INST OX‐05‐024: first line gemcitabine oxaliplatin and erlotinib for primary hepatocellular carcinoma and bile duct cancers: a multicenter Phase II trial

摘要

Hepatocellular Carcinoma (HCC) incidence is increasing in the USA. Gemcitabine (G) and oxaliplatin (O) are active in HCC and biliary duct cancer (BDC). Erlotinib (E) is an EGFR tyrosine kinase inhibitor (TKI) with known activity against both. We sought to evaluate the efficacy of the combination G+O+E. Patients with either of the two diagnosis were treated in a phase II trial. Simons 2 stage design was used. A disease‐control rate (DCR), complete response (CR) + partial response (PR)+ stable disease (SD) at 24 weeks of ≤20% and >40% (P0 and P1 of 0.2 and 0.4, respectively) were set as undesirable (null) and desirable results. 26 HCC and 7 BDC patients were accrued. In HCC, 1 PR, 10 SD, and 9 style="fixed-case">PDs were seen. style="fixed-case">DCR in style="fixed-case">HCC was 42%. Among seven (7) patients with style="fixed-case">BDC, one patient was not evaluable; one achieved a long lasting style="fixed-case">PR, and five patients had style="fixed-case">SD and style="fixed-case">DCR was 86%. Median overall survival ( style="fixed-case">OS) times and progression‐free survivals ( style="fixed-case">PFS) were 196 and 149 days in style="fixed-case">HCC and 238 days and not reached in style="fixed-case">BDC. style="fixed-case">PFS at 26 weeks in style="fixed-case">HCC was 41% and at 21 weeks in style="fixed-case">BDC was 60%. Grade 3 toxicities in >5% of patients were fatigue (12.9%), neutropenia (9.6%), thrombocytopenia (9.6%), and diarrhea (6.4%). G+O+E exceeded both preset P0a and P1 of the primary objective with a style="fixed-case">PFS of 41% at 26 weeks for style="fixed-case">HCC and preliminary style="fixed-case">BDC data may warrant further investigations.