Table 1. Summary of clinical and... | American Society of Hematology

Table 1.

Summary of clinical and recombinant data of FVIII mimetic–rescuable HB FIX variants

Variant	Chymotrypsin no.	FIX domain	Recombinant FIX				Clinical data
Variant	Chymotrypsin no.	FIX domain	Activity (% WT)	Antigen (% WT)	Clot time Enh∗	TGA Enh∗	Activity (%)	Antigen (%)	Reported patients
N2I	NA	Gla	3	100	3	4	1	60	1
K5E	NA	Gla	5	190	3	4	4-7	NR	2
L6S	NA	Gla	2	140	3	6	<1	NR	3
F25C	NA	Gla	6	50	2	2	10	NR	1
F25S	NA	Gla	13	150	3	8	2-5	25-32	5
D47E	NA	EGF1	9	100	7	4	1-14	80-90	2
D47G	NA	EGF1	8	30	4	1	10-24	100	2
N67K	NA	EGF1	35	60	2	1	<1-12	NR	2
E78K	NA	EGF1	2	40	2	2	<1	<1-84	5
E83K	NA	EGF1	<1	10	11	2	<1	<10	1
N92K	NA	EGF2	<1	30	30	18	<1-2	86	4
N92S	NA	EGF2	3	50	11	25	NR	NR	2
V182A	17	Protease	6	70	3	1	23	100	1
V182F	17	Protease	<1	100	3	2	<1	120	2
V182L	17	Protease	2	30	9	3	4-15	132	3
D269E	102	Protease	<1	5	3	2	<1	5	1
Y295C	128	Protease	<1	10	4	4	<1	NR	2
D332Y	164	Protease	3	20	4	1	3-4	NR	3
R333L	165	Protease	<1	30	87	17	2	150	2
R333P	165	Protease	1	40	49	6	1.7	NR	1
R333Q	165	Protease	1	110	49	8	<1-16	32-148	69
R338P	170	Protease	7	80	6	3	10-16	27	3
T340I	172	Protease	1	30	17	4	NR	NR	2
T340R	172	Protease	<1	80	9	6	<1	NR	1
K341E	173	Protease	5	100	26	2	<1-12	NR	8
K341N	173	Protease	<1	80	64	11	<1	55	1
I344F	176	Protease	1	70	13	2	<1-4	87	4
I344N	176	Protease	<1	60	22	13	NR	NR	1
I344S	176	Protease	<1	60	13	15	<1-4	112-119	2
I344T	176	Protease	<1	60	13	5	1-2	28	2
M348I	180	Protease	<1	80	4	11	<1	40-93	4
M348K	180	Protease	<1	40	3	1	<1-2	12	2
M348R	180	Protease	<1	50	14	3	NR	NR	1
G356R	187	Protease	1	160	24	2	<1-4	75	9
P368H	198	Protease	<1	50	7	6	<1	102	1
P368R	198	Protease	10	60	8	1	<1	NR	1
P368T	198	Protease	1	130	7	7	<1-7	100-156	13
E387A	217	Protease	2	170	7	2	<1-4	90-118	6
E387G	217	Protease	2	390	24	16	2	95	2
E387K	217	Protease	1	60	17	4	<1-2	63-74	10
I397T	227	Protease	2	70	6	6	<1-12	27-160	44

Variant	Chymotrypsin no.	FIX domain	Recombinant FIX				Clinical data
Variant	Chymotrypsin no.	FIX domain	Activity (% WT)	Antigen (% WT)	Clot time Enh∗	TGA Enh∗	Activity (%)	Antigen (%)	Reported patients
N2I	NA	Gla	3	100	3	4	1	60	1
K5E	NA	Gla	5	190	3	4	4-7	NR	2
L6S	NA	Gla	2	140	3	6	<1	NR	3
F25C	NA	Gla	6	50	2	2	10	NR	1
F25S	NA	Gla	13	150	3	8	2-5	25-32	5
D47E	NA	EGF1	9	100	7	4	1-14	80-90	2
D47G	NA	EGF1	8	30	4	1	10-24	100	2
N67K	NA	EGF1	35	60	2	1	<1-12	NR	2
E78K	NA	EGF1	2	40	2	2	<1	<1-84	5
E83K	NA	EGF1	<1	10	11	2	<1	<10	1
N92K	NA	EGF2	<1	30	30	18	<1-2	86	4
N92S	NA	EGF2	3	50	11	25	NR	NR	2
V182A	17	Protease	6	70	3	1	23	100	1
V182F	17	Protease	<1	100	3	2	<1	120	2
V182L	17	Protease	2	30	9	3	4-15	132	3
D269E	102	Protease	<1	5	3	2	<1	5	1
Y295C	128	Protease	<1	10	4	4	<1	NR	2
D332Y	164	Protease	3	20	4	1	3-4	NR	3
R333L	165	Protease	<1	30	87	17	2	150	2
R333P	165	Protease	1	40	49	6	1.7	NR	1
R333Q	165	Protease	1	110	49	8	<1-16	32-148	69
R338P	170	Protease	7	80	6	3	10-16	27	3
T340I	172	Protease	1	30	17	4	NR	NR	2
T340R	172	Protease	<1	80	9	6	<1	NR	1
K341E	173	Protease	5	100	26	2	<1-12	NR	8
K341N	173	Protease	<1	80	64	11	<1	55	1
I344F	176	Protease	1	70	13	2	<1-4	87	4
I344N	176	Protease	<1	60	22	13	NR	NR	1
I344S	176	Protease	<1	60	13	15	<1-4	112-119	2
I344T	176	Protease	<1	60	13	5	1-2	28	2
M348I	180	Protease	<1	80	4	11	<1	40-93	4
M348K	180	Protease	<1	40	3	1	<1-2	12	2
M348R	180	Protease	<1	50	14	3	NR	NR	1
G356R	187	Protease	1	160	24	2	<1-4	75	9
P368H	198	Protease	<1	50	7	6	<1	102	1
P368R	198	Protease	10	60	8	1	<1	NR	1
P368T	198	Protease	1	130	7	7	<1-7	100-156	13
E387A	217	Protease	2	170	7	2	<1-4	90-118	6
E387G	217	Protease	2	390	24	16	2	95	2
E387K	217	Protease	1	60	17	4	<1-2	63-74	10
I397T	227	Protease	2	70	6	6	<1-12	27-160	44

Clinical data extracted from FIX-variant database.¹⁵

NR, not reported.

Clot time and TGA enhancement (Enh) are the ratios of the parameter with and without emicizumab as detailed in article text.