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

From: 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

  All patients continuously treated with miglustat (N = 92) Patients with age at neurological onset and treatment data
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
  1. *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