Table 1 Baseline characteristics of the patients with ‘symptoms’ before the development of a complication, that received ERT (n = 58) and the NH cohort (n = 42)
From: Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain
| Â | NH cohort | ERT cohort | p-value (NH vs ERT cohort) |
|---|---|---|---|
N | 42 | 58 | Â |
Male (%) | 21 (50) | 27 (46.6) | 0.74 |
Atypical (%) | 13 (30.9) | 3 (5.2) | 0.001 |
Age at first presentation at the AMC, mean ± SD and median (range) | 45.0 ± 14.7 | 36.8 ± 14.1 | 0.009* |
| Â | 44.5 (10.8-72.2) | 40.3 (13.6-71.2) | Â |
Plasma lysoGb3 (nM) | 5 (0-137) | 11 (4-124) | <0.001 |
Proteinuria (%) | 8/36 (22.2) | 25/57 (43.9) | 0.03 |
ACE-inhibitors/ ARBs at presentation (%) | 8 (19.0) | 15 (25.9) | 0.43 |
Current smoking (%) | 6 (14.3) | 10 (17.2) | 0.51 |
Other co-morbidity (%) | 5 | 3 (5.2) | 0.20 |
Hypertension (%) | 9/40 (22.5) | 12 (20.7) | 0.18 |
Dyslipidemia (%) | 3 (7.1) | 0 (0) | 0.07 |
One symptom only at presentation (%) | 16 (38.1) | 30 (51.7) | 0.58 |
More than one symptom or presenting with a first complication (ERT not available yet/ before diagnosis) (%) | 26 (61.9) | 26 (44.8) | 0.58 |