Table 2. Comparative outcomes at... | American Society of Hematology

Table 2.

Comparative outcomes at baseline and at 3 years after etranacogene dezaparvovec treatment in the 3 participants

Outcome	Participant 1		Participant 2		Participant 3
Outcome	At baseline	At 3 y	At baseline	At 3 y	At baseline	At 3 y
ABR (all bleeds)	3∗,†	0	1∗	0	6∗	0.64
Mean on-demand FIX use (IU/y)	24 000∗	0	7 681∗	0	63 000∗	2 143.9‡
Total HJHS score	35§	31	36§	32	1§	NA
Hem-A-QoL total score	26.7§	8.3	13.0§	5.0	13.5§	15.7

Outcome	Participant 1		Participant 2		Participant 3
Outcome	At baseline	At 3 y	At baseline	At 3 y	At baseline	At 3 y
ABR (all bleeds)	3∗,†	0	1∗	0	6∗	0.64
Mean on-demand FIX use (IU/y)	24 000∗	0	7 681∗	0	63 000∗	2 143.9‡
Total HJHS score	35§	31	36§	32	1§	NA
Hem-A-QoL total score	26.7§	8.3	13.0§	5.0	13.5§	15.7

ABR, annualized bleeding rate; NA, not available.

Data collected retrospectively 1 year before screening from medical records.

One bleed occurred after enrollment but before dosing.

Except for FIX use for the prevention of bleeds during operations.

At day of dosing