Biliary tract cancer (BTC) is a rare malignancy with increasing incidence and mortality over the past two decades. Despite its unique anatomical and molecular features, BTC is typically diagnosed at advanced stages, thereby limiting treatment options and resulting in poor prognosis. Recent advances in molecular profiling and immunotherapy have revolutionized the BTC management, illustrating its molecular and immunologic underpinnings. Immune checkpoint inhibitors such as durvalumab and pembrolizumab, when combined with gemcitabine/ cisplatin, provide considerable survival benefits, with phase 3 trials such as TOPAZ-1 and KEYNOTE-966 demonstrating improved overall and progression-free survivals with immune checkpoint inhibitors compared to the conventional gemcitabine/cisplatin therapy. Therapies targeting FGFR2 fusions, IDH1 mutations, and HER2 overexpression have further expanded the therapeutic landscape, offering precision medicine opportunities for patients with actionable mutations. However, challenges persist owing to the rarity of BTC and inherent limitations of small cohort studies. Future research should focus on large, multicenter, prospective trials, establish new treatment paradigms, and expand therapeutic applications. In addition, integrating next-generation sequencing into routine practice will enhance molecular-based treatment approaches. This review explores the latest advancements in BTC immunotherapy and targeted therapies, emphasizing their clinical applicability in improving patient outcomes.

Biliary tract cancer (BTC) is a rare malignancy with increasing incidence and mortality over the past two decades. Despite its unique anatomical and molecular features, BTC is typically diagnosed at advanced stages, thereby limiting treatment options and resulting in poor prognosis. Recent advances in molecular profiling and immunotherapy have revolutionized the BTC management, illustrating its molecular and immunologic underpinnings. Immune checkpoint inhibitors such as durvalumab and pembrolizumab, when combined with gemcitabine/ cisplatin, provide considerable survival benefits, with phase 3 trials such as TOPAZ-1 and KEYNOTE-966 demonstrating improved overall and progression-free survivals with immune checkpoint inhibitors compared to the conventional gemcitabine/cisplatin therapy. Therapies targeting FGFR2 fusions, IDH1 mutations, and HER2 overexpression have further expanded the therapeutic landscape, offering precision medicine opportunities for patients with actionable mutations. However, challenges persist owing to the rarity of BTC and inherent limitations of small cohort studies. Future research should focus on large, multicenter, prospective trials, establish new treatment paradigms, and expand therapeutic applications. In addition, integrating next-generation sequencing into routine practice will enhance molecular-based treatment approaches. This review explores the latest advancements in BTC immunotherapy and targeted therapies, emphasizing their clinical applicability in improving patient outcomes.

Biliary tract cancer (BTC) is a rare malignancy with increasing incidence and mortality over the past two decades. Despite its unique anatomical and molecular features, BTC is typically diagnosed at advanced stages, thereby limiting treatment options and resulting in poor prognosis. Recent advances in molecular profiling and immunotherapy have revolutionized the BTC management, illustrating its molecular and immunologic underpinnings. Immune checkpoint inhibitors such as durvalumab and pembrolizumab, when combined with gemcitabine/ cisplatin, provide considerable survival benefits, with phase 3 trials such as TOPAZ-1 and KEYNOTE-966 demonstrating improved overall and progression-free survivals with immune checkpoint inhibitors compared to the conventional gemcitabine/cisplatin therapy. Therapies targeting FGFR2 fusions, IDH1 mutations, and HER2 overexpression have further expanded the therapeutic landscape, offering precision medicine opportunities for patients with actionable mutations. However, challenges persist owing to the rarity of BTC and inherent limitations of small cohort studies. Future research should focus on large, multicenter, prospective trials, establish new treatment paradigms, and expand therapeutic applications. In addition, integrating next-generation sequencing into routine practice will enhance molecular-based treatment approaches. This review explores the latest advancements in BTC immunotherapy and targeted therapies, emphasizing their clinical applicability in improving patient outcomes.

Background/Aims: Endoscopic ultrasound-guided fine-needle aspiration/biopsy (EUS-FNA/B) is essential in diagnosing solid pancreatic lesions (SPLs), but without rapid on-site evaluation (ROSE), a repeat EUS-FNA/B is crucial for clarifying an inconclusive diagnosis. We aimed to evaluate factors associated with improved diagnostic performance of repeat EUS-FNA/B for initially inconclusive SPL diagnoses without ROSE. Methods: Of 5,894 patients subjected to EUS-FNA/B, 237 (4.0%) with an initially inconclusive diagnosis of SPLs were retrospectively enrolled from five tertiary medical centers between January 2016 and June 2021. Diagnostic performance and procedural factors of EUS-FNA/B were analyzed. Results: The diagnostic accuracies of first and repeat EUS-FNA/B were 96.2% and 67.6%, respectively. Of 237 patients with an inconclusive diagnosis from initial EUS-FNA/B, 150were pathologically diagnosed after repeat EUS-FNA/B. In multivariate analysis of repeat EUS-FNA/B, tumor location (body/tail vs head: odds ratio [OR], 3.74; 95% confidence inter-val [CI], 1.48 to 9.46), number of needle passes (≥4 vs ≤3: OR, 4.80; 95% CI, 1.44 to 15.99),needle type (FNB vs FNA: OR, 3.26; 95% CI, 1.44 to 7.36), needle size (22 gauge vs 19/20 gauge: OR, 2.35; 95% CI, 1.19 to 4.62), and suction method (suction vs others: OR, 5.19;95% CI, 1.30 to 20.75) were associated with a significantly improved diagnostic performance. Conclusions Repeat EUS-FNA/B is essential for patients with an inconclusive EUS-FNA/B without ROSE. To improve the diagnostic performance of repeated EUS-FNA/B, it is recom-mended that 22-gauge FNB needles, ≥4 needle passes, and suction methods are used.

Purpose: Hypoxaemia is a significant adverse event during endoscopic retrograde cholangiopancreatography (ERCP) under monitored anaesthesia care (MAC); however, no model has been developed to predict hypoxaemia. We aimed to develop and compare logistic regression (LR) and machine learning (ML) models to predict hypoxaemia during ERCP under MAC. Materials and Methods: We collected patient data from our institutional ERCP database. The study population was randomly divided into training and test sets (7:3). Models were fit to training data and evaluated on unseen test data. The training set was further split into k-fold (k=5) for tuning hyperparameters, such as feature selection and early stopping. Models were trained over k loops; the i-th fold was set aside as a validation set in the i-th loop. Model performance was measured using area under the curve (AUC). Results: We identified 6114 cases of ERCP under MAC, with a total hypoxaemia rate of 5.9%. The LR model was established by combining eight variables and had a test AUC of 0.693. The ML and LR models were evaluated on 30 independent data splits. The average test AUC for LR was 0.7230, which improved to 0.7336 by adding eight more variables with an l 1 regularisation-based selection technique and ensembling the LRs and gradient boosting algorithm (GBM). The high-risk group was discriminated using the GBM ensemble model, with a sensitivity and specificity of 63.6% and 72.2%, respectively. Conclusion We established GBM ensemble model and LR model for risk prediction, which demonstrated good potential for preventing hypoxaemia during ERCP under MAC.

Malignant hilar biliary obstruction (MHBO) frequently accompanies cholestasis and cholangitis, and requires biliary stent placement. To prevent stent occlusion and prolong survival, local ablation therapy can be considered adjunctive to stent placement. Intraductal radiofrequency ablation (ID-RFA) is a recently developed local therapy for malignant biliary obstruction that can be easily performed employing endoscopic retrograde cholangiography. The use of ID-RFA to treat MHBO (as distinct from distal biliary obstruction) was suggested to be associated with severe adverse events. However, recent comparative studies have shown that ID-RFA is feasible and safe, and acceptably efficacious, in patients with advanced MHBO; newer temperature-controlled ID-RFA devices may enhance safety further. Regularly repeated ID-RFA with stent exchange affords better survival than stenting alone. However, the optimal ID-RFA strategy for MHBO remains inconclusive given the lack of data. Further large-scale clinical trials are needed.

no abstract available.