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A Syngeneic Mouse Model of Metastatic Renal Cell Carcinoma for Quantitative and Longitudinal Assessment of Preclinical Therapies
Published on: April 12, 2017
Targeted agents in metastatic Xp11 translocation/TFE3 gene fusion renal cell carcinoma (RCC): a report from the
G G Malouf1, P Camparo2, S Oudard3
1Department of Medicine, Institut Gustave Roussy, Villejuif.
Background:
Xp11 translocation renal cell carcinoma (RCC) is an RCC subtype affecting 15% of RCC patients <45 years. We analyzed the benefit of targeted therapy [vascular endothelial growth factor receptor (VEGFR)-targeted agents and/or mammalian target of rapamycin (mTOR) inhibitors] in these patients.
Patients And Methods:
Patients with Xp11 translocation/TFE3 fusion gene metastatic RCC who had received targeted therapy were identified. Nuclear TFE3 positivity was confirmed by reviewing pathology slides. Responses according to RECIST criteria, progression-free survival (PFS), and overall survival (OS) were analyzed.
Results:
Overall, 53 patients were identified; 23 had metastatic disease, and of these 21 had received targeted therapy (median age 34 years). Seven patients achieved an objective response. In first line, median PFS was 8.2 months [95% confidence interval (CI) 2.6-14.7 months] for sunitinib (n = 11) versus 2 months (95% CI 0.8-3.3 months) for cytokines (n = 9) (log-rank P = 0.003). Results for further treatment (second, third, or fourth line) were as follows: all three patients receiving sunitinib had a partial response (median PFS 11 months). Seven of eight patients receiving sorafenib had stable disease (median PFS 6 months). One patient receiving mTOR inhibitors had a partial response and six patients had stable disease. Median OS was 27 months with a 19 months median follow-up.
Conclusion:
In Xp11 translocation RCC, targeted therapy achieved objective responses and prolonged PFS similar to those reported for clear-cell RCC.
Insights
Targeted therapy, including vascular endothelial growth factor receptor (VEGFR) agents and mammalian target of rapamycin (mTOR) inhibitors, shows promise for Xp11 translocation renal cell carcinoma (RCC). This treatment achieved objective responses and extended progression-free survival in patients with this rare RCC subtype.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- Xp11 translocation renal cell carcinoma (RCC) is a distinct subtype affecting younger patients (<45 years).
- This subtype represents approximately 15% of RCC cases in this demographic.
- Understanding treatment efficacy in Xp11 translocation RCC is crucial due to its unique genetic drivers.
Purpose of the Study:
- To evaluate the efficacy of targeted therapies in patients with Xp11 translocation/TFE3 fusion gene metastatic RCC.
- To assess objective response rates, progression-free survival (PFS), and overall survival (OS) with VEGFR inhibitors and/or mTOR inhibitors.
- To compare treatment outcomes with those reported for clear-cell RCC.
Main Methods:
- Retrospective analysis of metastatic Xp11 translocation RCC patients who received targeted therapy.
- Confirmation of TFE3 fusion gene by nuclear TFE3 positivity on pathology slides.
- Evaluation of response using RECIST criteria, and analysis of PFS and OS.
Main Results:
- Twenty-one patients with metastatic Xp11 translocation RCC received targeted therapy (median age 34 years).
- First-line sunitinib showed a median PFS of 8.2 months versus 2 months for cytokines (P=0.003).
- Objective responses were observed with sunitinib, sorafenib, and mTOR inhibitors, with a median OS of 27 months.
Conclusions:
- Targeted therapy, including VEGFR inhibitors and mTOR inhibitors, demonstrates efficacy in Xp11 translocation RCC.
- Objective responses and prolonged PFS were achieved, comparable to clear-cell RCC.
- These findings support the use of targeted agents in managing this specific RCC subtype.
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