Purpose of reviewTo describe the design and validation of current outcome measures for antineutrophil cytoplasmic antibody-associated vasculitis, and provide insight into ongoing outcome measure development for future randomized controlled trials (RCTs).Recent findingsSubstantial evidence now supports that patient-reported outcomes (PROs) capture unique disease components not represented in current physician-based measures. Further incorporation of formal disease assessment in the setting of RCT will both improve disease assessment in terms of adding new important disease domains and could improve psychometric properties of outcome measures and, therefore, increase trial efficiency.SummaryOptimal outcome measures for AAV promote identification of therapeutic agents that treat all important disease manifestations.
机构:
Addenbrookes Hosp, Vasculitis & Lupus Clin, Cambridge, England
Lund Univ, Dept Clin Sci Lund, Rheumatol Sect, Lund, SwedenLinkoping Univ, Dept Med & Nephrol, Linkoping, Sweden
Mohammad, Aladdin J.
Segelmark, Marten
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Linkoping Univ, Dept Med & Nephrol, Linkoping, SwedenLinkoping Univ, Dept Med & Nephrol, Linkoping, Sweden
Segelmark, Marten
Smith, Rona
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Addenbrookes Hosp, Vasculitis & Lupus Clin, Cambridge, EnglandLinkoping Univ, Dept Med & Nephrol, Linkoping, Sweden
Smith, Rona
Englund, Martin
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Boston Univ, Sch Med, Clin Epidemiol Res & Training Unit, Boston, MA 02118 USA
Lund Univ, Dept Clin Sci Lund, Clin Epidemiol Unit, Orthoped, Lund, SwedenLinkoping Univ, Dept Med & Nephrol, Linkoping, Sweden
Englund, Martin
Nilsson, Jan-Ake
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Lund Univ, Dept Clin Sci Lund, Sect Rheumatol, Lund, SwedenLinkoping Univ, Dept Med & Nephrol, Linkoping, Sweden
Nilsson, Jan-Ake
Westman, Kerstin
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Lund Univ, Dept Clin Sci Lund, Sect Rheumatol, Lund, SwedenLinkoping Univ, Dept Med & Nephrol, Linkoping, Sweden
Westman, Kerstin
Merkel, Peter A.
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Univ Penn, Penn Vasculitis Ctr, Div Rheumatol, Philadelphia, PA 19104 USA
Univ Penn, Dept Biostat & Epidemiol, Philadelphia, PA 19104 USALinkoping Univ, Dept Med & Nephrol, Linkoping, Sweden
Merkel, Peter A.
Jayne, David R. W.
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Addenbrookes Hosp, Vasculitis & Lupus Clin, Cambridge, EnglandLinkoping Univ, Dept Med & Nephrol, Linkoping, Sweden
机构:
Thomas Jefferson Univ, Div Rheumatol, Dept Med, Philadelphia, PA 19107 USAThomas Jefferson Univ, Div Rheumatol, Dept Med, Philadelphia, PA 19107 USA
Jacobs-Kosmin, D
Derk, CT
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Thomas Jefferson Univ, Div Rheumatol, Dept Med, Philadelphia, PA 19107 USAThomas Jefferson Univ, Div Rheumatol, Dept Med, Philadelphia, PA 19107 USA
Derk, CT
Sandorfi, N
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机构:
Thomas Jefferson Univ, Div Rheumatol, Dept Med, Philadelphia, PA 19107 USAThomas Jefferson Univ, Div Rheumatol, Dept Med, Philadelphia, PA 19107 USA