Reference Detail

Ref Type

Journal Article

PMID

(38661071)

Authors

Deschler-Baier B, Konda B, Massarelli E, Hu MI, Wirth LJ, Xu X, Wright J, Clifton-Bligh RJ

Title

Clinical Activity of Selpercatinib in RET-mutant Pheochromocytoma.

Journal	Vol	Issue	Date	Pages	Journal Short Title
The Journal of clinical endocrinology and metabolism			2024 Apr 25		J Clin Endocrinol Metab

URL

Abstract Text

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 anti-tumor activity in diverse RET-activated solid tumors in the LIBRETTO-001 study (NCT03157128).We describe the first six pheochromocytoma cases treated with selpercatinib in the LIBRETTO-001 study.Of the six patients (one sporadic and five reported as part of MEN2 syndromes) in this case report, four had a partial response/complete response and two 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.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.

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Molecular Profile	Treatment Approach

Gene Name	Source	Synonyms	Protein Domains	Gene Description	Gene Role

Therapy Name	Drugs	Efficacy Evidence	Clinical Trials

Drug Name	Trade Name	Synonyms	Drug Classes	Drug Description

Gene	Variant	Impact	Protein Effect	Variant Description	Associated with drug Resistance

Molecular Profile	Indication/Tumor Type	Response Type	Therapy Name	Approval Status	Evidence Type	Efficacy Evidence	References
RET C618S	malignant pheochromocytoma	predicted - sensitive	Selpercatinib	Case Reports/Case Series	Actionable	In a Phase I/II trial (LIBRETTO-001), Retevmo (selpercatinib) treatment resulted in a partial response lasting 3.8 months in a patient with metastatic pheochromocytoma harboring RET C618S (PMID: 38661071; NCT03157128).	38661071
RET C634R	malignant pheochromocytoma	predicted - sensitive	Selpercatinib	Case Reports/Case Series	Actionable	In a Phase I/II trial (LIBRETTO-001), Retevmo (selpercatinib) treatment resulted in stable disease lasting 9.4 months in a patient with metastatic pheochromocytoma harboring germline RET C634R and resulted in a partial response with treatment lasting 28.4 months in a second patient with metastatic pheochromocytoma harboring germline RET C634R (PMID: 38661071; NCT03157128).	38661071
RET C634F	malignant pheochromocytoma	predicted - sensitive	Selpercatinib	Case Reports/Case Series	Actionable	In a Phase I/II trial (LIBRETTO-001), Retevmo (selpercatinib) treatment resulted in a partial response with treatment lasting 37 months in a patient with metastatic pheochromocytoma harboring germline RET C634F (PMID: 38661071; NCT03157128).	38661071
RET M918T	malignant pheochromocytoma	predicted - sensitive	Selpercatinib	Case Reports/Case Series	Actionable	In a Phase I/II trial (LIBRETTO-001), Retevmo (selpercatinib) treatment resulted in stable disease with progression-free survival for at least 56.3 months in a patient with metastatic pheochromocytoma harboring somatic RET M918T and resulted in a complete response by IRC assessment and partial response by investigator assessment in a second patient with concomitant pheochromocytoma and medullary thyroid carcinoma harboring germline RET M918T (PMID: 38661071;NCT03157128).	38661071