Changes in hemostatic parameters over the course of the study
. | Study arm . | Baseline; median (IQR) . | T1; median (IQR) . | Fold change (vs baseline) . | T3; median (IQR) . | Fold change (vs baseline) . | T4; median (IQR) . | Fold change (vs baseline) . |
---|---|---|---|---|---|---|---|---|
FVIII:C (%) | Arm A | 23.8 (7-50.7) | 48.3 (16.7-66.4) | 2 | 75.9 (23.2-101.7) | 3.2 | 66.7 (17.5-124.9) | 2.8 |
Arm B | 21.4 (6.9-50.4) | 37.2 (21.6-111.3) | 1.7 | 47.4 (20.4-111.2) | 2.2 | 42.8 (19.6-207.5) | 2 | |
Arm C | 32 (7.3-65.1) | 47.7 (15.6-155.2) | 1.5 | 63.3 (22.6-168.6) | 2 | 77 (30.1-166.1) | 2.4 | |
Arm D | 14.6 (8.2-70.6) | 29.6 (10.9-151.9) | 2 | 23.9 (9.3-133) | 1.4 | 20.3 (9.1-101.8) | 1.4 | |
VWG:Ag (%) | Arm A | 84.9 (80.4-121.3) | 118.8 (107.3-176) | 1.4 | 173.9 (145.2-266.8) | 2*† | 200.8 (128.2-266.8) | 2.4*† |
Arm B | 131.5 (89-172) | 156.6 (117.3-197.7) | 1.2 | 181.6 (139.6-227.1) | 1.4*† | 265.6 (145.2-288.9) | 2*† | |
Arm C | 88.7 (64.7-123.9) | 101.4 (71.1-112.5) | 1.1 | 107.5 (89.1-119.5) | 1.2 | 97.3 (93.8-165.5) | 1.1 | |
Arm D | 78.8 (61.2-89) | 80.3 (65.6-134.7) | 1 | 72.8 (56.4-130) | 1 | 76.8 (57.4-125.3) | 1 | |
VWF:Gp1bM (%) | Arm A | 88.7 (68.2-126.9) | 145.7 (114.9-169.3) | 1.6 | 207.4 (169.7-286.8) | 2.3* | 221.8 (233.8-152.1) | 2.5* |
Arm B | 139.7 (77.3-157.7) | 179.3 (145.9-210.6) | 1.3 | 192.8 (99-251.2) | 1.4* | 185.7 (136.4-228.6) | 1.3* | |
Arm C | 77.9 (56.3-127.2) | 113.5 (82.8-141.8) | 1.5 | 141.3 (91.7-156.1) | 1.8 | 146.9 (119.1-214.7) | 1.9* | |
Arm D | 75.9 (58.3-94.9) | 76.2 (67.4-138.7) | 1 | 73.6 (63.4-137) | 1 | 72.3 (63.9-124.1) | 1 | |
Platelet count (x103/L) | Arm A | 233.5 (212-275) | 236.5 (203.5-289) | 1 | 297 (234-326.5) | 1.3 | 226.5 (195-260.8) | 1 |
Arm B | 282 (175.3-298.3) | 263 (171-302.8) | 0.9 | 265.5 (165.8-293) | 0.9 | 256.5 (167.3-296.3) | 0.9 | |
Arm C | 235 (200-253) | 246 (219-275) | 1 | 216 (199-267) | 0.9 | 222 (200-241) | 0.9 | |
Arm D | 260 (255.5-292.5) | 281 (245.5-332) | 1.1 | 235 (216-274.5) | 0.9 | 264 (223.5-286) | 1 | |
Col/Epi closure time (s) | Arm A | 143.5 (122.3-153.5) | 82 (75-89) | 0.6 | 77.5 (70.8-83.8) | 0.5* | 99.5 (75.3-110) | 0.7* |
Arm B | 139 (111.3-148.3) | 78.5 (74.3-111.3) | 0.6 | 91 (71.5-100.5) | 0.7* | 94.5 (77.5-117.3) | 0.7* | |
Arm C | 129.5 (97.8-149.8) | 121 (106-173) | 0.9 | 102 (76-119) | 0.8* | 96 (82-112) | 0.7* | |
Arm D | 142 (128.5-177.5) | 143 (113-162.5) | 1 | 150 (113-177.5) | 1 | 166 (110-183) | 1.1 | |
Col/ADP closure time (s) | Arm A | 100.5 (74.3-119.5) | 77 (60-81) | 0.8 | 58 (53-76.3) | 0.6* | 76.5 (65.5-87.3) | 0.8* |
Arm B | 80.5 (61.5-100.8) | 64.5 (48.8-81) | 0.8 | 63 (59.3-66) | 0.8* | 62 (56-69.8) | 0.8* | |
Arm C | 104 (85-115.5) | 83 (62-97) | 0.8 | 75 (65-78) | 0.7* | 67 (64-79) | 0.6* | |
Arm D | 105 (90-120.5) | 103 (79.5-108.5) | 1 | 91 (85-113.5) | 0.9 | 103 (91.5-130) | 1 | |
Hematocrit (%) | Arm A | 41.7 (40.6-44.6) | 41 (37.8-42.3) | 1 | 41.6 (40.3-44.9) | 1 | 40.2 (36.7-40.6) | 1 |
Arm B | 43.6 (42.3-45.6) | 41.4 (40-41.4) | 0.9 | 40.5 (39.6-43.1) | 0.9 | 40.4 (39.4-42.7) | 0.9 | |
Arm C | 44.2 (39.8-45.4) | 44.9 (41.45.9) | 1 | 40.9 (37.7-42.6) | 0.9 | 41.4 (36.8-42.7) | 0.9 | |
Arm D | 43.3 (41.3-47.7) | 44.5 (42.6-46.6) | 1 | 40.4 (39.2-43.2) | 0.9 | 40.6 (39.3-44.5) | 0.9 |
. | Study arm . | Baseline; median (IQR) . | T1; median (IQR) . | Fold change (vs baseline) . | T3; median (IQR) . | Fold change (vs baseline) . | T4; median (IQR) . | Fold change (vs baseline) . |
---|---|---|---|---|---|---|---|---|
FVIII:C (%) | Arm A | 23.8 (7-50.7) | 48.3 (16.7-66.4) | 2 | 75.9 (23.2-101.7) | 3.2 | 66.7 (17.5-124.9) | 2.8 |
Arm B | 21.4 (6.9-50.4) | 37.2 (21.6-111.3) | 1.7 | 47.4 (20.4-111.2) | 2.2 | 42.8 (19.6-207.5) | 2 | |
Arm C | 32 (7.3-65.1) | 47.7 (15.6-155.2) | 1.5 | 63.3 (22.6-168.6) | 2 | 77 (30.1-166.1) | 2.4 | |
Arm D | 14.6 (8.2-70.6) | 29.6 (10.9-151.9) | 2 | 23.9 (9.3-133) | 1.4 | 20.3 (9.1-101.8) | 1.4 | |
VWG:Ag (%) | Arm A | 84.9 (80.4-121.3) | 118.8 (107.3-176) | 1.4 | 173.9 (145.2-266.8) | 2*† | 200.8 (128.2-266.8) | 2.4*† |
Arm B | 131.5 (89-172) | 156.6 (117.3-197.7) | 1.2 | 181.6 (139.6-227.1) | 1.4*† | 265.6 (145.2-288.9) | 2*† | |
Arm C | 88.7 (64.7-123.9) | 101.4 (71.1-112.5) | 1.1 | 107.5 (89.1-119.5) | 1.2 | 97.3 (93.8-165.5) | 1.1 | |
Arm D | 78.8 (61.2-89) | 80.3 (65.6-134.7) | 1 | 72.8 (56.4-130) | 1 | 76.8 (57.4-125.3) | 1 | |
VWF:Gp1bM (%) | Arm A | 88.7 (68.2-126.9) | 145.7 (114.9-169.3) | 1.6 | 207.4 (169.7-286.8) | 2.3* | 221.8 (233.8-152.1) | 2.5* |
Arm B | 139.7 (77.3-157.7) | 179.3 (145.9-210.6) | 1.3 | 192.8 (99-251.2) | 1.4* | 185.7 (136.4-228.6) | 1.3* | |
Arm C | 77.9 (56.3-127.2) | 113.5 (82.8-141.8) | 1.5 | 141.3 (91.7-156.1) | 1.8 | 146.9 (119.1-214.7) | 1.9* | |
Arm D | 75.9 (58.3-94.9) | 76.2 (67.4-138.7) | 1 | 73.6 (63.4-137) | 1 | 72.3 (63.9-124.1) | 1 | |
Platelet count (x103/L) | Arm A | 233.5 (212-275) | 236.5 (203.5-289) | 1 | 297 (234-326.5) | 1.3 | 226.5 (195-260.8) | 1 |
Arm B | 282 (175.3-298.3) | 263 (171-302.8) | 0.9 | 265.5 (165.8-293) | 0.9 | 256.5 (167.3-296.3) | 0.9 | |
Arm C | 235 (200-253) | 246 (219-275) | 1 | 216 (199-267) | 0.9 | 222 (200-241) | 0.9 | |
Arm D | 260 (255.5-292.5) | 281 (245.5-332) | 1.1 | 235 (216-274.5) | 0.9 | 264 (223.5-286) | 1 | |
Col/Epi closure time (s) | Arm A | 143.5 (122.3-153.5) | 82 (75-89) | 0.6 | 77.5 (70.8-83.8) | 0.5* | 99.5 (75.3-110) | 0.7* |
Arm B | 139 (111.3-148.3) | 78.5 (74.3-111.3) | 0.6 | 91 (71.5-100.5) | 0.7* | 94.5 (77.5-117.3) | 0.7* | |
Arm C | 129.5 (97.8-149.8) | 121 (106-173) | 0.9 | 102 (76-119) | 0.8* | 96 (82-112) | 0.7* | |
Arm D | 142 (128.5-177.5) | 143 (113-162.5) | 1 | 150 (113-177.5) | 1 | 166 (110-183) | 1.1 | |
Col/ADP closure time (s) | Arm A | 100.5 (74.3-119.5) | 77 (60-81) | 0.8 | 58 (53-76.3) | 0.6* | 76.5 (65.5-87.3) | 0.8* |
Arm B | 80.5 (61.5-100.8) | 64.5 (48.8-81) | 0.8 | 63 (59.3-66) | 0.8* | 62 (56-69.8) | 0.8* | |
Arm C | 104 (85-115.5) | 83 (62-97) | 0.8 | 75 (65-78) | 0.7* | 67 (64-79) | 0.6* | |
Arm D | 105 (90-120.5) | 103 (79.5-108.5) | 1 | 91 (85-113.5) | 0.9 | 103 (91.5-130) | 1 | |
Hematocrit (%) | Arm A | 41.7 (40.6-44.6) | 41 (37.8-42.3) | 1 | 41.6 (40.3-44.9) | 1 | 40.2 (36.7-40.6) | 1 |
Arm B | 43.6 (42.3-45.6) | 41.4 (40-41.4) | 0.9 | 40.5 (39.6-43.1) | 0.9 | 40.4 (39.4-42.7) | 0.9 | |
Arm C | 44.2 (39.8-45.4) | 44.9 (41.45.9) | 1 | 40.9 (37.7-42.6) | 0.9 | 41.4 (36.8-42.7) | 0.9 | |
Arm D | 43.3 (41.3-47.7) | 44.5 (42.6-46.6) | 1 | 40.4 (39.2-43.2) | 0.9 | 40.6 (39.3-44.5) | 0.9 |