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Table 2 The LVMI and serum lyso-Gb3 change after ERT in IVS4 + 919G > A Fabry disease male and female patients

From: Globotriaosylsphingosine (lyso-Gb3) might not be a reliable marker for monitoring the long-term therapeutic outcomes of enzyme replacement therapy for late-onset Fabry patients with the Chinese hotspot mutation (IVS4+919G>A)

Cardiac variant, IVS4 + 919G > A FD patients Parameters, patient number Baseline (Mean ± SD) After ERT (Mean ± SD) P value
Male LVMI (g/H2.7) N = 16 81.1 ± 32.2 71.0 ± 25.6 0.016*
Compare between baseline and 1st Lyso-Gb3 (nM) N = 16 8.5 ± 3.3 6.7 ± 2.2 0.012*
Compare between baseline and the last Lyso-Gb3 (nM) N = 16 8.5 ± 3.3 7.4 ± 2.8 0.163
Female LVMI (g/H2.7) N = 7 59.4 ± 12.8 42.0 ± 10.2 <0.001*
Compare between baseline and 1st Lyso-Gb3 (nM) N = 7 2.6 ± 1.0 1.2 ± 0.9 0.018*
Compare between baseline and the last Lyso-Gb3 (nM) N = 7 2.6 ± 1.0 2.6 ± 1.6 0.938
  1. *Asterisk represents statistically significant difference between the data of baseline and that after ERT (p < 0.05). ERT: enzyme replacement therapy; FD: Fabry disease; LVMI: left ventricular mass index; lyso-Gb3: globotriaosylsphingosine; SD: standard deviation.