Guidelines for the conduct of pharmacological clinical trials in hand osteoarthritis: Consensus of a Working Group of the European Society on Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO).
Reginster, Jean-Yves; Arden, Nigel K.; Haugen, Ida K.et al.
2018 • In Seminars in Arthritis and Rheumatism, 48, p. 1-8
Clinical trials; Guidelines; Hand osteoarthritis; Pharmacological treatment
Abstract :
[en] OBJECTIVES: To gather expert opinion on the conduct of clinical trials that will facilitate regulatory review and approval of appropriate efficacious pharmacological treatments for hand osteoarthritis (OA), an area of high unmet clinical need. METHODS: The European Society on Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal diseases (ESCEO) organized a working group under the auspices of the International Osteoporosis Foundation (IOF) and the World Health Organization (WHO). RESULTS: This consensus guideline is intended to provide a reference tool for practice, and should allow for better standardization of the conduct of clinical trials in hand OA. Hand OA is a heterogeneous disease affecting different, and often multiple, joints of the thumb and fingers. It was recognized that the various phenotypes and limitations of diagnostic criteria may make the results of hand OA trials difficult to interpret. Nonetheless, practical recommendations for the conduct of clinical trials of both symptom and structure modifying drugs are outlined in this consensus statement, including guidance on study design, execution, and analysis. CONCLUSIONS: While the working group acknowledges that the methodology for performing clinical trials in hand OA will evolve as knowledge of the disease increases, it is hoped that this guidance will support the development of new pharmacological treatments targeting hand OA.
Disciplines :
Public health, health care sciences & services Laboratory medicine & medical technology Rheumatology
Author, co-author :
Reginster, Jean-Yves ; Université de Liège - ULiège > Département des sciences de la santé publique > Santé publique, Epidémiologie et Economie de la santé
Arden, Nigel K.
Haugen, Ida K.
Rannou, Francois
Cavalier, Etienne ; Université de Liège - ULiège > Département de pharmacie > Chimie médicale
Bruyère, Olivier ; Université de Liège - ULiège > Département des sciences de la santé publique > Santé publique, Epidémiologie et Economie de la santé
Guidelines for the conduct of pharmacological clinical trials in hand osteoarthritis: Consensus of a Working Group of the European Society on Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO).
Publication date :
2018
Journal title :
Seminars in Arthritis and Rheumatism
ISSN :
0049-0172
eISSN :
1532-866X
Publisher :
W. B. Saunders Co., United Kingdom
Volume :
48
Pages :
1-8
Peer reviewed :
Peer Reviewed verified by ORBi
Commentary :
Copyright (c) 2017 The Authors. Published by Elsevier Inc. All rights reserved.
Pereira, D., Peleteiro, B., Araujo, J., Branco, J., Santos, R.A., Ramos, E., The effect of osteoarthritis definition on prevalence and incidence estimates: a systematic review. Osteoarthritis Cartilage 19 (2011), 1270–1285.
Haugen, I.K., Englund, M., Aliabadi, P., Niu, J., Clancy, M., Kvien, T.K., et al. Prevalence, incidence and progression of hand osteoarthritis in the general population: the Framingham Osteoarthritis Study. Ann Rheum Dis 70 (2011), 1581–1586.
Kloppenburg, M., Kwok, W.Y., Hand osteoarthritis—a heterogeneous disorder. Nat Rev Rheumatol 8 (2011), 22–31.
van der Pas, S., Castell, M.V., Cooper, C., Denkinger, M., Dennison, E.M., Edwards, M.H., et al. European project on osteoarthritis: design of a six-cohort study on the personal and societal burden of osteoarthritis in an older European population. BMC Musculoskelet Disord, 14, 2013, 138.
van Schoor, N.M., Zambon, S., Castell, M.V., Cooper, C., Denkinger, M., Dennison, E.M., et al. Impact of clinical osteoarthritis of the hip, knee and hand on self-rated health in six European countries: the European Project on OSteoArthritis. Qual Life Res 25 (2016), 1423–1432.
Lue, S., Koppikar, S., Shaikh, K., Mahendira, D., Towheed, T.E., Systematic review of non-surgical therapies for osteoarthritis of the hand: an update. Osteoarthritis Cartilage 25 (2017), 1379–1389.
Mahendira, D., Towheed, T.E., Systematic review of non-surgical therapies for osteoarthritis of the hand: an update. Osteoarthritis Cartilage 17 (2009), 1263–1268.
Kloppenburg, M., Maheu, E., Kraus, V.B., Cicuttini, F., Doherty, M., Dreiser, R.L., et al. OARSI Clinical Trials Recommendations: design and conduct of clinical trials for hand osteoarthritis. Osteoarthritis Cartilage 23 (2015), 772–786.
European Agency for the Evaluation of Medicinal Products (EMA), Committee for Medicinal Products for Human Use (CHMP), Committee for Proprietary Medicinal Products (CPMP). Guideline on Clinical Investigation of Medicinal Products Used in the Treatment of Osteoarthritis. CPMP/EWP/784/97 Rev.1, 2010, EMA, London.
Reginster, J.Y., Reiter-Niesert, S., Bruyere, O., Berenbaum, F., Brandi, M.L., Branco, J., et al. Recommendations for an update of the 2010 European regulatory guideline on clinical investigation of medicinal products used in the treatment of osteoarthritis and reflections about related clinically relevant outcomes: expert consensus statement. Osteoarthritis Cartilage 23 (2015), 2086–2093.
Bruyere, O., Cooper, C., Pelletier, J.P., Branco, J., Luisa Brandi, M., Guillemin, F., et al. An algorithm recommendation for the management of knee osteoarthritis in Europe and internationally: a report from a task force of the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO). Semin Arthritis Rheum 44 (2014), 253–263.
Zhang, W., Doherty, M., Leeb, B.F., Alekseeva, L., Arden, N.K., Bijlsma, J.W., et al. EULAR evidence based recommendations for the management of hand osteoarthritis: report of a Task Force of the EULAR Standing Committee for International Clinical Studies Including Therapeutics (ESCISIT). Ann Rheum Dis 66 (2007), 377–388.
Hochberg, M.C., Altman, R.D., April, K.T., Benkhalti, M., Guyatt, G., McGowan, J., et al. American College of Rheumatology 2012 recommendations for the use of nonpharmacologic and pharmacologic therapies in osteoarthritis of the hand, hip, and knee. Arthritis Care Res (Hoboken) 64 (2012), 465–474.
Marshall, M., van der Windt, D., Nicholls, E., Myers, H., Hay, E., Dziedzic, K., Radiographic hand osteoarthritis: patterns and associations with hand pain and function in a community-dwelling sample. Osteoarthritis Cartilage 17 (2009), 1440–1447.
Cooper, C., Egger, P., Coggon, D., Hart, D.J., Masud, T., Cicuttini, F., et al. Generalized osteoarthritis in women: pattern of joint involvement and approaches to definition for epidemiological studies. J Rheumatol 23 (1996), 1938–1942.
Zhang, W., Doherty, M., Leeb, B.F., Alekseeva, L., Arden, N.K., Bijlsma, J.W., et al. EULAR evidence-based recommendations for the diagnosis of hand osteoarthritis: report of a task force of ESCISIT. Ann Rheum Dis 68 (2009), 8–17.
Marshall, M., Peat, G., Nicholls, E., van der Windt, D., Myers, H., Dziedzic, K., Subsets of symptomatic hand osteoarthritis in community-dwelling older adults in the United Kingdom: prevalence, inter-relationships, risk factor profiles and clinical characteristics at baseline and 3-years. Osteoarthritis Cartilage 21 (2013), 1674–1684.
Bijsterbosch, J., Visser, W., Kroon, H.M., Stamm, T., Meulenbelt, I., Huizinga, T.W., et al. Thumb base involvement in symptomatic hand osteoarthritis is associated with more pain and functional disability. Ann Rheum Dis 69 (2010), 585–587.
Rannou, F., Dimet, J., Boutron, I., Baron, G., Fayad, F., Mace, Y., et al. Splint for base-of-thumb osteoarthritis: a randomized trial. Ann Intern Med 150 (2009), 661–669.
Kjeken, I., Smedslund, G., Moe, R.H., Slatkowsky-Christensen, B., Uhlig, T., Hagen, K.B., Systematic review of design and effects of splints and exercise programs in hand osteoarthritis. Arthritis Care Res (Hoboken) 63 (2011), 834–848.
Ye, L., Kalichman, L., Spittle, A., Dobson, F., Bennell, K., Effects of rehabilitative interventions on pain, function and physical impairments in people with hand osteoarthritis: a systematic review. Arthritis Res Ther, 13, 2011, R28.
Jonsson, H., Valtysdottir, S.T., Hypermobility features in patients with hand osteoarthritis. Osteoarthritis Cartilage 3 (1995), 1–5.
Kwok, W.Y., Kloppenburg, M., Marshall, M., Nicholls, E., Rosendaal, F.R., van der Windt, D.A., et al. Comparison of clinical burden between patients with erosive hand osteoarthritis and inflammatory arthritis in symptomatic community-dwelling adults: the Keele clinical assessment studies. Rheumatology (Oxford) 52 (2013), 2260–2267.
Kwok, W.Y., Kloppenburg, M., Marshall, M., Nicholls, E., Rosendaal, F.R., Peat, G., The prevalence of erosive osteoarthritis in carpometacarpal joints and its clinical burden in symptomatic community-dwelling adults. Osteoarthritis Cartilage 22 (2014), 756–763.
Haugen, I.K., Slatkowsky-Christensen, B., Boyesen, P., van der Heijde, D., Kvien, T.K., Cross-sectional and longitudinal associations between radiographic features and measures of pain and physical function in hand osteoarthritis. Osteoarthritis Cartilage 21 (2013), 1191–1198.
Haugen, I.K., Mathiessen, A., Slatkowsky-Christensen, B., Magnusson, K., Boyesen, P., Sesseng, S., et al. Synovitis and radiographic progression in non-erosive and erosive hand osteoarthritis: is erosive hand osteoarthritis a separate inflammatory phenotype?. Osteoarthritis Cartilage 24 (2016), 647–654.
Haugen, I.K., Boyesen, P., Slatkowsky-Christensen, B., Sesseng, S., van der Heijde, D., Kvien, T.K., Associations between MRI-defined synovitis, bone marrow lesions and structural features and measures of pain and physical function in hand osteoarthritis. Ann Rheum Dis 71 (2012), 899–904.
Haugen, I.K., Slatkowsky Christensen, B., Boyesen, P., Sesseng, S., van der Heijde, D., Kvien, T.K., Increasing synovitis and bone marrow lesions are associated with incident joint tenderness in hand osteoarthritis. Ann Rheum Dis 75 (2016), 702–708.
Kortekaas, M.C., Kwok, W.Y., Reijnierse, M., Huizinga, T.W., Kloppenburg, M., In erosive hand osteoarthritis more inflammatory signs on ultrasound are found than in the rest of hand osteoarthritis. Ann Rheum Dis 72 (2013), 930–934.
Gazeley, D.J., Yeturi, S., Patel, P.J., Rosenthal, A.K., Erosive osteoarthritis: A systematic analysis of definitions used in the literature. Semin Arthritis Rheum 46 (2017), 395–403.
Kwok, W.Y., Kloppenburg, M., Rosendaal, F.R., van Meurs, J.B., Hofman, A., Bierma-Zeinstra, S.M., Erosive hand osteoarthritis: its prevalence and clinical impact in the general population and symptomatic hand osteoarthritis. Ann Rheum Dis 70 (2011), 1238–1242.
Verbruggen, G., Veys, E.M., Numerical scoring systems for the anatomic evolution of osteoarthritis of the finger joints. Arthritis Rheum 39 (1996), 308–320.
Altman, R.D., Gold, G.E., Atlas of individual radiographic features in osteoarthritis, revised. Osteoarthritis Cartilage 15:Suppl. A (2007), A1–A56.
Altman, R., Alarcon, G., Appelrouth, D., Bloch, D., Borenstein, D., Brandt, K., et al. The American College of Rheumatology criteria for the classification and reporting of osteoarthritis of the hand. Arthritis Rheum 33 (1990), 1601–1610.
Kellgren, J.H., Lawrence, J.S., Radiological assessment of osteo-arthrosis. Ann Rheum Dis 16 (1957), 494–502.
Hunter, D.J., Arden, N., Cicuttini, F., Crema, M.D., Dardzinski, B., Duryea, J., et al. OARSI Clinical Trials Recommendations: hand imaging in clinical trials in osteoarthritis. Osteoarthritis Cartilage 23 (2015), 732–746.
Maheu, E., Altman, R.D., Bloch, D.A., Doherty, M., Hochberg, M., Mannoni, A., et al. Design and conduct of clinical trials in patients with osteoarthritis of the hand: recommendations from a task force of the Osteoarthritis Research Society International. Osteoarthritis Cartilage 14 (2006), 303–322.
Visser, A.W., Boyesen, P., Haugen, I.K., Schoones, J.W., van der Heijde, D.M., Rosendaal, F.R., et al. Instruments measuring pain, physical function, or patient's global assessment in hand osteoarthritis: a systematic literature search. J Rheumatol 42 (2015), 2118–2134.
Duruoz, M.T., Poiraudeau, S., Fermanian, J., Menkes, C.J., Amor, B., Dougados, M., et al. Development and validation of a rheumatoid hand functional disability scale that assesses functional handicap. J Rheumatol 23 (1996), 1167–1172.
Bellamy, N., Campbell, J., Haraoui, B., Buchbinder, R., Hobby, K., Roth, J.H., et al. Dimensionality and clinical importance of pain and disability in hand osteoarthritis: Development of the Australian/Canadian (AUSCAN) Osteoarthritis Hand Index. Osteoarthritis Cartilage 10 (2002), 855–862.
Dreiser, R.L., Maheu, E., Guillou, G.B., Sensitivity to change of the functional index for hand osteoarthritis. Osteoarthritis Cartilage 8:Suppl. A (2000), S25–S28.
Dreiser, R.L., Maheu, E., Guillou, G.B., Caspard, H., Grouin, J.M., Validation of an algofunctional index for osteoarthritis of the hand. Rev Rhum Engl Ed 62 (1995), 43S–53S.
Fries, J.F., Spitz, P., Kraines, R.G., Holman, H.R., Measurement of patient outcome in arthritis. Arthritis Rheum 23 (1980), 137–145.
Dominick, K.L., Jordan, J.M., Renner, J.B., Kraus, V.B., Relationship of radiographic and clinical variables to pinch and grip strength among individuals with osteoarthritis. Arthritis Rheum 52 (2005), 1424–1430.
Bellamy, N., Hochberg, M., Tubach, F., Martin-Mola, E., Awada, H., Bombardier, C., et al. Development of multinational definitions of minimal clinically important improvement and patient acceptable symptomatic state in osteoarthritis. Arthritis Care Res (Hoboken) 67 (2015), 972–980.
Lotz, M., Martel-Pelletier, J., Christiansen, C., Brandi, M.L., Bruyere, O., Chapurlat, R., et al. Value of biomarkers in osteoarthritis: current status and perspectives. Ann Rheum Dis 72 (2013), 1756–1763.
Kraus, V.B., Collins, J.E., Hargrove, D., Losina, E., Nevitt, M., Katz, J.N., et al. Predictive validity of biochemical biomarkers in knee osteoarthritis: data from the FNIH OA Biomarkers Consortium. Ann Rheum Dis 76 (2017), 186–195.
Bijsterbosch, J., Haugen, I.K., Malines, C., Maheu, E., Rosendaal, F.R., Watt, I., et al. Reliability, sensitivity to change and feasibility of three radiographic scoring methods for hand osteoarthritis. Ann Rheum Dis 70 (2011), 1465–1467.
Damman, W., Kortekaas, M.C., Stoel, B.C., van't Klooster, R., Wolterbeek, R., Rosendaal, F.R., et al. Sensitivity-to-change and validity of semi-automatic joint space width measurements in hand osteoarthritis: a follow-up study. Osteoarthritis Cartilage 24 (2016), 1172–1179.
Visser, A.W., Boyesen, P., Haugen, I.K., Schoones, J.W., van der Heijde, D.M., Rosendaal, F.R., et al. Radiographic scoring methods in hand osteoarthritis—a systematic literature search and descriptive review. Osteoarthritis Cartilage 22 (2014), 1710–1723.
Verbruggen, G., Wittoek, R., Vander Cruyssen, B., Elewaut, D., Morbid anatomy of ‘erosive osteoarthritis’ of the interphalangeal finger joints: an optimised scoring system to monitor disease progression in affected joints. Ann Rheum Dis 69 (2010), 862–867.
Kloppenburg, M., Boyesen, P., Visser, A.W., Haugen, I.K., Boers, M., Boonen, A., et al. Report from the OMERACT Hand Osteoarthritis Working Group: set of core domains and preliminary set of instruments for use in clinical trials and observational studies. J Rheumatol 42 (2015), 2190–2197.
Haugen, I.K., Lillegraven, S., Slatkowsky-Christensen, B., Haavardsholm, E.A., Sesseng, S., Kvien, T.K., et al. Hand osteoarthritis and MRI: development and first validation step of the proposed Oslo Hand Osteoarthritis MRI score. Ann Rheum Dis 70 (2011), 1033–1038.
Haugen, I.K., Ostergaard, M., Eshed, I., McQueen, F.M., Bird, P., Gandjbakhch, F., et al. Iterative development and reliability of the OMERACT hand osteoarthritis MRI scoring system. J Rheumatol 41 (2014), 386–391.
Kroon, F.P., Conaghan, P.G., Foltz, V., Gandjbakhch, F., Peterfy, C., Eshed, I., et al. Development and reliability of the OMERACT thumb base osteoarthritis magnetic resonance imaging scoring system. J Rheumatol 44 (2017), 1694–1698.
Haugen, I.K., Boyesen, P., Imaging modalities in hand osteoarthritis—and perspectives of conventional radiography, magnetic resonance imaging, and ultrasonography. Arthritis Res Ther, 13, 2011, 248.
Haugen, I.K., Hammer, H.B., Role of modern imaging techniques in hand osteoarthritis research and clinical practice. Curr Rheumatol Rep, 16, 2014, 399.
Cooper, C., Adachi, J.D., Bardin, T., Berenbaum, F., Flamion, B., Jonsson, H., et al. How to define responders in osteoarthritis. Curr Med Res Opin 29 (2013), 719–729.
Maheu, E., Cadet, C., Gueneugues, S., Ravaud, P., Dougados, M., Reproducibility and sensitivity to change of four scoring methods for the radiological assessment of osteoarthritis of the hand. Ann Rheum Dis 66 (2007), 464–469.
Hart, D.J., Spector, T.D., Kellgren & Lawrence grade 1 osteophytes in the knee—doubtful or definite?. Osteoarthritis Cartilage 11 (2003), 149–150.
Kallman, D.A., Wigley, F.M., Scott, W.W. Jr, Hochberg, M.C., Tobin, J.D., New radiographic grading scales for osteoarthritis of the hand. Reliability for determining prevalence and progression. Arthritis Rheum 32 (1989), 1584–1591.
