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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

  NH cohort ERT cohort p-value (NH vs ERT cohort)
N 42 58  
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*
  44.5 (10.8-72.2) 40.3 (13.6-71.2)  
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
  1. Comorbidity refers to other (chronic) illnesses possibly affecting disease course. *In the analysis for treatment effect the confounder age was adjusted for.