Secondary outcomes
| Binary outcomes . | Randomized controlled trials . | Observational studies . | ||||
|---|---|---|---|---|---|---|
| Risk difference (95% CI) . | Risk difference interpretation per 1000∗ . | RR (95% CI) . | Risk difference (95% CI) . | Risk difference interpretation per 1000∗ . | RR (95% CI) . | |
| TTP exacerbation | −0.29 (−0.42 to −0.14)† | 290 fewer (from 420 fewer to 140 fewer)† | 0.16 (0.07-0.47)† | −0.35 (−0.43 to −0.27)† | 350 fewer (from 430 fewer to 270 fewer)† | 0.10 (0.04-0.42)† |
| TTP relapse | 0.14 (−0.00 to 0.27) | 140 more (from equal to 270 more) | 3.81 (1.58-14.28)† | 0.00 (−0.29 to 0.29)∗∗ | equal (from 290 fewer to 290 more) | 1.00 (0.07-13.64)∗∗ |
| Refractory TTP | −0.08 (−0.13 to −0.02)† | 80 fewer (from 130 fewer to 20 fewer)† | 0.11 (0.01-0.81)† | −0.22 (−0.45 to −0.03)† | 220 fewer (from 450 fewer to 30 fewer)† | 0.12 (0.02-0.61)† |
| Thrombosis | 0.00 (−0.07 to 0.07) | equal (from 70 fewer to 70 more) | 1.02 (0.40-2.63) | 0.00 (−0.05 to 0.06) | equal (from 50 fewer to 60 more) | 1.07 (0.57-2.03) |
| Major bleeding | 0.02 (−0.02 to 0.07) | 20 more (from 20 fewer to 70 more) | 1.73 (0.39-7.07) | 0.00 (−0.19 to 0.19)∗∗ | equal (from 190 fewer to 190 more)∗∗ | 1.00 (0.02-45.13) ∗∗ |
| Intracranial bleeding | 0.00 (−0.03 to 0.03) | equal (from 30 fewer to 30 more) | 1.04 (0.15-7.25) | Not reported | ||
| Binary outcomes . | Randomized controlled trials . | Observational studies . | ||||
|---|---|---|---|---|---|---|
| Risk difference (95% CI) . | Risk difference interpretation per 1000∗ . | RR (95% CI) . | Risk difference (95% CI) . | Risk difference interpretation per 1000∗ . | RR (95% CI) . | |
| TTP exacerbation | −0.29 (−0.42 to −0.14)† | 290 fewer (from 420 fewer to 140 fewer)† | 0.16 (0.07-0.47)† | −0.35 (−0.43 to −0.27)† | 350 fewer (from 430 fewer to 270 fewer)† | 0.10 (0.04-0.42)† |
| TTP relapse | 0.14 (−0.00 to 0.27) | 140 more (from equal to 270 more) | 3.81 (1.58-14.28)† | 0.00 (−0.29 to 0.29)∗∗ | equal (from 290 fewer to 290 more) | 1.00 (0.07-13.64)∗∗ |
| Refractory TTP | −0.08 (−0.13 to −0.02)† | 80 fewer (from 130 fewer to 20 fewer)† | 0.11 (0.01-0.81)† | −0.22 (−0.45 to −0.03)† | 220 fewer (from 450 fewer to 30 fewer)† | 0.12 (0.02-0.61)† |
| Thrombosis | 0.00 (−0.07 to 0.07) | equal (from 70 fewer to 70 more) | 1.02 (0.40-2.63) | 0.00 (−0.05 to 0.06) | equal (from 50 fewer to 60 more) | 1.07 (0.57-2.03) |
| Major bleeding | 0.02 (−0.02 to 0.07) | 20 more (from 20 fewer to 70 more) | 1.73 (0.39-7.07) | 0.00 (−0.19 to 0.19)∗∗ | equal (from 190 fewer to 190 more)∗∗ | 1.00 (0.02-45.13) ∗∗ |
| Intracranial bleeding | 0.00 (−0.03 to 0.03) | equal (from 30 fewer to 30 more) | 1.04 (0.15-7.25) | Not reported | ||
| Continuous outcomes . | Mean difference (95% CI) . | Mean difference interpretation . | Mean difference (95% CI) . | Mean difference interpretation . |
|---|---|---|---|---|
| Time to platelet count recovery (d) | −0.66 (−0.90 to −0.43)† | 0.66 d fewer (from 0.90 fewer to 0.43 fewer)† | −4.84 (−7.91 to −1.76)† | 4.84 d fewer (from 7.91 fewer to 1.76 fewer)† |
| Hospital LOS (d) | −6.00 (−8.92 to −3.08)† | 6 d fewer (from 8.92 fewer to 3.08 fewer)† | −9.06 (−18.96 to 0.84) | 9.06 d fewer (from 18.96 fewer to 0.84 fewer) |
| Duration of TPE (d) | −3.60 (−3.82 to −3.38)† | 3.60 d fewer (from 3.82 fewer to 3.38 fewer)† | −6.63 (−11.83 to −1.43)† | 6.63 d fewer (from 11.83 fewer to 1.43 fewer)† |
| Continuous outcomes . | Mean difference (95% CI) . | Mean difference interpretation . | Mean difference (95% CI) . | Mean difference interpretation . |
|---|---|---|---|---|
| Time to platelet count recovery (d) | −0.66 (−0.90 to −0.43)† | 0.66 d fewer (from 0.90 fewer to 0.43 fewer)† | −4.84 (−7.91 to −1.76)† | 4.84 d fewer (from 7.91 fewer to 1.76 fewer)† |
| Hospital LOS (d) | −6.00 (−8.92 to −3.08)† | 6 d fewer (from 8.92 fewer to 3.08 fewer)† | −9.06 (−18.96 to 0.84) | 9.06 d fewer (from 18.96 fewer to 0.84 fewer) |
| Duration of TPE (d) | −3.60 (−3.82 to −3.38)† | 3.60 d fewer (from 3.82 fewer to 3.38 fewer)† | −6.63 (−11.83 to −1.43)† | 6.63 d fewer (from 11.83 fewer to 1.43 fewer)† |
All-cause mortality and any bleeding are reported separately in Figure 2. We present the event rates and corresponding forest plots for each outcome in supplemental Data 4.
Presented as the difference in absolute risk of the outcome among patients treated with caplacizumab plus SOC (compared with those treated with SOC alone) per 1000 patients treated with caplacizumab.
Statistically significant (P < .05).
Based on only 1 study (see supplemental Data for details).