Predictors of outcome in a Spanish cohort of patients with Fabry disease on enzyme replacement therapy |
| |
| Institution: | 1. Servicio de Nefrología Hospital General Universitario Gregorio Marañon, Spain;2. Red de Investigación Renal (REDinRen), Fondos FEDER, Spain;3. Servicio de Nefrología Hospital Universitario de Bellvitge, Spain;4. Servicio de Nefrología Fundacion Puigvert, Spain;5. Servicio de Nefrología Hospital Universitario Puerta del Hierro Majadahonda, Spain;6. Servicio de Cardiología Hospital Universitario Puerta de Hierro, Spain;7. Servicio de Nefrología Hospital Clinic de Barcelona, Spain;8. Servicio de Nefrología Complexo Hospitalario Universitario A Coruña, Spain;9. Servicio de Nefrología Hospital Vall d’Hebron, Spain;10. Servicio de Nefrología Hospital Universitario Virgen del Rocío, Spain;11. Servicio de Nefrología Hospital General Universitario de Guadalajara, Universidad de Alcalá de Henares, Spain;12. Servicio de Nefrología Hospital Infanta Leonor, Spain;13. Servicio de Nefrología Hospital Universitario de la Princesa, Spain;14. Servicio de Nefrología del Hospital Universitario del Tajo, Spain;15. Servicio de Nefrología del Hospital Universitari Arnau de Vilanova, Spain;p. Servicio de Nefrología del Hospital Universitario Ramon y Cajal, Spain;q. Servicio de Nefrología del Hospital Clínico Universitario de Valencia, Spain;r. Servicio de Cardiología del Hospital General Universitario Gregorio Marañon, Spain;s. Servicio de Nefrología de la Fundación Jimenez Diaz, Spain |
| |
| Abstract: | 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 (242–128 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 ≤ 60 ml/min/1.73 m2 (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. |
| |
| Keywords: | Fabry disease Chronic kidney disease Enzyme replacement therapy Renal events Enfermedad de Fabry Enfermedad renal crónica Tratamiento enzimático sustitutivo Eventos renales |
| 本文献已被 ScienceDirect 等数据库收录！ |
|
