Table 3. Clinical data for subjects.
Subject | Age | HA Severity | Baseline FVIII | Peak Titer(BU/ml)a | Inhibitor Treatment History | Hemophilia Genotype(if known) |
Predominantly IgG1 Response | ||||||
17A | 24 | mild | 6–14% | 250 | ITI failedb | A2201P |
N-008 | 2 | moderate | 3% | 11 | no ITI | 14–21 delc |
L-006 | 2 | moderate | 1% | 87 | ITI initiated | R2304C |
Predominantly IgG4 Response | ||||||
F-014 | 19 | severe | <1% | 32 | ITI partly successful | int-22 invd |
B-002 | 20 | severe | <1% | 667 | ITI failed | 9–11 dele |
A-002 | 14 | severe | <1% | 256 | ITI failed | not inversionf |
Mixed IgG Subtype Response | ||||||
G-004 | 16 | severe | <1% | 1000+ | no ITI | int-22 inv |
C-010 | 27 | severe | <1% | 80 | ITI partly successful | not inversion |
D-006 | 10 | severe | <1% | 496 | ITI failed | not inversion |
L-025 | 35 | severe | <1% | 191 | no ITI | not inversion |
P-011 | 8 | severe | <1% | 1084.4 | ITI failed | int-22 inv |
P-001 | 12 | severe | <1% | 308.7 | ITI failed | int-22 inv |
F-006 | 27 | severe | <0.25% | 44 | no ITI | int-22 inv |
A-008 | 31 | severe | <1% | 86 | ITI successful | int-22 inv |
F-025 | 21 | severe | <1% | 43.8 | ITI failed | int-22 inv |
C-019 | 60 | severe | <1% | 336 | ITI failed | int-22 inv |
C-028 | 2 | severe | <1% | 96 | ITI failed | not inversion |
Secondary and primary SPR binding signals (in RU) do not match | ||||||
H-001 | 50 | severe | <1% | 742 | no ITI | int-22 inv |
Autoimmune subjects | ||||||
Q-011 | 77 | autoimmune | normal | 6 | prednisone | autoimmune |
Q-012 | 77 | autoimmune | normal | 2 | prednisone | autoimmune |
Q-033 | 79 | autoimmune | normal | 39 | prednisone | autoimmune |
Q-016 | 62 | autoimmune | normal | 20 | prednisone | autoimmune |
BU/ml = Bethesda Units/milliliter;
ITI = Immune Tolerance Induction;
14–21del = exons 14–21 deleted;
int-22 inv = intron 22 inversion;
9–11del = exons 9–11 deleted;
not inversion = not an intron-22 or intron-1 inversion mutation.