Pilot study: rapamycin in advanced hepatocellular carcinoma

Alimentary Pharmacology & Therapeutics
M Schöniger-Hekele, C Müller

Abstract

The PI3K/Akt/mTOR signal pathway is involved in hepatocarcinogenesis. Rapamycin (=sirolimus), a specific mTOR inhibitor, leads to G(1) arrest of many malignant cell lines and currently, analogues of rapamycin are being investigated as a cancer chemotherapeutic adjuvant. To study the toxicity and tolerability of rapamycin therapy in patients with advanced hepatocellular carcinoma (HCC). Between June 2005 and February 2007, patients with advanced HCC, not eligible for any established therapy, were included in the study. Eighteen patients (F/M: 5/13) with compensated liver cirrhosis (Child A n = 11, Child B n = 5, Child C n = 2) and histologically proven HCC were included in this study. According to the BCLC staging system, most of the patients enrolled had an advanced HCC: BCLC stage B: n = 2, Barcelona Clinic Liver-Cancer (BCLC) stage C: n = 14, BCLC stage D: n = 2. Overall, therapy with rapamycin was well tolerated. Most common toxicities were thrombocytopaenia and anaemia. We did not observe any partial or complete tumour response. At 3 months, two patients had stable disease and at 6 months, all patients had progressed. The median overall survival was 5.27 months, median time to progression was 3 months. Rapamycin is well tol...Continue Reading

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Citations

Oct 14, 2011·Future Oncology·Qian ZhouWinnie Yeo
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Nov 16, 2019·European Journal of Pharmacology·Michał AntoszczakAdam Huczyński

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