Summary of findings 2. Prophylaxis with standard therapeutic factor concentrate compared to pegylated liposome FVIII formulation.
| Prophylaxis with standard clotting factor concentrate compared with pegylated liposome FVIII formulation for previously treated individuals with haemophilia A | ||||||
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Patient or population: children or adults with hemophilia A Settings: outpatient Intervention: prophylaxis using investigational BAY 79‐4980 Comparison: standard secondary prophylaxis | ||||||
| Outcomes | Illustrative comparative risks* (95% CI) | Relative effect (95% CI) | No of Participants (studies) | Certainty of the evidence (GRADE) | Comments | |
| Assumed risk | Corresponding risk | |||||
| Prophylaxis using investigational BAY 79‐4980 | Standard prophylaxis | |||||
| AJBR Follow‐up: 12 months |
The mean number of joint bleeding in the prophylaxis arm using investigational drug BAY 79‐4980 was 12.2. | The mean number of joint bleeding in the standard prophylaxis regimen (5.0), was 7.20 lower (11.01 lower to 3.39 lower) | MD ‐7.20 (‐11.01 to ‐3.39) |
143 participants (1 trial) |
⊕⊕⊝⊝
lowa,b |
More participants withdrew consent in the investigational drug arm. The trial was prematurely discontinued by the sponsor based on the recommendation of an independent data and safety monitoring board. |
| ABR Follow‐up: 12 months |
The mean number of total bleeds in the prophylaxis arm using investigational drug BAY 79‐4980 was 15. | The mean number of total bleeds in the standard prophylaxis regimen (5.8), was 9.20 lower (13.07 lower to 5.33 lower) | MD ‐7.20 (‐13.07 to ‐5.33 ) |
143 participants (1 trial) |
⊕⊕⊝⊝ lowa,b | More participants withdrew consent in the investigational drug arm. The trial was prematurely discontinued by the sponsor based on the recommendation of an independent data and safety monitoring board. |
| Any reported adverse effects Follow‐up: 12 months |
No specific information was given about the presence/absence of adverse events in the BAY 70‐4980 group. | One participant in the prophylaxis group reported three serious adverse events, which were deemed to be drug related. | Not estimable | 143 participants (1 trial) |
⊕⊕⊝⊝ lowa,b | |
| *The basis for the assumed risk (e.g. the median control group risk across studies) is provided in footnotes. The corresponding risk (and its 95% CI) is based on the assumed risk in the comparison group and the relative effect of the intervention (and its 95% CI). ABR: annualised bleed rate; AJBR: annualised joint bleed rate; CI: confidence interval; MD: mean difference. | ||||||
| GRADE Working Group grades of evidence High certainty: further research is very unlikely to change our confidence in the estimate of effect. Moderate certainty: further research is likely to have an important impact on our confidence in the estimate of effect and may change the estimate. Low certainty: further research is very likely to have an important impact on our confidence in the estimate of effect and is likely to change the estimate. Very low cerainty: we are very uncertain about the estimate. | ||||||
a. Downgraded once due to high risk of bias due to attrition bias from incomplete outcome data. b. Downgraded once due to premature study discontinuation.