Table 2 NPC survey sample characteristics by Country
From: Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale
| Sample characteristic | Surveys | Interviews | |||||
|---|---|---|---|---|---|---|---|
| US | UK | Total | US | UK | Total | ||
| Total | n (% of row total) |
37 (75.5) |
12 (24.5) | 49 |
23 (71.9) (20 [71.4])a |
9 (28.1) (8 [28.6])a | 32a |
|
Current age n (% of column total) | 6 months to < 4 years* |
5 (13.5) | 0 |
5 (10.2) |
1 (4.3) | 0 | 1 (3.1) |
| 4–17 years |
13 (35.1) |
4 (33.3) |
17 (34.7) |
7 (30.4) |
3 (33.3) | 10 (31.3) | |
| ≥ 18 years |
19 (51.4) |
8 (66.7) |
27 (55.1) |
15 (65.2) |
6 (66.7) | 21 (65.6) | |
|
Reported age of onset of first NPC related symptom n (% of column total) | < 3 months |
6 (16.2) |
2 (16.7) |
8 (16.3) |
5 (21.7) |
1 (11.1) | 6 (18.8) |
| 3 months to < 2 years |
7 (18.9) | 0 |
7 (14.3) |
3 (13.0) | 0 | 3 (9.4) | |
| 2 to < 6 years |
6 (16.2) |
1 (8.3) |
7 (14.3) |
2 (8.7) | 0 | 2 (6.3) | |
| 6 to 15 years |
10 (27.0) |
4 (33.3) |
14 (28.6) |
8 (34.8) |
4 (44.4) | 12 (37.5) | |
| > 15 years |
8 (21.6) |
5 (41.7) |
13 (26.5) |
5 (21.7) |
4 (44.4) | 9 (28.1) | |
|
5-domain NPCCSS Score Total n (% of column total) | ≤ 4 | 6 (16.2) | 1 (8.3) | 7 (14.3) | 5 (21.7) | 1 (11.1) | 6 (18.8) |
| 5 to 19 | 29 (78.4) | 7 (58.3) | 36 (73.5) | 17 (73.9) | 6 (66.7) | 23 (71.9) | |
| ≥ 20 | 2 (5.4) | 4 (33.3) | 6 (12.2) | 1 (4.3) | 2 (22.2) | 3 (9.4) | |