Prospective, randomized clinical trials of BAP in patients with hemophilia
| Reference/study design . | No. patients, age at start of prophylaxis, prestudy bleeding frequency . | BAP dose . | Duration of BAP (range) . | Efficacy . |
|---|---|---|---|---|
| 55; prospective study with 3-mo lead-in on-demand period (control period) followed by randomization to 2 doses of rFVIIa for a 3-mo treatment period | 22 | rFVIIa 90 µg/kg per day or | 3 mo | 45% reduction in bleeding in patients treated with 90 µg/kg per day |
| 5.1-50.5 y | rFVIIa 270 µg/kg per day | 59% reduction in bleeding in patients treated with 270 µg/kg per day (not statistically significant compared with 90 µg/kg dose) | ||
| ≥2 bleeds/mo during 3-mo preprophylaxis period | Significantly fewer hospital admissions and absences from school/work during prophylaxis | |||
| 56; randomized crossover study of 6 mo of aPCC prophylaxis followed by 6 mo of on-demand therapy or vice versa | 26 | aPCC 85 U/kg ± 15% on 3 nonconsecutive d/wk | 6 mo | 62% reduction in all bleeding events* |
| 2.8-62.8 y | 61% reduction in hemarthroses* | |||
| ≥6 bleeds requiring bypassing therapy in 6 mo before study enrollment | 72% reduction in target joint bleeding* | |||
| Significantly fewer absences from school/work during prophylaxis* | ||||
| 57; randomized control trial comparing 12 mo of aPCC prophylaxis with 12 mo of on-demand therapy | 36 | aPCC 85 ± 15 U/kg every other day | 12 mo | 72.5% reduction in annual bleeding rate |
| 7-56 y | ||||
| ≥12 bleeds in 12 mo before study enrollment |
| Reference/study design . | No. patients, age at start of prophylaxis, prestudy bleeding frequency . | BAP dose . | Duration of BAP (range) . | Efficacy . |
|---|---|---|---|---|
| 55; prospective study with 3-mo lead-in on-demand period (control period) followed by randomization to 2 doses of rFVIIa for a 3-mo treatment period | 22 | rFVIIa 90 µg/kg per day or | 3 mo | 45% reduction in bleeding in patients treated with 90 µg/kg per day |
| 5.1-50.5 y | rFVIIa 270 µg/kg per day | 59% reduction in bleeding in patients treated with 270 µg/kg per day (not statistically significant compared with 90 µg/kg dose) | ||
| ≥2 bleeds/mo during 3-mo preprophylaxis period | Significantly fewer hospital admissions and absences from school/work during prophylaxis | |||
| 56; randomized crossover study of 6 mo of aPCC prophylaxis followed by 6 mo of on-demand therapy or vice versa | 26 | aPCC 85 U/kg ± 15% on 3 nonconsecutive d/wk | 6 mo | 62% reduction in all bleeding events* |
| 2.8-62.8 y | 61% reduction in hemarthroses* | |||
| ≥6 bleeds requiring bypassing therapy in 6 mo before study enrollment | 72% reduction in target joint bleeding* | |||
| Significantly fewer absences from school/work during prophylaxis* | ||||
| 57; randomized control trial comparing 12 mo of aPCC prophylaxis with 12 mo of on-demand therapy | 36 | aPCC 85 ± 15 U/kg every other day | 12 mo | 72.5% reduction in annual bleeding rate |
| 7-56 y | ||||
| ≥12 bleeds in 12 mo before study enrollment |
Among good responders.