Cochrane · systematic review · CD014544
Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B
Olasupo, OO, Noronha, N, Lowe, MS, et al. · Cochrane Database of Systematic Reviews · 2024 · Cochrane Library
Cystic Fibrosis and Genetic DisordersAMSTAR 2: not assessed
SoF tableOnly the outcomes this review put in its Summary of Findings table are listed (the Cochrane Handbook caps them at about seven per comparison). Its other analyses are not shown here.
Questions
49
current version
GRADE rated
49
No studies
0
Included studies
NR
No studies 0 · 0%Ungraded 0 · 0%Very low 2 · 4.1%Low 13 · 26.5%Moderate 34 · 69.4%High 0 · 0%
Ungraded means an effect was reported without a GRADE rating; it is not a gap. No studies means the review found nothing to answer the question. The six categories are a display order, not a score.
Questions and results
Summary of findings table ‐ Emicizumab prophylaxis compared to on‐demand therapy in people with inhibitors
Population: people with inhibitors
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Annualized Bleeding Rate (ABR) ‐ All treated bleeds Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 53 participants | Moderate Downgraded: risk of bias |
Annualized spontaneous Bleeding Rate (AsBR) Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 53 participants | Moderate Downgraded: risk of bias |
Annualized joint Bleeding Rate (AjBR) Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 53 participants | Moderate Downgraded: risk of bias |
Proportion with zero bleeds Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy | RR 11.31 [1.66, 77.3] | 1 study · 53 participants | Moderate Downgraded: risk of bias |
Change from baseline in total score ‐ Haem‐A‐QoL Scale from: 0 (better) to 100 Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 40 participants | Low Downgraded: risk of bias |
All adverse events Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy | RR 1.97 [1.26, 3.1] | 1 study · 52 participants | Moderate Downgraded: risk of bias |
Serious adverse events Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy | RR 1.32 [0.48, 3.63] | 1 study · 52 participants | Moderate Downgraded: risk of bias |
Summary of findings table ‐ Fitusiran prophylaxis compared to on‐demand therapy in people with inhibitors
Population: people with inhibitors
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Annualized Bleeding Rate (ABR) ‐ All treated bleeds Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 57 participants | Moderate Downgraded: risk of bias |
Annualized spontaneous Bleeding Rate (AsBR) Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 57 participants | Moderate Downgraded: risk of bias |
Annualized joint Bleeding Rate (AjBR) Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 57 participants | Moderate Downgraded: risk of bias |
Change from baseline in total health score, Haem‐A‐QoLScale from: 0 (better) to 100 Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 48 participants | Low Downgraded: risk of bias |
Proportion with zero bleeds Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | RR 12.5 [1.83, 85.38] | 1 study · 57 participants | Moderate Downgraded: risk of bias |
All adverse events Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | RR 1.6 [1.08, 2.37] | 1 study · 60 participants | Moderate Downgraded: risk of bias |
Serious adverse events Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | RR 0.65 [0.24, 1.78] | 1 study · 60 participants | Moderate Downgraded: risk of bias |
Summary of findings table ‐ Concizumab prophylaxis compared to on‐demand therapy in people with inhibitors
Population: people with inhibitors
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Annualized Bleeding Rate (ABR), All bleeds Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy | no estimate extracted | 2 studies · 68 participants | Low Downgraded: risk of bias |
Annualized spontaneous Bleeding Rate (AsBR) Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy | no estimate extracted | 2 studies · 78 participants | Very low Downgraded: risk of bias, inconsistency |
Annualized joint Bleeding Rate (AjBR) Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy | no estimate extracted | 2 studies · 78 participants | Low Downgraded: risk of bias |
Proportion with zero bleeds Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy | RR 6.05 [1.59, 23] | 1 study · 52 participants | Low Downgraded: risk of bias |
Change from baseline in SF‐36v2 ‐ mental component scoreScale from: 0 to 100 (better) Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias, imprecision |
Adverse events ‐ All adverse events Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy | RR 1.14 [0.76, 1.7] | 2 studies · 78 participants | Low Downgraded: risk of bias |
Adverse events ‐ Serious adverse events Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy | RR 0.45 [0.06, 3.38] | 2 studies · 78 participants | Low Downgraded: risk of bias |
Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis compared to on‐demand therapy in people without inhibitors
Population: people without inhibitors
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Annualized Bleeding Rate (ABR) ‐ All treated bleeds Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 54 participants | Moderate Downgraded: risk of bias |
Annualized joint Bleeding Rate (AjBR) Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 54 participants | Moderate Downgraded: risk of bias |
Annualized spontaneous Bleeding Rate (AsBR) Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 54 participants | Moderate Downgraded: risk of bias |
Proportion of participants with zero bleeds Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy | RR 19 [1.21, 298.4] | 1 study · 54 participants | Low Downgraded: risk of bias, imprecision |
Change in Haem‐A‐QoL total score Scale from: 0 (better) to 100 Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 47 participants | Low Downgraded: risk of bias |
All adverse events Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy | RR 2.83 [1.47, 5.47] | 1 study · 54 participants | Moderate Downgraded: risk of bias |
Serious adverse events Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy | RR 0.5 [0.03, 7.54] | 1 study · 54 participants | Moderate Downgraded: risk of bias |
Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis compared to on‐demand therapy in people without inhibitors
Population: people without inhibitors
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Annualized Bleeding Rate (ABR) ‐ All treated bleeds Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 53 participants | Moderate Downgraded: risk of bias |
Annualized joint Bleeding Rate (AjBR) Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 53 participants | Moderate Downgraded: risk of bias |
Annualized spontaneous Bleeding Rate (AsBR) Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 53 participants | Moderate Downgraded: risk of bias |
Proportion of participants with zero bleeds Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy | RR 15.31 [0.96, 242.76] | 1 study · 53 participants | Low Downgraded: risk of bias, imprecision |
Change in total score, Haem‐A‐QoLScale from: 0 (better) to 100 Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 53 participants | Low Downgraded: risk of bias |
All adverse events Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy | RR 1.71 [1.06, 2.77] | 1 study · 53 participants | Moderate Downgraded: risk of bias |
Serious adverse events Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy | RR 1.54 [0.17, 13.79] | 1 study · 53 participants | Moderate Downgraded: risk of bias |
Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly compared to emicizumab 3.0 mg/kg every 2 weeks in people without inhibitors
Population: people without inhibitors
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Annualized Bleeding Rate (ABR) ‐ All treated bleeds Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks | no estimate extracted | 1 study · 71 participants | Moderate Downgraded: risk of bias |
Annualized joint Bleeding Rate (AjBR) Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks | no estimate extracted | 1 study · 71 participants | Moderate Downgraded: risk of bias |
Annualized spontaneous Bleeding Rate (AsBR) Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks | no estimate extracted | 1 study · 71 participants | Moderate Downgraded: risk of bias |
Proportion of participants with zero bleeds Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks | RR 1.25 [0.74, 2.1] | 1 study · 71 participants | Moderate Downgraded: risk of bias |
Change in total score, Haem‐A‐QoL Scale from: 0 (better) to 100 Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks | no estimate extracted | 1 study · 63 participants | Low Downgraded: risk of bias |
All adverse events Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks | RR 1.1 [0.04, 2.97] | 1 study · 71 participants | Moderate Downgraded: risk of bias |
Serious adverse events Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks | RR 0.32 [0.04, 2.97] | 1 study · 71 participants | Moderate Downgraded: risk of bias |
Summary of findings table ‐ Fitusiran prophylaxis compared to on‐demand therapy in people without inhibitors
Population: people without inhibitors
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Annualized Bleeding Rate (ABR) ‐ All treated bleeds Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 119 participants | Moderate Downgraded: risk of bias |
Annualized joint Bleeding Rate (AjBR) Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 119 participants | Moderate Downgraded: risk of bias |
Annualized spontaneous Bleeding Rate (AsBR) Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 119 participants | Moderate Downgraded: risk of bias |
Proportion with zero bleeds Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | RR 8.1 [2.04, 32.11] | 1 study · 119 participants | Moderate Downgraded: risk of bias |
Change in total scores, Haem‐A‐QoL Scale from: 0 (better) to 100 Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | no estimate extracted | 1 study · 103 participants | Low Downgraded: risk of bias |
All adverse events Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | RR 1.74 [1.21, 2.5] | 1 study · 119 participants | Moderate Downgraded: risk of bias |
Adverse events ‐ Serious adverse events Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy | RR 0.5 [0.15, 1.63] | 1 study · 120 participants | Moderate Downgraded: risk of bias |