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dc.date.accessioned | 2021-06-02T19:06:32Z | |
dc.date.available | 2021-06-02T19:06:32Z | |
dc.date.issued | 2020 | |
dc.identifier.uri | http://sedici.unlp.edu.ar/handle/10915/119662 | |
dc.description.abstract | 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. | en |
dc.language | en | es |
dc.subject | Early indicators | es |
dc.subject | Fabry disease | es |
dc.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 | en |
dc.type | Articulo | es |
sedici.identifier.other | https://doi.org/10.1136/ bmjopen-2019-035182 | es |
sedici.identifier.issn | 2044-6055 | es |
sedici.creator.person | Hughes, Derralynn A. | es |
sedici.creator.person | Aguiar, Patricio | es |
sedici.creator.person | Deegan, Patrick B. | es |
sedici.creator.person | Ezgu, Fatih | es |
sedici.creator.person | Frustaci, Andrea | es |
sedici.creator.person | Lidove, Olivier | es |
sedici.creator.person | Linhart, Aleš | es |
sedici.creator.person | Lubanda, Jean-Claude | es |
sedici.creator.person | Moon, James C. | es |
sedici.creator.person | Nicholls, Kathleen | es |
sedici.creator.person | Niu, Dau-Ming | es |
sedici.creator.person | Nowak, Albina | es |
sedici.creator.person | Ramaswami, Uma | es |
sedici.creator.person | Reisin, Ricardo | es |
sedici.creator.person | Rozenfeld, Paula Adriana | es |
sedici.creator.person | Schiffmann, Raphael | es |
sedici.creator.person | Svarstad, Einar | es |
sedici.creator.person | Thomas, Mark | es |
sedici.creator.person | Torra, Roser | es |
sedici.creator.person | Vujkovac, Bojan | es |
sedici.creator.person | Warnock, David G. | es |
sedici.creator.person | West, Michael L. | es |
sedici.creator.person | Johnson, Jack | es |
sedici.creator.person | Rolfe , Mark J. | es |
sedici.creator.person | Feriozzi, Sandro | es |
sedici.subject.materias | Biología | es |
sedici.description.fulltext | true | es |
mods.originInfo.place | Instituto de Estudios Inmunológicos y Fisiopatológicos | es |
sedici.subtype | Articulo | es |
sedici.rights.license | Creative Commons Attribution-NonCommercial 4.0 International (CC BY-NC 4.0) | |
sedici.rights.uri | http://creativecommons.org/licenses/by-nc/4.0/ | |
sedici.description.peerReview | peer-review | es |
sedici.relation.journalTitle | BMJ Open | es |
sedici.relation.journalVolumeAndIssue | vol. 10, no. 10 | es |