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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
- Source :
- e035182, BMJ Open, Dipòsit Digital de Documents de la UAB, Universitat Autònoma de Barcelona, SEDICI (UNLP), Universidad Nacional de La Plata, instacron:UNLP, BMJ Open, Vol 10, Iss 10 (2020), r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau, instname
- Publication Year :
- 2020
-
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.<br />Instituto de Estudios Inmunológicos y Fisiopatológicos
- Subjects :
- Early indicators
medicine.medical_specialty
Consensus
Delphi Technique
Cardiomyopathy
Biología
Delphi method
Computer-assisted web interviewing
Disease
chronic renal failure
Internal medicine
Surveys and Questionnaires
medicine
Genetics
Chronic renal failure
Humans
genetics
Response rate (survey)
Fabry disease
Renal Medicine
business.industry
General Medicine
Enzyme replacement therapy
medicine.disease
stroke medicine
Disease Progression
Biomarker (medicine)
Medicine
Fabry Disease
Stroke medicine
Microalbuminuria
business
cardiomyopathy
Subjects
Details
- Language :
- English
- ISSN :
- 20446055
- Database :
- OpenAIRE
- Journal :
- e035182, BMJ Open, Dipòsit Digital de Documents de la UAB, Universitat Autònoma de Barcelona, SEDICI (UNLP), Universidad Nacional de La Plata, instacron:UNLP, BMJ Open, Vol 10, Iss 10 (2020), r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau, instname
- Accession number :
- edsair.doi.dedup.....331d397aa4e03343500b80672fe0c4ba