ArticleThe Journal of clinical endocrinology and metabolism2025
Clinical Activity of Selpercatinib in RET-mutant Pheochromocytoma.
Article in The Journal of clinical endocrinology and metabolism, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03157128 (A Phase 1/2 Study of Oral Selpercatinib), which is not on this map. Cited by 10 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 1/2 Study of Oral Selpercatinib (LOXO-292) in Patients With Advanced Solid Tumors, Including RET Fusion-Positive Solid Tumors, Medullary Thyroid Cancer, and Other Tumors With RET Activation (LIBRETTO-001)
Who cites it
10 citing papers in PubMed, 12 citations in OpenAlex.
- Systemic management of adrenal malignancies: adrenocortical carcinoma and pheochromocytoma/paraganglioma.Endocrine connections · 2026Article
- Article
- Efficacy and safety of pralsetinib in multiple endocrine neoplasia type 2-associated pheochromocytoma: a case report.Therapeutic advances in endocrinology and metabolism · 2026Article
- Covalent organic framework-based electrochemical nanosensing: an emerging paradigm for early cancer diagnosis and longitudinal surveillance.Journal of nanobiotechnology · 2026Review
- Update on Systemic Therapies for Metastatic/Unresectable Pheochromocytomas and Paragangliomas and Future Directions.Cancers · 2025Review
- Approach to the Patient With Metastatic Pheochromocytoma and Paraganglioma.The Journal of clinical endocrinology and metabolism · 2025Article
- Inhibitory Effects of Vandetanib on Catecholamine Synthesis in Rat Pheochromocytoma PC12 Cells.International journal of molecular sciences · 2025Article
- The Perioperative Biochemical and Clinical Considerations of Pheochromocytoma Management.International journal of molecular sciences · 2025Review
- MEN2: surgical precision in the era of precision medicine.Endocrine-related cancer · 2025Review
- Adverse event profiles of selpercatinib: a real-world pharmacovigilance analysis based on FAERS database.BMC cancer · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 7 institutions in 3 countries.
Funding
Abstract
contextActivating 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).
methodsWe describe the first 6 pheochromocytoma cases treated with selpercatinib in the LIBRETTO-001 study.
resultsOf 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.
conclusionThese 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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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.