In an updated analysis of a Korean phase IIb trial (NIFTY) reported in JAMA Oncology, Hyung et al found that the addition of nanoliposomal irinotecan to fluorouracil and leucovorin significantly improved progression-free survival in patients with metastatic biliary tract cancer with disease progression on first-line treatment with gemcitabine/cisplatin.
The primary analysis of the trial showed significantly prolonged progression-free survival with the addition of nanoliposomal irinotecan to fluorouracil/leucovorin. The updated analysis included extended follow-up of 1.3 years and performance of masked independent central review by a new team of radiologists. In the multicenter trial, 174 patients (full analysis set) were randomly assigned between September 2018 and December 2021 to receive leucovorin at 400 mg/m2 and fluorouracil at 2,400 mg/m2 via 46-hour infusion every 2 weeks with (n = 88) or without (n = 86) nanoliposomal irinotecan at 70 mg/m2. Treatment continued until disease progression or unacceptable toxicity. The primary endpoint was progression-free survival on masked independent central review.
Median follow-up was 33.2 months (interquartile range = 27.6–35.7 months). Median progression-free survival on masked independent central review was 4.2 months (95% confidence interval [CI] = 2.8–5.3 months) in the nanoliposomal irinotecan group vs 1.7 months (95% CI = 1.4–2.6 months) in the control group (hazard ratio [HR] = 0.61, 95% CI = 0.44–0.86, P = .004). The rate at 6 months was 31.8% (95% CI = 21.7%–41.8%) vs 15.1% (95% CI = 7.5%–22.7%).
On investigator assessment, median progression-free survival was 3.9 months (95% CI = 2.7–5.2 months) in the nanoliposomal irinotecan group vs 1.6 months (95% CI = 1.3–2.2 months in the control group [HR = 0.51, 95% CI = 0.36–0.71, P < .001]). The ratesat 6 months was 30.0% (95% CI = 20.2%–39.8%) vs 11.6% (95% CI = 4.9%–18.4%).
Median overall survival was 8.6 months (95% CI = 5.4–10.5 months) in the nanoliposomal irinotecan group vs 5.3 months (95% CI = 4.7–7.2 months) in the control group (HR = 0.68, 95% CI = 0.48–0.95, P = .02). The rate at 6 months was 60.7% (95% CI = 50.3%–71.2%) vs 44.7% (95% CI = 34.2%–55.3%).
The investigators concluded: “The NIFTY randomized clinical trial demonstrated significant improvement in [progression-free survival] with treatment with [nanoliposomal irinotecan] plus [fluorouracil/leucovorin] compared with [fluorouracil/leucovorin] alone for patients with advanced [biliary tract cancer] after progression to gemcitabine plus cisplatin. The combination of [nanoliposomal irinotecan] plus [fluorouracil/leucovorin] could be considered as a second-line treatment option for patients with previously treated advanced [biliary tract cancer].”
Changhoon Yoo, MD, PhD, of the Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, is the corresponding author of the JAMA Oncology article.
Disclosure: The study was supported by Servier and HK inno.N. For full disclosures of the study authors, visit jamanetwork.com.The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.