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Clinical Activity of Selpercatinib in RET-mutant Pheochromocytoma
Barbara Deschler-Baier1, Bhavana Konda2, Erminia Massarelli3
1Comprehensive Cancer Center, University Hospital Würzburg, 97080 Würzburg, Germany.
Context:
Activating RET alterations have been reported in a variety of solid tumors, including pheochromocytoma where they occur both sporadically and as part of familial multiple endocrine neoplasia type 2 (MEN2) syndromes. Selpercatinib is a first-in-class, highly selective, and potent small molecule RET kinase inhibitor that has demonstrated marked and durable antitumor activity in diverse RET-activated solid tumors in the LIBRETTO-001 study (NCT03157128).
Methods:
We describe the first 6 pheochromocytoma cases treated with selpercatinib in the LIBRETTO-001 study.
Results:
Of the 6 patients (1 sporadic and 5 reported as part of MEN2 syndromes) in this case report, 4 had a partial response/complete response and 2 had stable disease per independent review committee. Treatment duration ranged from 9.2 months to more than 56.4 months. The safety profile of treatment was consistent with selpercatinib in other indications.
Conclusion:
These data support selpercatinib as an effective therapy against RET-mutant pheochromocytoma, adding to the diversity of RET-activated tumor types that may benefit from targeted RET inhibition.
Insights
Selpercatinib effectively treats RET-mutant pheochromocytoma in patients with sporadic or Multiple Endocrine Neoplasia type 2 (MEN2) syndromes. This RET kinase inhibitor shows significant antitumor activity and durable responses in these rare tumors.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- Activating RET alterations are found in various solid tumors, including pheochromocytoma, associated with sporadic cases and Multiple Endocrine Neoplasia type 2 (MEN2) syndromes.
- Selpercatinib is a selective RET kinase inhibitor demonstrating efficacy in RET-altered solid tumors within the LIBRETTO-001 study.
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