The phase 2 NIFE study compared a liposomal irinotecan plus fluorouracil and leucovorin regimen with standard-of-care gemcitabine/cisplatin in patients with advanced biliary tract cancer.
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.
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