TY - JOUR T1 - Predictors of outcome in a Spanish cohort of patients with Fabry disease on enzyme replacement therapy JO - Nefrología (English Edition) T2 - AU - Goicoechea,Marian AU - Gomez-Preciado,Francisco AU - Benito,Silvia AU - Torras,Joan AU - Torra,Roser AU - Huerta,Ana AU - Restrepo,Alejandra AU - Ugalde,Jessica AU - Astudillo,Daniela Estefania AU - Agraz,Irene AU - Lopez-Mendoza,Manuel AU - de Arriba,Gabriel AU - Corchete,Elena AU - Quiroga,Borja AU - Gutierrez,Maria Jose AU - Martin-Conde,Maria Luisa AU - Lopes,Vanessa AU - Ramos,Carmela AU - Mendez,Irene AU - Cao,Mercedes AU - Dominguez,Fernando AU - Ortiz,Alberto SN - 20132514 M3 - 10.1016/j.nefroe.2022.01.003 DO - 10.1016/j.nefroe.2022.01.003 UR - https://revistanefrologia.com/en-predictors-outcome-in-spanish-cohort-articulo-S2013251422000037 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 designMulticentre 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). ResultsIn 69 patients (42 males, 27 females, mean age 44.6±13.7 years), at the end of follow-up, eGFR and the left ventricular septum thickness remained stable and the urinary albumin: creatinine ratio tended to decrease, but this decrease only approached significance in patients on agalsidase-beta (242–128mg/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≤60ml/min/1.73m2 (log Rank 12.423, p=0.001), and this remained significant even after excluding incident renal events (log Rank 4.086, p=0.043) and in males and in females. Lower baseline eGFR was associated with a 3- to 7-fold increase the risk of clinical events in different Cox models. ConclusionsGFR at the initiation of ERT is the main predictor of clinical events, both in males and in females, suggesting that start of ERT prior to the development of CKD is associated with better outcomes. ER -