Table 3 Random coefficient mixed models results for longitudinal correlation between plasma lyso-Gb₃ and LVMi, eGFR, and pain during migalastat treatment.

Parameter	Overall (N = 97)	ERT-naive (n = 48)	ERT-experienced (n = 49)	Males (n = 37)	Females (n = 60)	Age ≤ 40 years (n = 31)	Age > 40 years (n = 66)
LVMi^a
n	66	25	41	27	39	14	52
Correlation coefficient	0.2056	0.2075	0.3389	−0.0412	0.1723	0.6263	0.6329
P value	0.2675	0.4407	0.2460	0.8824	0.4536	0.1391	0.0185
eGFR^b
n	97	48	49	37	60	31	66
Correlation coefficient	0.0851	0.5198	0.0511	−0.0342	0.1984	0.7603	−0.0954
P value	0.7342	0.4341	0.8484	0.9319	0.6393	0.1157	0.6913
Pain^c
n	96	47	49	37	59	31	65
Correlation coefficient	0.8230	0.4473	0.6868	0.9872	NE^d	0.3146	NE
P value	0.0032	0.3834	0.0380	0.0165	NE^d	0.4543	NE

eGFR estimated glomerular filtration rate, ERT enzyme replacement therapy, LVMi left ventricular mass index, lyso-Gb₃ globotriaosylsphingosine, NE not estimable.
^aLVMi data were derived from FACETS, ATTRACT, and the open-label extension study AT1001-042.
^beGFR data were derived from FACETS, ATTRACT, and the open-label extension study AT1001-042.
^cPain data were derived from FACETS and ATTRACT.
^dThese values were not estimated due to insufficient data.

Quick links

Search