Table 6.

AE type and outcomes for AEs grade 5 (fatal)

	Age	Sex	Gene	Indication for treatment			Pulmonary AVMs	Treatment response	Treatment duration at event	Fatal event (SOC)	Drug-related AE?
				Bleeding	Support	Mean Hemoglobin (Hb)
BZB	67	Male	ENG	Refractory GI bleeding	Regular blood transfusions	< 6 g/dL	Previously treated, no follow up for 5 ys	Excellent, mean Hb > 10 g/dL	65 months	Haemoptysis from ruptured PAVM (Vasc Disord)	Possibly related ; theoretical alternate cause: spontaneous rupture
TH	69	Male	ACVRL1	Refractory epistaxis	Regular blood transfusions	< 7 g/dL	no	Good: mean Hb > 9 g/dL; fewer tx	10 months	Cardiac failure (Card Disord)	Possibly related; theoretical alternate cause: ischemic cardiopathy
	62	Male	ENG	Refractory GI bleeding	Regular blood transfusions	< 7 g/dL	no	Partial, mean Hb > 8 g/dL; fewer tx	23 months	Ischemic stroke (Vasc Disord)	possibly related; theoretical alternate cause: atherosclerosis
	78	Male	ENG	Refractory epistaxis	Regular blood transfusions	< 7 g/dL	no	Good: mean Hb > 9 g/dL; fewer tx	2 months	Catastrophic epistaxis (Resp Disord)	possibly related; theoretical alternate cause: spontaneous nosebleed

Legend: Drug dosing schedules provided in the text. SOC System Organ Classification