Efficacy of FOLFOX in Patients with Aggressive Pancreatic Neuroendocrine Tumors After Prior Capecitabine/Temozolomide

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Abstract
Background 5‐fluorouracil/leucovorin/oxaliplatin (FOLFOX) has activity in pancreatic NETs (pNETs), but its use is limited, partly due to toxicities. pNETs can often become aggressive over time. We evaluated the efficacy of FOLFOX in patients with aggressive pNETs who had progressed after capecitabine/temozolomide among other treatments. Methods Retrospective study of all patients with well‐differentiated metastatic pNETs treated at an academic cancer center between 1/2008 and 6/2019 who received FOLFOX and had received cap/tem in the past. Primary endpoint was objective response rate. Results 31 patients met criteria. 25 received FOLFOX and 6 received FOLFOX with bev. Patients were heavily pre‐treated, having received a median of 3 prior lines of systemic therapy prior to FOLFOX (range 1 – 8). 3 (9.7%) patients had G1 tumors, 16 (51.6%) had G2, 6 (19.4%) had G3, and grade was unspecified in 6 (19.4%). 14 (45.2%) exhibited a best response of PR per RECIST 1.1 criteria, 15 (48.4%) SD, and 2 (6.4%) PD; ORR was 45.2% and DCR was 93.5%. Median PFS was 6 months (95% CI 5.0‐7.0) and median OS was 16 months from onset of study treatment (95% CI 11.3 – 20.7); 67 months from date of diagnosis (95% CI 49.8 – 84.2). Median duration of treatment was 3 months, and median duration of response was 2 months. Toxicity profile was consistent with known adverse events associated with this regimen. Conclusions FOLFOX is active in aggressive, heavily pretreated pNETs who have progressed on prior cap/tem chemotherapy; response durations are relatively short. Implications for Practice FOLFOX chemotherapy has robust activity in patients with aggressively progressive, heavily pretreated pancreatic NETs, a setting where few, if any, other options are likely to be effective. Durations of response, however, are relatively short, and new treatments are urgently needed for patients with aggressive transformation of pancreatic NETs.