Background: The National Comprehensive Cancer Network (NCCN) guidelines recommend chemotherapy for patients with inoperable biliary tract cancers (BTC), as well as patients following resection of BTC with lymph node metastasis (N1)/positive margins (R1). We sought to define overall adherence, as well as long-term outcomes, with the NCCN guidelines for BTC using the National Cancer Database (NCDB).
Methods: A total of 176,536 patients diagnosed with BTC at a hospital participating in the NCDB between 2004 and 2015 were identified.
Results: Among all patients, 63% of patients received medical therapy (chemotherapy or best supportive care), 11% underwent surgical palliation, and 26% underwent curative-intent surgery. According to the NCCN guidelines, 86% (n = 152,245) of patients were eligible for chemotherapy, yet, only 42.2% (n = 64,615) received chemotherapy. Factors associated with a lower adherence with NCCN guidelines included patient age (> 65 years: OR = 1.02), ethnicity (Black: OR = 1.14, Hispanic: OR = 1.21, Asian: OR = 1.24), and insurance status (non-private: OR = 1.45, all p < 0.001). A smaller subset of patients was either recommended chemotherapy but refused (n = 9269, 10.6%) or had medical factors that contraindicated chemotherapy (n = 8275, 9.4%). On multivariable analysis, adjusting for clinical and tumor-specific factors, adherence with NCCN guidelines was associated with a survival benefit for patients receiving medical therapies (HR = 0.74) or undergoing curative-intent surgery (HR = 0.73, both p < 0.001).
Conclusion: Less than half of patients with BTC received systemic chemotherapy in adherence with NCCN guidelines. While a subset of patients had contraindications or refused chemotherapy, other factors such as insurance status and ethnicity were associated with adherence. Adherence with chemotherapy guidelines may influence long-term outcomes.
Keywords: Biliary tract cancers; National Cancer Database; National Comprehensive Cancer Network guidelines.