Dinesh Khanna1, Daniel E Furst2, Yannick Allanore3, Sangmee Bae1, Vijay Bodukam1, Philip J Clements1, Maurizio Cutolo1, Laszlo Czirjak1, Christopher P Denton1, Oliver Distler1, Ulrich A Walker1, Marco Matucci-Cerinic3, Ulf Müller-Ladner1, James R Seibold1, Manjit Singh1, Alan Tyndall1. 1. Division of Rheumatology, University of Michigan Scleroderma Program, Ann Arbor, MI, Division of Rheumatology, University of California at Los Angeles, Los Angeles, CA, USA, Department of Rheumatology A, Paris Descartes University, Cochin Institut, INSERM U1016, Cochin Hospital, Paris, France, Department of Medicine, Crozer Chester Medical Center, Upland, PA, USA, Research Laboratory and Academic Unit of Clinical Rheumatology, Department of Internal Medicine, University of Genoa, Genoa, Italy, Department of Immunology and Rheumatology, University of Pécs, Pécs, Hungary, Centre for Rheumatology, Royal Free Hospital, London, UK, Department of Rheumatology, University Hospital Zurich, Zurich, Deparment of Rheumatology, Basel University, Basel, Switzerland, Department of Biomedicine, Division of Rheumatology, Azienda Ospedaliero Universitaria Careggi, Department of Medicine, Denothe Centre, University of Florence, Florence, Italy, Department of Rheumatology and Clinical Immunology, Justus-Liebrig University Giessen, Kerckhoff Clinic, Bad Beuheinn, Germany, Scleroderma Research Consultants, Avon, CT and Department of Internal Medicine, Rochester General Health System, Rochester, NY, USA. 2. Division of Rheumatology, University of Michigan Scleroderma Program, Ann Arbor, MI, Division of Rheumatology, University of California at Los Angeles, Los Angeles, CA, USA, Department of Rheumatology A, Paris Descartes University, Cochin Institut, INSERM U1016, Cochin Hospital, Paris, France, Department of Medicine, Crozer Chester Medical Center, Upland, PA, USA, Research Laboratory and Academic Unit of Clinical Rheumatology, Department of Internal Medicine, University of Genoa, Genoa, Italy, Department of Immunology and Rheumatology, University of Pécs, Pécs, Hungary, Centre for Rheumatology, Royal Free Hospital, London, UK, Department of Rheumatology, University Hospital Zurich, Zurich, Deparment of Rheumatology, Basel University, Basel, Switzerland, Department of Biomedicine, Division of Rheumatology, Azienda Ospedaliero Universitaria Careggi, Department of Medicine, Denothe Centre, University of Florence, Florence, Italy, Department of Rheumatology and Clinical Immunology, Justus-Liebrig University Giessen, Kerckhoff Clinic, Bad Beuheinn, Germany, Scleroderma Research Consultants, Avon, CT and Department of Internal Medicine, Rochester General Health System, Rochester, NY, USA. defurst@mednet.ucla.edu. 3. Division of Rheumatology, University of Michigan Scleroderma Program, Ann Arbor, MI, Division of Rheumatology, University of California at Los Angeles, Los Angeles, CA, USA, Department of Rheumatology A, Paris Descartes University, Cochin Institut, INSERM U1016, Cochin Hospital, Paris, France, Department of Medicine, Crozer Chester Medical Center, Upland, PA, USA, Research Laboratory and Academic Unit of Clinical Rheumatology, Department of Internal Medicine, University of Genoa, Genoa, Italy, Department of Immunology and Rheumatology, University of Pécs, Pécs, Hungary, Centre for Rheumatology, Royal Free Hospital, London, UK, Department of Rheumatology, University Hospital Zurich, Zurich, Deparment of Rheumatology, Basel University, Basel, Switzerland, Department of Biomedicine, Division of Rheumatology, Azienda Ospedaliero Universitaria Careggi, Department of Medicine, Denothe Centre, University of Florence, Florence, Italy, Department of Rheumatology and Clinical Immunology, Justus-Liebrig University Giessen, Kerckhoff Clinic, Bad Beuheinn, Germany, Scleroderma Research Consultants, Avon, CT and Department of Internal Medicine, Rochester General Health System, Rochester, NY, USA. Division of Rheumatology, University of Michigan Scleroderma Program, Ann Arbor, MI, Division of Rheumatology, University of California at Los Angeles, Los Angeles, CA, USA, Department of Rheumatology A, Paris Descartes University, Cochin Institut, INSERM U1016, Cochin Hospital, Paris, France, Department of Medicine, Crozer Chester Medical Center, Upland, PA, USA, Research Laboratory and Academic Unit of Clinical Rheumatology, Department of Internal Medicine, University of Genoa, Genoa, Italy, Department of Immunology and Rheumatology, University of Pécs, Pécs, Hungary, Centre for Rheumatology, Royal Free Hospital, London, UK, Department of Rheumatology, University Hospital Zurich, Zurich, Deparment of Rheumatology, Basel University, Basel, Switzerland, Department of Biomedicine, Div
Abstract
OBJECTIVE: SSc is clinically and aetiopathogenically heterogeneous. Consensus standards for more uniform trial design and selection of outcome measures are needed. The objective of this study was to develop evidence-based points to consider (PTCs) for future clinical trials in SSc. METHODS: Thirteen international SSc experts experienced in SSc clinical trial design were invited to participate. One researcher with experience in systematic literature review and three trainees were also included. A systematic review using PubMed and the Cochrane Central Register of Controlled Trials was conducted and PTCs when designing clinical trials in SSc were developed. As part of that development we conducted an Internet-based Delphi exercise regarding the main points to be made in the consensus statement. Consensus was defined as achieving a median score of ≥7 of 9. RESULTS: By consensus, the experts decided to develop PTCs for each individual organ system. The current document provides a unifying outline on PTCs regarding general trial design, inclusion/exclusion criteria and analysis. Consensus was achieved regarding all the main points of the PTCs. CONCLUSION: Using European League Against Rheumatism suggestions for PTCs, a general outline for PTCs for controlled clinical trials in SSc was developed. Specific outlines for individual organ systems are to be published separately. This general outline should lead to more uniform and higher-quality trials and clearly delineate areas where further research is needed.
OBJECTIVE: SSc is clinically and aetiopathogenically heterogeneous. Consensus standards for more uniform trial design and selection of outcome measures are needed. The objective of this study was to develop evidence-based points to consider (PTCs) for future clinical trials in SSc. METHODS: Thirteen international SSc experts experienced in SSc clinical trial design were invited to participate. One researcher with experience in systematic literature review and three trainees were also included. A systematic review using PubMed and the Cochrane Central Register of Controlled Trials was conducted and PTCs when designing clinical trials in SSc were developed. As part of that development we conducted an Internet-based Delphi exercise regarding the main points to be made in the consensus statement. Consensus was defined as achieving a median score of ≥7 of 9. RESULTS: By consensus, the experts decided to develop PTCs for each individual organ system. The current document provides a unifying outline on PTCs regarding general trial design, inclusion/exclusion criteria and analysis. Consensus was achieved regarding all the main points of the PTCs. CONCLUSION: Using European League Against Rheumatism suggestions for PTCs, a general outline for PTCs for controlled clinical trials in SSc was developed. Specific outlines for individual organ systems are to be published separately. This general outline should lead to more uniform and higher-quality trials and clearly delineate areas where further research is needed.
Authors: G Valentini; A Della Rossa; S Bombardieri; W Bencivelli; A J Silman; S D'Angelo; M M Cerinic; J F Belch; C M Black; P Bruhlmann; L Czirják; A De Luca; A A Drosos; C Ferri; A Gabrielli; R Giacomelli; G Hayem; M Inanc; N J McHugh; H Nielsen; M Rosada; R Scorza; J Stork; A Sysa; F H van den Hoogen; P J Vlachoyiannopoulos Journal: Ann Rheum Dis Date: 2001-06 Impact factor: 19.103
Authors: Daniel E Furst; Dinesh Khanna; Marco Mattucci-Cerinic; Alan J Silman; Peter A Merkel; Ivan Foeldvari Journal: J Rheumatol Date: 2005-12 Impact factor: 4.666
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Authors: Dinesh Khanna; Daniel E Furst; Paul Maranian; James R Seibold; Ann Impens; Maureen D Mayes; Philip J Clements; Terri Getzug; Ron D Hays Journal: J Rheumatol Date: 2011-07-01 Impact factor: 4.666
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Authors: P J Clements; D E Furst; W K Wong; M Mayes; B White; F Wigley; M H Weisman; W Barr; L W Moreland; T A Medsger; V Steen; R W Martin; D Collier; A Weinstein; E Lally; J Varga; S Weiner; B Andrews; M Abeles; J R Seibold Journal: Arthritis Rheum Date: 1999-06
Authors: J Avouac; J Fransen; U A Walker; V Riccieri; V Smith; C Muller; I Miniati; I H Tarner; S Bellando Randone; M Cutolo; Y Allanore; O Distler; G Valentini; L Czirjak; U Müller-Ladner; D E Furst; A Tyndall; M Matucci-Cerinic Journal: Ann Rheum Dis Date: 2010-11-15 Impact factor: 19.103
Authors: Dinesh Khanna; James Seibold; Jonathan Goldin; Donald P Tashkin; Daniel E Furst; Athol Wells Journal: Rheumatology (Oxford) Date: 2017-09-01 Impact factor: 7.580
Authors: Femke C C van Rhijn-Brouwer; Hendrik Gremmels; Joost O Fledderus; Arnold H Schuurman; Femke Bonte-Mineur; Madelon C Vonk; Alexandre E Voskuyl; Jeska K de Vries-Bouwstra; J Henk Coert; Timothy R D J Radstake; Jacob M van Laar; Marianne C Verhaar Journal: BMJ Open Date: 2018-08-20 Impact factor: 2.692