The Lynx Group
Cholangiocarcinoma News

Comparison of Liposomal Irinotecan (nal-IRI) plus 5-Fluorouracil/Leucovorin (5-FU/LV) versus Gemcitabine/Cisplatin as First-Line Therapy in Patients with Advanced Biliary Tract Cancer: The NIFE Trial

Web Exclusives — December 20, 2021

Clinical outcomes in advanced biliary tract cancer (BTC) are modest with the current standard of care gemcitabine/cisplatin. The NIFE study examined nanoliposomal-irinotecan (nal-IRI)/5-fluorouracil (5-FU)/leucovorin (LV) as an alternative first-line treatment option in advanced BTC.

NIFE was a prospective, randomized, 2-sided, phase 2 study in which 93 patients with advanced BTC in 21 centers in Germany were randomized (1:1) to receive either nal-IRI/5-FU/LV (arm A) or gemcitabine/cisplatin (arm B), with a stratification for tumor site (intrahepatic vs extrahepatic), gender, and Eastern Cooperative Oncology Group performance status (0 vs 1). Arm A was planned as a Simon’s optimal 2-stage design and arm B served as an internal control for selection bias. The primary end point was defined as the 4-month progression-free survival (PFS) rate ≥50% in the intent-to-treat (ITT) population.

The NIFE trial met its primary end point with a PFS rate of 51% at 4 months in the ITT population (arm A). Median PFS in arm A was 5.98 months (95% confidence interval [CI], 2.37-9.59) and 6.87 months (95% CI, 2.46-7.82) in arm B. Provisional median overall survival (OS) was 15.9 months (95% CI, 10.58-21.85) in arm A and 13.63 months (95% CI, 6.51-17.68) in arm B with ongoing follow-up at data closure.

Median PFS in intrahepatic cholangiocarcinoma (CCA) versus extrahepatic CCA was 3.45 months (95% CI, 2.10-6.05) versus 9.59 months (95% CI, 1.94-15.67) in arm A, and 7.72 months (95% CI, 6.05-9.46) versus 1.76 months (95% CI, 0.16-6.87) in arm B. Corresponding median OS was 14.19 months for intrahepatic CCA and 18.23 months for extrahepatic CCA in arm A; and 16.36 months for intrahepatic CCA and 6.34 months for extrahepatic CCA in arm B.

The investigators concluded that nal-IRI/5-FU/LV showed efficacy as first-line treatment of advanced BTC with no new safety findings. Extrahepatic CCA and intrahepatic CCA responded differently to drug interventions, with a clear benefit for nal-IRI/5-FU/LV in extrahepatic CCA.

Source

Perkhofer L, Striefler JK, Sinn M, et al. Nal-IRI with 5-fluorouracil (5-FU) and leucovorin or gemcitabine plus cisplatin in advanced biliary tract cancer: final results of the NIFE-trial (AIO-YMO HEP-0315), a randomized phase II study of the AIO biliary tract cancer group. Ann Oncol. 2021;32(suppl_5):S1283-S1346.

Related Items

Efficacy and Safety of Infigratinib in Patients with Previously Treated Advanced CCA with an FGFR2 Gene Fusion or Rearrangement
Web Exclusives
Infigratinib demonstrates significant efficacy and a manageable safety profile in patients with previously treated, locally advanced or metastatic CCA with an FGFR2 gene fusion or rearrangement.
Efficacy and Safety of Ivosidenib in Patients with Previously Treated CCA with an IDH1 Mutation: The ClarIDHy Trial
Web Exclusives
The ClarIDHy trial demonstrated safety and efficacy of ivosidenib in patients with previously treated metastatic CCA harboring an IDH1 mutation.
Futibatinib in iCCA with FGFR2 Fusions/Rearrangements: The FOENIX-CCA2 Study
Web Exclusives
Initial results of the phase 2 FOENIX-CCA2 study demonstrated that futibatinib demonstrated efficacy and a manageable safety profile in patients with previously treated iCCA harboring an FGFR2 fusion or rearrangement.
Efficacy and Safety of Derazantinib in Patients with Previously Treated iCCA with an FGFR2 Fusion or Rearrangement: The FIDES-01 Study
Web Exclusives
The phase 2 FIDES-01 study demonstrated that derazantinib resulted in objective responses and a manageable safety profile in patients with previously treated iCCA with an FGFR2 fusion or rearrangement.
Safety and Initial Efficacy of Zanidatamab (ZW25) in HER2-Overexpressing Biliary Tract Cancer
Web Exclusives
Results from a phase 1 study showed that the bispecific HER2-targeted antibody zanidatamab is well-tolerated and demonstrates clinical activity in patients with HER2-overexpressing biliary tract cancer.

Subscribe to CCA News

Stay up to date with personalized medicine by subscribing to receive the free CCA News print publication or weekly e‑Newsletter.

I'd like to receive: