Effect of carglumic acid with or without ammonia scavengers on hyperammonaemia in acute decompensation episodes of organic acidurias

Table 3 Treatment-emergent adverse events

	Carglumic acid (n = 38)	NH₃ scavengers (n = 33)	Carglumic acid + NH₃ scavengers (n = 27)
Number of TEAEs	54	85	18
Number of drug-related^a TEAEs	18	1	6
Number of serious TEAEs	13	31	7
Number of drug-related^b serious TEAEs	5	0	1
TEAEs leading to death	3	13	6
TEAEs occurring in ≥10% of episodes in any treatment group, n episodes (%)
Coagulopathy	1 (2.6)^b	5 (15.2)	0
Thrombocytopenia	2 (5.3)^b	7 (21.2)	1 (3.7)
Hyperglycaemia	1 (2.6)	5 (15.2)	1 (3.7)
Hypocalcaemia	1 (2.6)^b	4 (12.1)	0
Hypokalaemia	1 (2.6)	6 (18.2)	0
Respiratory distress	0	4 (12.1)	0

NH₃ Ammonia, TEAE Treatment-emergent adverse event
^aDrug-relatedness in the combination group refers exclusively to carglumic acid treatment and not NH₃ scavenger treatment
^bConsidered to be drug-related in one episode/patient. n represents the number of episodes evaluated

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