Table 2

Parameter changes of cardiac status and renal function, plasma lyso-Gb3 and Mainz Severity Score Index (MSSI) at baseline and after 6–39 months of enzyme replacement therapy (ERT) for 36 Taiwanese patients with Fabry disease

					Plasma lyso-Gb3 (nM)*		eGFR (mL/min/1.73 m²) (CKD-EPI)		Urine ACR (mg/mmol)		LVM (g)		LVMI (g/m^2.7)		IVS (mm)		LPW (mm)		MSSI (max score=76)		Adverse effects
No.	Type	Age at ERT (year)	Gender	ERT duration (months)	Baseline	After ERT	Baseline	After ERT	Baseline	After ERT	Baseline	After ERT	Baseline	After ERT	Baseline	After ERT	Baseline	After ERT	Baseline	After ERT	Adverse effects
1	Cardiac	25	M	14	4.1	1.8	73.3	101.1	0.46	0.49	221.3	NA	48.8	NA	8.7	NA	10	NA	9	9	N
2	Cardiac	43	M	39	4.44	3.36	109.6	115.1	0.35	0.48	392.1	270	92.1	63.4	16.7	13.7	12	12.2	21	17	N
3	Cardiac	53	M	39	5.19	4.18	99.7	92.5	0.85	0.30	298.5	198	76	50.4	14.6	12.8	15.2	11.7	24	20	N
4	Cardiac	54	M	10	6.13	4.71	93.9	93.3	1.70	0.28	303.0	304	74.5	74.8	14.7	12	14.7	12	16	16	N
5	Cardiac	55	M	17	8.22	8	72.2	80.1	13.31	0.66	272.1	342.8	66.5	83.8	15.2	14	12.8	12	27	27	N
6	Cardiac	55	M	19	5.44	3.67	71.3	69.9	2.43	0.27	188.8	148	50.5	39.6	12.7	12.5	7.3	9.5	24	24	N
7	Cardiac	57	M	13	6.77	NA	94.9	97.8	3.94	3.28	148.2	138.7	43.8	41.0	15.4	9.9	6.4	7	20	21	N
8	Cardiac	59	M	12	13.51	9.86	67.9	63.2	0.30	0.43	185.3	244.3	51.2	67.5	13.3	13.3	9	10.7	25	25	N
9	Cardiac	60	M	12	5.06	4.38	86.7	96	1.97	0.49	204.1	130	50.6	32.2	10.5	9	9.3	9.5	16	14	N
10	Cardiac	62	M	15	11.52	3.79	92.5	95.9	0.81	1.07	276.9	403.9	75.9	110.7	18.6	12	10.1	10	20	20	N
11	Cardiac	63	M	18	14.54	8.19	94.8	90	1.07	0.25	340.8	302.1	88.2	78.2	12	15.3	14	13.3	17	17	N
12	Cardiac	63	M	35	3.49	4.17	93.4	89.7	5.65	0.47	383.6	336	88.7	77.7	19.7	17.7	12.6	8.2	25	21	N
13	Cardiac	64	M	6	9.77	8.66	66.8	82.8	0.67	0.51	544.8	439	136.4	109.9	30	22.3	28.6	16.6	31	31	N
14	Cardiac	65	M	8	8.08	4.49	76.1	83.5	0.25	3.67	179.1	NA	55.2	NA	16	NA	8	NA	22	22	N
15	Cardiac	67	M	32	7.8	10.8	100.9	98.4	1.07	0.15	195.6	191	52.3	51.1	15.4	20.2	8	10.7	23	24	Y
16	Classic	14	M	27	178.4	75.64	133.8	141.2	0.99	0.74	225.2	NA	57.3	NA	10.1	9.9	11.7	9.9	17	17	Y
17	Classic	23	M	9	203.68	74.51	108	119.7	1.14	0.68	323.3	242	57.1	42.8	12	11.3	11.3	10.7	19	17	N
18	Classic	33	M	16	54.95	45.13	85.7	83.1	171.68	NA	282.2	181.9	68.5	44.2	13	9.5	11	10.6	27	25	Y
19	Classic	49	M	16	71.48	58.8	31.5	25.3	111.84	11.53	151.6	227.1	30.5	45.7	10.5	13.3	7	7.3	32	34	Y
20	Cardiac	44	F	8	2.23	1.34	106.7	112.7	0.77	0.89	204.6	104	57.5	29.2	11	5	9.5	8	17	15	N
21	Cardiac	53	F	15	2.59	1.29	103.6	NA	0.66	0.74	143.0	NA	45.4	NA	9	NA	6.4	NA	15	15	N
22	Cardiac	58	F	24	2.60	1.20	26	12.1	286.24	117.22	186.5	114	59.2	36.2	12.5	9.9	13	8.8	25	19	Y
23	Cardiac	59	F	17	3.49	3.11	77.2	94.3	13.11	4.09	169.0	133.8	53.4	42.3	13.3	11	8	6.5	21	15	N
24	Cardiac	63	F	9	4.24	1.47	96.2	96.6	6.14	0.67	179.0	94.4	52.1	27.5	11.5	7.6	11.5	7.6	17	11	N
25	Cardiac	67	F	12	3.67	NA	91.1	99.1	0.76	0.33	194.7	193.8	64	63.7	12	12.7	12.7	11.8	18	18	N
26	Classic	33	F	16	11.74	7.08	114.7	117.4	0.73	0.52	124.5	131.7	35.6	37.7	9.1	8	9.1	6	5	4	Y
27	Classic	34	F	25	24.8	12.57	107.9	120.2	355.33	105.94	122.2	155.7	38.1	48.5	7	8.6	8.2	9.5	18	20	Y
28	Classic	48	F	17	8.97	6.85	108.2	106	2.03	3.43	168.7	155.7	55.9	51.6	15.7	9.3	8.8	8.8	29	29	Y
29	Classic	53	F	6	4.66	4.56	97.2	100.1	40.08	55.31	195.6	158	62.6	50.6	12	9.5	10	10.8	21	21	N
30	Classic	54	F	26	17.26	11.70	105.6	104.2	4.10	1.81	216.8	182	72.5	60.9	15	10.1	14	10.8	27	15	N
31	Classic	56	F	12	11.41	12.16	109	84.1	0.69	1.86	211.8	233	51.1	56.2	9.3	16.3	9.9	11.5	22	26	Y
32	Classic	58	F	16	20.12	17.17	77.1	82.3	3.79	2.81	123.9	101.2	58.7	35.1	10.5	9.5	8.6	8	25	23	Y
33	Classic	58	F	20	31.77	21.00	86.7	78.1	5.53	0.31	255.7	250.4	82.6	80.8	16.4	14.3	11.1	11.3	38	38	Y
34	Classic	66	F	11	18.38	14.03	77	74.3	13.57	2.03	468.3	431.1	146.5	134.8	24.5	22.5	16.3	14.4	35	35	N
35	Classic	76	F	8	28.12	23.32	54.7	60.9	4.19	0.58	352.4	272	120.1	92.7	18	18.3	18	14.5	34	34	Y
36	Classic	79	F	12	11.55	10.36	72.5	73.2	36.34	1.41	NA	NA	NA	NA	21	16	9	11	34	34	N
	Mean SD	53.4 14.4		17 8.8	24.1 44.9	14.2 19.6	87.9 22	89.5 24.6	30.4 79.4	9.3 27.2	247.2 102.9	219.7 97.1	68.2 26.9	60 26.1	14.3 4.6	12.6 4.2	11.5 4.1	10.3 2.4	22.7 7.1	21.5 7.7
p value†					0.043		NS		NS		0.015		0.008		0.006		0.024		0.019
N					34		35		35		31		31		33		33		36

*Reference range: <0.01–0.5.
†Paired t test, significance at p<0.05.
ACR, albumin-to-creatine ratio; eGFR, estimated glomerular filtration rate; IVS, intraventricular septum; LPW, left posterior wall; LVM, left ventricular mass; LVMI, left-ventricular mass index; lyso-Gb3, globotriaosylsphingosine; NA, not available; NS, not significant.