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Zielgerichtete Therapie biliärer Tumoren

Targeted treatment of billary tract tumors

  • Leitthema
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Die Onkologie Aims and scope

Zusammenfassung

Hintergrund

Biliäre Tumoren gehören mit jährlich 8000 Neuerkrankungen in Deutschland zu den seltenen malignen Neoplasien. Die Prognose für Patient:innen mit biliären Tumorerkrankungen ist weiterhin sehr schlecht. Nach langjähriger Stagnation ist es jedoch in den vergangenen Jahren zu einer Erweiterung der therapeutischen Optionen in der Systemtherapie gekommen: Zum einen ist die Immunonkologie Teil des Standard of Care in der Erstlinie geworden, zum anderen haben Studien in genetisch selektionierten Subgruppen das Potenzial zielgerichteter Strategien aufgezeigt.

Ziel

In dieser Arbeit geben die Autoren einen Überblick über die zielgerichtete Systemtherapie bei biliären Tumoren.

Material und Methode

Diese Arbeit basiert auf einer selektiven Literaturrecherche in der Datenbank PubMed zum Thema genetische Alterationen und zielgerichtete Therapien bei biliären Tumoren.

Ergebnisse

In bis zu 40 % der biliären Tumoren können genetische Alterationen gefunden werden, für die heute schon molekulare Therapien zur Verfügung stehen. Bislang liegt vorwiegend jedoch lediglich Evidenz aus kleinen einarmigen Phase-II Studien vor, was der niedrigen Inzidenz einzelner genetischer Alterationen in dieser seltenen Tumorentität geschuldet ist.

Schlussfolgerung

Bei biliären Tumoren sollte frühzeitig eine umfassende genetische Analyse der Tumoren durchgeführt werden. Für viele genetische Alterationen besteht heute noch keine Zulassung für die zielgerichtete Therapie, sodass diese Patient:innen in einem molekularen Tumorboard vorgestellt werden sollten. Zudem sind die jeweiligen genetischen Subgruppen sehr klein, sodass es eminent wichtig ist, dass geeignete Patient:innen konsequent in Therapiestudien und molekulare Register eingeschlossen werden.

Abstract

Background

Biliary tract tumors are rare malignant neoplasms with 8000 new cases per year in Germany. The prognosis for patients with biliary tumors is still very poor. However, after many years of stagnation, the number of options regarding systemic therapy has been increasing in recent years: on the one hand, immuno-oncology has become part of first-line standard of care, and on the other hand, studies in genetically selected subgroups have shown the potential of targeted strategies.

Objective

The aim of this paper is to provide an overview of targeted systemic therapy in biliary tumors.

Materials and methods

This work is based on a selective literature search of the PubMed database on genetic alterations and targeted therapies in biliary tumors.

Results

Genetic alterations can be found in up to 40% of cases of patients with biliary tumors, for which molecular therapies are already available today. However, many studies in the small genetic subgroups of a rare tumor entity have been performed as single-arm phase II studies without control arms.

Conclusion

Comprehensive genetic analysis of biliary tumors should be performed at an early stage. For many genetic alterations, there is no approval for targeted therapy today, so these patients should be presented in a molecular tumor board. Moreover, the respective genetic subgroups are very small, so that it is paramount that suitable patients are consistently included in therapy studies and molecular registries.

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Authors and Affiliations

  1. Klinik für Gastroenterologie, Hepatologie, Infektiologie und Endokrinologie, Medizinische Hochschule Hannover, Carl-Neubersgstr. 1, 30625, Hannover, Deutschland

    Arndt Vogel,  Christoph Gerdes &  Anna Saborowski

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  1. Arndt Vogel
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Correspondence to Arndt Vogel.

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Interessenkonflikt

A. Vogel: Consultancy and advisory role: AstraZeneca, Amgen, BeiGene, Böhringer Mannheim, BMS, BTG, Daichi-Sankyo, EISAI, Incyte, Ipsen, MSD, PierreFabre, Roche, Servier, Sirtex, Tahio, Terumo. A. Saborowski: Honoraria from BMS, Roche, Servier, Ipsen, Lilly, AstraZeneca, MSD, Travel support: Ipsen, Servier, Pierre-Fabre, MSD, Adboard: Eisai, MSD. C. Gerdes gibt an, dass kein Interessenkonflikt besteht.

Für diesen Beitrag wurden von den Autor/-innen keine Studien an Menschen oder Tieren durchgeführt. Für die aufgeführten Studien gelten die jeweils dort angegebenen ethischen Richtlinien.

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Vogel, A., Gerdes, C. & Saborowski, A. Zielgerichtete Therapie biliärer Tumoren. Onkologie 29, 793–800 (2023). https://doi.org/10.1007/s00761-023-01386-x

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