Neuroendocrine neoplasms most commonly arise from the gastroenteropancreatic (GEP) system. The WHO classification of digestive system tumors describes four main types: Neuroendocrine tumor (NET) grades 1-3 and neuroendocrine carcinoma (NEC). NECs have an aggressive biology and are often diagnosed in an advanced stage associated with poor prognosis. First-line palliative chemotherapy is commonly carboplatin or cisplatin in combination with etoposide, while in second-line capecitabine-temozolomide, FOLFIRI or FOLFOX regimens are used. Few prospective studies have been done and data on efficacy is scarce. We conducted a retrospective study of patients with GEP-NEC treated with FOLFIRINOX, evaluating response to treatment and survival.
Patients diagnosed with GEP-NEC at three different centers and treated with FOLFIRINOX were identified and included in the study. Baseline demographics were collected at start of FOLFIRINOX. The Response Evaluation Criteria in Solid Tumors (RECIST v1.1) criteria were used to assess the treatment response at computed tomography (CT).
Thirty-seven patients between 2014 and 2020 were identified and included in the study. Median age was 53 years and female/male ratio was 1:1. Most of the patients were in WHO performance status 0 or 1 (86%). The most common primary tumor sites were colon (30%), pancreas (27%), oesophagus (10%)and rectum (10%). The median Ki67 was 80% (range 22%, 100%). Out of 37 patients treated, 8 (22%) patients received FOLFIRINOX as first-line treatment, 21 (57%) patients as second line treatment and 8 patients as third-line treatment or later. Overall response rate (ORR) (all lines) was 46% (17/37); i.e. complete response 0% (0/37), partial response 46% (17/37), stable disease 22% (8/37) and progressive disease 22% (8/37). Median overall survival (mOS) was 17,8 months (CI: 11,4 – 23,3). Median progression free survival from first course of FOLFIRINOX was 5,4 months (CI: 3,5 – 6,9).
FOLFIRINOX is an active regimen in the treatment of GEP-NEC and may be considered in the treatment of advanced disease. Prospective randomized trials are needed to compare efficacy among different chemotherapy regimens.
Clinical trial identification
Legal entity responsible for the study
Oslo University Hospital.
Has not received any funding.
All authors have declared no conflicts of interest.