Open AccessPhase II Trial of Trifluridine/Tipiracil in Patients with Advanced, Refractory Biliary Tract Carcinoma
Author(s) -
Chakrabarti Sakti,
Zemla Tyler J.,
Ahn Daniel H.,
Ou FangShu,
Fruth Briant,
Borad Mitesh J.,
Hartgers Mindy L.,
Wessling Jaclynn,
Walkes Rachel L.,
Alberts Steven R.,
McWilliams Robert R.,
Liu Minetta C.,
Durgin Lori M.,
BekaiiSaab Tanios S.,
Mahipal Amit
Publication year - 2020
Publication title -
the oncologist
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.176
H-Index - 164
eISSN - 1549-490X
pISSN - 1083-7159
DOI - 10.1634/theoncologist.2019-0874
Subject(s) - medicine , clinical endpoint , progression free survival , refractory (planetary science) , phases of clinical research , gastroenterology , population , oncology , chemotherapy , surgery , clinical trial , physics , environmental health , astrobiology
Lessons Learned Trifluridine/tipiracil (FTD/TPI) shows promising antitumor activity in heavily pretreated patients with advanced biliary tract carcinoma, including patients with 5‐fluorouracil refractory tumors. FTD/TPI has an acceptable safety profile and should be studied further in patients with advanced biliary tract carcinoma after progression on standard first‐line therapy.Background Patients with advanced biliary tract carcinoma (BTC) refractory to first‐line therapy lack an established second‐line option. Trifluridine/tipiracil (FTD/TPI) has activity in both fluoropyrimidine‐sensitive and ‐resistant tumors, which led us to conduct a single arm phase II trial to evaluate the safety and efficacy of FTD/TPI for patients previously treated for advanced BTC. Methods Patients with advanced BTC previously treated with at least one line of chemotherapy were enrolled and treated with FTD/TPI until disease progression or unacceptable toxicity. The primary endpoint target was to have at least 6 patients who were progression free and alive at 16 weeks among 25 evaluable patients. Secondary endpoints included overall survival (OS), overall response rate (ORR), progression‐free survival (PFS), and toxicity. Results Of 27 evaluable patients, 59.3% received at least three prior lines of therapy, and 81.5% had previous exposure to fluoropyrimidine. Eight (32%, 95% confidence interval [CI], 14.9%–53.5%) patients were progression free at 16 weeks in the primary analysis population ( n = 25), which met the predefined efficacy criteria. Median PFS and OS were 3.8 (95% CI, 2–5.8 months) and 6.1 (95% CI, 4.4–11.4 months) months, respectively. No objective responses were seen. There were no unexpected safety signals noted. Conclusion FTD/TPI demonstrated promising antitumor activity, with acceptable toxicity, in heavily pretreated patients with advanced BTC.