Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study

Table 3 Summary of adverse events by treatment time interval

	Treatment time interval
Event, n (%)	0–3 months^*(n = 9)	>3–6 months^*(n = 6)	>6–12 months^*(n = 6)
Any TEAE	8 (89)	5 (83)	6 (100)
Any related TEAE	4 (44)	1 (17)	3 (50)
Any IAR	3 (33)	2 (33)	3 (50)
Any serious TEAE	7 (78)	4 (67)	4 (67)
Any related serious TEAE	1 (11)	0	0
Dose modification due to a TEAE^†	3 (33)	1 (17)	1 (17)
Discontinuation due to a TEAE	0	0	0
Death	3 (33)	0	1 (17)
Death related to treatment	0	0	0

IAR infusion-associated reaction, TEAE treatment-emergent adverse event
^*Non-serious adverse event data were unavailable for one patient from week 0 to week 39 (~month 9)
^†All dose modifications were dose escalations based on inadequate clinical response. These symptoms of inadequate clinical response were also captured as adverse events (see Additional file 1)

ISSN: 1750-1172