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Attend this session at the
2018 ASCO Annual Meeting!

Session: Gastrointestinal (Noncolorectal) Cancer

Type: Poster Session

Time: Sunday June 3, 8:00 AM to 11:30 AM

Location: Hall A

Multi institutional phase II trial of single agent regorafenib in refractory advanced biliary cancers.

Hepatobiliary Cancer

Gastrointestinal (Noncolorectal) Cancer

2018 ASCO Annual Meeting

Abstract No:

Poster Board Number:
Poster Session (Board #271)

J Clin Oncol 36, 2018 (suppl; abstr 4082)

Author(s): Richard D. Kim, Andrew Stewart Poklepovic, Andrew B. Nixon, Dae Won Kim, Heloisa P. Soares, Jongphil Kim, Jun Min Zhou, Fatima Tariq, Natalie Burgess, Hanna Kelly Sanoff; H. Lee Moffitt Cancer Center & Research Institute, Tampa, FL; Virginia Commonwealth University Health System, Richmond, VA; Duke University Medical Center, Durham, NC; The University of Texas MD Anderson Cancer Center, Houston, TX; Moffitt Cancer Center, Tampa, FL; The University of North Carolina Lineberger Comprehensive Cancer Center, and Alliance, Chapel Hill, NC

Abstract Disclosures


Background: There is currently an unmet medical need for patients with advanced biliary cancer (BC) who have failed one prior gemcitabine based systemic therapy.Regorafenib is an oral multi-kinase inhibitor that targets both receptor tyrosine kinases (RTKs), as well as the tumor cell proliferation/survival signaling pathway kinases (RAS/RAF/MEK/ERK). Methods: Pts with histologically proven BC who progressed on at least one line of systemic therapy received regorafenib 160 mg daily 21 days on 7 days off in a 28 days cycle. The single arm design was used to access 6 month overall survival (OS) as a primary endpoint. The study tested the null hypothesis of ≤30% of OS at 6 month against the alternative of ≥50% of OS at 6 month (HR = 0.578 ). With one-sided α of 10% and 86% power, the experimental treatment was deemed to have good activity if ≥14 out of 32 evaluable patients (43.8%) survive 6 months or longer. The secondary objectives included median OS, RR and PFS. Results: Thirty nine pts received at least 1 dose of regorafenib of whom 32 pts were evaluable for efficacy. Twenty pts failed 1 line of therapy and 12 pts failed two lines of therapy. Median age was 62 (range: 27-88) years and the primary sites of tumor were intrahepatic cholangiocarcinoma (68.8%), extrahepatic (18.8%), and gallbladder (12.5%). Pts were considered evaluable for efficacy if patients received more than 1 cycle of regorafenib. Seven pts were not evaluable because one pt withdrew consent, 5 pts expired due to clinical progression within a month and 1 pt due to toxicity. For 32 evaluable pts, 6 month OS was 50.9% (95% CI: 32.1%-67.0%), 12 month OS was 35% (95% CI 16.2-53.7) and 18 month OS was 35% (95% CI 16.2-53.7). Median PFS was 3.7 months (95% CI: 2.3-5.5) and median OS was 9.9 months (95% CI: 3.7-20.1). PR was achieved in 2 (6.2%) pts, SD in 18 (56.2%) pts with DCR of 62.4%. The overall toxicity profile was as expected with grade 3/4 AE of 71.8%. The most common adverse events were fatigue (56.4%) and hypertension (53.8%). Dose modification was required in 49% of the patients. Plasma samples were collected in all pts with planned correlative studies underway. Conclusions: The primary endpoint was met in this study. Further randomized trials are warranted to confirm the efficacy Clinical trial information: NCT02115542

Other Abstracts in this Sub-Category:


1. REACH-2: A randomized, double-blind, placebo-controlled phase 3 study of ramucirumab versus placebo as second-line treatment in patients with advanced hepatocellular carcinoma (HCC) and elevated baseline alpha-fetoprotein (AFP) following first-line sorafenib.

Meeting: 2018 ASCO Annual Meeting Abstract No: 4003 First Author: Andrew X. Zhu
Category: Gastrointestinal (Noncolorectal) Cancer - Hepatobiliary Cancer


2. Randomized phase III study of gemcitabine plus S-1 combination therapy versus gemcitabine plus cisplatin combination therapy in advanced biliary tract cancer: A Japan Clinical Oncology Group study (JCOG1113, FUGA-BT).

Meeting: 2018 ASCO Annual Meeting Abstract No: 4014 First Author: Makoto Ueno
Category: Gastrointestinal (Noncolorectal) Cancer - Hepatobiliary Cancer


3. Randomized, open label, multicenter, phase II trial of transcatheter arterial chemoembolization (TACE) therapy in combination with sorafenib as compared with TACE alone in patients with hepatocellular carcinoma: TACTICS trial.

Meeting: 2018 ASCO Annual Meeting Abstract No: 4017 First Author: Masatoshi Kudo
Category: Gastrointestinal (Noncolorectal) Cancer - Hepatobiliary Cancer