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Measures of Response in Clinical Trials of Systemic Sclerosis: The Combined Response Index for Systemic Sclerosis (CRISS) and Outcome Measures in Pulmonary Arterial Hypertension Related to Systemic Sclerosis (EPOSS)

Identifieur interne : 008225 ( Main/Exploration ); précédent : 008224; suivant : 008226

Measures of Response in Clinical Trials of Systemic Sclerosis: The Combined Response Index for Systemic Sclerosis (CRISS) and Outcome Measures in Pulmonary Arterial Hypertension Related to Systemic Sclerosis (EPOSS)

Auteurs : Dinesh Khanna [États-Unis, Suisse, France, Allemagne, Royaume-Uni, Pologne, Italie, Australie, Canada] ; Oliver Distler ; Jerome Avouac ; Frank Behrens ; Philip J. Clements ; Christopher Denton ; Ivan Foeldvari ; Edward Giannini ; Doerte Huscher ; Otylia Kowal-Bielecka ; Daniel Lovell ; Marco Matucci-Cerinic ; Maureen Mayes ; Peter A. Merkel ; Peter Nash ; Christian F. Opitz ; David Pittrow ; Lewis Rubin ; James R. Seibold ; Virginia Steen ; C. Vibeke Strand ; Peter S. Tugwell ; John Varga ; Angela Zink ; Daniel E. Furst

Source :

RBID : Pascal:09-0429665

Descripteurs français

English descriptors

Abstract

There have been steady efforts to develop a combined response index for systemic sclerosis (CRISS). A parallel and equally successful effort has been made by an Expert Panel on Outcome Measures in PAH related to Systemic Sclerosis (EPOSS) to measure effect in treatment of pulmonary arterial hypertension of systemic sclerosis (PAH-SSc). CRISS conducted a Delphi process combined with expert review to identify 11 candidate domains for inclusion in a core set of outcomes for SSc clinical trials: soluble biomarkers, cardiac, digital ulcers, gastrointestinal, global health, health related quality of life (HRQOL) and function, musculoskeletal, pulmonary, Raynaud's, renal, and skin. Tools within domains were also agreed upon. Concentrating on one aspect of disease, PAH, EPOSS also conducted a Delphi process and judged the following domains as the most appropriate for randomized controlled trials in PAH-SSc: lung vascular/pulmonary arterial pressure, cardiac function, exercise testing; severity of dyspnea, discontinuation of treatment; quality of life/activities of daily living; global state; and survival. Possible useful tools within each domain were also agreed on. Patient derived, physician derived, and objective measures of response will be included and combined with the idea that each reflects different aspects of PAH (EPOSS) and overall disease (CRISS) although this assumption may not prove true and can be separated if statistically and clinically valid to do so. In either case, prospective studies will require measurement of all domains, and tools are required and will be developed to define appropriate combined measures of response. CRISS and EPOSS are being developed through the OMERACT process. Through Delphi process and literature review significant progress has been made for both indices, and prospective data are being collected.


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Le document en format XML

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<name sortKey="Zink, Angela" sort="Zink, Angela" uniqKey="Zink A" first="Angela" last="Zink">Angela Zink</name>
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<name sortKey="Furst, Daniel E" sort="Furst, Daniel E" uniqKey="Furst D" first="Daniel E." last="Furst">Daniel E. Furst</name>
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<s1>Division of Immunology, Stanfard University</s1>
<s2>Portola Valley, CA</s2>
<s3>USA</s3>
</inist:fA14>
<country>États-Unis</country>
<wicri:noRegion>Portola Valley, CA</wicri:noRegion>
</affiliation>
<affiliation wicri:level="1">
<inist:fA14 i1="20">
<s1>Institute of Population Health</s1>
<s2>Ottawa, ON</s2>
<s3>CAN</s3>
</inist:fA14>
<country>Canada</country>
<wicri:noRegion>Institute of Population Health</wicri:noRegion>
</affiliation>
<affiliation wicri:level="1">
<inist:fA14 i1="21">
<s1>Feinberg School of Medicine, Northwestern School of Medicine</s1>
<s2>Chicago, IL</s2>
<s3>USA</s3>
</inist:fA14>
<country>États-Unis</country>
<wicri:noRegion>Chicago, IL</wicri:noRegion>
</affiliation>
<affiliation wicri:level="3">
<inist:fA14 i1="22">
<s1>German Rheumatism Research Centre</s1>
<s2>Berlin</s2>
<s3>DEU</s3>
</inist:fA14>
<country>Allemagne</country>
<placeName>
<region type="land" nuts="3">Berlin</region>
<settlement type="city">Berlin</settlement>
</placeName>
</affiliation>
</author>
<author>
<name sortKey="Distler, Oliver" sort="Distler, Oliver" uniqKey="Distler O" first="Oliver" last="Distler">Oliver Distler</name>
</author>
<author>
<name sortKey="Avouac, Jerome" sort="Avouac, Jerome" uniqKey="Avouac J" first="Jerome" last="Avouac">Jerome Avouac</name>
</author>
<author>
<name sortKey="Behrens, Frank" sort="Behrens, Frank" uniqKey="Behrens F" first="Frank" last="Behrens">Frank Behrens</name>
</author>
<author>
<name sortKey="Clements, Philip J" sort="Clements, Philip J" uniqKey="Clements P" first="Philip J." last="Clements">Philip J. Clements</name>
</author>
<author>
<name sortKey="Denton, Christopher" sort="Denton, Christopher" uniqKey="Denton C" first="Christopher" last="Denton">Christopher Denton</name>
</author>
<author>
<name sortKey="Foeldvari, Ivan" sort="Foeldvari, Ivan" uniqKey="Foeldvari I" first="Ivan" last="Foeldvari">Ivan Foeldvari</name>
</author>
<author>
<name sortKey="Giannini, Edward" sort="Giannini, Edward" uniqKey="Giannini E" first="Edward" last="Giannini">Edward Giannini</name>
</author>
<author>
<name sortKey="Huscher, Doerte" sort="Huscher, Doerte" uniqKey="Huscher D" first="Doerte" last="Huscher">Doerte Huscher</name>
</author>
<author>
<name sortKey="Kowal Bielecka, Otylia" sort="Kowal Bielecka, Otylia" uniqKey="Kowal Bielecka O" first="Otylia" last="Kowal-Bielecka">Otylia Kowal-Bielecka</name>
</author>
<author>
<name sortKey="Lovell, Daniel" sort="Lovell, Daniel" uniqKey="Lovell D" first="Daniel" last="Lovell">Daniel Lovell</name>
</author>
<author>
<name sortKey="Matucci Cerinic, Marco" sort="Matucci Cerinic, Marco" uniqKey="Matucci Cerinic M" first="Marco" last="Matucci-Cerinic">Marco Matucci-Cerinic</name>
</author>
<author>
<name sortKey="Mayes, Maureen" sort="Mayes, Maureen" uniqKey="Mayes M" first="Maureen" last="Mayes">Maureen Mayes</name>
</author>
<author>
<name sortKey="Merkel, Peter A" sort="Merkel, Peter A" uniqKey="Merkel P" first="Peter A." last="Merkel">Peter A. Merkel</name>
</author>
<author>
<name sortKey="Nash, Peter" sort="Nash, Peter" uniqKey="Nash P" first="Peter" last="Nash">Peter Nash</name>
</author>
<author>
<name sortKey="Opitz, Christian F" sort="Opitz, Christian F" uniqKey="Opitz C" first="Christian F." last="Opitz">Christian F. Opitz</name>
</author>
<author>
<name sortKey="Pittrow, David" sort="Pittrow, David" uniqKey="Pittrow D" first="David" last="Pittrow">David Pittrow</name>
</author>
<author>
<name sortKey="Rubin, Lewis" sort="Rubin, Lewis" uniqKey="Rubin L" first="Lewis" last="Rubin">Lewis Rubin</name>
</author>
<author>
<name sortKey="Seibold, James R" sort="Seibold, James R" uniqKey="Seibold J" first="James R." last="Seibold">James R. Seibold</name>
</author>
<author>
<name sortKey="Steen, Virginia" sort="Steen, Virginia" uniqKey="Steen V" first="Virginia" last="Steen">Virginia Steen</name>
</author>
<author>
<name sortKey="Vibeke Strand, C" sort="Vibeke Strand, C" uniqKey="Vibeke Strand C" first="C." last="Vibeke Strand">C. Vibeke Strand</name>
</author>
<author>
<name sortKey="Tugwell, Peter S" sort="Tugwell, Peter S" uniqKey="Tugwell P" first="Peter S." last="Tugwell">Peter S. Tugwell</name>
</author>
<author>
<name sortKey="Varga, John" sort="Varga, John" uniqKey="Varga J" first="John" last="Varga">John Varga</name>
</author>
<author>
<name sortKey="Zink, Angela" sort="Zink, Angela" uniqKey="Zink A" first="Angela" last="Zink">Angela Zink</name>
</author>
<author>
<name sortKey="Furst, Daniel E" sort="Furst, Daniel E" uniqKey="Furst D" first="Daniel E." last="Furst">Daniel E. Furst</name>
</author>
</analytic>
<series>
<title level="j" type="main">Journal of rheumatology</title>
<title level="j" type="abbreviated">J. rheumatol.</title>
<idno type="ISSN">0315-162X</idno>
<imprint>
<date when="2009">2009</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
<seriesStmt>
<title level="j" type="main">Journal of rheumatology</title>
<title level="j" type="abbreviated">J. rheumatol.</title>
<idno type="ISSN">0315-162X</idno>
</seriesStmt>
</fileDesc>
<profileDesc>
<textClass>
<keywords scheme="KwdEn" xml:lang="en">
<term>Artery</term>
<term>Clinical trial</term>
<term>Prognosis</term>
<term>Pulmonary hypertension</term>
<term>Rheumatology</term>
<term>Scleroderma</term>
</keywords>
<keywords scheme="Pascal" xml:lang="fr">
<term>Essai clinique</term>
<term>Sclérodermie</term>
<term>Pronostic</term>
<term>Hypertension artérielle pulmonaire</term>
<term>Artère</term>
<term>Rhumatologie</term>
</keywords>
</textClass>
</profileDesc>
</teiHeader>
<front>
<div type="abstract" xml:lang="en">There have been steady efforts to develop a combined response index for systemic sclerosis (CRISS). A parallel and equally successful effort has been made by an Expert Panel on Outcome Measures in PAH related to Systemic Sclerosis (EPOSS) to measure effect in treatment of pulmonary arterial hypertension of systemic sclerosis (PAH-SSc). CRISS conducted a Delphi process combined with expert review to identify 11 candidate domains for inclusion in a core set of outcomes for SSc clinical trials: soluble biomarkers, cardiac, digital ulcers, gastrointestinal, global health, health related quality of life (HRQOL) and function, musculoskeletal, pulmonary, Raynaud's, renal, and skin. Tools within domains were also agreed upon. Concentrating on one aspect of disease, PAH, EPOSS also conducted a Delphi process and judged the following domains as the most appropriate for randomized controlled trials in PAH-SSc: lung vascular/pulmonary arterial pressure, cardiac function, exercise testing; severity of dyspnea, discontinuation of treatment; quality of life/activities of daily living; global state; and survival. Possible useful tools within each domain were also agreed on. Patient derived, physician derived, and objective measures of response will be included and combined with the idea that each reflects different aspects of PAH (EPOSS) and overall disease (CRISS) although this assumption may not prove true and can be separated if statistically and clinically valid to do so. In either case, prospective studies will require measurement of all domains, and tools are required and will be developed to define appropriate combined measures of response. CRISS and EPOSS are being developed through the OMERACT process. Through Delphi process and literature review significant progress has been made for both indices, and prospective data are being collected.</div>
</front>
</TEI>
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<country>
<li>Allemagne</li>
<li>Australie</li>
<li>Canada</li>
<li>France</li>
<li>Italie</li>
<li>Pologne</li>
<li>Royaume-Uni</li>
<li>Suisse</li>
<li>États-Unis</li>
</country>
<region>
<li>Angleterre</li>
<li>Berlin</li>
<li>Californie</li>
<li>District de Darmstadt</li>
<li>Grand Londres</li>
<li>Hesse (Land)</li>
<li>Massachusetts</li>
<li>Michigan</li>
<li>Ohio</li>
<li>Île-de-France</li>
</region>
<settlement>
<li>Berlin</li>
<li>Francfort-sur-le-Main</li>
<li>Londres</li>
<li>Paris</li>
<li>San Diego</li>
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<name sortKey="Behrens, Frank" sort="Behrens, Frank" uniqKey="Behrens F" first="Frank" last="Behrens">Frank Behrens</name>
<name sortKey="Clements, Philip J" sort="Clements, Philip J" uniqKey="Clements P" first="Philip J." last="Clements">Philip J. Clements</name>
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<name sortKey="Foeldvari, Ivan" sort="Foeldvari, Ivan" uniqKey="Foeldvari I" first="Ivan" last="Foeldvari">Ivan Foeldvari</name>
<name sortKey="Furst, Daniel E" sort="Furst, Daniel E" uniqKey="Furst D" first="Daniel E." last="Furst">Daniel E. Furst</name>
<name sortKey="Giannini, Edward" sort="Giannini, Edward" uniqKey="Giannini E" first="Edward" last="Giannini">Edward Giannini</name>
<name sortKey="Huscher, Doerte" sort="Huscher, Doerte" uniqKey="Huscher D" first="Doerte" last="Huscher">Doerte Huscher</name>
<name sortKey="Kowal Bielecka, Otylia" sort="Kowal Bielecka, Otylia" uniqKey="Kowal Bielecka O" first="Otylia" last="Kowal-Bielecka">Otylia Kowal-Bielecka</name>
<name sortKey="Lovell, Daniel" sort="Lovell, Daniel" uniqKey="Lovell D" first="Daniel" last="Lovell">Daniel Lovell</name>
<name sortKey="Matucci Cerinic, Marco" sort="Matucci Cerinic, Marco" uniqKey="Matucci Cerinic M" first="Marco" last="Matucci-Cerinic">Marco Matucci-Cerinic</name>
<name sortKey="Mayes, Maureen" sort="Mayes, Maureen" uniqKey="Mayes M" first="Maureen" last="Mayes">Maureen Mayes</name>
<name sortKey="Merkel, Peter A" sort="Merkel, Peter A" uniqKey="Merkel P" first="Peter A." last="Merkel">Peter A. Merkel</name>
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<name sortKey="Opitz, Christian F" sort="Opitz, Christian F" uniqKey="Opitz C" first="Christian F." last="Opitz">Christian F. Opitz</name>
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<name sortKey="Rubin, Lewis" sort="Rubin, Lewis" uniqKey="Rubin L" first="Lewis" last="Rubin">Lewis Rubin</name>
<name sortKey="Seibold, James R" sort="Seibold, James R" uniqKey="Seibold J" first="James R." last="Seibold">James R. Seibold</name>
<name sortKey="Steen, Virginia" sort="Steen, Virginia" uniqKey="Steen V" first="Virginia" last="Steen">Virginia Steen</name>
<name sortKey="Tugwell, Peter S" sort="Tugwell, Peter S" uniqKey="Tugwell P" first="Peter S." last="Tugwell">Peter S. Tugwell</name>
<name sortKey="Varga, John" sort="Varga, John" uniqKey="Varga J" first="John" last="Varga">John Varga</name>
<name sortKey="Vibeke Strand, C" sort="Vibeke Strand, C" uniqKey="Vibeke Strand C" first="C." last="Vibeke Strand">C. Vibeke Strand</name>
<name sortKey="Zink, Angela" sort="Zink, Angela" uniqKey="Zink A" first="Angela" last="Zink">Angela Zink</name>
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<country name="États-Unis">
<noRegion>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
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<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
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<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
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<region name="Île-de-France">
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
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<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
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<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
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<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
</region>
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<country name="Pologne">
<noRegion>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
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</country>
<country name="Italie">
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<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
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<country name="Australie">
<noRegion>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
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<country name="Canada">
<noRegion>
<name sortKey="Khanna, Dinesh" sort="Khanna, Dinesh" uniqKey="Khanna D" first="Dinesh" last="Khanna">Dinesh Khanna</name>
</noRegion>
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</affiliations>
</record>

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