Nab-paclitaxel plus S-1 in advanced pancreatic adenocarcinoma (NPSPAC): a single arm, single center, phase II trial

// Yan Shi 1 , Sui Zhang 2 , Quanli Han 1 , Jie Li 3 , Huan Yan 1 , Yao Lv 1 , Huaiyin Shi 3 , Rong Liu 4 and Guanghai Dai 1 1 Medical Oncology Department 2, Chinese PLA General Hospital and Chinese PLA Medical School, Beijing, P.R. China 2 Department of Medical Oncology, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA, USA 3 Pathology Department, Chinese PLA General Hospital and Chinese PLA Medical School, Beijing, P.R. China 4 Department of Hepatobiliary and Pancreatic Surgical Oncology, Chinese PLA General Hospital and Chinese PLA Medical School, Beijing, P.R. China Correspondence to: Guanghai Dai, email: daigh301@vip.sina.com Keywords: nab-paclitaxel, S-1, objective response rate, survival, advanced pancreatic adenocarcinoma Received: April 28, 2017 Accepted: August 28, 2017 Published: September 28, 2017 ABSTRACT This single-arm, phase II trial is to investigate efficacy and safety of nab-paclitaxel plus S-1 as first-line treatment in advanced pancreatic cancer. Nab-paclitaxel was administered at 120 mg/m 2 intravenously on day 1 and 8, S-1 was given twice a day orally on day 1-14 of each 21-day cycle, for 6 cycles. The primary endpoint was objective response rate (ORR), the secondary endpoints were progression-free survival (PFS), overall survival (OS) and safety. The ORR in intent-to-treat population (N=60) by either blinded independent review (BIR) or investigator assessment was 50.0%. Median PFS (mPFS) by BIR and median OS (mOS) were 5.6 months (95%CI, 4.6 to 6.6 m) and 9.4 months (95%CI, 8.0 to 10.8m), respectively. The most common grade 3 or 4 toxicities were leukopenia/neutropenia (35%) and fatigue (8.3%). Subgroup analyses based on BIR showed a remarkable ORR (>70%) was achieved in patients with female gender, ≥ 50% decline from baseline CA19-9, and developed grade 3 or 4 leukopenia/neutropenia. Remarkable survival benefit was statistically significant in female (mPFS: 7.7m, mOS: 18.2m), ≥ 50% decline from baseline CA19-9 (mPFS: 6.8m, mOS: 11.8m), objective responders (mPFS: 6.9m, mOS: 12.2m), and ECOG of 0 at baseline (mPFS: 7.5m, mOS: 16.1m). Nab-paclitaxel plus S-1 showed encouraging ORR and manageable toxicities, which is an effective alternative treatment regimen for advanced pancreatic cancer. ( https://clinicaltrials.gov/ number, NCT02124317)