Acute VTE treatment study: composite primary end point, thrombus assessment at EOT, and on-treatment bleeding events by treatment for patients with confirmed inherited thrombophilia
| . | Confirmed inherited thrombophilia (only FVL and/or PT gene) . | ||
|---|---|---|---|
| Dabigatran . | SOC . | Total . | |
| Efficacy end points (randomized set, intention-to-treat period), N | 23 | 9 | 32 |
| Composite primary end point,∗ n (%) | 8 (34.8) | 1 (11.1) | 9 (28.1) |
| Mantel-Haenszel–weighted difference in rates for SOC − dabigatran (95% CI) | −0.246 (−0.482 to −0.010) | ||
| Noninferiority P | 0.0009 | ||
| VTE recurrence rate at d 84 or EOT, n (%) | 1 (4.3) | 1 (11.1) | 2 (6.3) |
| Residual thrombotic burden at d 84 or EOT, n (%) | |||
| Thrombus progression† | 1 (4.3) | 1 (11.1) | 2 (6.3) |
| Stabilization | 3 (13.0) | 1 (11.1) | 4 (12.5) |
| Partial resolution | 9 (39.1) | 4 (44.4) | 13 (40.6) |
| Complete resolution | 8 (34.8) | 1 (11.1) | 9 (28.1) |
| Missing | 2 (8.7) | 2 (22.2) | 4 (12.5) |
| VTE-related death | 0 | 0 | 0 |
| On-treatment bleeding and PTS (treated set, on-treatment period), N | 23 | 9 | 32 |
| Any bleeding event, n (%) | 6 (26.1) | 4 (44.4) | 10 (31.3) |
| MBE, n (%) | 0 | 0 | 0 |
| PTS,‡ n (%) | 0 | 1 (11.1) | 1 (3.0) |
| . | Confirmed inherited thrombophilia (only FVL and/or PT gene) . | ||
|---|---|---|---|
| Dabigatran . | SOC . | Total . | |
| Efficacy end points (randomized set, intention-to-treat period), N | 23 | 9 | 32 |
| Composite primary end point,∗ n (%) | 8 (34.8) | 1 (11.1) | 9 (28.1) |
| Mantel-Haenszel–weighted difference in rates for SOC − dabigatran (95% CI) | −0.246 (−0.482 to −0.010) | ||
| Noninferiority P | 0.0009 | ||
| VTE recurrence rate at d 84 or EOT, n (%) | 1 (4.3) | 1 (11.1) | 2 (6.3) |
| Residual thrombotic burden at d 84 or EOT, n (%) | |||
| Thrombus progression† | 1 (4.3) | 1 (11.1) | 2 (6.3) |
| Stabilization | 3 (13.0) | 1 (11.1) | 4 (12.5) |
| Partial resolution | 9 (39.1) | 4 (44.4) | 13 (40.6) |
| Complete resolution | 8 (34.8) | 1 (11.1) | 9 (28.1) |
| Missing | 2 (8.7) | 2 (22.2) | 4 (12.5) |
| VTE-related death | 0 | 0 | 0 |
| On-treatment bleeding and PTS (treated set, on-treatment period), N | 23 | 9 | 32 |
| Any bleeding event, n (%) | 6 (26.1) | 4 (44.4) | 10 (31.3) |
| MBE, n (%) | 0 | 0 | 0 |
| PTS,‡ n (%) | 0 | 1 (11.1) | 1 (3.0) |
Complete thrombus resolution, freedom from recurrent VTE, and freedom from VTE-related death.
Defined as any symptomatic or asymptomatic contiguous progression of the index thrombus.
PTS (newly identified or worsening from baseline) was not specified as an outcome in the acute treatment study but was reported as an AE within the treated set.