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Perkins J , Boland P , Cohen SJ , Olszanski AJ , Zhou Y , Engstrom P , Astsaturov I
Successful Imatinib Therapy for Neuroendocrine Carcinoma With Activating KIT Mutation: A Case Study
J Natl Compr Canc Netw. 2014 Jun;12(6) :847-52
PMID: 24925195 PMCID: PMC4112647
AbstractNeuroendocrine tumors (NET) and gastrointestinal stromal tumors (GIST) are believed to originate from the cells of Cajal that are randomly dispersed along the aerodigestive tract. Despite their distinct morphologic appearance, NET and GIST may share oncogenic mechanisms. Often presenting in the metastatic setting, treatment options for patients with NET are limited. This case report presents a patient with refractory metastatic NET that did not respond conventional chemotherapy. The patient was treated with a KIF11 inhibitor in a phase I clinical trial and experienced a prolonged and clinically meaningful partial response. On progression at 20 months, the patient's tumor was sequenced to reveal a KIT exon 11 mutation. Institution of imatinib therapy achieved a rapid and sustained antitumor effect with profound clinical benefit. Despite previously reported KIT expression in NET, this is the first documented case of an activating KIT mutation in NET and of successful treatment with both a KIF11 inhibitor and imatinib, each of which was elucidated through molecular profiling of the patient's tumor. Imatinib may be a valuable therapy in NET harboring activating KIT mutations.
Notes1540-1413 Perkins, James Boland, Patrick Cohen, Steven J Olszanski, Anthony J Zhou, Yan Engstrom, Paul Astsaturov, Igor Journal Article United States J Natl Compr Canc Netw. 2014 Jun;12(6):847-52.