Table 2.
The efficacy results assessed by independent assessment committee in modified intention-to-treat population
| Aponermin Group (N = 276) | Placebo Group (N = 139) | Proportion difference between groups | Hazard Ratio (95%CI) | P-value | |
|---|---|---|---|---|---|
| Overall response —% (95% CI) | 30.4 (25.1–36.2) | 13.7 (8.4–20.5) | 16.8 (8.9–24.6) | ·· | < 0.001 |
| Clinical benefit — % (95% CI)a | 45.3 (39.3–51.4) | 29.5 (22.1–37.8) | 15.8 (6.2–25.4) | ·· | 0.002 |
| Very good partial response or better— % (95% CI) | 14.1 (10.2–18.8) | 2.2 (0.4–6.2) | 12.0 (7.2–16.7) | ·· | < 0.001 |
| Best response — % (95% CI)b | |||||
| Stringent complete response | 0 (0–1.3) | 0 (0–2.6) | ·· | ·· | ·· |
| Complete response | 2.2 (0.8–4.7) | 0.7 (0.0–3.9) | 1.5 (-0.8–3.7) | ·· | 0.43 |
| Very good partial response | 12.0 (8.4–16.4) | 1.4 (0.2–5.1) | 10.5 (6.2–14.8) | ·· | < 0.001 |
| Partial response | 16.3 (12.1–21.2) | 11.5 (6.7–18.0) | 4.8 (-2.1–11.7) | ·· | 0.24 |
| Minimal responsec | 14.9 (10.9–19.6) | 15.8 (10.2–23.0) | -1.0 (-8.3–6.4) | ·· | 0.77 |
| Stable disease | 38.0 (32.3–44.1) | 44.6 (36.2–53.3) | -6.6 (-16.6–3.5) | ·· | 0.21 |
| Progression | 12.3 (8.7–16.8) | 23.7 (16.9–31.7) | -11.4 (-19.5– -3.4) | ·· | 0.004 |
| Not evaluable | 4.3 (2.3–7.5) | 2.2 (0.4–6.2) | 2.2 (-1.2–5.6) | ·· | 0.40 |
| Median time to response — mo (95% CI) | 1.9 (1.2–1.9) | 1.9 (1.0–2.6) | ·· | 0.89 (0.53–1.51) | 0.67 |
| Median duration of response — mo (95% CI) | 15.2 (10.0–18.2) | 9.8 (4.4–14.7) | ·· | 0.55 (0.29–1.05) | 0.07 |
| Median time to progression — mo (95% CI) | 5.8 (4.8–7.4) | 3.5 (2.1–4.4) | ·· | 0.61 (0.48–0.78) | < 0.001 |
aA clinical benefit was defined as a minimal response or better
bThe best confirmed responses were assessed by the independent assessment committee in a blinded manner according to the International Myeloma Working Group criteria
cMinimal response was assessed according to the European Group for Blood and Bone Marrow Transplant criteria