Table 3. Efficacy results.
| Carfilzomib n=157 | Control n=158 | One-sided P-valuea | |
|---|---|---|---|
| Median OS, months (range; 95% CI) | 10.2 (8.4–14.4) | 10.0 (7.7–12.0) | 0.4172 |
| Hazard ratio (carfilzomib/control) (95% CI) | 0.975 (0.760–1.249) | ||
| Median PFS, years (95% CI) | 3.7 (2.8–4.2) | 3.3 (2.2–5.2) | 0.2479 |
| Hazard ratio (carfilzomib/control) (95% CI) | 1.091 (0.843–1.410) | ||
| Best overall responseb, n (%) | |||
| CR | 1 (1) | 0 (0) | NA |
| VGPR | 5 (3) | 5 (3) | NA |
| PR | 24 (15) | 13 (8) | NA |
| MR | 19 (12) | 15 (9) | NA |
| SD | 70 (45) | 74 (47) | NA |
| PD | 26 (17) | 35 (22) | NA |
| Unable to evaluate | 12 (8) | 16 (10) | NA |
| ORR, n (%) | 30 (19) | 18 (11) | NA |
| 95% CIc of ORR | 13–26 | 7–17 | 0.0305 |
| Median time to overall response, monthsd (range) | 2.0 (0.5–11.0) | 3.9 (1.0–28.0) | NA |
| Median duration of overall response, months (95% CI) | 7.2 (4.6–12.0) | 9.5 (3.7–NE) | NA |
| CBR, n (%) | 49 (31) | 33 (21) | NA |
| 95% CIc of CBR | 24–39 | 15–28 | 0.0212 |
| Median time to clinical benefit response, monthsd (range) | 1.5 (0.4–5.0) | 1.9 (0.9–17.0) | NA |
| Median duration of clinical benefit response, months (95% CI) | 6.4 (4.9–8.3) | 8.3 (6.5–12.9) | NA |
| DCR, n (%) | 119 (76) | 107 (68) | NA |
| 95% CIc of DCR | 68–82 | 60–75 | 0.0514 |
| Median duration of disease control, months (95% CI) | 5.5 (3.9–6.5) | 6.6 (5.4–8.8) | NA |
Abbreviations: CBR, clinical benefit rate; CI, confidence interval; CR, complete response; DCR, disease control rate; MR, minimal response; NA, not applicable; NE, not estimable; ORR, overall response rate; OS, overall survival; PD, progressive disease; PFS, progression-free survival; PR, partial response; sCR, stringent complete response; SD, stable disease; VGPR, very good partial response.
Overall response rate=sCR, CR, VGPR, or PR; clinical benefit response rate=sCR, CR, VGPR, PR, or MR; disease control=sCR, CR, VGPR, PR, MR, or SD.
One-sided P-values for OS and PFS were from stratified log-rank test with number of previous therapies (3 vs 4 vs ⩾5) and geographical region (Europe vs non-Europe) as stratification factors; one-sided P-values for ORR, CBR and DCR were from Cochran–Mantel–Haenszel chi-squared test with number of previous therapies (3 vs 4 vs⩾5) and geographical region (Europe vs non-Europe) as stratification factors; all P-values were unadjusted.
Best overall response was defined as a patient's best response during the study. A confirmed MR required a minimum duration of 8 weeks in addition to at least two consecutive assessment results of MR or higher. A confirmed SD required a minimum duration of 8 weeks.
Clopper–Pearson interval.
Medians, percentiles and their 95% CIs were estimated using the Kaplan−Meier method.