Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative
Objectives The PRoposing Early Disease Indicators for Clinical Tracking in Fabry Disease (PREDICT-FD) initiative aimed to reach consensus among a panel of global experts on early indicators of disease progression that may justify FD-specific treatment initiation. Design and setting Anonymous feedba...
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| Autores principales: | , , , , , , , , , , , , , , , , , , , , , , , , |
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| Formato: | Articulo |
| Lenguaje: | Inglés |
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2020
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| Acceso en línea: | http://sedici.unlp.edu.ar/handle/10915/119662 |
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I19-R120-10915-119662 |
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dspace |
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Universidad Nacional de La Plata |
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I-19 |
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R-120 |
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SEDICI (UNLP) |
| language |
Inglés |
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Biología Early indicators Fabry disease |
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Biología Early indicators Fabry disease Hughes, Derralynn A. Aguiar, Patricio Deegan, Patrick B. Ezgu, Fatih Frustaci, Andrea Lidove, Olivier Linhart, Aleš Lubanda, Jean-Claude Moon, James C. Nicholls, Kathleen Niu, Dau-Ming Nowak, Albina Ramaswami, Uma Reisin, Ricardo Rozenfeld, Paula Adriana Schiffmann, Raphael Svarstad, Einar Thomas, Mark Torra, Roser Vujkovac, Bojan Warnock, David G. West, Michael L. Johnson, Jack Rolfe , Mark J. Feriozzi, Sandro Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative |
| topic_facet |
Biología Early indicators Fabry disease |
| description |
Objectives The PRoposing Early Disease Indicators for Clinical Tracking in Fabry Disease (PREDICT-FD) initiative aimed to reach consensus among a panel of global experts on early indicators of disease progression that may justify FD-specific treatment initiation.
Design and setting Anonymous feedback from panellists via online questionnaires was analysed using a modified Delphi consensus technique. Questionnaires and data were managed by an independent administrator directed by two non-voting cochairs. First, possible early indicators of renal, cardiac and central/peripheral nervous system (CNS/PNS) damage, and other disease and patient-reported indicators assessable in routine clinical practice were compiled by the cochairs and administrator from panellists’ free-text responses. Second, the panel scored indicators for importance (5-point scale: 1=not important; 5=extremely important); indicators scoring ≥3 among >75% of panellists were then rated for agreement (5-point scale: 1=strongly disagree; 5=strongly agree).
Indicators awarded an agreement score ≥4 by >67% of panellists achieved consensus. Finally, any panel-proposed refinements to consensus indicator definitions were adopted if >75% of panellists agreed.
Results A panel of 21 expert clinicians from 15 countries provided information from which 83 possible current indicators of damage (kidney, 15; cardiac, 15; CNS/PNS, 13; other, 16; patient reported, 24) were compiled. Of 45 indicators meeting the importance criteria, consensus was reached for 29 and consolidated as 27 indicators (kidney, 6; cardiac, 10; CNS/PNS, 2; other, 6; patient reported, 3) including: (kidney) elevated albumin:creatinine ratio, histological damage, microalbuminuria; (cardiac) markers of early systolic/diastolic dysfunction, elevated serum cardiac troponin; (CNS/PNS) neuropathic pain, gastrointestinal symptoms suggestive of gastrointestinal neuropathy; (other) pain in extremities/neuropathy, angiokeratoma; (patient-reported) febrile crises, progression of symptoms/signs. Panellists revised and approved proposed chronologies of when the consensus indicators manifest. The panel response rate was >95% at all stages.
Conclusions PREDICT-FD captured global opinion regarding current clinical indicators that could prompt FD-specific treatment initiation earlier than is currently practised. |
| format |
Articulo Articulo |
| author |
Hughes, Derralynn A. Aguiar, Patricio Deegan, Patrick B. Ezgu, Fatih Frustaci, Andrea Lidove, Olivier Linhart, Aleš Lubanda, Jean-Claude Moon, James C. Nicholls, Kathleen Niu, Dau-Ming Nowak, Albina Ramaswami, Uma Reisin, Ricardo Rozenfeld, Paula Adriana Schiffmann, Raphael Svarstad, Einar Thomas, Mark Torra, Roser Vujkovac, Bojan Warnock, David G. West, Michael L. Johnson, Jack Rolfe , Mark J. Feriozzi, Sandro |
| author_facet |
Hughes, Derralynn A. Aguiar, Patricio Deegan, Patrick B. Ezgu, Fatih Frustaci, Andrea Lidove, Olivier Linhart, Aleš Lubanda, Jean-Claude Moon, James C. Nicholls, Kathleen Niu, Dau-Ming Nowak, Albina Ramaswami, Uma Reisin, Ricardo Rozenfeld, Paula Adriana Schiffmann, Raphael Svarstad, Einar Thomas, Mark Torra, Roser Vujkovac, Bojan Warnock, David G. West, Michael L. Johnson, Jack Rolfe , Mark J. Feriozzi, Sandro |
| author_sort |
Hughes, Derralynn A. |
| title |
Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative |
| title_short |
Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative |
| title_full |
Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative |
| title_fullStr |
Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative |
| title_full_unstemmed |
Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative |
| title_sort |
early indicators of disease progression in fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based predict-fd modified delphi consensus initiative |
| publishDate |
2020 |
| url |
http://sedici.unlp.edu.ar/handle/10915/119662 |
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