Table III.

Berotralstat effectiveness

Patient no.	General impact of berotralstat (physician reported)	No. of attacks per month				Average duration of attacks	Average severity of attacks		Response to acute treatment (physician reported)
		Before transition	Month 1	Month 3	Month 6		Before transition	After transition
1	Partially effective with decrease in AE frequency, but persistence of attacks reported; unclear if symptoms were due to berotralstat introduction vs tranexamic acid dose increase.	2∗	0.5	0.5	0.5	15 minutes†	Severe	Severe	Icatibant immediately effective (no repeat dosing needed)
2	Obvious rapid benefit. Attacks stopped almost completely without tranexamic acid. A few more hand swellings in first few weeks, but facial swellings stopped immediately.	4	4	2	1	Half a day	Moderate	Minor	NA—no significant attacks recently
3	Attacks now very occasional, mild, and self-limiting; able to attend school and take exams. After 1 year’s therapy, had orofacial surgery and had no associated angioedema attacks.	4	3	3	1	Quarter of a day	Moderate	Mild	No repeat dosing of icatibant required
4	Major impact—very few attacks after therapy for a few months since initiating berotralstat; any attacks are mostly self-limiting or respond to single 1.5 g dose of tranexamic acid.	8	4	2	1	Half a day	Moderate	Mild	Response in <4 hours
5	Reduction in frequency of HAE attacks.	3.5	1	1	2.5	<1 day	Severe	Severe	Good response to icatibant
6	NR	0.5	0	0‡	0	1 hour†	Severe	Severe	Icatibant very effective

NA, Not applicable; NR, not reported.

^∗Patient 1 attack rate before therapy.

^†After administration of icatibant.

^‡Just 1 attack reported at month 4.