Subtype
Malignant Hepatobiliary Neoplasm
CI-CAN-00001136Explore in graph →
- NCIt
- C8609
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Subtype
CI-CAN-00001136Explore in graph →
Variants & evidence
107 evidence items mapped to this entity or its descendants, grouped by molecular profile, then therapy. 50 items per page.
| Therapy | Cancer | Type | Level | Direction · significance | Rating (1–5) | Status | Evidence | Source |
|---|---|---|---|---|---|---|---|---|
| BICC1 Fusion1 | ||||||||
| Infigratinib + PD173074Substitutes | Cholangiocarcinoma | Predictive | D | Supports Sensitivity Response | 3 | accepted | EID1851Expression of the FGFR2-BICC1 fusion kinase in NIH3T3 cells demonstrated transforming ability that was effectively suppressed with the fibroblast growth factor receptor (FGFR) kinase inhibitors BGJ398… (full text at CIViC) PMID 24122810 · Arai et al., 2014 · Open in CIViC | civic |
| FGFR2 C383R1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | submitted | EID11631In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| CCDC6 Fusion1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | submitted | EID11646In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| DBP Fusion1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | submitted | EID11628In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| FILIP1 Fusion1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 1 | submitted | EID11630In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| FGFR2 Fusion1 | ||||||||
| Erdafitinib | Cholangiocarcinoma | Predictive | B | Supports Sensitivity Response | 3 | submitted | EID10406Phase I dose-escalation cohort of patients with advanced solid tumors in which antineoplastic therapy (NCT01703481) was no longer effective, using pan-FGFR inhibitor erdafitinib (JNJ-42756493). Among … (full text at CIViC) PMID 31088831 · Bahleda et al., 2019 · Open in CIViC | civic |
| KIAA1217 Fusion2 | ||||||||
| Futibatinib | Intrahepatic Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 3 | submitted | EID11611In a phase 2 trial (NCT02052778) of the FGFR1/2/3/4 inhibitor futibatinib in 103 patients with previously treated intrahepatic cholangiocarcinoma harboring FGFR2 fusions or rearrangements, the overal… (full text at CIViC) PMID 36652354 · Goyal et al., 2023 · Open in CIViC | civic |
| 〃 | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 1 | submitted | EID11647In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| FGFR2 M538I1 | ||||||||
| Sorafenib | Hepatocellular Carcinoma | Predictive | C | Supports Sensitivity Response | 1 | submitted | EID13271Among 45 patients with recurrent hepatocellular carcinoma treated with sorafenib, one patient whose tumor harbored FGFR2 M538I achieved a partial response. The same tumor also harbored FGFR4 G665E, pr… (full text at CIViC) PMID 27384874 · Kaibori et al., 2016 · Open in CIViC | civic |
| FGFR2 Mutation4 | ||||||||
| Erdafitinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 3 | submitted | EID10375Phase I dose-escalation cohort (NCT01703481) of patients with advanced solid tumors in which antineoplastic therapy was no longer effective using pan-FGFR inhibitor erdafitinib (JNJ-42756493). 1 chola… (full text at CIViC) PMID 31088831 · Bahleda et al., 2019 · Open in CIViC | civic |
| 〃 | Cholangiocarcinoma | Predictive | C | Does Not Support Sensitivity Response | 3 | submitted | EID10398Phase I dose-escalation cohort (NCT01703481) of patients with advanced solid tumors in which antineoplastic therapy was no longer effective using pan-FGFR inhibitor JNJ-42756493. 2 cholangiocarcinoma … (full text at CIViC) PMID 31088831 · Bahleda et al., 2019 · Open in CIViC | civic |
| Infigratinib | Cholangiocarcinoma | Predictive | B | Supports Sensitivity Response | 4 | |||
| CDH1 MUTATION + CDKN2A Mutation + CDKN2B Mutation + FGFR2 MutationFGFR2CDH1CDKN2ACDKN2B1 | ||||||||
| Crizotinib + Palbociclib Regimen + PazopanibCombination | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | submitted | EID12777In a case report of metastatic cholangiocarcinoma harboring an FGFR2 W290_P307>C (16.25% Variant allele frequency) alteration together with CDH1 Q677fs*7 (23.1% VAF) and CDKN2A/B frameshift mutations … (full text at CIViC) PMID 38645422 · Aydın et al., 2024 · Open in CIViC | civic |
| NRAP Fusion1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | submitted | EID11641In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| OGA Fusion1 | ||||||||
| Ponatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | accepted | EID295In a single patient with this fusion in intrahepatic cholangiocarcinoma. Ponatinib treatment resulted in necrosis and shrinkage of the primary and metastatic sites and overall stable disease. However,… (full text at CIViC) PMID 24550739 · Borad et al., 2014 · Open in CIViC | civic |
| FGFR2 Overexpression2 | ||||||||
| (prognostic) | Hepatocellular Carcinoma | Prognostic | B | Supports Poor Outcome | 4 | submitted | EID6456Patients with high FGFR2 expression had an overall survival rate of 72.2,40.6% at 1,3 years (p<0.05) and those with low FGFR2 expression had a 1,3 year survival rate of 92.0,82.8% (p<0.05). The diseas… (full text at CIViC) PMID 20798558 · Harimoto et al., 2010 · Open in CIViC | civic |
| 〃 | Hepatocellular Carcinoma | Prognostic | B | Supports Poor Outcome | 3 | submitted | EID6460FGFR1, -2, -3, and -4 were expressed in 5.3%, 11.1%, 3.8%, and 52.7% of HCCs, respectively. Among the development set of 153 patients, FGFR2 positivity in HCC was associated with a significantly short… (full text at CIViC) PMID 25834803 · Lee et al., 2015 · Open in CIViC | civic |
| POC1B Fusion1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 1 | submitted | EID11627In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| TACC3 Fusion1 | ||||||||
| Pazopanib + PonatinibSequential | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 3 | accepted | EID296In a 43 year old female patient with this fusion in intrahepatic cholangiocarcinoma. Both pazopanib and ponatinib separately resulted in minor tumor shrinkage. This fusion is also shown to be recurren… (full text at CIViC) PMID 24550739 · Borad et al., 2014 · Open in CIViC | civic |
| TNS1 Fusion1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | submitted | EID11644In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| TTC28 Fusion1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | submitted | EID11645In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| FGFR2 Fusion3 | ||||||||
| Derazantinib | Cholangiocarcinoma | Predictive | D | Supports Sensitivity Response | 4 | submitted | EID7969ARQ 087 exhibited inhibitory effects biochemically, in cell line and cell cycle study of FGFR2 functions. PMID 27627808 · Hall et al., 2016 · Open in CIViC | civic |
| Futibatinib | Cholangiocarcinoma | Predictive | B | Supports Sensitivity Response | 4 | submitted | EID12489The authors detail a phase 1 dose-expansion trial of a pan-FGFR inhibitor, fubatinib. Most FGFR inhibitors are reversible ATP-competitor inhibitors that tumors can acquire resistance to. Futibatinib, … (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| 〃 | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 3 | submitted | EID7322 | |
| FGFR2 Fusion + FGFR2 Fusion8 | ||||||||
| Derazantinib | Intrahepatic Cholangiocarcinoma | Predictive | B | Supports Sensitivity Response | 3 | accepted | EID7329Actionable fibroblast growth factor receptor 2 (FGFR2) fusions have been identified in intrahepatic cholangiocarcinomas (iCCA). Derazantinib, a multi-kinase inhibitor with potent pan-FGFR activity sho… (full text at CIViC) PMID 30420614 · Mazzaferro et al., 2019 · Open in CIViC | civic |
| Futibatinib | Intrahepatic Cholangiocarcinoma | Predictive | A | Supports Sensitivity Response | 4 | submitted | EID11609In the phase 2 trial (NCT02052778), 103 patients (22-79 years of age) with unresectable or metastatic intrahepatic cholangiocarcinoma harboring FGFR2 fusions or rearrangements were treated with the … (full text at CIViC) PMID 36652354 · Goyal et al., 2023 · Open in CIViC | civic |
| 〃 | Intrahepatic Cholangiocarcinoma | Predictive | B | Supports | ||||
| FGFR2 W290C1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 1 | submitted | EID11633In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| WAC Fusion1 | ||||||||
| Futibatinib | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 1 | submitted | EID11643In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| FGFR2 Y376C1 | ||||||||
| AZD-4547 | Intrahepatic Cholangiocarcinoma | Predictive | C | Does Not Support Sensitivity Response | 1 | submitted | EID8317The phase II NCI-MATCH trial (EAY131) subprotocol W assessed the FGFR1-3 selective TKI AZD-4547 in 48 patients with FGFR1/2 amplifications, FGFR2/3 SNV or FGFR1/3 fusions. The partial response overall… (full text at CIViC) PMID 32463741 · Chae et al., 2020 · Open in CIViC | civic |
| FGFR4 G665E1 | ||||||||
| Sorafenib | Hepatocellular Carcinoma | Predictive | C | Supports Sensitivity Response | 1 | submitted | EID13272Among 45 patients with recurrent hepatocellular carcinoma treated with sorafenib, one patient whose tumor harbored FGFR4 G665E achieved a partial response. The same tumor also harbored FGFR2 M538I, pr… (full text at CIViC) PMID 27384874 · Kaibori et al., 2016 · Open in CIViC | civic |
| GALNT14 rs96791621 | ||||||||
| (prognostic) | Hepatocellular Carcinoma | Prognostic | B | Supports Poor Outcome | 4 | submitted | EID10100From October 2015 to April 2019, 103 HCC patients scheduled to receive chemoembolization were screened. Of them, 84 met inclusion criteria and were assigned to TT (TACE) (n = 25), Non-TT (TACE) (n = 3… (full text at CIViC) PMID 34982369 · Chen et al., 2022 · Open in CIViC | civic |
| GNAS c.393T>C1 | ||||||||
| (prognostic) | Intrahepatic Cholangiocarcinoma | Prognostic | B | Supports Poor Outcome | 3 | accepted | EID1996Intrahepatic cholangiocarcinomas of patients with homozygous TT genotypes revealed a higher proliferation rate and a lower apoptotic rate.This study demonstrated a significant association of both dise… (full text at CIViC) PMID 17356712 · Schmitz et al., 2007 · Open in CIViC | civic |
| IDH1 Mutation1 | ||||||||
| Ivosidenib | Cholangiocarcinoma | Predictive | B | Supports Sensitivity Response | 3 | accepted | EID7447In a phase1 trial, 73 patients with cholangiocarcinoma harboring IDH1 mutations were treated with mutant IDH1 inhibitor ivosidenib. The response rate was 5%, median progression free survival was 3.8 m… (full text at CIViC) PMID 31300360 · Lowery et al., 2019 · Open in CIViC | civic |
| IDH1 R1322 | ||||||||
| Dasatinib | Intrahepatic Cholangiocarcinoma | Predictive | D | Supports Sensitivity Response | 4 | accepted | EID6439Through a high-throughput drug screen of a large panel of cancer cell lines, including 17 biliary tract cancers, this work found that IDH mutant (IDHm) ICC cells demonstrate a striking response to the… (full text at CIViC) PMID 27231123 · Saha et al., 2016 · Open in CIViC | civic |
| Ivosidenib | Cholangiocarcinoma | Predictive | A | Supports Sensitivity Response | 5 | accepted | EID10886The multicenter randomized double-blind phase 3 study included patients with IDH1-mutant cholangiocarcinoma who had progressed on previous therapy. Patients were randomly assigned to Ivosidenib or pla… (full text at CIViC) PMID 32416072 · Abou-Alfa et al., 2020 · Open in CIViC | civic |
| JAK1 A1086S1 | ||||||||
| Ruxolitinib | Hepatocellular Carcinoma | Predictive | D | Does Not Support Sensitivity Response | 3 | submitted | EID10058JAK1 mutations were identified by sequencing 60 HCC PDX tumour models. There mutations were then functionally characterized. HEK293FT cells were transfected with JAK1 WT or mutants (N451S, E483D, S703… (full text at CIViC) PMID 26701727 · Yang et al., 2016 · Open in CIViC | civic |
| JAK1 E483D1 | ||||||||
| Ruxolitinib | Hepatocellular Carcinoma | Predictive | D | Does Not Support Sensitivity Response | 3 | submitted | EID10057JAK1 mutations were identified by sequencing 60 HCC PDX tumour models. There mutations were then functionally characterized. HEK293FT cells were transfected with JAK1 WT or mutants (N451S, E483D, S703… (full text at CIViC) PMID 26701727 · Yang et al., 2016 · Open in CIViC | civic |
| JAK1 N451S1 | ||||||||
| Ruxolitinib | Hepatocellular Carcinoma | Predictive | D | Does Not Support Sensitivity Response | 3 | submitted | EID10056JAK1 mutations were identified by sequencing 60 HCC PDX tumour models. There mutations were then functionally characterized. HEK293FT cells were transfected with JAK1 WT or mutants (N451S, E483D, S703… (full text at CIViC) PMID 26701727 · Yang et al., 2016 · Open in CIViC | civic |
| JAK1 S703I1 | ||||||||
| Ruxolitinib | Hepatocellular Carcinoma | Predictive | D | Supports Sensitivity Response | 4 | accepted | EID1900A JAK1 S703I mutation was found to activate the JAK-STAT signaling pathway in vitro and in vivo in hepatocellular carcinoma. Ruxolitinib causes the suppression of JAK-STAT signaling pathway in PDX mod… (full text at CIViC) PMID 26701727 · Yang et al., 2016 · Open in CIViC | civic |
| KIF23 EXPRESSION1 | ||||||||
| (prognostic) | Hepatocellular Carcinoma | Prognostic | B | Supports Better Outcome | 3 | accepted | EID1745KIF23 variant 1 was found to be expressed in the cDNA of tumor tissues from a large cohort of HCC patients of chinese descent via PCR. This expression was absent in the normal tissues of these patient… (full text at CIViC) PMID 26674738 · Sun et al., 2015 · Open in CIViC | civic |
| KRAS Mutation1 | ||||||||
| Refametinib + SorafenibCombination | Hepatocellular Carcinoma | Predictive | B | Supports Sensitivity Response | 2 | accepted | EID1642Phase 2 study in Asian patients with unresectable hepatocellular carcinoma. 70 patients received refametinib (BAY 86-9766) plus sorafenib as study treatment. A retrospective biomarker analysis was per… (full text at CIViC) PMID 25294897 · Lim et al., 2014 · Open in CIViC | civic |
| MAGEH1 EXPRESSION1 | ||||||||
| Gemcitabine | Bile Duct Adenocarcinoma | Predictive | D | Supports Resistance | 2 | accepted | EID95010 biliary tract carcinoma cell lines were sorted based on their gemcitabine sensitivity. MAGEH1 protein expression was negatively correlated with sensitivity. In a clinical validation cohort, MAGEH p… (full text at CIViC) PMID 20088962 · Ojima et al., 2010 · Open in CIViC | civic |
| NEDD9 EXPRESSION1 | ||||||||
| (prognostic) | Hepatocellular Carcinoma | Prognostic | B | Supports Poor Outcome | 3 | accepted | EID1059IHC was performed in 123 hepatocellular carcinoma tissues and their adjacent normal liver tissues for protein expression of HEF1 (NEDD9). Overexpression of HEF1 protein was observed in HCC tissue when… (full text at CIViC) PMID 26893853 · Li et al., 2016 · Open in CIViC | civic |
Data updated 22 hours agoSource updated unknownsource: civic (CC0)
Evidence levels, directions and ratings are those assigned by CIViC curators. "Submitted" items have not completed curation review. This is not treatment guidance.
| accepted |
EID5912Eight patients with cholangiocarcinoma haboring FGFR2 mutations received BGJ398. No response was observed, but one tumor showed a 23% reduction in size. Water fall plot of this article showed 5 patie… (full text at CIViC) PMID 29182496 · Javle et al., 2018 · Open in CIViC |
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| 〃 | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | accepted | EID1912Phase 1 study of FGFR-inhibitor BGJ398 in advanced cancer with FGFR-aberrations. Among three patients with cholangiocarcinoma and FGFR2 aberrations (2 fusions, 1 mutation), all three had stable diseas… (full text at CIViC) PMID 27870574 · Nogova et al., 2017 · Open in CIViC | civic |
Among six patients with advanced FGFR2 fusion-positive ICC who received care at our institution after progression on BGJ398 or Debio1347 in clinical trials, four subsequently enrolled in the phase I t… (full text at CIViC)
PMID 31109923 · Goyal et al., 2019 · Open in CIViC
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| 3 |
| submitted |
EID11612In a phase 2 trial (NCT02052778) of the FGFR1/2/3/4 inhibitor futibatinib in 103 patients with previously treated intrahepatic cholangiocarcinoma harboring FGFR2 fusions or rearrangements, the overal… (full text at CIViC) PMID 36652354 · Goyal et al., 2023 · Open in CIViC |
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| 〃 | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 3 | submitted | EID11632In a phase 1 multihistology trial of the FGFR1/2/3/4 inhibitor futibatinib, an objective response rate of 13.7% was seen with responses in a broad spectrum of tumors and across a broad spectrum of FGF… (full text at CIViC) PMID 34551969 · Meric-Bernstam et al., 2022 · Open in CIViC | civic |
| Infigratinib | Cholangiocarcinoma | Predictive | A | Supports Sensitivity Response | 4 | accepted | EID11230This open-label phase 2 trial included 122 patients with histologically or cytologically confirmed, locally advanced or metastatic cholangiocarcinoma, 108 of whom had an FGFR2 fusion or rearrangement.… (full text at CIViC) PMID 34358484 · Javle et al., 2021 · Open in CIViC | civic |
| 〃 | Cholangiocarcinoma | Predictive | B | Supports Sensitivity Response | 4 | accepted | EID5908This Phase II trial of BGJ398 for FGFR2 aberant cholangiocarcinoma included 48 patients with FGFR2 fusions. Among these patients with FGFR2 fusions, eight showed tumor response (18.8%) and thirty-six … (full text at CIViC) PMID 29182496 · Javle et al., 2018 · Open in CIViC | civic |
| 〃 | Cholangiocarcinoma | Predictive | C | Supports Sensitivity Response | 2 | accepted | EID1913Phase 1 study of FGFR-inhibitor BGJ398 (infigratinib) in advanced cancer with FGFR-aberrations (n=132). Two cholangiocarcinoma patients harbored FGFR2 fusions. Both were treated at BGJ398 doses >100mg… (full text at CIViC) PMID 27870574 · Nogova et al., 2017 · Open in CIViC | civic |
| Pemigatinib | Cholangiolocellular Small Duct Intrahepatic CholangiocarcinomaALIAS | Predictive | A | Supports Sensitivity Response | 4 | accepted | EID8173In a multicentre, open-label, single-arm, phase 2 study (FIGHT-202), patients with disease progression following at least one previous treatment received a starting dose of 13·5 mg oral pemigatinib on… (full text at CIViC) PMID 32203698 · Abou-Alfa et al., 2020 · Open in CIViC | civic |