Trial reportCancer2019
A phase 2 trial of regorafenib as a single agent in patients with chemotherapy-refractory, advanced, and metastatic biliary tract adenocarcinoma.
Trial report in Cancer, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 71 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
71 citing papers in PubMed, 121 citations in OpenAlex.
- Regorafenib plus modified gemcitabine-oxaliplatin in patients with advanced biliary tract cancer. The randomized phase Ib/II BREGO study.The oncologist · 2025Trial
- Bortezomib in previously treated phosphatase and tension homology-deficient patients with advanced intrahepatic cholangiocarcinoma: An open-label, prospective and single-centre phase II trial.Clinical and translational medicine · 2024Trial
- Chemotherapy combined with regorafenib and immune checkpoint inhibitors as a first-line treatment for patients with advanced biliary tract cancer: a single arm phase II trial.Frontiers in immunology · 2024Trial
- Multicenter Phase II Trial of Axitinib Monotherapy for Gemcitabine-Based Chemotherapy Refractory Advanced Biliary Tract Cancer (AX-BC Study).The oncologist · 2021Trial
- Phase 2 study of lenvatinib monotherapy as second-line treatment in unresectable biliary tract cancer: primary analysis results.BMC cancer · 2020Trial
- A multi-institutional phase 2 trial of regorafenib in refractory advanced biliary tract cancer.Cancer · 2020Trial
- Antiangiogenic therapy for advanced biliary tract cancers: promising or illusory?Journal of gastrointestinal oncology · 2026Article
- Expanding Horizons in Cholangiocarcinoma: Emerging Targets Beyond FGFR2 and IDH1.International journal of molecular sciences · 2025Review
- Global gene expression landscape of gallbladder cancer and advances in targeted therapeutic strategies.World journal of clinical oncology · 2025Review
- Targeted therapies and immunotherapies for unresectable cholangiocarcinoma.Chinese medical journal · 2025Review
- Analysis of the effectiveness and safety of lenvatinib/bevacizumab combined with PD-1/PD-L1 inhibitors and GEMOX in the first-line treatment of advanced biliary tract carcinoma.Clinical and experimental medicine · 2025Article
- Review
- Advances in targeted therapy of cholangiocarcinoma.Annals of medicine · 2024Review
- Efficacy and safety of lenvatinib combined with anti-PD-1 antibodies plus GEMOX chemotherapy as non-first-line systemic therapy in advanced gallbladder cancer.Cancer immunology, immunotherapy : CII · 2024Article
- Article
- Current and Future Therapeutic Targets for Directed Molecular Therapies in Cholangiocarcinoma.Cancers · 2024Review
- Recent Advances in Systemic Therapy for Advanced Intrahepatic Cholangiocarcinoma.Liver cancer · 2024Review
- Current Status of Targeted Therapy for Biliary Tract Cancer in the Era of Precision Medicine.Cancers · 2024Review
- Review
- Current and Emerging Therapeutic Targets for the Treatment of Cholangiocarcinoma: An Updated Review.International journal of molecular sciences · 2023Review
11 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 7 institutions in 1 country.
Funding
Abstract
backgroundBiliary tract cancers are rare, aggressive neoplasms. Most patients present with advanced/unresectable or metastatic disease at diagnosis, and no second-line regimen has demonstrated clinical benefit. This was a phase 2 study evaluating the efficacy and safety of regorafenib in patients who had advanced/unresectable or metastatic disease after receiving standard therapy.
methodsIn this single arm-study, patients with advanced/unresectable or metastatic biliary tract cancer who failed at least 1 line of systemic chemotherapy received regorafenib once daily on a schedule of 21-days on/7-days off in a 28-day cycle. Patients initially received a standard 160 mg dose. After toxicity assessments in the first 3 patients, the dose was reduced to 120 mg for subsequent patients, as preplanned. The primary endpoint was progression-free survival (PFS). Secondary objectives included overall survival (OS), the objective response rate, and the disease control rate.
resultsForty-three patients received at least 1 dose of regorafenib, and 34 patients who received at least 1 cycle of treatment were evaluable for tumor response. The median PFS was 15.6 weeks (90% confidence interval, 12.9-24.7 weeks), and the median OS was 31.8 weeks (90% confidence interval, 13.3-74.3 weeks), with survival rates 40% at 12 months and 32% at 18 months. A partial response was achieved in 5 patients (11%), and 19 had stable disease (44%), for a disease control rate of 56%. The toxicity profile was as expected, with grade 3 and 4 adverse events reported in 40% of patients. The most common toxicities were hypophosphatemia (40%), hyperbilirubinemia (26%), hypertension (23%), and hand-foot skin reaction (7%).
conclusionsThe current results suggest promising efficacy of regorafenib in patients with chemotherapy-refractory, advanced/metastatic biliary tract cancer, warranting further studies to confirm its clinical efficacy. There is a clear unmet need for effective therapies in patients who have advanced and metastatic biliary tract cancer.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.