Login
Search
Search
0 Dates
2026
2025
2024
2023
2022
2021
2020
2019
2018
0 Events
CPC 2018
CPC 2019
Curso de Atualização em Medicina Cardiovascular 2019
Reunião Anual Conjunta dos Grupos de Estudo de Cirurgia Cardíaca, Doenças Valvulares e Ecocardiografia da SPC
CPC 2020
CPC 2021
CPC 2022
CPC 2023
CPC 2024
CPC 2025
CPC 2026
0 Topics
A. Basics
B. Imaging
C. Arrhythmias and Device Therapy
D. Heart Failure
E. Coronary Artery Disease, Acute Coronary Syndromes, Acute Cardiac Care
F. Valvular, Myocardial, Pericardial, Pulmonary, Congenital Heart Disease
G. Aortic Disease, Peripheral Vascular Disease, Stroke
H. Interventional Cardiology and Cardiovascular Surgery
I. Hypertension
J. Preventive Cardiology
K. Cardiovascular Disease In Special Populations
L. Cardiovascular Pharmacology
M. Cardiovascular Nursing
N. E-Cardiology / Digital Health, Public Health, Health Economics, Research Methodology
O. Basic Science
P. Other
0 Themes
01. History of Cardiology
02. Clinical Skills
03. Imaging
04. Arrhythmias, General
05. Atrial Fibrillation
06. Supraventricular Tachycardia (non-AF)
07. Syncope and Bradycardia
08. Ventricular Arrhythmias and Sudden Cardiac Death (SCD)
09. Device Therapy
10. Chronic Heart Failure
11. Acute Heart Failure
12. Coronary Artery Disease (Chronic)
13. Acute Coronary Syndromes
14. Acute Cardiac Care
15. Valvular Heart Disease
16. Infective Endocarditis
17. Myocardial Disease
18. Pericardial Disease
19. Tumors of the Heart
20. Congenital Heart Disease and Pediatric Cardiology
21. Pulmonary Circulation, Pulmonary Embolism, Right Heart Failure
22. Aortic Disease
23. Peripheral Vascular and Cerebrovascular Disease
24. Stroke
25. Interventional Cardiology
26. Cardiovascular Surgery
27. Hypertension
28. Risk Factors and Prevention
29. Rehabilitation and Sports Cardiology
30. Cardiovascular Disease in Special Populations
31. Pharmacology and Pharmacotherapy
32. Cardiovascular Nursing
33. e-Cardiology / Digital Health
34. Public Health and Health Economics
35. Research Methodology
36. Basic Science
37. Miscellanea
0 Resources
Abstract
Slides
Vídeo
Report
CLEAR FILTERS
followME Fabry Pathfinders real-world registry in Spain and Portugal: cardiac and renal outcomes with migalastat in patients with Fabry disease
Session:
Sessão de Posters 21 - Por dentro do miocárdio: da genética aos resultados
Speaker:
Olga Azevedo
Congress:
CPC 2026
Topic:
F. Valvular, Myocardial, Pericardial, Pulmonary, Congenital Heart Disease
Theme:
17. Myocardial Disease
Subtheme:
17.4 Myocardial Disease – Treatment
Session Type:
Posters Eletrónicos
FP Number:
---
Authors:
Olga Azevedo; Patrício Aguiar; Vicente Climent-Payá; Roser Torra; Maria Camprodon-Gomez; Elena Fortuny; João Paulo Oliveira; Ana Aguinaga-Barrilero; Vipul Jain; Ana Luísa Vaz; Biliana O Veleva-Rotse; Tomas Ripoll-Vera
Abstract
<p><span style="font-size:11pt"><span style="font-family:Arial,sans-serif">Fabry disease (FD) is a progressive lysosomal disorder caused by <em>GLA</em> gene variants and characterised by multisystemic dysfunction, predominantly affecting the renal, cardiac and nervous systems. We present data from migalastat-treated patients in Spain and Portugal from the real-world followME Fabry Pathfinders registry (EUPAS20599). Patients ≥12 years with confirmed FD and estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73m<sup>2</sup> were enrolled. Retrospective and prospective data were analysed from patients with amenable <em>GLA</em> variants who received migalastat for ≥2 years. Left ventricular hypertrophy (LVH) was defined as left ventricular mass index (LVMi) >115 g/m<sup>2</sup> in males and >95 g/m<sup>2</sup> in females. In total, 73 patients (69.9% male) were included. At enrolment, median age was 58.0 years. The three most common <em>GLA</em> variants were p.F113L (n=37; 50.7%), p.S238N (n=17; 23.3%) and p.R301Q (n=6; 8.2%). As of August 2025, median migalastat exposure was 4.4 years (range 2.0–6.8). At baseline, median LVMi was 132.4 g/m<sup>2</sup> (n=46; range 62.3–296.2 [with LVH: 152.6 g/m<sup>2</sup>, n=32, range 109.1–296.2; without LVH: 92.6 g/m<sup>2</sup>, n=14, range 62.3–109.7]) and median eGFR calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (eGFR<sub>CKD-EPI</sub>) was 92.1 mL/min/1.73m<sup>2</sup> (n=66; range 30.5–126.9 [males: 94.7 mL/min/1.73m<sup>2</sup>, n=46, range 42.6–126.9; females: 85.2 mL/min/1.73m<sup>2</sup>, n=20, range 30.5–107.0]). Overall LVMi median annualised rate of change (ARC) was 0.20 g/m<sup>2</sup> (n=59; 95% confidence interval [95% CI] −2.31 to 2.98 [LVH: −0.47 g/m<sup>2</sup>, n=41, 95% CI −7.67 to 3.24; without LVH: 0.89 g/m<sup>2</sup>, n=18, 95% CI −1.18 to 5.29]). Overall median eGFR<sub>CKD</sub><sub>-</sub><sub>EPI</sub> ARC was −1.32 mL/min/1.73m<sup>2</sup> (n=73; 95% CI −1.79 to −0.65 [males: −1.58 mL/min/1.73m<sup>2</sup>, n=51, 95% CI −2.06 to −0.70; females: −1.01 mL/min/1.73m<sup>2</sup>, n=22, 95% CI −2.92 to 0.15]). No treatment-related treatment-emergent serious adverse events or discontinuations were reported. During a median 4.4 years, these results appear consistent with previously published followME registry data. Cardiac </span></span>geometry <span style="font-size:11pt"><span style="font-family:Arial,sans-serif">and renal function were stable and migalastat was well tolerated in a population of patients with primarily late-onset variants and cardiac involvement at baseline.</span></span></p>
Slides
Our mission: To reduce the burden of cardiovascular disease
Visit our site