Table 2.
Clinical outcomes in patients treated with melphalan/Hepatic Delivery System (treated population, assessed by Independent Review Committee)
| Characteristic | Melphalan/HDS (n = 91) n (%) |
|---|---|
| Primary end point | |
| Objective response rate: % (95 % CI)a | 36.3 (26.44–47.01) |
| No. of patients who achieved objective response | 33 |
| Best overall responseb,c | |
| Complete response | 7 (7.7) |
| Partial response | 26 (28.6) |
| Stable disease | 34 (37.4) |
| Progressive disease | 23 (25.3) |
| Not evaluable | 1 (1.1) |
| Secondary end points | |
| Median duration of response in responders: months (95 % CI)d | 14.0 (8.31–17.74); n = 33 |
| Disease control rate: % (95 % CI)a | 73.6 (63.35–82.31) |
| No. of patients who achieved disease control | 67 |
| Median progression-free survival: months (95 % CI)d | 9.0 (6.34–11.56) |
| Progression-free survival at 6 months: % (95 % CI)d | 65 (54–74) |
| Progression-free survival at 1 year: % (95 % CI)d | 38 (27–48) |
| Median overall survival: months (95 % CI)d | 20.5 (16.79–25.26) |
| Overall survival at 1 year: % (95 % CI)d | 80 (70–87) |
| Overall survival at 2 years: % (95 % CI)d | 43 (32–53) |
| Exploratory end points | |
| Median time to objective response: months (95 % CI)d | 3.3 (2.86–5.59); n = 33 |
| Median hepatic progression-free survival: months (95 % CI)d | 13.9 (9.30–16.66) |
| Hepatic objective response rate: % (95 % CI)a | 41.8 (31.50–52.57); n = 38 |
HDS, hepatic delivery system; CI, confidence interval
aPatients without at least 1 post-baseline response assessment were designated as non-responders
bBest overall response per Independent Review Committee (Response Evaluation Criteria in Solid Tumors v1.1) from the date of randomization/eligibility until disease progression
cFor complete response or partial response, confirmation was required by repeat assessment ≥4 weeks after initial documentation. To qualify as stable disease, the image must have been taken at least 9 weeks after start of therapy
dKaplan-Meier estimate