Table 2.

Clinical Severity, Cross-Reactive Immunologic Material Status, and Anti–Adeno-Associated Virus Serotype 8 Antibody Titers in Mucopolysaccharidosis VI Patients

				Involvement
Patient	Age at the last evaluation (years)/gender	Age at diagnosis (years)	Age at start of ERT (years)	Eye	ENT	Heart	Growth	Bone and joints	Spinal cord	Motor	Clinical score (0–24)	CRIM	Nab anti-AAV8
1	5/M	1.0	1.0	2	0	2	0	2	0	2	8	+	n.a.
2	5/M	1.0	2.0	1	0	2	1	2	0	2	8	+	<5
3	5/F	4.0	4.0	1	2	2	1	1	0	1	8	n.a	<5
4	6/M	1.9	2.3	2	1	1	1	2	0	1	8	+	160
5	7/M	2.5	3.0	1	1	1	2	2	0	2	9	+	5
6	7/F	4.0	4.0	1	2	3	2	2	0	2	12	+	40
7	7/M	1.7	2.5	1	2	2	1	1	0	1	8	−	<5
8	7/F	1.8	2.1	2	1	2	3	2	0	2	12	+	<5
9	8/F	4.5	n.a	1	2	2	2	1	0	2	10	+	n.a
10	8/M	3.0	3.0	1	2	2	2	1	0	1	9	+	<5
11	8/M	2.0	4.0	2	2	2	2	2	0	2	12	−	10
12	8/M	0.6	2.0	1	3	1	3	3	2	2	15	−	<5
13	9/F	2.8	2.9	2	0	2	2	2	1	2	11	+	<5
14	10/F	2.0	3.5	2	1	2	2	3	3	2	15	+	<5
15	10/M	1.0	4.0	2	1	2	2	3	2	2	14	+	<5
16	12/M	2.0	5.0	3	1	2	2	2	0	2	12	+	10
17	12/M	7.5	7.5	2	2	2	2	2	0	2	12	−	20
18	12/M	8.0	8.0	2	1	2	2	3	0	2	12	−	20
19	12/F	5.0	6.0	2	1	2	2	1	0	2	10	+	n.a
20	12/M	5.0	5.4	2	2	1	3	3	3	3	17	−	<5
21	12/M	5.8	6.1	1	0	0	0	2	0	2	5	+	20
22	12/M	1.2	6.1	2	2	2	3	3	3	3	18	n.a	<5
23	12/M	1.6	4.0	2	2	2	2	2	1	2	13	−	<5
24	13/F	4.0	7.0	3	2	2	2	1	2	2	14	+	5
25	13/M	5.1	5.9	0	0	1	2	2	0	3	8	+	<5
26	13/F	7.4	7.8	0	0	0	2	2	0	2	6	+	640
27	13.5/F	3.4	6.8	2	2	3	3	3	0	3	16	−	<5
28	14/F	7.8	8.3	1	1	1	2	2	0	1	8	+	<5
29	14/M	0.7	7.6	0	0	1	0	2	0	2	5	+	<5
30	14/M	10.2	10.6	0	0	1	1	2	0	3	7	+	<5
31	14/F	4.0	6.0	1	0	1	1	1	1	0	5	+	<5
32	17/M	12	13.0	0	1	2	2	3	3	2	13	+	20
33	17/M	3.5	11.5	2	3	2	2	3	2	2	16	−	160
34	21/F	4.5	10.0	2	2	3	3	3	1	2	16	−	320
35	23/M	10.1	18.3	0	1	1	2	2	0	3	9	+	<5
36	25/F	23	24	0	1	2	2	3	2	2	12	+	20

CRIM, cross-reactive ARSB immunologic material; ENT, ear, nose, and throat; ERT, enzyme replacement therapy; F, female; M, male; n.a, not available.

Involvement was graded as absent (0), mild (1), moderate (2), or severe (3).