Background: Hereditary Hemorrhagic Telangiectasia (HHT) is a rare autosomal dominant vascular disorder causing systemic arteriovenous malformations (AVMs). Hepatocellular carcinoma (HCC) is a common malignancy, and liver transplantation (LT) serves as a curative option for advanced HCC meeting specific criteria. However, the coexistence of HHT and HCC is extremely rare, and HHT-related hepatic vascular abnormalities combined with the risk of AVM recurrence after LT pose unique management challenges. Few cases of LT for HHT-associated HCC have been reported worldwide; herein, we present one successful case. Case description: A 62-year-old Chinese man was diagnosed with HHT type 2 (harboring an ACVRL1 gene mutation) and Barcelona Clinic Liver Cancer (BCLC) stage B HCC. Preoperative imaging revealed a 55 mm × 32 mm HCC lesion in the right hepatic lobe, along with HHT-related intrahepatic vascular anomalies. He underwent orthotopic LT using a deceased donor liver. Intraoperatively, a “tension-free end-to-end anastomosis” technique was applied to address anomalous hepatic arteries, with real-time Doppler ultrasound monitoring to ensure vascular patency. Postoperatively, an immunosuppressive regimen based on mammalian target of rapamycin (mTOR) inhibitor was administered. The patient recovered uneventfully, with normalized liver function indicators and no evidence of HCC recurrence or HHT-related complications at the 6-month follow-up. Conclusions: Liver transplantation is safe and effective for HCC complicated by HHT. Successful management relies on comprehensive preoperative evaluation of vascular anomalies and AVMs, intraoperative individualized vascular management, and postoperative immunosuppression combined with close monitoring. Favorable short-term outcomes can be achieved with this strategy.

Background: Gemcitabine–cisplatin (GC) combined with a programmed death-ligand 1 (PD-L1) inhibitor has become an important first-line regimen for advanced intrahepatic cholangiocarcinoma (ICC). However, overall efficacy remains modest, and inter-patient heterogeneity in outcomes is substantial, highlighting the need for simple biomarkers for pretreatment risk stratification. The systemic immune-inflammation index (SII), derived from peripheral neutrophil, lymphocyte, and platelet counts, has been associated with prognosis in various malignancies, but its clinical relevance in advanced ICC treated with first-line GC plus PD-L1 inhibitor remains unclear. Aims: To evaluate the association of baseline SII with objective response and survival outcomes in patients with advanced ICC receiving first-line GC plus PD-L1 inhibitor. Methods: We retrospectively analyzed 193 consecutive patients with advanced ICC who received first-line GC plus a PD-L1 inhibitor at our center. Baseline clinicopathologic characteristics and laboratory parameters were collected, and SII was calculated as platelet count (×10⁹/L) × neutrophil count (×10⁹/L)/lymphocyte count (×10⁹/L). Receiver operating characteristic (ROC) analysis was performed to assess the discriminative ability of baseline SII for objective response and to determine an internally derived cut-off value. Patients were categorized into low- and high-SII groups accordingly. Logistic regression was used to identify factors associated with objective response rate (ORR). Progression-free survival (PFS) and overall survival (OS) were estimated by the Kaplan–Meier method and compared using the log-rank test. Multivariable Cox proportional hazards models were constructed to evaluate the independent prognostic significance of SII for PFS and OS. Results: Among the 193 patients included, 55 achieved complete or partial response and 138 had stable or progressive disease, yielding an ORR of 28.5%. Baseline SII showed good discrimination for objective response (AUC = 0.91), and the optimal cut-off value was 495.75. Patients in the low-SII group had a significantly higher ORR than those in the high-SII group (p < 0.001). Kaplan–Meier analysis demonstrated that both PFS and OS were longer in the low-SII group than in the high-SII group (median OS: 13.0 vs. 8.0 months, log-rank p < 0.001; median PFS: 8.5 vs. 6.0 months, p = 0.025). In multivariable Cox models adjusting for differentiation, CA19-9, tumor multiplicity, and distant metastasis, SII grouping remained independently associated with PFS and OS, and distant metastasis was consistently associated with increased risks of progression and death. Conclusions: Baseline SII is a readily available prognostic biomarker associated with objective response and survival in patients with advanced ICC treated with first-line GC plus PD-L1 inhibitor. Given the retrospective single-center design, the absence of a non-immunotherapy comparator cohort, and internal cut-off derivation, these findings should be interpreted as hypothesis-generating and warrant external validation.