Background
Methods
Trial design and patients
Safety and preliminary efficacy assessments
Immune biomarkers and modulation activities in myeloid cells
Statistical analysis methods
Results
Patient demographics
Characteristic | Icaritin | ||
---|---|---|---|
600 mg bid | 800mg bid | Total | |
(N = 14) | (N = 6) | (N = 20) | |
Median age, years (range) | 61 (31–74) | 43 (33–73) | 58 (31–74) |
Male, no.(%) | 12 (85.7) | 5 (83.3) | 17 (85.0) |
ECOG performance status, no.(%) | |||
0 | 2 (14.3) | 0 (0.0) | 2 (10.0) |
1 | 11 (78.6) | 6 (100.0) | 17 (85.0) |
2 | 1 (7.1) | 0 (0.0) | 1 (5.0) |
Macroscopic vascular invasion, no.(%) | |||
Yes | 5 (35.7) | 2 (33.3) | 7 (35.0) |
No | 9 (64.3) | 4 (66.7) | 13 (65.0) |
Portal vein tumor thrombus, no.(%) | |||
Yes | 3 (21.4) | 2 (33.3) | 5 (25.0) |
No | 11 (78.6) | 4 (66.7) | 15 (75.0) |
Bulky tumor*, no.(%) | |||
Yes | 1 (7.1) | 1 (16.7) | 2 (10.0) |
No | 13 (92.9) | 5 (83.3) | 18(90.0) |
Extrahepatic metastasis, no.(%) | |||
Yes | 13 (92.9) | 5 (83.3) | 18 (90.0) |
No | 1 (7.1) | 1 (16.7) | 2 (10.0) |
Extrahepatic metastatic site, no.(%) | |||
Lymph node | 7 (50.0) | 4 (66.7) | 11 (55.0) |
Lung | 7 (50.0) | 3 (50.0) | 10 (50.0) |
BCLC stage, no.(%) | |||
B | 1 (7.1) | 0 (0.0) | 1 (5.0) |
C | 13 (92.9) | 6 (100.0) | 19 (95.0) |
Number of tumor sites, no.(%) | |||
1 | 2 (14.3) | 1 (16.7) | 3 (15.0) |
2 | 6 (42.9) | 1 (16.7) | 7 (35.0) |
3 | 3 (21.4) | 2 (33.3) | 5 (25.0) |
≥ 4 | 3 (21.4) | 2 (33.3) | 5 (25.0) |
Hepatitis virus status, no.(%) | |||
HBV infection | 12(85.7) | 6 (100.0) | 18 (90.0) |
HCV infection | 1 (7.1) | 0 (0.0) | 1 (5.0) |
Liver cirrhosis, no.(%) | |||
Yes | 10 (71.4) | 5 (83.3) | 15 (75.0) |
No | 4 (28.6) | 1 (16.7) | 5 (25.0) |
Child-Pugh classification, no.(%) | |||
A | 13 (92.9) | 5 (83.3) | 18 (90.0) |
B | 1 (7.1) | 1 (16.7) | 2 (10.0) |
AFP > ULN (laboratory), no.(%) | 13 (92.9) | 6 (100.0) | 19 (95.0) |
Previous treatment, no.(%) | |||
Surgery | 3 (21.4) | 4 (66.7) | 7 (35.0) |
TACE/RFA | 11 (78.6) | 3 (50.0) | 14 (70.0) |
Sorafenib or Chemo | 1 (7.1) | 3 (50.0) | 4 (20.0) |
Safety and adverse events
Drug-related AE# (≥5%) | 600 mg bid (N = 14) | 800mg bid (N = 6) | Total (N = 20) | ||
---|---|---|---|---|---|
Grade 1 | Grade2 | Grade 1 | Grade 2 | N (%) | |
Laboratory abnormality | |||||
Leukopenia | 2 | 1 | 0 | 0 | 3(15.0) |
Neutropenia | 2 | 1 | 0 | 0 | 3(15.0) |
LDH increased | 1 | 0 | 0 | 0 | 1(5.0) |
Thrombocytopenia | 1 | 0 | 0 | 0 | 1(5.0) |
Skin and subcutaneous tissue disease | |||||
Rash | 0 | 1 | 0 | 0 | 1(5.0) |
Gastrointestinal disorders | |||||
Constipation | 0 | 0 | 1 | 0 | 1(5.0) |
Diarrhea | 0 | 0 | 0 | 1 | 1(5.0) |
Serious Adverse Event (SAE)* | 600 mg bid | 800 mg bid | Outcome | ||
Gastrointestinal bleeding | 1 | 1 | Resolved/Death | ||
Dyspnea | 2 | 0 | Death | ||
Seroperitoneum | 1 | 0 | Unresolved | ||
Liver abscess | 1 | 0 | Unresolved | ||
Cardiac sudden death | 1 | 0 | Death | ||
Liver function damage aggravated | 0 | 1 | Death |