PhD Defense: Neuroendocrine Neoplasia; Tailoring Treatment and Prognosis

Neuroendocrine neoplasms are cancers that arise from various parts of the body. They can be divided in relatively indolent neuroendocrine tumours (NET) and relatively aggressive neuroendocrine carcinoma (NEC). 

The first part of this thesis describes tumours of the small intestine. These are tumours that have the ability to cause fibrotic complications in the abdomen and in the heart. We found that a previously established nomogram to predict survival was unable to accurately predict survival but was able to differentiate between risk groups. Further, we saw that removing the primary tumour when metastatic disease was already present provided survival benefit and possibly less complications of fibrosis in the gut. Also, we identified a biomarker that was useful in the prediction and detection of cardiac complications of the neuroendocrine tumour, and described a very large cohort of patients with this complication.

Another part where NET may arise is the pulmonary tract. In this thesis we describe the disease course of these tumours that exist in a sporadic context or in the context of specific syndromes, namely the multiple endocrine neoplasia syndrome and the diffuse idiopathic pulmonary neuroendocrine cell hyperplasia, and found that the sporadic kind has a worse survival than the other two.
Finally, we describe different treatment strategies in neuroendocrine carcinoma. We identified that postoperative radiotherapy does not always provide survival benefit in patients with Merkel cell carcinoma (MCC), a NEC of the skin, and that immunotherapy for MCC showed favourable responses and survival, similar to results from clinical trials.

Lastly, we performed a study in NEC that investigated the effect of a new treatment combination, and found that it had similar survival results as conventional therapy, without the serious side effect of hair loss.