Impacts and Burden of Niemann pick Type-C: a patient and caregiver perspective

Table 1 Baseline characteristics by study activity

Baseline characteristic		Survey	Interview
Total	n	49	28^a
Gender of patient, n (% of column total)	Female	24 (49.0)	10 (35.7)
Gender of patient, n (% of column total)	Male	25 (51.0)	18 (64.3)
Current age of patient, n (% of column total)	< 18 years	22 (44.9)	10 (35.7)
	18–29 years	13 (24.5)	9 (28.6)
	≥ 30 years	14 (30.6)	9 (35.7)
Reported age of onset of first NPC-related symptom, n (% of column total)^b	< 3 months	8 (16.3)	6 (21.4)
	3 months to < 2 years	7 (14.3)	2 (7.1)
	2 to < 6 years	7 (14.3)	2 (7.1)
	6 to 15 years	14 (28.6)	10 (35.7)
	> 15 years	13 (26.5)	8 (28.6)
5-domain NPCCSS score total (0–25), n (% of column total)^c	≤ 4	7 (14.3)	4 (14.3)
	5–9	13 (26.5)	7 (25.0)
	10–14	12 (24.5)	7 (25.0)
	15–19	11 (22.4)	7 (25.0)
	≥ 20	6 (12.2)	3 (10.7)

^aFour of the 28 interview participants were caregivers of two individuals with NPC. However, the interviews focused on the first individual diagnosed with NPC. The characteristics reported here are of the 28 patients who were the focus of the interview
^bAge of symptom onset based on patient self-report or caregiver report
^cHigher scores indicate greater severity

ISSN: 1750-1172