Show simple item record

dc.contributor.authorHughes, DA
dc.contributor.authorAguiar, P
dc.contributor.authorDeegan, PB
dc.contributor.authorEzgu, F
dc.contributor.authorFrustaci, A
dc.contributor.authorLidove, O
dc.contributor.authorLinhart, A
dc.contributor.authorLubanda, J-C
dc.contributor.authorMoon, JC
dc.contributor.authorNicholls, K
dc.contributor.authorNiu, D-M
dc.contributor.authorNowak, A
dc.contributor.authorRamaswami, U
dc.contributor.authorReisin, R
dc.contributor.authorRozenfeld, P
dc.contributor.authorSchiffmann, R
dc.contributor.authorSvarstad, E
dc.contributor.authorThomas, M
dc.contributor.authorTorra, R
dc.contributor.authorVujkovac, B
dc.contributor.authorWarnock, DG
dc.contributor.authorWest, ML
dc.contributor.authorJohnson, J
dc.contributor.authorRolfe, MJ
dc.contributor.authorFeriozzi, S
dc.date.accessioned2020-11-17T04:02:57Z
dc.date.available2020-11-17T04:02:57Z
dc.date.issued2020-01-01
dc.identifierpii: bmjopen-2019-035182
dc.identifier.citationHughes, D. A., Aguiar, P., Deegan, P. B., Ezgu, F., Frustaci, A., Lidove, O., Linhart, A., Lubanda, J. -C., Moon, J. C., Nicholls, K., Niu, D. -M., Nowak, A., Ramaswami, U., Reisin, R., Rozenfeld, P., Schiffmann, R., Svarstad, E., Thomas, M., Torra, R. ,... Feriozzi, S. (2020). 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. BMJ OPEN, 10 (10), https://doi.org/10.1136/bmjopen-2019-035182.
dc.identifier.issn2044-6055
dc.identifier.urihttp://hdl.handle.net/11343/251682
dc.description.abstractOBJECTIVES: 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.
dc.languageEnglish
dc.publisherBMJ PUBLISHING GROUP
dc.titleEarly 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
dc.typeJournal Article
dc.identifier.doi10.1136/bmjopen-2019-035182
melbourne.affiliation.departmentMedicine and Radiology
melbourne.source.titleBMJ Open
melbourne.source.volume10
melbourne.source.issue10
melbourne.source.pagese035182-
dc.rights.licenseCC BY-NC
melbourne.elementsid1468424
melbourne.openaccess.pmchttp://www.ncbi.nlm.nih.gov/pmc/articles/PMC7549469
melbourne.contributor.authorNicholls, Kathleen
dc.identifier.eissn2044-6055
melbourne.accessrightsOpen Access


Files in this item

Thumbnail

This item appears in the following Collection(s)

Show simple item record