Journal article

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

Derralynn A Hughes, Patricio Aguiar, Patrick B Deegan, Fatih Ezgu, Andrea Frustaci, Olivier Lidove, Ales Linhart, Jean-Claude Lubanda, James C Moon, Kathleen Nicholls, Dau-Ming Niu, Albina Nowak, Uma Ramaswami, Ricardo Reisin, Paula Rozenfeld, Raphael Schiffmann, Einar Svarstad, Mark Thomas, Roser Torra, Bojan Vujkovac Show all

BMJ OPEN | BMJ PUBLISHING GROUP | Published : 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 pr..

View full abstract

Grants

Awarded by Shire International GmbH, Zug, Switzerland


Funding Acknowledgements

Administration and coordination of the PREDICT-FD initiative and medical writing support were funded by an unrestricted independent educational grant (IME-GBR-15474) from Shire International GmbH, Zug, Switzerland, now part of Takeda Pharmaceutical Company. Shire/Takeda was not involved in the planning, design or delivery of the initiative. No reimbursement or funding was offered to the voting panel, to the cochairs or to any other organisation participating in the initiative.