Evidence map›Paper›PMID 41801283›Full record

ArticleEndocrine2026

Efficacy of RET inhibitors in the management of advanced, RET mutated, pheochromocytoma. Pooled analysis of published cases with the addition of 2 new cases.

Valentina Cremaschi, Marta Laganà, Antonella Turla, Francesco Dondi, Benedetta Trevisan, Mara Giacchè, Salvatore Grisanti, Valentina Zilioli, Francesco Bertagna, Alfredo Berruti and 1 more

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In one paragraph

Article in Endocrine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

The trial behind it

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Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors.

Valentina Cremaschi *Medical Oncology Unit, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy.
Marta Laganà *Medical Oncology Unit, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy. martagana@gmail.com.
Antonella TurlaMedical Oncology Unit, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy.
Francesco DondiNuclear Medicine, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy.
Benedetta TrevisanMedical Oncology Unit, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy.
Mara GiacchèDepartment of Medical and Surgical Specialties, Division of Clinical Genetics, ASST Spedali Civili, Brescia, Italy.
Salvatore GrisantiMedical Oncology Unit, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy.
Valentina ZilioliNuclear Medicine, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy.
Francesco BertagnaNuclear Medicine, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy.
Alfredo BerrutiMedical Oncology Unit, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy.
Deborah CosentiniMedical Oncology Unit, Department of Medical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia at ASST Spedali Civili, Brescia, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeSelective RET inhibitors are approved for treatment of RET-mutant lung and thyroid cancers. RET mutation is a druggable molecular driver of malignant pheochromocytomas/paragangliomas (PPGLs), but few data have been published regarding the efficacy of RET inhibitors in this clinical setting.

methodsWe performed a pooled analysis of RET-mutated PPGLs treated with RET-inhibitors, including both literature published case reports and patients treated at our Institution.

resultsNine patients with advanced pheochromocytoma were collected (7 published cases and 2 patients followed in our department). Eight patients had metastatic disease and 6 of them were pretreated. Eight patients received selpercatinib and one patient received pralsetinib. All patients obtained clinical benefit, 5 of them reached a partial response, 2 a durable stable disease and 2 a complete response. Median progression free survival ranged between 5.5 – 56.3 months. Urinary catecholamine and metanephrine levels improved or normalized in 7 cases. The treatment was well tolerated but in 2 patients a dose reduction was needed, due to G3 adverse events.

conclusionRET inhibitors are efficacious in patients affected by PPGL with RET mutation or fusion. Based on these findings, these drugs represent a promising strategy and these data support the development of prospective clinical trials in this setting.

Indexed as

Adrenal Gland NeoplasmsAntineoplastic AgentsPheochromocytomaProtein Kinase InhibitorsProto-Oncogene Proteins c-retPyrazolesPyridinesPyrimidinesAdultAgedFemaleHumansMaleMiddle AgedMutationPyrimidinonesAntineoplastic AgentspralsetinibProtein Kinase InhibitorsProto-Oncogene Proteins c-retPyrazolesPyridinesPyrimidinesPyrimidinonesRET protein, humanselpercatinibPheochromocytomaPralsetinibRET-mutationSelpercatinibTargeted therapy

Identifiers

PMID41801283

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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.