Summary of findings 2. Berotralstat compared with placebo or active control for preventing hereditary angioedema attacks.
| Berotralstat compared with placebo or active control for preventing HAE attacks | ||||||
|
Patient or population: children or adults with Types I or II HAE Settings: outpatient setting Intervention: berotralstat Comparison: placebo | ||||||
| Outcomes | Anticipated absolute effects* (95% CI) | Relative effect (95% CI) | No of participants (studies) | Certainty of the evidence (GRADE) | Comments | |
| Risk with placebo | Risk with berotralstat | |||||
|
Risk of HAE attacks (during follow‐up) |
Study population | RR 0.63 (0.39 to 1.00) | 37 (1) |
⊕⊕⊝⊝ Lowa | — | |
| 910 per 1000 | 573 per 1000 (355 to 910) | |||||
|
Change in number of HAE attacks (per week) |
Study population | — | 130 (3) |
⊕⊕⊝⊝ Lowa | — | |
| The number of HAE attacks per week ranged across control groups from 0.55 to 0.95 | The number of HAE attacks per week in the intervention groups was 0.39 attacks lower (0.74 lower to 0.05 lower) | |||||
|
Mortality (during follow‐up) |
Study population | N/A | N/A | N/A | No deaths reported. | |
| N/A | N/A | |||||
|
Serious adverse events (during follow‐up) |
Study population | RR 0.77 (0.02 to 24.03) | 128 (3) | ⊕⊕⊝⊝ Lowa | — | |
| 45 per 1000 | 35 per 1000 (1 to 1000) | |||||
| Quality of life Angioedema Quality of Life scale (lower score is better) (during follow‐up) | Study population | — | 130 (3) |
⊕⊕⊕⊝ Moderateb | — | |
| The mean change in quality of life ranged across control groups from 3.18 points to −9.69 points | The mean change in quality of life in the intervention group was 15.28 points lower (29.42 lower to 1.14 lower) | |||||
|
Disability
Standardised mean difference (lower is better) (during follow‐up) |
Study population | — | 50 (2) |
⊕⊕⊝⊝ Lowa | — | |
| The mean change in disability ranged across control groups from 1.51 to −1.95 | The mean change in disability in the intervention groups was 1.01 units lower (1.62 lower to 0.40 lower) | |||||
| Adverse events (during follow‐up) | Study population | RR 1.03 (0.88 to 1.22) | 128 (3) |
⊕⊕⊕⊝ Moderateb | — | |
| 761 per 1000 | 784 per 1000 (670 to 1000) | |||||
| *The risk in the intervention group (and its 95% confidence interval) is based on the assumed risk in the comparison group and the relative effect of the intervention (and its 95% CI). CI: confidence interval; HAE: hereditary angioedema; N/A: not applicable; RR: risk ratio. | ||||||
| GRADE Working Group grades of evidence High certainty: we are very confident that the true effect lies close to that of the estimate of the effect. Moderate certainty: we are moderately confident in the effect estimate. The true effect is likely to be close to the estimate of the effect, but there is a possibility that it is substantially different. Low certainty: our confidence in the effect estimate is limited. The true effect may be substantially different from the estimate of the effect. Very low certainty: we have very little confidence in the effect estimate. The true effect is likely to be substantially different from the estimate of effect. | ||||||
aDowngraded two levels for imprecision. bDowngraded one level for imprecision.