Utility of Transpapillary Biopsy and Endoscopic Ultrasound-Guided Tissue Acquisition for Comprehensive Genome Profiling of Unresectable Biliary Tract Cancer

Simple Summary There are only a few reports on comprehensive genome profiling (CGP) analysis using transpapillary biopsy (TPB) and endoscopic ultrasound-guided tissue acquisition (EUS-TA) specimens from primary lesions in biliary tract cancers (BTCs). This study compared TPB and EUS-TA for their suitability in CGP analysis of unresectable BTC. We used the OncoGuide NCC Oncopanel System (NCCOP) and FoundationOne CDx (F1CDx) as criteria for CGP analysis. Among 78 patients (35 TPB, 43 EUS-TA) EUS-TA showed significantly higher suitability for NCCOP analysis (34.9% vs. 8.6%, p = 0.007), whereas F1CDx analysis suitability was 0% in both groups. EUS-TA was identified as an independent factor contributing to NCCOP analysis suitability. EUS-TA specimens were more suitable for analysis in mass lesions (43.8% vs. 9.1%, p = 0.065) and less suitable in perihilar cholangiocarcinoma (0% vs. 41.7%, p = 0.077), whereas TPB showed potential for analysis in papillary-type lesions and peroral cholangioscopy-assisted biopsies. Overall, EUS-TA is preferable for CGP analysis in unresectable BTC, with TPB potentially serving as a complementary method.Abstract Tissue sampling in biliary tract cancer (BTC) is generally performed through transpapillary biopsy (TPB) or endoscopic ultrasound-guided tissue acquisition (EUS-TA). For the first time, we compared the suitability of specimens obtained using TPB and EUS-TA to determine the optimal tissue-sampling method for comprehensive genome profiling (CGP) analysis in patients with unresectable BTC (UR-BTC). Pathology precheck criteria for CGP analysis comprised the OncoGuide NCC Oncopanel System (NCCOP) and FoundationOne CDx (F1CDx). Seventy-eight patients with UR-BTC (35 TPB and 43 EUS-TA) were included. The NCCOP analysis suitability achievement rate was higher in EUS-TA specimens than in TPB specimens (34.9% vs. 8.6%, p = 0.007), whereas that of F1CDx was 0% in both groups. EUS-TA was identified as an independent factor that contributed to the suitability of the NCCOP analysis. The suitability of the NCCOP analysis of EUS-TA specimens showed a tendency to be higher for mass lesions (43.8% vs. 9.1%, p = 0.065), especially for target size >= 18.5 mm, and lower for perihilar cholangiocarcinoma (0% vs. 41.7%, p = 0.077). In TPB, papillary-type lesions (66.7% vs. 3.2%, p = 0.016) and peroral cholangioscopy-assisted biopsies (50.0% vs. 3.3%, p = 0.029) showed better potential for successful NCCOP analysis. EUS-TA is suitable for NCCOP analysis in UR-BTC and may be partially complemented by TPB.