RT Journal Article
T1 Predictors of outcome in a Spanish cohort of patients with Fabry disease on enzyme replacement therapy
A1 Goicoechea, Marian
A1 Gomez-Preciado, Francisco
A1 Benito, Silvia
A1 Torras, Joan
A1 Torra, Roser
A1 Huerta, Ana
A1 Restrepo, Alejandra
A1 Ugalde, Jessica
A1 Estefania Astudillo, Daniela
A1 Agraz, Irene
A1 Lopez-Mendoza, Manuel
A1 de Arriba, Gabriel
A1 Corchete, Elena
A1 Quiroga, Borja
A1 Jose Gutierrez, Maria
A1 Luisa Martin-Conde, Maria
A1 Lopes, Vanessa
A1 Ramos, Carmela
A1 Mendez, Irene
A1 Cao, Mercedes
A1 Dominguez, Fernando
A1 Ortiz, Alberto
K1 Fabry disease
K1 Chronic kidney disease Enzyme replacement therapy
K1 Renal events
K1 Natural-history data
K1 Agalsidase beta
K1 Renal-disease
K1 Lyso-gb3
K1 Events
AB Fabry disease may be treated by enzyme replacement therapy (ERT), but the impact of chronic kidney disease (CKD) on the response to therapy remains unclear. The aim of the present study was to analyse the incidence and predictors of clinical events in patients on ERT.Study design: Multicentre retrospective observational analysis of patients diagnosed and treated with ERT for Fabry disease. The primary outcome was the first renal, neurological or cardiological events or death during a follow-up of 60 months (24-120).Results: In 69 patients (42 males, 27 females, mean age 44.6 +/- 13.7 years), at the end of followup, eGFR and the left ventricular septum thickness remained stable and the urinary albumin: creatinine ratio tended to decrease, but this decrease only approached significance inpatients on agalsidase-beta (242-128 mg/g (p = 0.05). At the end of follow-up, 21 (30%) patients had suffered an incident clinical event: 6 renal, 2 neurological and 13 cardiological (including 3 deaths). Events were more frequent in patients with baseline eGFR
PB Soc espanola nefrologia dr rafael matesanz
SN 0211-6995
YR 2021
FD 2021-11-01
LK https://hdl.handle.net/10668/27447
UL https://hdl.handle.net/10668/27447
LA en
DS RISalud
RD Apr 17, 2025