Ottaviano, Margaret (2021) Thymic Epithelial Tumors: Model of rare tumors for the identification of new biomarkers and novel therapeutic opportunities. [Tesi di dottorato]

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In this thesis, I discuss the identification of new insights and the evaluation of novel treatment opportunities in thymic epithelial tumors, which are widely recognized as rare cancers. Rare cancers are a heterogeneous group of diseases, which share similar problems: uncertainty of diagnosis, lack of therapies, poor research opportunities, difficulties in clinical trials, lack of expertise and of centres of reference. Despite their low incidence, not greater than 6 cases per 100000 in habitants, in Europe a considerable fraction of all cancers is represented by rare cancers (24%),making their clinical relevance far from being insignificant. Indeed, most of rare malignancies require complex surgical treatment, thus a multidisciplinary approach is essential and treatment should be provided in centres of expertise and/or in networks including expert centres. This research focuses on a peculiar model of rare cancer, thymic epithelial tumors which are characterized by: the platinum-sensitivity in the majority of cases; the lack of effective treatments in case of platinum-resistance cases; the lack of novel, reproducible and minim-invasive biomarkers and the dramatic survival variations depending on the oncological expertise. Actually, an opportunity to improve knowledge and outcomes of rare tumors and reduce disparities, is provided by the centralization of their diagnosis and management in reference centres and the cooperation with recognized expert Networks for these diseases. Thus, the main aim of this thesis is to describe the most updated scientific findings concerning diagnosis, molecular characterization and treatment of thymic epithelial tumors, in relation to my research activity in both expert reference centre and national and international expert networks. Thymic epithelial tumors are rare thoracic cancers, characterized by unpredictable oncological outcomes and unique association with immunological dysregulations. Here, I report the last researches that I carried out, focused on: the identification of new biomarkers using the emerging approach of liquid biopsy; the evaluation of novel systemic treatments in progressing/relapsing platinum-resistant tumors and the immunological characterization of patients with both autoimmunity and immunodeficiency. The work I have presented, therefore, has shown that, despite the several problems due to the rarity of these malignancies, the achievement of new molecular insights and novel treatment opportunities is feasible. The centralization of diagnosis and management in expert reference centers, as well as the cooperation with international dedicated networks is mandatory.

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