Distribution of current treatment products used for prophylaxis and age of prophylaxis initiation between birth cohorts 2011 to 2021
| . | Total . | 2011-2015 . | 2016-2018 . | 2019-2021 . | P value . |
|---|---|---|---|---|---|
| # (%) . | # (%) . | # (%) . | # (%) . | ||
| Hemophilia A | 691 | 203 | 325 | 163 | |
| Continuous prophylaxis (including continuous prophylaxis with bypassing agents plus ITI) | 252 (36.5%) | 71 (35.0%) | 119 (36.6%) | 62 (38.0%) | |
| Severe hemophilia | 229 (90.9%) | 64 (90.1%) | 107 (89.9%) | 58 (93.5%) | |
| Age at initiation of prophylaxis mean (mo) | |||||
| Mean (SD) | 10.1 ± 6.2 | 10.8 ± 6.8 | 11.1 ± 5.6 | 7.1 ± 5.6 | |
| 0-6 mo | 50 (25.4%) | 17 (24.6%) | 15 (17.6%) | 18 (41.9%) | <.0001 |
| 7-23 mo | 147 (74.6%) | 52 (75.4%) | 70 (82.4%) | 25 (58.1%) | |
| 7-12 mo | 123 (62.4%) | 41 (59.4%) | 58 (68.2%) | ∗ | |
| 13-23 mo | 24 (12.2%) | 11 (15.9%) | 12 (14.1%) | ∗ | |
| N/A or unknown | 55 (-) | 2 (-) | 34 (-) | 19 (-) | |
| Current product type | |||||
| Recombinant, SHL clotting factor | ∗ | 44 (62.0) | 32 (26.9) | ∗ | <.0001 |
| Recombinant, EHL clotting factor | ∗ | 10 (14.1) | 18 (15.1) | ∗ | |
| Plasma derived clotting factor | ∗ | 11 (15.5) | 24 (20.2) | ∗ | |
| Bypassing agents | ∗ | 6 (8.5) | ∗ | ∗ | |
| Emicizumab | ∗ | N/A | 16 (13.4) | 40 (64.5) | |
| Unknown | ∗ | 0 (-) | ∗ | ∗ | |
| CVAD | |||||
| No | 130 (52.2%) | 30 (42.3%) | 53 (44.9%) | 47 (78.3%) | <.0001 |
| Yes | 119 (47.8%) | 41 (57.7%) | 65 (55.1%) | 13 (21.7%) | |
| Current product type | |||||
| Recombinant, SHL clotting factor | 47 (39.5%) | ∗ | ∗ | ∗ | |
| Recombinant, EHL clotting factor | 18 (15.1%) | ∗ | ∗ | ∗ | |
| Plasma derived clotting factor | 28 (23.5%) | ∗ | ∗ | ∗ | |
| Bypassing agents | 7 (5.9%) | ∗ | ∗ | ∗ | |
| Emicizumab | 13 (10.9%) | ∗ | ∗ | ∗ | |
| Unknown | 6 (-) | ∗ | ∗ | ∗ | |
| ICH | |||||
| No | 209 (83.6%) | 56 (80.0%) | 102 (86.4%) | 51 (82.3%) | .4873 |
| Yes | 41 (16.4%) | 14 (20.0%) | 16 (13.6%) | 11 (17.7%) | |
| Current product type | |||||
| Recombinant, SHL clotting factor | 13 (31.7%) | ∗ | ∗ | ∗ | |
| Recombinant, EHL clotting factor | ∗ | ∗ | ∗ | ∗ | |
| Plasma derived clotting factor | 7 (17.1%) | ∗ | ∗ | ∗ | |
| Bypassing agents | ∗ | ∗ | ∗ | ∗ | |
| Emicizumab | 11 (26.8%) | ∗ | ∗ | ∗ | |
| Hemophilia B | 192 | 63 | 80 | 49 | |
| Continuous prophylaxis (including continuous prophylaxis with bypassing agents plus ITI) | 38 (19.8%) | 12 (19.0%) | 16 (20.0%) | 10 (20.4%) | |
| Severe hemophilia | 33 (86.8%) | 11 (91.7%) | 13 (81.3%) | 9 (90.0%) | |
| Age at initiation of prophylaxis mean (mo) | |||||
| Mean (SD) | 11.6 ± 5.7 | 12.6 ± 6.4 | 10.5 ± 4.5 | 12.0 ± 6.9 | |
| 0-6 mo | ∗ | ∗ | ∗ | ∗ | |
| 7-23 mo | 29 (76.3%) | 10 (0.0%) | 13 (0.0%) | 6 (0.0%) | |
| Current product type | |||||
| Recombinant, SHL clotting factor | ∗ | 8 (66.7) | ∗ | ∗ | .059 |
| Recombinant, EHL clotting factor | ∗ | ∗ | 11 (68.8) | ∗ | |
| CVAD | |||||
| No | 13 (35.1%) | ∗ | ∗ | ∗ | .047 |
| Yes | 24 (64.9%) | 9 (75.0%) | 10 (66.7%) | ∗ | |
| Current product type | |||||
| Recombinant, SHL clotting factor | 10 (41.7%) | ∗ | ∗ | ∗ | |
| Recombinant, EHL clotting factor | 13 (54.2%) | ∗ | ∗ | ∗ | |
| Unknown/other | 1 (-) | ∗ | ∗ | ∗ | |
| ICH | |||||
| No | 31 (81.6%) | ∗ | ∗ | ∗ | |
| Yes | 7 (18.4%) | ∗ | ∗ | ∗ |
| . | Total . | 2011-2015 . | 2016-2018 . | 2019-2021 . | P value . |
|---|---|---|---|---|---|
| # (%) . | # (%) . | # (%) . | # (%) . | ||
| Hemophilia A | 691 | 203 | 325 | 163 | |
| Continuous prophylaxis (including continuous prophylaxis with bypassing agents plus ITI) | 252 (36.5%) | 71 (35.0%) | 119 (36.6%) | 62 (38.0%) | |
| Severe hemophilia | 229 (90.9%) | 64 (90.1%) | 107 (89.9%) | 58 (93.5%) | |
| Age at initiation of prophylaxis mean (mo) | |||||
| Mean (SD) | 10.1 ± 6.2 | 10.8 ± 6.8 | 11.1 ± 5.6 | 7.1 ± 5.6 | |
| 0-6 mo | 50 (25.4%) | 17 (24.6%) | 15 (17.6%) | 18 (41.9%) | <.0001 |
| 7-23 mo | 147 (74.6%) | 52 (75.4%) | 70 (82.4%) | 25 (58.1%) | |
| 7-12 mo | 123 (62.4%) | 41 (59.4%) | 58 (68.2%) | ∗ | |
| 13-23 mo | 24 (12.2%) | 11 (15.9%) | 12 (14.1%) | ∗ | |
| N/A or unknown | 55 (-) | 2 (-) | 34 (-) | 19 (-) | |
| Current product type | |||||
| Recombinant, SHL clotting factor | ∗ | 44 (62.0) | 32 (26.9) | ∗ | <.0001 |
| Recombinant, EHL clotting factor | ∗ | 10 (14.1) | 18 (15.1) | ∗ | |
| Plasma derived clotting factor | ∗ | 11 (15.5) | 24 (20.2) | ∗ | |
| Bypassing agents | ∗ | 6 (8.5) | ∗ | ∗ | |
| Emicizumab | ∗ | N/A | 16 (13.4) | 40 (64.5) | |
| Unknown | ∗ | 0 (-) | ∗ | ∗ | |
| CVAD | |||||
| No | 130 (52.2%) | 30 (42.3%) | 53 (44.9%) | 47 (78.3%) | <.0001 |
| Yes | 119 (47.8%) | 41 (57.7%) | 65 (55.1%) | 13 (21.7%) | |
| Current product type | |||||
| Recombinant, SHL clotting factor | 47 (39.5%) | ∗ | ∗ | ∗ | |
| Recombinant, EHL clotting factor | 18 (15.1%) | ∗ | ∗ | ∗ | |
| Plasma derived clotting factor | 28 (23.5%) | ∗ | ∗ | ∗ | |
| Bypassing agents | 7 (5.9%) | ∗ | ∗ | ∗ | |
| Emicizumab | 13 (10.9%) | ∗ | ∗ | ∗ | |
| Unknown | 6 (-) | ∗ | ∗ | ∗ | |
| ICH | |||||
| No | 209 (83.6%) | 56 (80.0%) | 102 (86.4%) | 51 (82.3%) | .4873 |
| Yes | 41 (16.4%) | 14 (20.0%) | 16 (13.6%) | 11 (17.7%) | |
| Current product type | |||||
| Recombinant, SHL clotting factor | 13 (31.7%) | ∗ | ∗ | ∗ | |
| Recombinant, EHL clotting factor | ∗ | ∗ | ∗ | ∗ | |
| Plasma derived clotting factor | 7 (17.1%) | ∗ | ∗ | ∗ | |
| Bypassing agents | ∗ | ∗ | ∗ | ∗ | |
| Emicizumab | 11 (26.8%) | ∗ | ∗ | ∗ | |
| Hemophilia B | 192 | 63 | 80 | 49 | |
| Continuous prophylaxis (including continuous prophylaxis with bypassing agents plus ITI) | 38 (19.8%) | 12 (19.0%) | 16 (20.0%) | 10 (20.4%) | |
| Severe hemophilia | 33 (86.8%) | 11 (91.7%) | 13 (81.3%) | 9 (90.0%) | |
| Age at initiation of prophylaxis mean (mo) | |||||
| Mean (SD) | 11.6 ± 5.7 | 12.6 ± 6.4 | 10.5 ± 4.5 | 12.0 ± 6.9 | |
| 0-6 mo | ∗ | ∗ | ∗ | ∗ | |
| 7-23 mo | 29 (76.3%) | 10 (0.0%) | 13 (0.0%) | 6 (0.0%) | |
| Current product type | |||||
| Recombinant, SHL clotting factor | ∗ | 8 (66.7) | ∗ | ∗ | .059 |
| Recombinant, EHL clotting factor | ∗ | ∗ | 11 (68.8) | ∗ | |
| CVAD | |||||
| No | 13 (35.1%) | ∗ | ∗ | ∗ | .047 |
| Yes | 24 (64.9%) | 9 (75.0%) | 10 (66.7%) | ∗ | |
| Current product type | |||||
| Recombinant, SHL clotting factor | 10 (41.7%) | ∗ | ∗ | ∗ | |
| Recombinant, EHL clotting factor | 13 (54.2%) | ∗ | ∗ | ∗ | |
| Unknown/other | 1 (-) | ∗ | ∗ | ∗ | |
| ICH | |||||
| No | 31 (81.6%) | ∗ | ∗ | ∗ | |
| Yes | 7 (18.4%) | ∗ | ∗ | ∗ |
Categorical are presented as n (%).
Responses of “Unknown” are not shown or included in the statistical analyses.
P values result from χ2 test for birth cohort across the respective patient characteristics.
ITI, immune tolerance induction; N/A, not applicable.
Known fields with N of greater than 0 but less than 5 have been suppressed to protect patient confidentiality. Additional cells may be suppressed to prevent derivation of these counts by subtraction.