Journal Article

Targeted agents in metastatic Xp11 translocation/TFE3 gene fusion renal cell carcinoma (RCC): a report from the Juvenile RCC Network

G. G. Malouf, P. Camparo, S. Oudard, G. Schleiermacher, C. Theodore, A. Rustine, J. Dutcher, B. Billemont, O. Rixe, E. Bompas, A. Guillot, L. Boccon-Gibod, J. Couturier, V. Molinié and B. Escudier

in Annals of Oncology

Published on behalf of European Society for Medical Oncology

Volume 21, issue 9, pages 1834-1838
Published in print September 2010 | ISSN: 0923-7534
Published online February 2010 | e-ISSN: 1569-8041 | DOI:
Targeted agents in metastatic Xp11 translocation/TFE3 gene fusion renal cell carcinoma (RCC): a report from the Juvenile RCC Network

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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.

Keywords: interferon; pediatric renal cell carcinoma; sunitinib; targeted agents; TFE3; translocation renal cell carcinoma; Xp11.2

Journal Article.  2534 words.  Illustrated.

Subjects: Medical Oncology

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