Skip to main content

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)
  1. a The interviews are counted by patient, however 4 caregivers (3 in US and 1 in UK) interviewed had 2 children in the family with NPC. Number of participants interviewed indicated in parentheses
  2. *Youngest participant 13 months old