| Alive, n (%)b |
122 (87.1) |
76 (87.4) |
46 (86.8) |
| Mean (SD) |
11.5 (7.7) |
10.9 (5.1) |
12.3 (10.8) |
| Median (range) |
9.8 (0.6, 62.5) |
10.2 (2.3, 22.8) |
8.6 (0.6, 62.5) |
| Deceased, n (%) |
18 (12.9) |
11 (12.6) |
7 (13.2) |
| Last documented ages, years (deceased) |
|
|
|
| Mean (SD) |
12.5 (4.9) |
12.6 (4.1) |
12.2 (6.3) |
| Median (range) |
13.4 (3.3, 20.2) |
12.9 (3.3, 18.6) |
14.2 (4.6, 20.2) |
| Cause of death, n (%) |
|
|
|
| Related to MPS II |
7 (38.9) |
4 (36.4) |
3 (42.9) |
| Unclear if related to MPS II |
5 (27.8) |
2 (18.2) |
3 (42.9) |
| Unknown |
6 (33.3) |
5 (45.5) |
1 (14.3) |
| Age at diagnosis, yearsc |
n = 136 |
n = 85 |
n = 51 |
| Mean (SD) |
3.8 (5.8) |
3 (2.1) |
5.2 (8.9) |
| Median (range)d |
2.8 (−0.1, 59.7) |
2.8 (0.0, 12.8) |
3.2 (−0.1, 59.7) |
| Family history of MPS II, n (%)e |
53 (37.9) |
33 (37.9) |
20 (37.7) |
| Sibling(s) with MPS II |
33 (62.3) |
20 (60.6) |
13 (65.0) |
| Uncle(s) with MPS II |
15 (28.3) |
10 (30.3) |
5 (25.0) |
| Ethnicity, n (%) |
|
|
|
| White/non-Hispanic |
76 (54.3) |
40 (46.0) |
36 (67.9) |
| Black/African American |
25 (17.9) |
20 (23.0) |
5 (9.4) |
| Hispanic/Latino |
21 (15.0) |
15 (17.2) |
6 (11.3) |
| Mixed |
7 (5.0) |
6 (6.9) |
1 (1.9) |
| Asian |
5 (3.6) |
3 (3.4) |
2 (3.8) |
| Other |
5 (3.6) |
2 (2.3) |
3 (5.7) |
| Unknown |
1 (0.7) |
1 (1.1) |
0 (0.0) |
| Insurance status, n (%) |
|
|
|
| Private |
58 (41.4) |
32 (36.8) |
26 (49.1) |
| Medicaid |
57 (40.7) |
39 (44.8) |
18 (34.0) |
| None |
1 (0.7) |
0 (0) |
1 (1.9) |
| Other |
9 (6.4) |
6 (6.9) |
3 (5.7) |
| Multiple insurances |
13 (9.3) |
9 (10.3) |
4 (7.5) |
| Unknown |
2 (1.4) |
1 (1.1) |
1 (1.9) |
| US region |
|
|
|
| East |
26 (18.6) |
21 (24.1) |
5 (9.4) |
| South |
32 (22.9) |
16 (18.4) |
16 (30.2) |
| Midwest |
52 (37.1) |
30 (34.5) |
22 (41.5) |
| Southwest |
17 (12.1) |
12 (13.8) |
5 (9.4) |
| West |
13 (9.3) |
8 (9.2) |
5 (9.4) |
| Patients receiving ERTf |
n = 139 |
n = 87 |
n = 52 |
| Number (%) |
108 (77.7) |
69 (79.3) |
39 (75.0) |