Journal article
Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: A 10-year fabry outcome survey (FOS) analysis
U Ramaswami, M Beck, D Hughes, C Kampmann, J Botha, G Pintos-Morell, ML West, DM Niu, K Nicholls, R Giugliani
Drug Design Development and Therapy | DOVE MEDICAL PRESS LTD | Published : 2019
DOI: 10.2147/DDDT.S207856
Abstract
Purpose: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods: FOS (ClinicalTrials.gov identifier: NCT03289065) data (April 2001 to August 2018) were retrospectively analyzed. Estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) were analyzed after treatment start (baseline) for patients with ≥3 measurements, including baseline and year 10. Results: Median (range) age (years) of the evaluable treated renal cohort at treatment start was 48.8 (17.9–67.3) for females (n=62), 34.4 (18.0–66.8) for males (n=90). With eGFR ≥60 ..
View full abstractGrants
Funding Acknowledgements
The Fabry Outcome Survey is sponsored by Shire Human Genetic Therapies, Inc., a member of the Takeda group of companies. Shire International GmbH, a member of the Takeda group of companies, provided funding to Excel Medical Affairs for support in writing and editing this manuscript and participated in data collection and analysis.