kirwan J., Boers M., Brooks P., Strand V., Tugwell P., et al. Recommendations for a core set of outcome measures for future phase III clinical trials in knee, hip, and hand osteoarthritis. Consensus development at OMERACT III. JRheumatol 1997, 24:799-802.
Lo G., McAlindon T.E., Hawker G., Driban J., Price L.L., Song J., et al. Knee osteoarthritis symptom assessments that combine pain and activity level are superior. Arthritis Rheum 2012, 64:103.
Bruce B., Fries J.F. The Stanford Health Assessment Questionnaire: a review of its history, issues, progress, and documentation. JRheumatol 2003, 30:167-178.
Bruce B., Fries J.F. The Stanford Health Assessment Questionnaire: dimensions and practical applications. Health Qual Life Outcomes 2003, 1:20.
Bruce B., Fries J.F. The Health Assessment Questionnaire (HAQ). Clin Exp Rheumatol 2005, 23:S14-S18.
Bellamy N., Buchanan W.W., Goldsmith C.H., Campbell J., Stitt L.W. Validation study of WOMAC: a health status instrument for measuring clinically important patient relevant outcomes to antirheumatic drug therapy in patients with osteoarthritis of the hip or knee. JRheumatol 1988, 15:1833-1840.
Roos E.M., Lohmander L.S. The Knee injury and Osteoarthritis Outcome Score (KOOS): from joint injury to osteoarthritis. Health Qual Life Outcomes 2003, 1:64.
Roos E.M., Roos H.P., Lohmander L.S., Ekdahl C., Beynnon B.D. Knee Injury and Osteoarthritis Outcome Score (KOOS)-development of a self-administered outcome measure. JOrthop Sports Phys Ther 1998, 28:88-96.
Cella D., Riley W., Stone A., Rothrock N., Reeve B., Yount S., et al. The Patient-Reported Outcomes Measurement Information System (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005-2008. JClin Epidemiol 2010, 63:1179-1194.
Collins N.J., Misra D., Felson D.T., Crossley K.M., Roos E.M. Measures of knee function: International Knee Documentation Committee (IKDC) subjective knee evaluation form, Knee Injury and Osteoarthritis Outcome Score (KOOS), Knee Injury and Osteoarthritis Outcome Score Physical Function Short Form (KOOS-PS), Knee Outcome Survey Activities of Daily Living Scale (KOS-ADL), Lysholm Knee Scoring Scale, Oxford Knee Score (OKS), Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), Activity Rating Scale (ARS), and Tegner Activity Score (TAS). Arthritis Care Res (Hoboken) 2011, 63(Suppl 11):S208-S228.
Ware J.E., Sherbourne C.D. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care 1992, 30:473-483.
Ware J.E., Kosinski M., Dewey J.E., Gandek B. SF-36 Health Survey: Manual and Interpretation Guide 2000.
Bergner M., Bobbitt R.A., Carter W.B., Gilson B.S. The Sickness Impact Profile: development and final revision of a health status measure. Med Care 1981, 19:787-805.
Szende A., Williams A. Measuring Self-reported Population Health: An International Perspective Based on EQ-5D 2004, SpringMed Publishing, Budapest, Hungar.
EuroQol: a new facility for the measurement of health related quality of life. Health Policy 1990, 16:199-208. Group E.
Feeny D., Furlong W., Boyle M., Torrance G.W. Multi-attribute health status classification systems. Health Utilities Index. Pharmacoeconomics 1995, 7:490-502.
Torrance G.W., Furlong W., Feeny D., Boyle M. Multi-attribute preference functions. Health utilities Index. Pharmacoeconomics 1995, 7:503-520.
McDowell I., Newell C. Measuring Health. A Guide to Rating Scales and Questionnaires 1996, Oxford University Press Inc, New York. 2nd edn.
Streiner D.L., Norman G.R. Health Measurement Scales. A Practical Guide to Their Development and Use 2003, Oxford University Press Inc, New York. 3rd edn.
Rothstein J.M. On defining subjective and objective measurements. Phys Ther 1989, 69:577-579.
Foundations of Behavioral Research: Educational, Psychological, and Sociological Inquiry 1973, 491. Holt, Rinehart, & Winston, New York, General Book. F.N. Kerlinger (Ed.).
Dobson F., Hinman R.S., Roos E.M., Abbott J.H., Stratford P., Davis A.M., et al. OARSI recommended performance-based tests to assess physical function in people diagnosed with hip or knee osteoarthritis. Osteoarthritis Cartilage 2013, 21:1042-1052.
Whittaker J.L., Woodhouse L.J., Nettel-Aguirre A., Emery C.A. Outcomes associated with early post-traumatic osteoarthritis and other negative health consequences 3-10 years following knee joint injury in youth sport. Osteoarthritis Cartilage 2015.
United States Food and Drug Administration Guidance for Industry Clinical Development Programs for Drugs, Devices, and Biological Products Intended for the Treatment of Osteoarthritis (OA) 1999, (Accessed 24.07.14). http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071577.pdf.
Guidance for Industry: Clinical Development Programs for Drugs, Devices, and Biological Products Intended for the Treatment of Osteoarthritis (OA) 1999, United States Food and Drug Administration, Rockville, MD.
Weinstein A.M., Rome B.N., Reichmann W.M., Collins J.E., Burbine S.A., Thornhill T.S., et al. Estimating the Burden of total knee replacement in the United States. JBone Joint Surg Am 2013, 95:385-392.
Allen K.D., Golightly Y.M., Callahan L.F., Helmick C.G., Ibrahim S.A., Kwoh C.K., et al. Race and sex differences in willingness to undergo total joint replacement: the Johnston County Osteoarthritis Project. Arthritis Care Res (Hoboken) 2014, 66:1193-1202.
Mota R.E., Tarricone R., Ciani O., Bridges J.F., Drummond M. Determinants of demand for total hip and knee arthroplasty: a systematic literature review. BMC Health Serv Res 2012, 12:225.
Bederman S.S., Rosen C.D., Bhatia N.N., Kiester P.D., Gupta R. Drivers of surgery for the degenerative hip, knee, and spine: a systematic review. Clin Orthop Relat Res 2012, 470:1090-1105.
Escobar A., Quintana J.M., Bilbao A., Arostegui I., Lafuente I., Vidaurreta I. Responsiveness and clinically important differences for the WOMAC and SF-36 after total knee replacement. Osteoarthritis Cartilage 2007, 15:273-280.
Henrotin Y., Addison S., Kraus V., Deberg M. Type II collagen markers in osteoarthritis: what do they indicate?. Curr Opin Rheumatol 2007, 19:444-450.
Bauer D.C., Hunter D.J., Abramson S.B., Attur M., Corr M., Felson D., et al. Classification of osteoarthritis biomarkers: a proposed approach. Osteoarthritis Cartilage 2006, 14:723-727.
Wagner J.A., Williams S.A., Webster C.J. Biomarkers and surrogate end points for fit-for-purpose development and regulatory evaluation of new drugs. Clin Pharmacol Ther 2007, 81:104-107.
Kraus V.B., Burnett B., Coindreau J., Cottrell S., Eyre D., Gendreau M., et al. Application of biomarkers in the development of drugs intended for the treatment of osteoarthritis. Osteoarthritis Cartilage 2011, 19:515-542.
Iannone F., De B.C., Dell'Accio F., Covelli M., Patella V., Lo B.G., et al. Increased expression of nerve growth factor (NGF) and high affinity NGF receptor (p140 TrkA) in human osteoarthritic chondrocytes. Rheumatology 2002, 41:1413-1418.
Tse T., Williams R.J., Zarin D.A. Reporting "basic results" in ClinicalTrials.gov. Chest 2009, 136:295-303.