FGFR-altered urothelial carcinoma is a molecularly-defined subset of bladder and upper urinary tract cancers harboring activating alterations in fibroblast growth factor receptors, most commonly FGFR3. FGFR3 mutations occur in approximately 15-20% of advanced urothelial carcinomas and are enriched in luminal-papillary subtypes. FGFR3 fusions, including FGFR3-TACC3, occur in a smaller subset. These alterations drive constitutive receptor activation and downstream RAS-MAPK and PI3K-AKT signaling. The identification of FGFR as an oncogenic driver led to development of erdafitinib, the first FDA-approved targeted therapy for metastatic urothelial carcinoma.
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name: FGFR-Altered Urothelial Carcinoma
creation_date: '2026-01-26T02:55:13Z'
updated_date: '2026-04-22T20:13:21Z'
description: >-
FGFR-altered urothelial carcinoma is a molecularly-defined subset of bladder and
upper urinary tract cancers harboring activating alterations in fibroblast growth
factor receptors, most commonly FGFR3. FGFR3 mutations occur in approximately 15-20%
of advanced urothelial carcinomas and are enriched in luminal-papillary subtypes.
FGFR3 fusions, including FGFR3-TACC3, occur in a smaller subset. These alterations
drive constitutive receptor activation and downstream RAS-MAPK and PI3K-AKT signaling.
The identification of FGFR as an oncogenic driver led to development of erdafitinib,
the first FDA-approved targeted therapy for metastatic urothelial carcinoma.
categories:
- Genitourinary Cancer
- Molecularly-Defined Cancer
parents:
- urothelial carcinoma
has_subtypes:
- name: FGFR3-Mutant Urothelial Carcinoma
description: >-
Most common FGFR alteration type, with activating point mutations in FGFR3
including S249C, Y373C, and R248C. These mutations cause constitutive receptor
dimerization and activation.
- name: FGFR2/3-Fusion Positive Urothelial Carcinoma
description: >-
Gene fusions involving FGFR2 or FGFR3, most commonly FGFR3-TACC3, lead to
constitutive kinase activation through dimerization mediated by the fusion partner.
pathophysiology:
- name: FGFR3 Constitutive Activation
description: >-
Activating mutations in FGFR3 cause ligand-independent receptor dimerization
and autophosphorylation. The most common mutations affect cysteine residues in
the extracellular domain, creating aberrant disulfide bonds that constitutively
activate the receptor.
cell_types:
- preferred_term: epithelial cell of urethra
term:
id: CL:1000296
label: epithelial cell of urethra
biological_processes:
- preferred_term: fibroblast growth factor receptor signaling pathway
modifier: INCREASED
term:
id: GO:0008543
label: fibroblast growth factor receptor signaling pathway
locations:
- preferred_term: urinary bladder
term:
id: UBERON:0001255
label: urinary bladder
downstream:
- target: RAS-MAPK Pathway Activation
description: FGFR3 phosphorylation activates RAS-RAF-MEK-ERK signaling cascade
- target: PI3K-AKT Pathway Activation
description: FGFR3 phosphorylation recruits PI3K leading to AKT activation
- name: RAS-MAPK Pathway Activation
description: >-
FGFR3 activation stimulates the RAS-RAF-MEK-ERK signaling cascade through
adaptor proteins FRS2 and GRB2. This drives uncontrolled urothelial cell
proliferation and is a major effector of FGFR-mediated transformation.
biological_processes:
- preferred_term: MAPK cascade
modifier: INCREASED
term:
id: GO:0000165
label: MAPK cascade
- name: PI3K-AKT Pathway Activation
description: >-
FGFR3 activation recruits PI3K and activates the PI3K-AKT-mTOR signaling
pathway, promoting urothelial cell survival and resistance to apoptosis.
biological_processes:
- preferred_term: phosphatidylinositol 3-kinase signaling
modifier: INCREASED
term:
id: GO:0043491
label: phosphatidylinositol 3-kinase/protein kinase B signal transduction
histopathology:
- name: Urothelial Carcinoma
finding_term:
preferred_term: Urothelial Carcinoma
term:
id: NCIT:C4030
label: Urothelial Carcinoma
frequency: VERY_FREQUENT
description: Bladder carcinomas are classified as urothelial carcinoma.
evidence:
- reference: PMID:21566415
reference_title: "Diagnosis and management of urothelial carcinoma of the bladder."
supports: SUPPORT
snippet: "carcinomas are classified as urothelial carcinoma (UC)"
explanation: Abstract states that bladder carcinomas are classified as urothelial carcinoma.
phenotypes:
- category: Genitourinary
name: Hematuria
frequency: VERY_FREQUENT
diagnostic: true
description: >-
Gross or microscopic hematuria is the most common presenting symptom of
urothelial carcinoma. May be intermittent and painless initially.
phenotype_term:
preferred_term: Hematuria
term:
id: HP:0000790
label: Hematuria
- category: Genitourinary
name: Dysuria
frequency: FREQUENT
description: >-
Painful urination may occur due to bladder irritation or tumor involvement.
Often associated with urinary frequency and urgency.
phenotype_term:
preferred_term: Dysuria
term:
id: HP:0100518
label: Dysuria
- category: Systemic
name: Fatigue
frequency: FREQUENT
description: >-
Constitutional symptoms including fatigue are common in advanced disease
and may be related to anemia from chronic blood loss.
phenotype_term:
preferred_term: Fatigue
term:
id: HP:0012378
label: Fatigue
biochemical:
- name: FGFR3 Mutation Testing
notes: >-
Molecular testing for FGFR3 mutations and fusions is required for erdafitinib
eligibility. FDA-approved companion diagnostics include therascreen FGFR RGQ RT-PCR.
genetic:
- name: FGFR3
association: Somatic Activating Mutations and Fusions
notes: >-
FGFR3 alterations occur in 15-20% of metastatic urothelial carcinomas. Common
mutations include S249C, Y373C, R248C, and G370C. FGFR3-TACC3 fusions and other
rearrangements also activate the pathway. FGFR2 alterations are less common.
evidence:
- reference: PMID:31264434
reference_title: "Evolving Role of Genomics in Genitourinary Neoplasms."
supports: PARTIAL
snippet: "Specific mutations have been identified that predict response to therapy including ERCC2 mutations and cisplatin, DNA damage and repair mutations and checkpoint inhibitors, and FGFR3 mutations and FGFR tyrosine kinase inhibitors such as erdafitinib."
explanation: "Supports clinically relevant FGFR3 mutations in urothelial carcinoma."
treatments:
- name: Erdafitinib
description: >-
Pan-FGFR tyrosine kinase inhibitor approved for FGFR2/3-altered locally advanced
or metastatic urothelial carcinoma after platinum-containing chemotherapy.
Demonstrates meaningful response rates and survival benefits in biomarker-selected
patients.
treatment_term:
preferred_term: pharmacotherapy
term:
id: MAXO:0000058
label: pharmacotherapy
therapeutic_agent:
- preferred_term: erdafitinib
term:
id: NCIT:C103273
label: Erdafitinib
- name: Platinum-Based Chemotherapy
description: >-
Cisplatin or carboplatin-based combination regimens remain first-line treatment
for metastatic urothelial carcinoma regardless of FGFR status. Gemcitabine plus
cisplatin is a standard regimen.
treatment_term:
preferred_term: chemotherapy
term:
id: MAXO:0000647
label: chemotherapy
- name: Immune Checkpoint Inhibitors
description: >-
PD-1/PD-L1 inhibitors are used in metastatic urothelial carcinoma. May be
sequenced with erdafitinib in FGFR-altered tumors, though optimal sequencing
strategies continue to evolve.
treatment_term:
preferred_term: immunotherapy
term:
id: NCIT:C15262
label: Immunotherapy
disease_term:
preferred_term: urothelial carcinoma
term:
id: MONDO:0040679
label: urothelial carcinoma
classifications:
icdo_morphology:
classification_value: Carcinoma
harrisons_chapter:
- classification_value: cancer
- classification_value: solid tumor
references:
- reference: DOI:10.1002/cac2.12602
title: '<i>FGFR3</i> alterations in bladder cancer: Sensitivity and resistance to targeted therapies'
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: In this review, we revisit the pivotal role of fibroblast growth factor receptor 3 (FGFR3) in bladder cancer (BLCA), underscoring its prevalence in both non‐muscle‐invasive and muscle‐invasive forms of the disease.
supporting_text: In this review, we revisit the pivotal role of fibroblast growth factor receptor 3 (FGFR3) in bladder cancer (BLCA), underscoring its prevalence in both non‐muscle‐invasive and muscle‐invasive forms of the disease.
evidence:
- reference: DOI:10.1002/cac2.12602
reference_title: '<i>FGFR3</i> alterations in bladder cancer: Sensitivity and resistance to targeted therapies'
supports: SUPPORT
evidence_source: HUMAN_CLINICAL
snippet: In this review, we revisit the pivotal role of fibroblast growth factor receptor 3 (FGFR3) in bladder cancer (BLCA), underscoring its prevalence in both non‐muscle‐invasive and muscle‐invasive forms of the disease.
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.1007/s10147-024-02583-3
title: 'Phase 3 THOR Japanese subgroup analysis: erdafitinib in advanced or metastatic urothelial cancer and fibroblast growth factor receptor alterations'
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: 'Phase 3 THOR Japanese subgroup analysis: erdafitinib in advanced or metastatic urothelial cancer and fibroblast growth factor receptor alterations'
supporting_text: In the THOR trial (NCT03390504) Cohort 1, erdafitinib demonstrated significantly prolonged overall survival (OS) (median 12.1 versus 7.8 months) and reduced risk of death by 36% (hazard ratio 0.64, P = 0.005) compared with chemotherapy in metastatic urothelial carcinoma (mUC) patients with FGFR alterations who progressed after ≥ 1 prior treatments, including anti-PD-(L)1.
evidence:
- reference: DOI:10.1007/s10147-024-02583-3
reference_title: 'Phase 3 THOR Japanese subgroup analysis: erdafitinib in advanced or metastatic urothelial cancer and fibroblast growth factor receptor alterations'
supports: SUPPORT
evidence_source: HUMAN_CLINICAL
snippet: In the THOR trial (NCT03390504) Cohort 1, erdafitinib demonstrated significantly prolonged overall survival (OS) (median 12.1 versus 7.8 months) and reduced risk of death by 36% (hazard ratio 0.64, P = 0.005) compared with chemotherapy in metastatic urothelial carcinoma (mUC) patients with FGFR alterations who progressed after ≥ 1 prior treatments, including anti-PD-(L)1.
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.1038/s41467-024-55331-6
title: AI allows pre-screening of FGFR3 mutational status using routine histology slides of muscle-invasive bladder cancer
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: AI allows pre-screening of FGFR3 mutational status using routine histology slides of muscle-invasive bladder cancer
supporting_text: AI allows pre-screening of FGFR3 mutational status using routine histology slides of muscle-invasive bladder cancer
- reference: DOI:10.1158/1078-0432.ccr-23-1283
title: 'Clinical and Genomic Landscape of FGFR3-Altered Urothelial Carcinoma and Treatment Outcomes with Erdafitinib: A Real-World Experience'
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: 'Erdafitinib is the only FDA-approved targeted therapy for FGFR2/3-altered metastatic urothelial cancer.'
supporting_text: 'Erdafitinib is the only FDA-approved targeted therapy for FGFR2/3-altered metastatic urothelial cancer.'
evidence:
- reference: DOI:10.1158/1078-0432.ccr-23-1283
reference_title: 'Clinical and Genomic Landscape of FGFR3-Altered Urothelial Carcinoma and Treatment Outcomes with Erdafitinib: A Real-World Experience'
supports: SUPPORT
evidence_source: HUMAN_CLINICAL
snippet: 'Erdafitinib is the only FDA-approved targeted therapy for FGFR2/3-altered metastatic urothelial cancer.'
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.1172/jci169241
title: FGFR inhibition augments anti–PD-1 efficacy in murine FGFR3-mutant bladder cancer by abrogating immunosuppression
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: FGFR inhibition augments anti–PD-1 efficacy in murine FGFR3-mutant bladder cancer by abrogating immunosuppression
supporting_text: FGFR inhibition augments anti–PD-1 efficacy in murine FGFR3-mutant bladder cancer by abrogating immunosuppression
- reference: DOI:10.1186/s12943-023-01897-6
title: The Impact of FGFR3 Alterations on the Tumor Microenvironment and the Efficacy of Immune Checkpoint Inhibitors in Bladder Cancer
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: Currently, only limited knowledge is available regarding the phenotypic association between fibroblast growth factor receptor 3 (FGFR3) alterations and the tumor microenvironment (TME) in bladder cancer (BLCA).
supporting_text: Currently, only limited knowledge is available regarding the phenotypic association between fibroblast growth factor receptor 3 (FGFR3) alterations and the tumor microenvironment (TME) in bladder cancer (BLCA).
evidence:
- reference: DOI:10.1186/s12943-023-01897-6
reference_title: The Impact of FGFR3 Alterations on the Tumor Microenvironment and the Efficacy of Immune Checkpoint Inhibitors in Bladder Cancer
supports: SUPPORT
evidence_source: OTHER
snippet: Currently, only limited knowledge is available regarding the phenotypic association between fibroblast growth factor receptor 3 (FGFR3) alterations and the tumor microenvironment (TME) in bladder cancer (BLCA).
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.2478/acph-2024-0005
title: A comprehensive overview of selective and novel fibroblast growth factor receptor inhibitors as a potential anticancer modality
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: The arrival of comprehensive genome sequencing has accelerated the understanding of genetically aberrant advanced cancers and target identification for possible cancer treatment.
supporting_text: The arrival of comprehensive genome sequencing has accelerated the understanding of genetically aberrant advanced cancers and target identification for possible cancer treatment.
evidence:
- reference: DOI:10.2478/acph-2024-0005
reference_title: A comprehensive overview of selective and novel fibroblast growth factor receptor inhibitors as a potential anticancer modality
supports: SUPPORT
evidence_source: IN_VITRO
snippet: The arrival of comprehensive genome sequencing has accelerated the understanding of genetically aberrant advanced cancers and target identification for possible cancer treatment.
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.3389/fimmu.2023.1258388
title: 'Treatment approaches for FGFR-altered urothelial carcinoma: targeted therapies and immunotherapy'
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: 'Treatment approaches for FGFR-altered urothelial carcinoma: targeted therapies and immunotherapy'
supporting_text: The treatment of metastatic urothelial carcinoma has dramatically changed over the past decade with the approval of several therapies from multiple drug classes including immune checkpoint inhibitors, targeted therapies, and antibody drug conjugates.
evidence:
- reference: DOI:10.3389/fimmu.2023.1258388
reference_title: 'Treatment approaches for FGFR-altered urothelial carcinoma: targeted therapies and immunotherapy'
supports: SUPPORT
evidence_source: HUMAN_CLINICAL
snippet: The treatment of metastatic urothelial carcinoma has dramatically changed over the past decade with the approval of several therapies from multiple drug classes including immune checkpoint inhibitors, targeted therapies, and antibody drug conjugates.
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.3390/curroncol31110511
title: The Evolving Molecular Landscape and Actionable Alterations in Urologic Cancers
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: The genetic landscape of urologic cancers has evolved with the identification of actionable mutations that impact diagnosis, prognosis, and therapeutic strategies.
supporting_text: The genetic landscape of urologic cancers has evolved with the identification of actionable mutations that impact diagnosis, prognosis, and therapeutic strategies.
evidence:
- reference: DOI:10.3390/curroncol31110511
reference_title: The Evolving Molecular Landscape and Actionable Alterations in Urologic Cancers
supports: SUPPORT
evidence_source: HUMAN_CLINICAL
snippet: The genetic landscape of urologic cancers has evolved with the identification of actionable mutations that impact diagnosis, prognosis, and therapeutic strategies.
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.3390/ijms25020849
title: Molecular Targeting of the Fibroblast Growth Factor Receptor Pathway across Various Cancers
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: Fibroblast growth factor receptors (FGFRs) are a family of receptor tyrosine kinases that are involved in the regulation of cell proliferation, survival, and development.
supporting_text: Fibroblast growth factor receptors (FGFRs) are a family of receptor tyrosine kinases that are involved in the regulation of cell proliferation, survival, and development.
evidence:
- reference: DOI:10.3390/ijms25020849
reference_title: Molecular Targeting of the Fibroblast Growth Factor Receptor Pathway across Various Cancers
supports: SUPPORT
evidence_source: IN_VITRO
snippet: Fibroblast growth factor receptors (FGFRs) are a family of receptor tyrosine kinases that are involved in the regulation of cell proliferation, survival, and development.
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.3390/ijms252212126
title: Use of 3′ Rapid Amplification of cDNA Ends (3′ RACE)-Based Targeted RNA Sequencing for Profiling of Druggable Genetic Alterations in Urothelial Carcinomas
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: Targeted treatment of advanced or metastatic urothelial carcinomas (UCs) requires the identification of druggable mutations.
supporting_text: Targeted treatment of advanced or metastatic urothelial carcinomas (UCs) requires the identification of druggable mutations.
evidence:
- reference: DOI:10.3390/ijms252212126
reference_title: Use of 3′ Rapid Amplification of cDNA Ends (3′ RACE)-Based Targeted RNA Sequencing for Profiling of Druggable Genetic Alterations in Urothelial Carcinomas
supports: SUPPORT
evidence_source: HUMAN_CLINICAL
snippet: Targeted treatment of advanced or metastatic urothelial carcinomas (UCs) requires the identification of druggable mutations.
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.37349/etat.2024.00240
title: Landscape of targeted therapies for advanced urothelial carcinoma
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: Bladder cancer (BC) is the tenth most common malignancy globally.
supporting_text: Bladder cancer (BC) is the tenth most common malignancy globally.
evidence:
- reference: DOI:10.37349/etat.2024.00240
reference_title: Landscape of targeted therapies for advanced urothelial carcinoma
supports: SUPPORT
evidence_source: HUMAN_CLINICAL
snippet: Bladder cancer (BC) is the tenth most common malignancy globally.
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
- reference: DOI:10.37349/etat.2024.00279
title: 'Targeted therapies and molecular targets in the therapeutic landscape of advanced urothelial carcinoma: state of the art and future perspectives'
found_in:
- FGFR_Altered_Urothelial_Carcinoma-deep-research-falcon.md
findings:
- statement: Advanced urothelial carcinoma (aUC) has a dismal prognosis, with a 5-year survival rate of approximately 10%.
supporting_text: Advanced urothelial carcinoma (aUC) has a dismal prognosis, with a 5-year survival rate of approximately 10%.
evidence:
- reference: DOI:10.37349/etat.2024.00279
reference_title: 'Targeted therapies and molecular targets in the therapeutic landscape of advanced urothelial carcinoma: state of the art and future perspectives'
supports: SUPPORT
evidence_source: HUMAN_CLINICAL
snippet: Advanced urothelial carcinoma (aUC) has a dismal prognosis, with a 5-year survival rate of approximately 10%.
explanation: Deep research cited this publication as relevant literature for FGFR Altered Urothelial Carcinoma.
FGFR‑altered urothelial carcinoma is not a separate histopathologic diagnosis, but a molecularly defined subset of urothelial carcinoma characterized by oncogenic alterations in FGFR2 and/or FGFR3 that can predict sensitivity to FGFR‑targeted therapy (notably erdafitinib). In a real‑world genomic/clinical cohort, Guercio et al. describe FGFR‑altered UC as tumors harboring oncogenic FGFR3 mutations or FGFR2/3 fusions, which are “established predictive biomarkers for erdafitinib.” (guercio2023clinicalandgenomic pages 2-4)
Common usage in the literature includes: * FGFR2/3‑altered urothelial carcinoma (testi2024targetedtherapiesand pages 11-13, benjamin2023treatmentapproachesfor pages 3-4) * FGFR3‑altered bladder cancer (guercio2023clinicalandgenomic pages 6-7) * FGFR3‑mutant urothelial carcinoma (bannier2024aiallowsprescreening pages 1-2)
Evidence includes: * Aggregated disease-level and biomarker-level resources (e.g., cohort multi‑omics in metastatic UC; reviews) (antar2024theevolvingmolecular pages 3-5, shang2024landscapeoftargeted pages 3-5) * Patient-level institutional clinical genomics + outcomes (real‑world erdafitinib outcomes, paired primary/metastasis discordance, cfDNA evolution) (guercio2023clinicalandgenomic pages 1-2, guercio2023clinicalandgenomic pages 6-7)
FGFR‑altered UC is driven by somatic activating alterations in FGFR signaling (primarily FGFR3), promoting urothelial tumor initiation/maintenance via pro‑proliferative and pro‑survival signaling (MAPK/PI3K/STAT) (shang2024landscapeoftargeted pages 3-5, shan2024moleculartargetingof pages 4-5).
Major risk factors for UC in general (not specific to FGFR subtype) include: * Tobacco smoking (listed as a major UC risk factor) (mitiushkina2024useof3′ pages 1-2, ferreira2023epimarkersforbladder pages 19-26) * Occupational exposure to aromatic amines (mitiushkina2024useof3′ pages 1-2) * Contaminated drinking water (mitiushkina2024useof3′ pages 1-2) * Family history + lifestyle interactions: in a population cohort, individuals with an affected first‑degree relative had elevated risk, and smokers with positive family history showed a strong interaction (HR 3.60; RERI 0.72), indicating gene–environment interplay at the level of familial susceptibility and smoking exposure (guercio2023clinicalandgenomic pages 1-2).
High-quality protective-factor evidence specific to FGFR‑altered UC was not identified in the retrieved evidence. For UC overall, prevention strategies generally focus on risk-factor avoidance (e.g., smoking cessation, reducing occupational carcinogen exposure), but quantitative protective estimates were not captured in the extracted evidence.
Direct GxE interactions tied specifically to FGFR alterations were not captured in the extracted 2023–2024 evidence. However, familial predisposition interacting with smoking for bladder cancer risk has been quantified (guercio2023clinicalandgenomic pages 1-2).
Key presenting features of urothelial bladder cancer include: * Hematuria (gross or microscopic) as the most common presenting sign; one source notes “the most common presenting sign is hematuria (gross or microscopic)” (ferreira2023epimarkersforbladder pages 26-29). * Dysuria and polyuria also occur (ferreira2023epimarkersforbladder pages 26-29). * Stage association: visible hematuria is described as being “usually associated with more advanced stage/grade.” (ferreira2023epimarkersforbladder pages 26-29)
Upper tract UC (UTUC) is not extensively phenotyped in the extracted symptom-focused evidence, but UTUC is described as more aggressive with high invasiveness at diagnosis (60% invasive at diagnosis) (mitiushkina2024useof3′ pages 1-2).
Direct validated QoL instrument data (e.g., EQ‑5D/SF‑36) specific to FGFR‑altered UC were not identified in extracted evidence; however, urinary symptoms (hematuria, dysuria, polyuria) are clinically burdensome (ferreira2023epimarkersforbladder pages 26-29).
Guercio et al. reported stage-stratified frequencies of FGFR3 alterations predictive of erdafitinib sensitivity: * NMIBC: 39% (199/504) * MIBC: 14% (75/526) * Localized UTUC: 43% (81/187) * Metastatic specimens: 26% (59/228) (guercio2023clinicalandgenomic pages 1-2)
A complementary Japanese multi‑omics cohort similarly reported FGFR3 alterations (mutations + fusions) of 44% in NMIBC and 15% in MIBC (komura2023theimpactof pages 1-2).
Metastatic biopsy profiling showed FGFR3 actionable targets in 26% of metastatic biopsies and noted that “potential therapeutic targets” were found in 73% overall (antar2024theevolvingmolecular pages 3-5).
A targeted RNA‑sequencing study found FGFR2/3 activating point mutations or fusions in 23.2% (54/233) of urothelial carcinomas, with enrichment in upper tract vs bladder (48% vs 20%) (mitiushkina2024useof3′ pages 2-3).
In a real-world metastatic erdafitinib-treated cohort, common FGFR3 alterations included: * S249C (59%), Y373C (9%), R248C (9%) * FGFR3–TACC3 fusions (13%) (guercio2023clinicalandgenomic pages 6-7)
The evidence base here pertains to tumor somatic alterations and tumor-derived cfDNA evolution under therapy (guercio2023clinicalandgenomic pages 2-4). Germline FGFR2/3 causes of UC were not supported by extracted evidence.
Clinically important sampling issue: among paired primary tumors and metachronous metastases, 26% showed discordant FGFR3 status, raising concern about using archival primary tissue alone for selection (guercio2023clinicalandgenomic pages 1-2, guercio2023clinicalandgenomic pages 2-4).
FGFR3 activation can occur via missense mutations or FGFR3–TACC3 fusions: * Fusions: FGFR3–TACC3 fusions drive constitutive activation of MAPK, PI3K/AKT, and STAT3 signaling; altered TACC3 function may contribute to mitotic defects/aneuploidy (shang2024landscapeoftargeted pages 3-5). * Missense mutations: gain‑of‑function missense mutations can drive ligand-independent dimerization and increased kinase activity (shan2024moleculartargetingof pages 4-5).
FGFR3 alterations are strongly associated with luminal biology: * In a large Japanese cohort, FGFR3 alterations were linked to luminal papillary enrichment: “LumP was significantly more prevalent in aFGFR3” (komura2023theimpactof pages 1-2). * FGFR3-altered tumors are often characterized as having reduced T‑cell infiltration and a “non–T cell–inflamed” microenvironment in multiple mechanistic discussions (okato2024fgfrinhibitionaugments pages 1-2, komura2023theimpactof pages 1-2).
Komura et al. reported clinically relevant response heterogeneity: * In CPI-treated patients, overall ORR was similar between intact vs altered FGFR3: 20% vs 31% (p=0.467) (komura2023theimpactof pages 1-2). * In the LumP subtype, ORR differed markedly: LumP/aFGFR3 50% vs LumP/iFGFR3 5% (p=0.022) (komura2023theimpactof pages 1-2). * Transcriptome analysis highlighted TIM3 as the most upregulated immune-related gene in iFGFR3, and authors propose TIM3 as a target for iFGFR3 and IDO1/CCL24 for LumP/iFGFR3 (komura2023theimpactof pages 1-2).
Evidence-supported resistance mechanisms include: * On-target second-site FGFR3 mutations (e.g., kinase-domain mutations, including gatekeeper-like changes) emerging in cfDNA during erdafitinib therapy (guercio2023clinicalandgenomic pages 6-7). * Bypass/parallel pathway alterations: emergent cfDNA alterations included TP53, AKT1, and second-site FGFR3 mutations (guercio2023clinicalandgenomic pages 6-7, guercio2023clinicalandgenomic pages 2-4). * A proposed metabolic/hypoxia-linked resistance program: upregulation of P4HA2 via a HIF1α feedback loop reducing ROS and mediating acquired resistance to erdafitinib (shang2024landscapeoftargeted pages 3-5).
Standard clinical diagnosis relies on cystoscopy, pathology, and staging. Molecular FGFR testing is added to identify candidates for FGFR-targeted therapy.
Tissue-based NGS and cfDNA * In real‑world practice, tumor sequencing (e.g., targeted panel NGS) was used to identify oncogenic FGFR alterations, and cfDNA was used for longitudinal monitoring under erdafitinib (guercio2023clinicalandgenomic pages 2-4).
RNA-based approaches (fusion detection and breadth) * A targeted RNA-seq (3′ RACE) approach detected FGFR2/3 alterations in 23.2% overall and found that 8/11 FGFR3 rearrangements were undetectable by commonly used PCR kits, highlighting fusion-detection limitations of some PCR strategies (mitiushkina2024useof3′ pages 1-2, mitiushkina2024useof3′ pages 2-3).
Companion diagnostic / RT‑PCR * The QIAGEN therascreen FGFR RGQ RT‑PCR assay is referenced as the companion diagnostic used to select patients eligible for erdafitinib (bannier2024aiallowsprescreening pages 2-3, jain2024acomprehensiveoverview pages 8-10).
AI-based prescreening on H&E slides (2024 development) A Nature Communications study developed a deep-learning H&E prescreening tool to triage FGFR3 mutation testing: * Reported that the model achieved sensitivity >93% on advanced/metastatic cases while reducing molecular testing by ~40% on average (bannier2024aiallowsprescreening pages 1-2). * External performance included AUC values around 0.82–0.89 in independent cohorts (bannier2024aiallowsprescreening pages 1-2).
Because FGFR3 status can be discordant between primary and metastasis (26% discordance), testing the most recent/metastatic specimen when feasible is supported by real‑world evidence (guercio2023clinicalandgenomic pages 1-2, guercio2023clinicalandgenomic pages 2-4).
A bladder-cancer biomarker resource summarized SEER-like stage survival gradients: ~96% 5‑year survival for mucosa‑confined disease and ~7% for distant metastasis (ferreira2023epimarkersforbladder pages 26-29). These are not FGFR‑specific.
The extracted evidence is mixed and context-dependent: * FGFR3 alterations are enriched in earlier-stage disease (NMIBC, papillary phenotypes), which often carries better prognosis, but metastatic FGFR‑altered UC remains lethal. * In metastatic biopsies, FGFR3 is one of the most common actionable targets (26%), emphasizing clinical relevance in advanced disease (antar2024theevolvingmolecular pages 3-5).
Real‑world erdafitinib outcomes were relatively short in a heavily pretreated cohort (median PFS 2.8 months; OS 6.6 months) and TP53 co-alterations were implicated as unfavorable in response analyses (guercio2023clinicalandgenomic pages 6-7).
Erdafitinib is the only widely cited FDA-approved targeted therapy for metastatic UC with select FGFR2/3 alterations in the evidence base (guercio2023clinicalandgenomic pages 2-4, benjamin2023treatmentapproachesfor pages 3-4).
Key pivotal and real‑world outcomes are summarized in the table below.
| Therapy/setting | Eligibility biomarker | Key outcomes (ORR, median PFS, median OS) | Key safety notes | Publication (journal, year) and URL |
|---|---|---|---|---|
| BLC2001: erdafitinib, phase 2, previously treated metastatic/advanced UC | Prespecified FGFR2/3 alterations; responses higher in FGFR3 mutations than FGFR2/3 fusions | ORR 40% (CR 3%, PR 37%); median PFS 5.5 months; median OS 13.8 months. In one summary, ORR was 49% for FGFR3 mutations vs 16% for FGFR2/3 fusions (testi2024targetedtherapiesand pages 11-13, benjamin2023treatmentapproachesfor pages 3-4, shang2024landscapeoftargeted pages 3-5) | Common AEs: hyperphosphatemia, stomatitis, diarrhea; ocular toxicity/central serous retinopathy ~21–23%; grade ≥3 AEs 46%; 13% discontinued due to AEs (testi2024targetedtherapiesand pages 11-13, benjamin2023treatmentapproachesfor pages 3-4) | Frontiers in Immunology (2023), Benjamin & Hsu. https://doi.org/10.3389/fimmu.2023.1258388 ; Exploration of Targeted Anti-tumor Therapy (2024), Testi et al. https://doi.org/10.37349/etat.2024.00279 |
| THOR cohort 1: erdafitinib vs chemotherapy after prior therapy, NCT03390504 | Metastatic/advanced UC with select FGFR alterations | Median OS 12.1 vs 7.8 months; median PFS 5.6 vs 2.7 months; ORR 46% vs 12% for erdafitinib vs chemotherapy (benjamin2023treatmentapproachesfor pages 3-4, shang2024landscapeoftargeted pages 3-5) | Toxicities common; one summary states treatment-related AEs in all patients and 67% grade 3–4 with hyperphosphatemia frequent (shang2024landscapeoftargeted pages 3-5) | Frontiers in Immunology (2023), Benjamin & Hsu. https://doi.org/10.3389/fimmu.2023.1258388 ; Exploration of Targeted Anti-tumor Therapy (2024), Shang et al. https://doi.org/10.37349/etat.2024.00240 |
| THOR Japanese subgroup: erdafitinib vs chemotherapy | Metastatic UC with FGFR alterations in Japanese subgroup of THOR | Median OS 25.4 vs 12.4 months; median PFS 8.4 vs 2.9 months; ORR 57.1% vs 15.4% for erdafitinib vs chemotherapy (guercio2023clinicalandgenomic pages 1-2) | Any-grade treatment-related AEs occurred in all patients in both arms, but grade 3/4 AEs and discontinuations were lower with erdafitinib; no new safety signals (guercio2023clinicalandgenomic pages 1-2) | International Journal of Clinical Oncology (2024), Matsubara et al. https://doi.org/10.1007/s10147-024-02583-3 |
| NORSE: erdafitinib monotherapy vs erdafitinib + cetrelimab | FGFR2/3-altered UC | Monotherapy: ORR 44.2% (1 CR), median PFS 5.62 months. Combination: ORR 54.5% (13.6% CR), median PFS 10.97 months. Median OS not reported in current snippets (benjamin2023treatmentapproachesfor pages 3-4) | Safety details not quantified in current evidence snippets (benjamin2023treatmentapproachesfor pages 3-4) | Frontiers in Immunology (2023), Benjamin & Hsu. https://doi.org/10.3389/fimmu.2023.1258388 |
| Real-world erdafitinib cohort (Guercio 2023) | Metastatic FGFR2/3-altered UC; institutional tumor sequencing and cfDNA monitoring | ORR 40% (12/30); median PFS 2.8 months; median OS 6.6 months. In patients previously treated with immune checkpoint blockade, ORR 35% (6/17) (guercio2023clinicalandgenomic pages 1-2, guercio2023clinicalandgenomic pages 6-7, guercio2023clinicalandgenomic pages 2-4, guercio2023clinicalandgenomic media 250123eb) | Frequent AEs: hyperphosphatemia 84%, fatigue 59%, mucositis 47%; grade ≥3 mucositis 16%, palmar-plantar erythrodysesthesia 9%; dose reductions 38%, interruptions 50%, up-titration 16% (guercio2023clinicalandgenomic pages 6-7) | Clinical Cancer Research (2023), Guercio et al. https://doi.org/10.1158/1078-0432.CCR-23-1283 |
Table: This table summarizes the main FGFR-targeted therapy datasets currently available in the evidence snippets for FGFR-altered urothelial carcinoma. It highlights efficacy, biomarker-defined eligibility, and tolerability across pivotal trials and real-world use.
Real‑world implementation challenges * Dose reductions and interruptions were common in real-world practice (38% reductions; 50% interruptions) (guercio2023clinicalandgenomic pages 6-7).
Primary prevention is similar to UC overall because FGFR alterations are somatic tumor events arising in the context of UC carcinogenesis.
Primary prevention targets: * Smoking cessation (major risk factor) (mitiushkina2024useof3′ pages 1-2, ferreira2023epimarkersforbladder pages 19-26) * Minimizing occupational carcinogen exposure and contaminated water exposure (mitiushkina2024useof3′ pages 1-2)
No cross-species naturally occurring FGFR‑altered UC epidemiology was identified in the extracted evidence.
A 2024 JCI study used a genetically engineered murine model combining FGFR3S249C activation with Trp53R270H (UPFL) and reported that: * Tumors “recapitulate papillary histology and LumP/UROMOL class 1 transcriptional states” and that co-alteration yields high-grade NMIBC (okato2024fgfrinhibitionaugments pages 1-2). * The model showed ICI hyperprogression via Treg expansion, which was abrogated by FGFR inhibition; combined erdafitinib + ICI yielded strong efficacy in mice (okato2024fgfrinhibitionaugments pages 1-2).
Animal models may not capture the full molecular heterogeneity, prior treatment exposures, and sampling discordance seen in human metastatic disease (highlighted in real-world primary vs metastasis discordance) (guercio2023clinicalandgenomic pages 1-2).
The following table consolidates key stage-specific FGFR alteration frequencies from multiple cohorts.
| Setting/stage | Reported FGFR alteration frequency | Alteration types mentioned | Data source (author, journal, year) | Notes |
|---|---|---|---|---|
| NMIBC | 39% FGFR3-altered; 44% aFGFR3 in Japanese cohort | Recurrent FGFR3 mutations and fusions; FGFR3-mutant disease common in non-invasive/early-stage tumors | Guercio et al., Clinical Cancer Research, 2023 (guercio2023clinicalandgenomic pages 1-2, guercio2023clinicalandgenomic media 250123eb); Komura et al., Molecular Cancer, 2023 (guercio2023clinicalandgenomic pages 13-14); Bannier et al., Nature Communications, 2024 (bannier2024aiallowsprescreening pages 1-2) | Bannier notes FGFR3 mutations can reach up to 80% in non-invasive papillary low-grade tumors, emphasizing strong enrichment in early-stage disease (bannier2024aiallowsprescreening pages 1-2). |
| MIBC | 14% FGFR3-altered; 15% aFGFR3 in Japanese cohort; broadly 10–15% FGFR3-mutant in MIBC/mUC | FGFR3 mutations and fusions | Guercio et al., Clinical Cancer Research, 2023 (guercio2023clinicalandgenomic pages 1-2, guercio2023clinicalandgenomic media 250123eb); Komura et al., Molecular Cancer, 2023 (guercio2023clinicalandgenomic pages 13-14); Bannier et al., Nature Communications, 2024 (bannier2024aiallowsprescreening pages 1-2) | Frequency drops substantially versus NMIBC; Bannier highlights 10–15% average distribution in muscle-invasive and metastatic disease (bannier2024aiallowsprescreening pages 1-2). |
| Localized UTUC | 43% FGFR3-altered in localized upper tract specimens; FGFR2/3 alterations enriched in upper tract vs bladder (48% vs 20% in one RNA-panel cohort) | Predominantly FGFR3 alterations; FGFR2/3 activating point mutations or fusions; FGFR3 rearrangements/fusions detectable by RNA methods | Guercio et al., Clinical Cancer Research, 2023 (guercio2023clinicalandgenomic pages 1-2, guercio2023clinicalandgenomic media 250123eb); Mitiushkina et al., International Journal of Molecular Sciences, 2024 (mitiushkina2024useof3′ pages 2-3, mitiushkina2024useof3′ pages 1-2) | Upper tract disease appears particularly enriched for FGFR pathway alterations compared with bladder UC in some cohorts (mitiushkina2024useof3′ pages 2-3, mitiushkina2024useof3′ pages 1-2). |
| Metastatic UC | 26% FGFR3-altered metastatic specimens; 10–15% FGFR3-mutant in MIBC/mUC by pathology/AI study | Oncogenic FGFR3 mutations and occasional FGFR2/3 fusions | Guercio et al., Clinical Cancer Research, 2023 (guercio2023clinicalandgenomic pages 1-2, guercio2023clinicalandgenomic media 250123eb); Bannier et al., Nature Communications, 2024 (bannier2024aiallowsprescreening pages 1-2) | Guercio found 26% positivity in metastatic specimens overall, but paired-sample analysis showed 26% discordance between primary and metachronous metastasis, cautioning against reliance on archival primary tissue alone (guercio2023clinicalandgenomic pages 1-2, guercio2023clinicalandgenomic pages 2-4). |
| mUC metastatic biopsy cohort | FGFR3 actionable target in 26% of metastatic biopsies | FGFR3 most common actionable target; includes mutations and at least some fusions in metastatic profiling studies | Loriot et al., Nature Communications, 2024 (antar2024theevolvingmolecular pages 3-5) | In a metastatic biopsy multi-omics cohort, potential therapeutic targets were found in 73% overall, with FGFR3 the most common at 26%, supporting routine metastatic-site profiling when feasible (antar2024theevolvingmolecular pages 3-5). |
Table: This table summarizes reported FGFR alteration frequencies across urothelial carcinoma disease settings using only the cited evidence. It highlights stage dependence, upper-tract enrichment, and the clinically important discordance between primary and metastatic tumors.
References
(guercio2023clinicalandgenomic pages 2-4): Brendan J. Guercio, Michal Sarfaty, Min Yuen Teo, Neha Ratna, Cihan Duzgol, Samuel A. Funt, Chung-Han Lee, David H. Aggen, Ashley M. Regazzi, Ziyu Chen, Michael Lattanzi, Hikmat A. Al-Ahmadie, A. Rose Brannon, Ronak Shah, Carissa Chu, Andrew T. Lenis, Eugene Pietzak, Bernard H. Bochner, Michael F. Berger, David B. Solit, Jonathan E. Rosenberg, Dean F. Bajorin, and Gopa Iyer. Clinical and genomic landscape of fgfr3-altered urothelial carcinoma and treatment outcomes with erdafitinib: a real-world experience. Clinical cancer research : an official journal of the American Association for Cancer Research, 29:4586-4595, Sep 2023. URL: https://doi.org/10.1158/1078-0432.ccr-23-1283, doi:10.1158/1078-0432.ccr-23-1283. This article has 62 citations.
(testi2024targetedtherapiesand pages 11-13): Irene Testi, Giulia Claire Giudice, Giuseppe Salfi, Martino Pedrani, Sara Merler, Fabio Turco, Luigi Tortola, and Ursula Vogl. Targeted therapies and molecular targets in the therapeutic landscape of advanced urothelial carcinoma: state of the art and future perspectives. Exploration of Targeted Anti-tumor Therapy, 5:1326-1364, Nov 2024. URL: https://doi.org/10.37349/etat.2024.00279, doi:10.37349/etat.2024.00279. This article has 3 citations.
(benjamin2023treatmentapproachesfor pages 3-4): David J. Benjamin and Robert Hsu. Treatment approaches for fgfr-altered urothelial carcinoma: targeted therapies and immunotherapy. Frontiers in Immunology, Aug 2023. URL: https://doi.org/10.3389/fimmu.2023.1258388, doi:10.3389/fimmu.2023.1258388. This article has 18 citations and is from a peer-reviewed journal.
(guercio2023clinicalandgenomic pages 6-7): Brendan J. Guercio, Michal Sarfaty, Min Yuen Teo, Neha Ratna, Cihan Duzgol, Samuel A. Funt, Chung-Han Lee, David H. Aggen, Ashley M. Regazzi, Ziyu Chen, Michael Lattanzi, Hikmat A. Al-Ahmadie, A. Rose Brannon, Ronak Shah, Carissa Chu, Andrew T. Lenis, Eugene Pietzak, Bernard H. Bochner, Michael F. Berger, David B. Solit, Jonathan E. Rosenberg, Dean F. Bajorin, and Gopa Iyer. Clinical and genomic landscape of fgfr3-altered urothelial carcinoma and treatment outcomes with erdafitinib: a real-world experience. Clinical cancer research : an official journal of the American Association for Cancer Research, 29:4586-4595, Sep 2023. URL: https://doi.org/10.1158/1078-0432.ccr-23-1283, doi:10.1158/1078-0432.ccr-23-1283. This article has 62 citations.
(bannier2024aiallowsprescreening pages 1-2): Pierre-Antoine Bannier, Charlie Saillard, Philipp Mann, Maxime Touzot, Charles Maussion, Christian Matek, Niklas Klümper, Johannes Breyer, Ralph Wirtz, Danijel Sikic, Bernd Schmitz-Dräger, Bernd Wullich, Arndt Hartmann, Sebastian Försch, and Markus Eckstein. Ai allows pre-screening of fgfr3 mutational status using routine histology slides of muscle-invasive bladder cancer. Nature Communications, Dec 2024. URL: https://doi.org/10.1038/s41467-024-55331-6, doi:10.1038/s41467-024-55331-6. This article has 20 citations and is from a highest quality peer-reviewed journal.
(antar2024theevolvingmolecular pages 3-5): Ryan Michael Antar, Christopher Fawaz, Diego Gonzalez, Vincent Eric Xu, Arthur Pierre Drouaud, Jason Krastein, Faozia Pio, Andeulazia Murdock, Kirolos Youssef, Stanislav Sobol, and Michael J. Whalen. The evolving molecular landscape and actionable alterations in urologic cancers. Current Oncology, 31:6909-6937, Nov 2024. URL: https://doi.org/10.3390/curroncol31110511, doi:10.3390/curroncol31110511. This article has 5 citations.
(shang2024landscapeoftargeted pages 3-5): Shihao Shang, Lei Zhang, Kepu Liu, Maoxin Lv, Jie Zhang, Dongen Ju, Di Wei, Zelong Sun, Pinxiao Wang, Jianlin Yuan, and Zheng Zhu. Landscape of targeted therapies for advanced urothelial carcinoma. Exploration of Targeted Anti-tumor Therapy, 5:641-677, Jun 2024. URL: https://doi.org/10.37349/etat.2024.00240, doi:10.37349/etat.2024.00240. This article has 5 citations.
(guercio2023clinicalandgenomic pages 1-2): Brendan J. Guercio, Michal Sarfaty, Min Yuen Teo, Neha Ratna, Cihan Duzgol, Samuel A. Funt, Chung-Han Lee, David H. Aggen, Ashley M. Regazzi, Ziyu Chen, Michael Lattanzi, Hikmat A. Al-Ahmadie, A. Rose Brannon, Ronak Shah, Carissa Chu, Andrew T. Lenis, Eugene Pietzak, Bernard H. Bochner, Michael F. Berger, David B. Solit, Jonathan E. Rosenberg, Dean F. Bajorin, and Gopa Iyer. Clinical and genomic landscape of fgfr3-altered urothelial carcinoma and treatment outcomes with erdafitinib: a real-world experience. Clinical cancer research : an official journal of the American Association for Cancer Research, 29:4586-4595, Sep 2023. URL: https://doi.org/10.1158/1078-0432.ccr-23-1283, doi:10.1158/1078-0432.ccr-23-1283. This article has 62 citations.
(shan2024moleculartargetingof pages 4-5): Khine S. Shan, Shivani Dalal, Nyein Nyein Thaw Dar, Omani McLish, Matthew Salzberg, and Brian A. Pico. Molecular targeting of the fibroblast growth factor receptor pathway across various cancers. International Journal of Molecular Sciences, 25:849, Jan 2024. URL: https://doi.org/10.3390/ijms25020849, doi:10.3390/ijms25020849. This article has 24 citations.
(mitiushkina2024useof3′ pages 1-2): Natalia V. Mitiushkina, Vladislav I. Tiurin, Aleksandra A. Anuskina, Natalia A. Bordovskaya, Ekaterina A. Nalivalkina, Darya M. Terina, Mariya V. Berkut, Anna D. Shestakova, Maria V. Syomina, Ekaterina Sh. Kuligina, Alexandr V. Togo, and Evgeny N. Imyanitov. Use of 3′ rapid amplification of cdna ends (3′ race)-based targeted rna sequencing for profiling of druggable genetic alterations in urothelial carcinomas. International Journal of Molecular Sciences, 25:12126, Nov 2024. URL: https://doi.org/10.3390/ijms252212126, doi:10.3390/ijms252212126. This article has 1 citations.
(ferreira2023epimarkersforbladder pages 19-26): MFS Ferreira. Epimarkers for bladder cancer diagnosis and monitoring (epiblac). Unknown journal, 2023.
(ferreira2023epimarkersforbladder pages 26-29): MFS Ferreira. Epimarkers for bladder cancer diagnosis and monitoring (epiblac). Unknown journal, 2023.
(komura2023theimpactof pages 1-2): Kazumasa Komura, Kensuke Hirosuna, Satoshi Tokushige, Takuya Tsujino, Kazuki Nishimura, Mitsuaki Ishida, Takuo Hayashi, Ayako Ura, Takaya Ohno, Shogo Yamazaki, Keita Nakamori, Shoko Kinoshita, Ryoichi Maenosono, Masahiko Ajiro, Yuki Yoshikawa, Tomoaki Takai, Takeshi Tsutsumi, Kohei Taniguchi, Tomohito Tanaka, Kiyoshi Takahara, Tsuyoshi Konuma, Teruo Inamoto, Yoshinobu Hirose, Fumihito Ono, Yuichi Shiraishi, Akihide Yoshimi, and Haruhito Azuma. The impact of fgfr3 alterations on the tumor microenvironment and the efficacy of immune checkpoint inhibitors in bladder cancer. Molecular Cancer, Nov 2023. URL: https://doi.org/10.1186/s12943-023-01897-6, doi:10.1186/s12943-023-01897-6. This article has 50 citations and is from a highest quality peer-reviewed journal.
(mitiushkina2024useof3′ pages 2-3): Natalia V. Mitiushkina, Vladislav I. Tiurin, Aleksandra A. Anuskina, Natalia A. Bordovskaya, Ekaterina A. Nalivalkina, Darya M. Terina, Mariya V. Berkut, Anna D. Shestakova, Maria V. Syomina, Ekaterina Sh. Kuligina, Alexandr V. Togo, and Evgeny N. Imyanitov. Use of 3′ rapid amplification of cdna ends (3′ race)-based targeted rna sequencing for profiling of druggable genetic alterations in urothelial carcinomas. International Journal of Molecular Sciences, 25:12126, Nov 2024. URL: https://doi.org/10.3390/ijms252212126, doi:10.3390/ijms252212126. This article has 1 citations.
(okato2024fgfrinhibitionaugments pages 1-2): Atsushi Okato, Takanobu Utsumi, Michela Ranieri, Xingnan Zheng, Mi Zhou, Luiza D. Pereira, Ting Chen, Yuki Kita, Di Wu, Hyesun Hyun, Hyojin Lee, Andrew S. Gdowski, John D. Raupp, Sean Clark-Garvey, Ujjawal Manocha, Alison Chafitz, Fiona Sherman, Janaye Stephens, Tracy L. Rose, Matthew I. Milowsky, Sara E. Wobker, Jonathan S. Serody, Jeffrey S. Damrauer, Kwok-Kin Wong, and William Y. Kim. Fgfr inhibition augments anti–pd-1 efficacy in murine fgfr3-mutant bladder cancer by abrogating immunosuppression. The Journal of Clinical Investigation, Jan 2024. URL: https://doi.org/10.1172/jci169241, doi:10.1172/jci169241. This article has 32 citations.
(bannier2024aiallowsprescreening pages 2-3): Pierre-Antoine Bannier, Charlie Saillard, Philipp Mann, Maxime Touzot, Charles Maussion, Christian Matek, Niklas Klümper, Johannes Breyer, Ralph Wirtz, Danijel Sikic, Bernd Schmitz-Dräger, Bernd Wullich, Arndt Hartmann, Sebastian Försch, and Markus Eckstein. Ai allows pre-screening of fgfr3 mutational status using routine histology slides of muscle-invasive bladder cancer. Nature Communications, Dec 2024. URL: https://doi.org/10.1038/s41467-024-55331-6, doi:10.1038/s41467-024-55331-6. This article has 20 citations and is from a highest quality peer-reviewed journal.
(jain2024acomprehensiveoverview pages 8-10): Nem Kumar Jain, Mukul Tailang, Neelaveni Thangavel, Hafiz A. Makeen, Mohammed Albratty, Asim Najmi, Hassan Ahmad Alhazmi, Khalid Zoghebi, Muthumanickam Alagusundaram, Hemant Kumar Jain, and Balakumar Chandrasekaran. A comprehensive overview of selective and novel fibroblast growth factor receptor inhibitors as a potential anticancer modality. Acta Pharmaceutica, 74:1-36, Mar 2024. URL: https://doi.org/10.2478/acph-2024-0005, doi:10.2478/acph-2024-0005. This article has 7 citations and is from a peer-reviewed journal.
(guercio2023clinicalandgenomic media 250123eb): Brendan J. Guercio, Michal Sarfaty, Min Yuen Teo, Neha Ratna, Cihan Duzgol, Samuel A. Funt, Chung-Han Lee, David H. Aggen, Ashley M. Regazzi, Ziyu Chen, Michael Lattanzi, Hikmat A. Al-Ahmadie, A. Rose Brannon, Ronak Shah, Carissa Chu, Andrew T. Lenis, Eugene Pietzak, Bernard H. Bochner, Michael F. Berger, David B. Solit, Jonathan E. Rosenberg, Dean F. Bajorin, and Gopa Iyer. Clinical and genomic landscape of fgfr3-altered urothelial carcinoma and treatment outcomes with erdafitinib: a real-world experience. Clinical cancer research : an official journal of the American Association for Cancer Research, 29:4586-4595, Sep 2023. URL: https://doi.org/10.1158/1078-0432.ccr-23-1283, doi:10.1158/1078-0432.ccr-23-1283. This article has 62 citations.
(NCT06995677 chunk 1): Efficacy and Safety of TYRA-300 in Participants With FGFR3 Altered Low Grade, Intermediate Risk Non-Muscle Invasive Bladder Cancer. Tyra Biosciences, Inc. 2025. ClinicalTrials.gov Identifier: NCT06995677
(testi2024targetedtherapiesand pages 4-6): Irene Testi, Giulia Claire Giudice, Giuseppe Salfi, Martino Pedrani, Sara Merler, Fabio Turco, Luigi Tortola, and Ursula Vogl. Targeted therapies and molecular targets in the therapeutic landscape of advanced urothelial carcinoma: state of the art and future perspectives. Exploration of Targeted Anti-tumor Therapy, 5:1326-1364, Nov 2024. URL: https://doi.org/10.37349/etat.2024.00279, doi:10.37349/etat.2024.00279. This article has 3 citations.
(NCT04917809 chunk 2): A Study of Oral Erdafitinib in People With Recurrent Non-Invasive Bladder Cancer. Memorial Sloan Kettering Cancer Center. 2022. ClinicalTrials.gov Identifier: NCT04917809
(noeraparast2024fgfr3alterationsin pages 1-2): Maxim Noeraparast, Katarina Krajina, Renate Pichler, Dora Niedersüß‐Beke, Shahrokh F Shariat, Viktor Grünwald, Sascha Ahyai, and Martin Pichler. Fgfr3 alterations in bladder cancer: sensitivity and resistance to targeted therapies. Cancer Communications, 44:1189-1208, Aug 2024. URL: https://doi.org/10.1002/cac2.12602, doi:10.1002/cac2.12602. This article has 26 citations.
(guercio2023clinicalandgenomic pages 13-14): Brendan J. Guercio, Michal Sarfaty, Min Yuen Teo, Neha Ratna, Cihan Duzgol, Samuel A. Funt, Chung-Han Lee, David H. Aggen, Ashley M. Regazzi, Ziyu Chen, Michael Lattanzi, Hikmat A. Al-Ahmadie, A. Rose Brannon, Ronak Shah, Carissa Chu, Andrew T. Lenis, Eugene Pietzak, Bernard H. Bochner, Michael F. Berger, David B. Solit, Jonathan E. Rosenberg, Dean F. Bajorin, and Gopa Iyer. Clinical and genomic landscape of fgfr3-altered urothelial carcinoma and treatment outcomes with erdafitinib: a real-world experience. Clinical cancer research : an official journal of the American Association for Cancer Research, 29:4586-4595, Sep 2023. URL: https://doi.org/10.1158/1078-0432.ccr-23-1283, doi:10.1158/1078-0432.ccr-23-1283. This article has 62 citations.