Demographics and clinical characteristics of participants with severe HA
| . | Total cohort N = 171 . | 50 EDs end point n = 98 . | Confirmed inhibitor end point n = 39 . | Early study exit∗ n = 34 . |
|---|---|---|---|---|
| Birth year cohort, n (%) | ||||
| 1 Jan 2010 to 31 Dec 2012 | 34 (19.88) | 25 (25.51) | 8 (20.51) | 1 (2.94) |
| 1 Jan 2013 to 3 Oct 2018 | 106 (61.99) | 66 (67.35) | 29 (74.36) | 11 (32.35) |
| After 3 Oct 2018 | 31 (18.13) | 7 (7.14) | 2 (5.13) | 22 (64.71) |
| Ethnicity, n (%) | ||||
| Hispanic, Latino/a, or Spanish origin | 34 (19.88) | 21 (21.43) | 6 (15.38) | 7 (20.59) |
| Not Hispanic, Latino/a, or Spanish origin | 137 (80.12) | 77 (78.57) | 33 (84.62) | 27 (79.41) |
| Unknown | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Race, n (%) | ||||
| White | 120 (70.18) | 69 (70.41) | 27 (69.23) | 24 (70.59) |
| Black or African American | 25 (14.62) | 13 (13.27) | 8 (20.51) | 4 (11.76) |
| Asian | 7 (4.09) | 4 (4.08) | 1 (2.56) | 2 (5.88) |
| Native Hawaiian or other Pacific Islander | 1 (0.58) | 0 (0.0) | 0 (0.0) | 1 (2.94) |
| American Indian or Alaska Native | 2 (1.17) | 1 (1.02) | 0 (0.0) | 1 (2.94) |
| Multiple races | 9 (5.26) | 7 (7.14) | 1 (2.56) | 1 (2.94) |
| Unknown | 7 (4.09) | 4 (4.08) | 2 (5.13) | 1 (2.94) |
| Ever CFC exposure, n (%) | ||||
| Yes | 164 (95.91) | 98 (100.0) | 39 (100.0) | 27 (79.41) |
| No‡ | 7 (4.09) | 0 (0.0) | 0 (0.0) | 7 (20.59) |
| Ever prophylaxis, n (%) | ||||
| Yes | 123 (71.93) | 86 (87.76) | 24 (61.54) | 13 (38.24) |
| No | 48 (28.07) | 12 (12.24) | 15 (38.46) | 21 (61.76) |
| Age at first CFC exposure, n (%) | ||||
| <1 mo | 55 (32.16) | 26 (26.53) | 16 (41.03) | 13 (38.24) |
| 1-12 mo | 78 (45.61) | 56 (57.14) | 12 (30.77) | 10 (29.41) |
| 1-10 y | 27 (15.79) | 16 (16.33) | 7 (17.95) | 4 (11.76) |
| Unknown | 4 (2.34) | 0 (0.0) | 4 (10.26) | 0 (0.0) |
| Never had CFC exposure | 7 (4.09) | 0 (0.0) | 0 (0.0) | 7 (20.59) |
| Treatment regimen before study exit, n (%) | ||||
| On-demand | 54 (31.58) | 17 (17.35) | 19 (48.72) | 18 (52.94) |
| Prophylaxis | 110 (64.33) | 81 (82.65) | 17 (43.59) | 12 (35.29) |
| No product usages | 4 (2.34) | 0 (0.0) | 0 (0.0) | 4 (11.76) |
| Unknown | 3 (1.75) | 0 (0.0) | 3 (7.69) | 0 (0.0) |
| Treatment class before study exit, n (%) | ||||
| Plasma derived | 29 (16.96) | 13 (13.27) | 8 (20.51) | 8 (23.53) |
| SHL recombinant | 95 (55.56) | 61 (62.24) | 25 (64.1) | 9 (26.47) |
| EHL recombinant | 25 (14.62) | 22 (22.45) | 2 (5.13) | 1 (2.94) |
| FVIII mimetic | 15 (8.77) | 2 (2.04) | 1 (2.56) | 12 (35.29) |
| No product usages | 4 (2.34) | 0 (0.0) | 0 (0.0) | 4 (11.76) |
| Missing data§ | 3 (1.75) | 0 (0.0) | 3 (7.69) | 0 (0.0) |
| Known CFC EDs | ||||
| N | 167 | 98 | 35 | 34 |
| Mean (standard deviation) | 33.2 (21.01) | 50 (0) | 12.09 (8.87) | 6.53 (9.81) |
| Median | 50 | 50 | 11 | 2 |
| IQR | 41 (9-50) | N/A | 12 (5-17) | 6 (1-7) |
| Range | 0-50 | 50 | 1-38 | 0-41 |
| . | Total cohort N = 171 . | 50 EDs end point n = 98 . | Confirmed inhibitor end point n = 39 . | Early study exit∗ n = 34 . |
|---|---|---|---|---|
| Birth year cohort, n (%) | ||||
| 1 Jan 2010 to 31 Dec 2012 | 34 (19.88) | 25 (25.51) | 8 (20.51) | 1 (2.94) |
| 1 Jan 2013 to 3 Oct 2018 | 106 (61.99) | 66 (67.35) | 29 (74.36) | 11 (32.35) |
| After 3 Oct 2018 | 31 (18.13) | 7 (7.14) | 2 (5.13) | 22 (64.71) |
| Ethnicity, n (%) | ||||
| Hispanic, Latino/a, or Spanish origin | 34 (19.88) | 21 (21.43) | 6 (15.38) | 7 (20.59) |
| Not Hispanic, Latino/a, or Spanish origin | 137 (80.12) | 77 (78.57) | 33 (84.62) | 27 (79.41) |
| Unknown | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Race, n (%) | ||||
| White | 120 (70.18) | 69 (70.41) | 27 (69.23) | 24 (70.59) |
| Black or African American | 25 (14.62) | 13 (13.27) | 8 (20.51) | 4 (11.76) |
| Asian | 7 (4.09) | 4 (4.08) | 1 (2.56) | 2 (5.88) |
| Native Hawaiian or other Pacific Islander | 1 (0.58) | 0 (0.0) | 0 (0.0) | 1 (2.94) |
| American Indian or Alaska Native | 2 (1.17) | 1 (1.02) | 0 (0.0) | 1 (2.94) |
| Multiple races | 9 (5.26) | 7 (7.14) | 1 (2.56) | 1 (2.94) |
| Unknown | 7 (4.09) | 4 (4.08) | 2 (5.13) | 1 (2.94) |
| Ever CFC exposure, n (%) | ||||
| Yes | 164 (95.91) | 98 (100.0) | 39 (100.0) | 27 (79.41) |
| No‡ | 7 (4.09) | 0 (0.0) | 0 (0.0) | 7 (20.59) |
| Ever prophylaxis, n (%) | ||||
| Yes | 123 (71.93) | 86 (87.76) | 24 (61.54) | 13 (38.24) |
| No | 48 (28.07) | 12 (12.24) | 15 (38.46) | 21 (61.76) |
| Age at first CFC exposure, n (%) | ||||
| <1 mo | 55 (32.16) | 26 (26.53) | 16 (41.03) | 13 (38.24) |
| 1-12 mo | 78 (45.61) | 56 (57.14) | 12 (30.77) | 10 (29.41) |
| 1-10 y | 27 (15.79) | 16 (16.33) | 7 (17.95) | 4 (11.76) |
| Unknown | 4 (2.34) | 0 (0.0) | 4 (10.26) | 0 (0.0) |
| Never had CFC exposure | 7 (4.09) | 0 (0.0) | 0 (0.0) | 7 (20.59) |
| Treatment regimen before study exit, n (%) | ||||
| On-demand | 54 (31.58) | 17 (17.35) | 19 (48.72) | 18 (52.94) |
| Prophylaxis | 110 (64.33) | 81 (82.65) | 17 (43.59) | 12 (35.29) |
| No product usages | 4 (2.34) | 0 (0.0) | 0 (0.0) | 4 (11.76) |
| Unknown | 3 (1.75) | 0 (0.0) | 3 (7.69) | 0 (0.0) |
| Treatment class before study exit, n (%) | ||||
| Plasma derived | 29 (16.96) | 13 (13.27) | 8 (20.51) | 8 (23.53) |
| SHL recombinant | 95 (55.56) | 61 (62.24) | 25 (64.1) | 9 (26.47) |
| EHL recombinant | 25 (14.62) | 22 (22.45) | 2 (5.13) | 1 (2.94) |
| FVIII mimetic | 15 (8.77) | 2 (2.04) | 1 (2.56) | 12 (35.29) |
| No product usages | 4 (2.34) | 0 (0.0) | 0 (0.0) | 4 (11.76) |
| Missing data§ | 3 (1.75) | 0 (0.0) | 3 (7.69) | 0 (0.0) |
| Known CFC EDs | ||||
| N | 167 | 98 | 35 | 34 |
| Mean (standard deviation) | 33.2 (21.01) | 50 (0) | 12.09 (8.87) | 6.53 (9.81) |
| Median | 50 | 50 | 11 | 2 |
| IQR | 41 (9-50) | N/A | 12 (5-17) | 6 (1-7) |
| Range | 0-50 | 50 | 1-38 | 0-41 |
Birth cohorts chosen based on approval dates for new products.
Dec, December; Jan, January; IQR, interquartile range; SHL, standard half-life.
Severe HA: 1 Jan 2010 to 31 Dec 2012: 1 of 34 exited early and had 1 ED at study exit. 1 Jan 2013 to 3 Oct 2018: 11 of 106 participants exited early with mean 8.45 ± 12.47 EDs; median, 2 EDs; range, 0-41 EDs; IQR, (Q1, Q3) 6 (1.5, 7.5) EDs. After 3 Oct 2018: 22 of 31 participants with mean 5.82 ± 8.56 EDs; median, 2 EDs; range, 0-29 EDs; IQR, (Q1, Q3) 7 (0, 7) EDs.
Includes participants on prophylaxis taking FVIII mimetic.
Three participants with confirmed inhibitor did not have any recorded factor products.