A classification prognostic score to predict OS in stage IV well-differentiated neuroendocrine tumors

Pusceddu S, Barretta F, Trama A, Botta L, Milione M, Buzzoni R, De Braud F, Mazzaferro V, Pastorino U, Seregni E, Mariani L, Gatta G, Di Bartolomeo M, Femia D, Prinzi N, Coppa J, Panzuto F, Antonuzzo L, Bajetta E, Brizzi MP, Campana D, Catena L, Comber H, Dwane F, Fazio N, Faggiano A, Giuffrida D, Henau K, Ibrahim T, Marconcini R, Massironi S, Žakelj MP, Spada F, Tafuto S, Van Eycken E, Van der Zwan JM, Žagar T, Giacomelli L, Miceli R, NEPscore Working Group
Publicatiedatum
Naam tijdschrift
Endocrine-related cancer
No validated prognostic tool is available for predicting overall survival (OS) of patients with well-differentiated neuroendocrine tumors (WDNETs). This study, conducted in three independent cohorts of patients from five different European countries, aimed to develop and validate a classification prognostic score for OS in patients with stage IV WDNETs. We retrospectively collected data on 1387 patients: (i) patients treated at the Istituto Nazionale Tumori (Milan, Italy; n = 515); (ii) European cohort of rare NET patients included in the European RARECAREnet database (n = 457); (iii) Italian multicentric cohort of pancreatic NET (pNETs) patients treated at 24 Italian institutions (n = 415). The score was developed using data from patients included in cohort (i) (training set); external validation was performed by applying the score to the data of the two independent cohorts (ii) and (iii) evaluating both calibration and discriminative ability (Harrell C statistic). We used data on age, primary tumor site, metastasis (synchronous vs metachronous), Ki-67, functional status and primary surgery to build the score, which was developed for classifying patients into three groups with differential 10-year OS: (I) favorable risk group: 10-year OS ≥70%; (II) intermediate risk group: 30% ≤ 10-year OS < 70%; (III) poor risk group: 10-year OS <30%. The Harrell C statistic was 0.661 in the training set, and 0.626 and 0.601 in the RARECAREnet and Italian multicentric validation sets, respectively. In conclusion, based on the analysis of three 'field-practice' cohorts collected in different settings, we defined and validated a prognostic score to classify patients into three groups with different long-term prognoses.