Precision Oncology in Rare Endocrine and Neuroendocrine Neoplasms - Experiences and Challenges of the CCCMunichLMU Molecular Tumor Board

Sep 23, 2026

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Comprehensive genomic profiling (CGP) is increasingly available for patients with rare cancers, yet evidence on outcomes and clinical benefits remains limited. This retrospective cohort study analyzes CGP results and real-world clinical data for patients with neuroendocrine tumors, neuroendocrine carcinomas, adrenocortical carcinomas, pheochromocytomas, and carcinoids discussed by the CCCMunichLMU Molecular Tumor Board between 2017 and 2023, evaluating molecular findings and treatment decision pathways.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages10

Reading Time25 min

AccessFree to download

Abstract

Background

Objective

Patients and Methods

Results

Conclusions

Key Points

Molecular alteration spectrum

Treatment recommendations and outcomes

Introduction

Definition and classification of NENs

Subgroups and clinical context

Key treatment considerations

Chemotherapy, immunotherapy, and targeted options

Role of CGP in treatment decisions

Targeted Oncology (2025) 20:715–724 [https://doi.org/10.1007/s1](https://doi.org/10.1007/s1) 1523-025-01152-6 Precision Oncology in Rare Endocrine and Neuroendocrine Neoplasms: Experiences and Challenges ofthe CCCMunichLMU Molecular Tumor Board Klara Dorman1,2 · Christoph J. Auernhammer3,11 · Christine Spitzweg3,11 · Ralf Schmidmaier3,12 · Svenja Nölting3,14 · Matthias Kroiss3,12 · Martin Reincke3,12 · Christian Schulz4,11 · Martin Angele5,11 · Jens Werner5,11 · Christine Schmid‑Tannwald6,11,12 · Josefine Rauch7,11 · Mathias Zacherl8,11 · Thomas Knösel2,9,11 · Jörg Kumbrink2,9 · Andreas Jung2,9 · Frederick Klauschen2,9 · Amanda Tufman13 · Danmei Zhang1,2 · Lena Weiss1 · Stefan Boeck1,2,10 · Michael von Bergwelt‑Baildon1,2,11 · Volker Heinemann1,2 · C. Benedikt Westphalen1,2,11 · Kathrin Heinrich1,2,11 Accepted: 4 May 2025 / Published online: 30 June 2025 © The Author(s) 2025, corrected publication 2025 Abstract Background Comprehensive genomic profiling (CGP) has become more generally accessible to patients with rare cancer, but data on the results and benefits are limited. Objective Our objective was to gain a real-world understanding of the molecular landscape and targeted treatment options in neuroendocrine tumors, neuroendocrine carcinomas, adrenocortical carcinomas, pheochromocytomas, and carcinoids. Patients and Methods In this retrospective cohort study, we analyzed CGP results and clinical data from patients with neuroendocrine tumors, neuroendocrine carcinomas, adrenocortical carcinomas, pheochromocytomas, and carcinoids who were discussed in the CCCMunichLMU Molecular Tumor Board (MTB) between May 2017 and April 2023. Results In total, 104 patients with endocrine and neuroendocrine neoplasms were discussed in the MTB. CGP was technically successful in 99 patients. The most commonly mutated genes were TP53 (29.3%), RB1 (11.1%), and KRAS (10.1%) . The highest overall prevalence of pathogenic alterations was detected in neuroendocrine carcinomas (76.9%) and carcinoids (83.3%), and the lowest prevalence of pathogenic alterations was seen in adrenocortical carcinoma (37.5%). Of the 99 patients with successful CGP, 35 received a treatment recommendation from the MTB based on the CGP results. Of these, ten patients ultimately received the recommended treatment. Of the ten treated patients, four experienced a longer progression-free survival under the targeted treatment than under their previous treatment. Conclusions One-third of patients with rare endocrine and neuroendocrine neoplasms who underwent CGP had a druggable alteration and received a treatment recommendation from the MTB. However, only 28.6% of these patients were treated accordingly. Our experience highlights the unmet medical need for targeted treatment options in patients with rare cancers. 1 Introduction Neuroendocrine neoplasms (NENs) are a rare and heterogeneous subset of tumors that originate from cells with aneuroendocrine phenotype and can occur at many different anatomical sites [1]. The International Agency for Research on Cancer and World Health Organization expert consensus proposal recommends a uniform classification for NENs. Depending on their differentiation, NENs can be divided Extended author information available on the last page of the article into neuroendocrine tumors (NETs, well-differentiated) and neuroendocrine carcinomas (NECs, poorly differentiated) . Furthermore, NETs should be graded as low grade, intermediate grade, and high grade (G1, G2, and G3, respectively), depending on mitotic count, ki67, and presence of necrosis, whereas NECs are always high grade [2] . Pheochromocytomas are another subgroup of NETs that originate in the adrenal medulla and can produce catecholamines [3] . The most frequent anatomical site for NENs is the digestive system, followed by the lung [4]. Along with the profound heterogeneity of NENs, oncogenic molecular drivers and epigenetic profiles also vary greatly [5, 6] . Key P

FAQs

What was the study objective regarding CGP in rare neuroendocrine and endocrine cancers?

To provide a real-world understanding of the molecular landscape and targeted treatment options in neuroendocrine tumors, neuroendocrine carcinomas, adrenocortical carcinomas, pheochromocytomas, and carcinoids.

How many patients were included, and how successful was CGP?

A total of 104 patients were discussed in the Molecular Tumor Board, and CGP was technically successful in 99 patients.

What proportion of patients received a MTB-recommended treatment, and what outcomes were observed?

Of 99 patients with successful CGP, 35 received a treatment recommendation and 10 ultimately received the recommended treatment. Among these treated patients, four experienced longer progression-free survival than with prior therapy.

Sep 23, 2026

Uploaded by Stanford