Heart failure & cardiomyopathies
Fabry disease: development and progression of left ventricular hypertrophy despite long-term enzyme replacement therapy
Authors' conclusion
Over a median follow-up of >10 years under ERT, 36% of the patients still had LVMI cardiac progression, and 32%, predominantly older men, experienced major AFD-related events. LVH at treatment initiation was a strong predictor of LVMI progression and adverse events on ERT.
Study context
- Population
- Patients with Anderson-Fabry disease receiving enzyme replacement therapy (ERT), followed up ≥5 years
- Participants
- 60 patients
- Outcome
- Cardiac progression: increase of >10 g/m² in LVMI between first and last follow-up transthoracic echocardiography
Methodological detailsconsecutive patients, Independent Swiss-Fabry Cohort
- Consecutive patients
- Independent Swiss-Fabry Cohort
Main finding
As reported in the abstract
Patients with left ventricular mass index (LVMI) progression
22
36%
Rate of LVMI progression
12.1 (7-17.6) g/m² per 100 patient-years
Progressors with no left ventricular hypertrophy (LVH) at baseline
7
11%
Progressed to LVH
3
Observational data: associations, not effects.
Further findings
LVMI progression by sex
P < 0.01
LVH at ERT start as predictor of LVMI progression
| OR (95% CI) | P value | ||
|---|---|---|---|
| LVH at ERT start | 1.3 (1.1–2.6) | P = 0.02 | |
| 0.20.5125Odds ratio (log scale) |
- LVMI progression by sex — Progression after age 30 years
- 17 of 22 (77%)
Major Anderson-Fabry disease (AFD)-related events
- Patients with major AFD-related event
- 19 (31%)
- Men among those with events
- 17 of 19, 89%
- Age, with vs without events
- 45±11 vs 32±9 years
- Baseline LVH among those with events
- 12 of 19, 63%
- LVMI progression among those with events
- 10 of 19 (52%)
Original publication
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