Open-label study to evaluate trifluridine/tipiracil safety, tolerability and pharmacokinetics in patients with advanced solid tumours and hepatic impairment

Br J Clin Pharmacol. 2019 Jun;85(6):1239-1246. doi: 10.1111/bcp.13856. Epub 2019 Mar 28.

Abstract

Aims: Trifluridine/tipiracil (FTD/TPI) prolongs survival in refractory metastatic colorectal cancer, but limited data exist on its use in patients with hepatic impairment. This Phase I, open-label, nonrandomized study investigated the safety, tolerability and pharmacokinetics of FTD/TPI in patients with advanced solid tumours (except breast cancer) and varying degrees of hepatic impairment, to provide dosing recommendations.

Methods: Patients aged ≥18 years with advanced solid tumours and normal hepatic function, or mild, moderate or severe hepatic impairment according to National Cancer Institute criteria, were planned to be enrolled. Patients received FTD/TPI 35 mg/m2 orally twice daily on days 1-5 and 8-12 of each 28-day cycle.

Results: Twenty-four patients were enrolled to the normal hepatic function (n = 8) and mild (n = 10) and moderate (n = 6) hepatic impairment cohorts. Overall, 12 patients (50.0%) had at least 1 adverse event leading to study discontinuation. In the moderate hepatic impairment cohort, 5 of 6 patients experienced grade ≥ 3 elevation in bilirubin. No patients with severe hepatic impairment were enrolled. FTD area under the curve at steady state decreased by 18% and 22% in the mild and moderate cohorts, respectively; however, no clear change was observed in TPI area under the curve.

Conclusions: FTD/TPI can be safely administered in patients with normal hepatic function and mild hepatic impairment, with no initial dose adjustment. FTD/TPI is not recommended for use in patients with moderate hepatic impairment because of findings of grade 3 or 4 increased blood bilirubin. Therefore, FTD/TPI is not recommended for patients with moderate or severe hepatic impairment.

Keywords: chemotherapy; drug development; gastroenterology; liver; oncology; pharmacokinetics; phase I.

Publication types

  • Clinical Trial, Phase I
  • Multicenter Study
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Adult
  • Aged
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / pharmacokinetics*
  • Bilirubin / blood
  • Drug Administration Schedule
  • Drug Combinations
  • Drug Dosage Calculations
  • Female
  • Humans
  • Liver / metabolism*
  • Liver Diseases / complications*
  • Liver Diseases / diagnosis
  • Liver Diseases / metabolism
  • Male
  • Middle Aged
  • Models, Biological
  • Neoplasms / complications
  • Neoplasms / diagnosis
  • Neoplasms / drug therapy*
  • Pyrrolidines / administration & dosage
  • Pyrrolidines / adverse effects
  • Pyrrolidines / pharmacokinetics*
  • Severity of Illness Index
  • Thymine
  • Treatment Outcome
  • Trifluridine / administration & dosage
  • Trifluridine / adverse effects
  • Trifluridine / pharmacokinetics*
  • United States
  • Uracil / administration & dosage
  • Uracil / adverse effects
  • Uracil / analogs & derivatives*
  • Uracil / pharmacokinetics

Substances

  • Antineoplastic Agents
  • Drug Combinations
  • Pyrrolidines
  • trifluridine tipiracil drug combination
  • Uracil
  • Thymine
  • Bilirubin
  • Trifluridine