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Published on: September 25, 2018
PD-L1 - inhibitors in neuroendocrine neoplasia: Results from a real-life study
Burcin Özdirik1, Henning Jann1, Philip Bischoff2
1Charité - University Medicine Berlin, Department of Hepatology & Gastroenterology, Campus Virchow Klinikum and Charité Campus Mitte.
Abstract:
Immune check-point inhibitors (ICIs) have changed our view on how to treat cancer. Despite their approval in treatment of many different cancers, efficacy of immune check-point inhibitors (ICI) in neuroendocrine neoplasia is limited and poorly understood. Established treatment options of neuroendocrine tumors (NET) and neuroendocrine carcinomas (NECs) are based on surgery, tumor-targeted medical treatments, Peptide Receptor Radionuclide Therapy (PRRT), and locoregional therapies. However, in many patients these treatments lose efficacy over time, and novel therapies are urgently needed. We report on 8 patients diagnosed with neuroendocrine neoplasms (NEN) that were treated with ICI (pembrolizumab, avelumab, nivolumab plus ipilimumab) as salvage therapy. In this cohort, we observed tumor response with partial remission in 3 patients and stable disease in 1 patient. Four patients showed progressive disease. Of note, responses were observed both in PD-L1 positive and PD-L1 negative patients. Here, we discuss clinical courses of these patients in the context of available literature to highlight limitations and drawbacks currently preventing the use of ICI in routine management of patients with NEN.
Insights
Immune check-point inhibitors (ICIs) show limited efficacy in neuroendocrine neoplasms (NEN). This study on 8 patients found partial remission in 3, but 4 had progressive disease, highlighting the need for novel therapies.
Area of Science:
- Oncology
- Immunotherapy
Background:
- Immune check-point inhibitors (ICIs) have revolutionized cancer treatment but show limited efficacy in neuroendocrine neoplasms (NEN).
- Established treatments for neuroendocrine tumors (NET) and neuroendocrine carcinomas (NECs) often lose efficacy over time, necessitating novel therapeutic strategies.
Purpose of the Study:
- To evaluate the efficacy and safety of immune check-point inhibitors (ICIs) as salvage therapy in patients with neuroendocrine neoplasms (NEN).
Main Methods:
- Retrospective analysis of 8 neuroendocrine neoplasm (NEN) patients treated with ICIs (pembrolizumab, avelumab, nivolumab plus ipilimumab) as salvage therapy.
- Tumor response assessment including partial remission, stable disease, and progressive disease.
- Analysis of PD-L1 expression in relation to treatment response.
Main Results:
- Tumor response observed in 4 out of 8 patients (3 partial remission, 1 stable disease).
- Four patients experienced progressive disease.
- Treatment responses were noted in both PD-L1 positive and PD-L1 negative patients.
Conclusions:
- Immune check-point inhibitors (ICIs) demonstrate a limited but present role as salvage therapy in neuroendocrine neoplasms (NEN).
- Further research is needed to understand the mechanisms of ICI resistance and identify predictive biomarkers for improved patient selection.
- Limitations and drawbacks currently hinder the routine use of ICIs in NEN management.

