Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study

Table 2 Miglustat exposure among continuously treated patients by neurological onset category

	All patients continuously treated with miglustat (N = 92)	Patients with age at neurological onset and treatment data
	All patients continuously treated with miglustat (N = 92)	EI (<2 years)	LI (2 – <6 years)	JUV (6 – <15 years)	AA (≥15 years)
Age at 1st miglustat dose, years
n*	82	9	23	27	15
Mean (SD)	16.1 (10.8)	2.9 (1.9)	9.1 (6.9)	19.6 (8.2)	27.1 (8.0)
Range	0.7–44.6	0.8 − 7.4	2.1 − 28.5	9.8 − 41.2	16.3 − 44.6
Pre-enrolment miglustat exposure, years
n*	91	9	27	29	18
Mean (SD)	1.9 (1.8)	1.7 (1.5)	2.4 (2.1)	1.5 (1.5)	1.7 (1.9)
Range	0–7.6	0.3 − 4.5	0 − 7.6	0 − 4.6	0 − 6.0
Miglustat exposure during follow up ^† , years
n*	92	9	27	30	18
Mean (SD)	2.0 (0.7)	1.8 (0.4)	1.9 (0.6)	2.3 (0.7)	1.9 (0.5)
Range	1.0–3.7	1.4–2.8	1.1–3.3	1.0–3.7	1.0–3.2
Overall miglustat exposure ^‡ , years
n*	91	9	27	29	18
Mean (SD)	3.9 (1.9)	3.4 (1.42)	4.3 (2.2)	3.9 (1.7)	3.6 (1.9)
Range	1.1–9.8	2.1 − 6.0	1.6 − 9.8	1.3 − 6.8	1.3 − 7.9

*Number of patients with available data (eight patients who had no age at neurological onset data are not included in the age-at-onset subgroup columns); **exposure period from most recent miglustat treatment start date up to and including NPC Registry enrolment day; ^†period from Registry start to last follow-up visit; ^‡period from most recent miglustat treatment start date prior to enrolment up to last follow-up visit in the NPC Registry. EI, early-infantile onset; LI, late-infantile onset; JUV; juvenile onset; AA, adolescent/adult onset

ISSN: 1750-1172