Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry

Table 6 Safety information for patients continuously treated with miglustat before miglustat initiation and during NPC Registry follow-up (N = 241)

Safety information/event	Time period	Patient, N	Patient with events, n (%^e)
Seizures	Pretreatment: present	241	81 (33.6)
Seizures	During follow-up: new or worsened^d	103	48 (46.6)
Thrombocytopenia^a	Pretreatment: present	241	49 (20.3)
Thrombocytopenia^a	During follow-up: present	211	109 (51.7)
Tremor	Pretreatment: present	241	86 (35.7)
Tremor	During follow-up: new or worsened^d	241	37 (15.4)
Neuropathy	Pretreatment: present	240	18 (7.5)
Neuropathy	During follow-up: new or worsened^d	241	19 (7.9)
Chronic diarrhea^b	Pretreatment: present	241	11 (4.6)
Chronic diarrhea^b	During follow-up: new or worsened^d	241	27 (11.2)
Other^c	Pretreatment: present	231	13 (5.6)

^a101 patients had mild thrombocytopenia (101–150 × 10⁹/L) and 32 had moderate thrombocytopenia (51–100 × 10⁹/L)
^bDiarrhea lasting > 3 months
^cAny other possibly related adverse event not considered as thrombocytopenia, neuropathy, seizure, tremor, or gastrointestinal-related event
^dEvent occurred at least once during follow-up
^ePercentages based on patients with available data; for some patients this information is unknown or missing

ISSN: 1750-1172