Table 2. PFS and OS in relation to clinical characteristics and toxicity in the two independent cohorts.
| Training set | Validation set | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| No. of Patients | No. of Events | Median PFS (95% CI) | P | No. of Events | Median OS (95% CI) | P | No. of Patients | No. of Events | Median PFS (95% CI) | P | No. of Events | Median OS (95% CI) | P | |
| Gender | ||||||||||||||
| Male | 32 | 31 | 3.7 (2.6-4.7) | 28 | 8.7 (3.9-14.9) | 60 | 50 | 3.9 (2.4-6.2) | 43 | 11.3 (8.2-17.2) | ||||
| Female | 9 | 7 | 8.2 (2.2-11.2) | 0.166 | 7 | 14.6 (6.6-23.0) | 0.266 | 27 | 23 | 5.0 (2.1-6.3) | 0.516 | 19 | 14.1 (5.6-27.0) | 0.842 |
| Smoking habits | ||||||||||||||
| No | 18 | 17 | 4.3 (2.6-8.5) | 16 | 10.8 (6.8-14.6) | 12 | 9 | 13.8 (2.1-27.1) | 6 | 27.8 (14.1-35.0) | ||||
| Yes | 18 | 17 | 4.2 (2.6-6.2) | 0.997 | 17 | 9.8 (2.9-15.8) | 0.578 | 16 | 11 | 2.4 (1.1-15.2) | 0.112 | 9 | 5.2 (2.5-39.0) | 0.125 |
| Etiology | ||||||||||||||
| Metabolic syndrome | 8 | 8 | 3.0 (0.9-6.0) | 6 | 6.8 (1.0-nr) | 5 | 4 | 2.4 (2.1-8.7) | 3 | 7.5 (3.7-9.9) | ||||
| Alcohol | 6 | 6 | 6.7 (2.6-34.2) | 6 | 15.3 (2.7-34.2) | 6 | 5 | 13.5 (2.0-31.4) | 5 | 14.4 (3.0-50.8) | ||||
| Viral | 27 | 24 | 4.7 (2.6-5.8) | 23 | 11.2 (6.9-15.1) | 71 | 59 | 3.9 (2.5-5.7) | 50 | 12.4 (8.2-18.0) | ||||
| Cryptogenic | 0 | 0 | - | 0.195 | 0 | - | 0.458 | 5 | 5 | 3.4 (1.7-27.0) | 0.599 | 4 | 10.5 (2.5-nr) | 0.729 |
| BCLC stage | ||||||||||||||
| B | 8 | 7 | 4.3 (0.5-21.6) | 6 | 10.0 (0.5-23.0) | 11 | 6 | 9.0 (4.8-27.1) | 2 | 18.0 (14.4-nr) | ||||
| C | 32 | 30 | 3.8 (2.7-5.7) | 0.780 | 28 | 12.0 (6.7-14.9) | 0.747 | 38 | 30 | 2.5 (2.0-8.7) | 0.084 | 24 | 19.5 (3.7-28.8) | 0.100 |
| MELD score | ||||||||||||||
| ≤10 | 30 | 29 | 3.9 (2.6-6.0) | 26 | 12.0 (3.9-14.9) | 37 | 32 | 5.7 (3.9-12.8) | 29 | 13.6 (9.7-27.8) | ||||
| >10 | 7 | 6 | 3.8 (1.2-34.2) | 0.706 | 6 | 8.5 (2.9-34.2) | 0.863 | 22 | 22 | 1.6 (1.1-2.4) | <0.0001 | 20 | 4.4 (2.5-10.9) | 0.004 |
| Serum α-FP level | ||||||||||||||
| ≤400 KUI/L | 20 | 18 | 3.8 (2.6-6.0) | 16 | 10.1 (3.9-23.6) | 52 | 44 | 5.0 (3.4-6.3) | 38 | 12.4 (7.2-19.5) | ||||
| >400 KUI/L | 18 | 17 | 4.6 (2.6-10.8) | 0.327 | 16 | 12.0 (3.9-14.9) | 0.809 | 23 | 20 | 2.4 (1.3-4.8) | 0.044 | 18 | 7.5 (2.5-13.9) | 0.043 |
| Hypertension* | ||||||||||||||
| No | 21 | 20 | 2.8 (2.2-3.9) | 19 | 7.5 (3.2-14.9) | - | 0 | - | 0 | - | ||||
| Yes | 18 | 17 | 6.1 (3.7-10.8) | 0.005 | 15 | 14.6 (11.2-23.0) | 0.027 | - | 0 | - | - | 0 | - | |
| Skin toxicity | ||||||||||||||
| No | 23 | 21 | 3.8 (2.3-5.2) | 19 | 6.9 (3.2-14.9) | 53 | 43 | 2.8 (2.4-5.6) | 34 | 13.6 (6.1-17.2) | ||||
| Yes | 18 | 17 | 6.2 (2.6-10.8) | 0.065 | 16 | 13.9 (7.5-16.7) | 0.124 | 34 | 30 | 5.3 (3.7-12.8) | 0.035 | 28 | 11.3 (7.2-28.6) | 0.349 |
| Diarrhea | ||||||||||||||
| No | 35 | 32 | 3.8 (2.6-5.8) | 29 | 10.4 (6.7-14.9) | 65 | 52 | 3.7 (2.5-6.2) | 45 | 10.9 (6.4-18.0) | ||||
| Yes | 6 | 6 | 5.0 (2.7-21.6) | 0.447 | 6 | 13.9 (2.7-23.6) | 0.876 | 22 | 21 | 5.0 (2.1-9.9) | 0.365 | 17 | 13.9 (10.5-28.8) | 0.301 |
| Asthenia | ||||||||||||||
| No | 28 | 27 | 3.9 (2.6-5.3) | 26 | 10.8 (5.2-14.9) | 86 | 72 | 4.7 (2.6-5.7) | 61 | 12.4 (8.2-17.2) | ||||
| Yes | 13 | 11 | 4.7 (1.9-8.7) | 0.681 | 9 | 13.7 (3.9-16.7) | 0.997 | 1 | 1 | 2.1 (−) | 0.281 | 1 | 3.0 (−) | 0.045 |
| Mucositis | ||||||||||||||
| No | 40 | 37 | 3.8 (2.6-5.7) | 34 | 11.2 (6.7-14.9) | 81 | 68 | 4.6 (2.6-5.7) | 57 | 11.8 (8.2-17.2) | ||||
| Yes | 1 | 1 | 15.3 (−) | 0.397 | 1 | 15.8 (−) | 0.762 | 6 | 5 | 4.8 (1.1-nr) | 0.439 | 5 | 9.8 (2.2-nr) | 0.917 |
PFS, progression-free survival; OS, overall survival; BCLC, Barcelona-Clinic Liver Cancer; MELD, Model For End-Stage Liver Disease
*
diastolic pressure >90 mmHg or systolic pressure >140 mmHg recorded 15 days after the start of sorafenib treatment