Long-term dose-dependent agalsidase effects on kidney histology in Fabry disease
R Skrunes, C Tøndel, S Leh, KK Larsen… - Clinical Journal of the …, 2017 - journals.lww.com
Results No statistical differences were found in baseline or final GFR or albuminuria. Kidney
biopsies showed significant reduction of podocyte globotriaosylceramide in both the lower
fixed dose group (− 1.39 [SD= 1.04]; P= 0.004) and the higher dose group (− 3.16 [SD=
2.39]; P= 0.002). Podocyte globotriaosylceramide (Gb3) reduction correlated with cumulative
agalsidase dose (r= 0.69; P= 0.001). Arterial/arteriolar intima Gb3 cleared significantly in the
higher dose group, all seven patients with baseline intimal Gb3 cleared the intima, one …
biopsies showed significant reduction of podocyte globotriaosylceramide in both the lower
fixed dose group (− 1.39 [SD= 1.04]; P= 0.004) and the higher dose group (− 3.16 [SD=
2.39]; P= 0.002). Podocyte globotriaosylceramide (Gb3) reduction correlated with cumulative
agalsidase dose (r= 0.69; P= 0.001). Arterial/arteriolar intima Gb3 cleared significantly in the
higher dose group, all seven patients with baseline intimal Gb3 cleared the intima, one …