| General information |
| Database: | DB00153 |
| Objective: | Rapalogs are emerging as promising targeted anticancer drugs. Activation of the PI3K/Akt/mTOR pathway has been observed in 1550% of hepatocellular carcinomas. |
| Authors: | Decaens T, et al |
| Title: | Phase II study of sirolimus in treatmentnaive patients with advanced hepatocellular carcinoma. |
| Journal: | Dig Liver Dis. |
| Year: | 2012 |
| PMID: | 22459565 |
| Trial Design |
| Clinical Trial Id: | NA |
| Agent: | sirolimus/rapamycin
|
| Target: | Serine/threonineprotein kinase mTOR
|
| Cancer Type: | liver cancer |
| Cancer Subtype: | advanced hepatocellular carcinoma and underlying cirrhosis |
| Therapy Type: | mono |
| Therapeutic Combination Type: | NA |
| Therapeutic Combination Content: | NA |
| Study Type: | a singlecentre openlabelphase II study |
| Key Patients Feature: | patients with advanced hepatocellular carcinoma and underlying cirrhosis |
| Biomarker: | NA |
| Biomark Analysis: | NA |
| Control Group Info: | NA |
| Treatment Info: | pts received sirolimus (20 mg/week for 1 month then 30 mg/week). Tumour response was assessed every 8 weeks. |
| Primary End Point: | the objective tumour response rate. |
| Secondary End Point: | NA |
| Patients Number: | 25 |
| Trial Results |
| DLT_MTD: | NA |
| Objective Response Rate: | NA |
| Disease Control Rate: | NA |
| Median Time to Progression: | NA |
| Median PFS A vs. C: | Median time to radiological progression was 15.3 weeks (range: 8.2173.9) |
| Median OS A vs. C: | median overall survival was 26.4 weeks (range: 8.2173.9) |
| Adverse Event(agent arm): | There were 2 cases of grade 5 toxicity (infections) and 5 cases of grade 3 toxicity. The main grade 1/2 toxicity was mild transient fatigue (76%). |
| Conclusions: | These data suggest that firstline sirolimus shows antitumoural efficacy in advanced hepatocellular carcinoma. Larger trials with Child A patients are needed. |