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Abstract Details

Clinical Response to Pazopanib in a Patient with Endolymphatic Sac Tumor Not Associated with von Hippel-Lindau Syndrome
Neuro-oncology
Neuro-oncology Posters (7:00 AM-5:00 PM)
023
We present the case of a sporadic ELST, previously minimally responsive to bevacizumab, treated with pazopanib, a multi-kinase inhibitor and antiangiogenic, with good clinical and radiologic response. 
Endolymphatic sac tumors (ELSTs) are rare, locally invasive, vascular tumors of the temporal bone. These lesions are associated with von Hippel-Lindau syndrome but may arise sporadically. Early surgical intervention is recommended to prevent permanent neurologic deficits; however, many ELSTs are unresectable or are subtotally resected due to neurovascular compromise. Chemotherapeutic salvage therapy in trials of neoplasms of associated syndromes has targeted angiogenesis with variable response. 
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The sites of action of pazopanib may in part explain our patient’s improved response to this therapy. In particular, disruption of tyrosine kinase signaling cascades rather than scavenging VEGF-A – the mechanism of bevacizumab – may provide longer-term reduction in tumor burden. Additionally, there may be decreased development of resistance to this therapy. The authors acknowledge that this is a single case report and that no clinical trial has yet been performed to assess the response of ELSTs to pazopanib therapy. Given the rare nature of this condition, a prospective clinical trial is likely not feasible. There remains a dearth of available systemic therapies for vHL syndrome, but as more trials are conducted, the frontiers of treatment options for vHL that may extend to ESLT will continue to shift. However, cases such as our patient may demonstrate the utility of novel antiangiogenics in the treatment of these rare neoplasms, particularly when the tumor is unresectable or necessitates subtotal resection.

Authors/Disclosures
Thomas A. Nelson, MD (University of California, San Francisco)
PRESENTER
Dr. Nelson has nothing to disclose.
No disclosure on file
No disclosure on file
Jeremy Rudnick, MD Dr. Rudnick has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Novocure.
No disclosure on file
Jethro Hu, MD (Cedars-Sinai Medical Center) Dr. Hu has nothing to disclose.