Search Results (150)

Background/Objectives: Succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumors (GISTs) are a rare subgroup of GISTs and respond poorly to conventional systemic therapies. This study describes long-term outcomes after yttrium-90 (Y-90) selective internal radiation therapy (SIRT) for progression of unresectable SDH-deficient GIST hepatic metastases. Methods: We performed a retrospective review of consecutive patients treated with SIRT at three tertiary referral centers in Europe and the United States. Data collection included demographics, tumor profiling, prior therapies, Y-90 dosimetry approach, imaging response, adverse events, and long-term outcomes. Results: Twelve patients (66.7% female) with a median age of 27 years (range, 17–57 years) were included. One patient had a complete response (8.3%) and seven had partial responses (58.3%) by modified Response Evaluation Criteria in Solid Tumors. Four patients (33.3%) had tumor shrinkage that did not meet partial response criteria. The objective response rate was 66.7%, with a disease control rate of 100%. One grade 3 or higher adverse event was observed (cholecystitis requiring cholecystectomy). At a median follow-up of 32 months (range, 3–77 months), two patients experienced disease progression. Median overall survival was not reached, with one death during follow-up. Conclusions: SIRT appears safe and effective for patients with progressive, unresectable SDH-deficient GIST hepatic metastases, with durable responses and limited serious toxicity. These findings suggest that SDH-deficient GIST may be more sensitive to radiation than previously appreciated and that SIRT may be a useful liver-directed approach for patients with limited systemic options. Full article

Background: Locally advanced thyroid cancer is associated with a poor prognosis and high surgical morbidity. This study evaluated the feasibility and outcomes of neoadjuvant treatment including targeted therapy with locoregional interventions. Methods: We retrospectively analyzed 17 patients with locally advanced thyroid cancer who received neoadjuvant treatment, including tyrosine kinase inhibitors (TKIs), transarterial embolization (TAE), and radiofrequency ablation (RFA), either alone or in combination. The primary outcome was the proportion of patients who proceeded to R0 or R1 surgical resection following neoadjuvant treatment. Secondary outcomes included tumor response assessed using the Response Evaluation Criteria in Solid Tumors (RECIST), version 1.1, and changes in surgical morbidity evaluated using the MGH/MEEI–MSK–MD Anderson surgical morbidity complexity score (MMM score) and the Invasive Thyroid Class. Results: Five patients (29%) achieved R0/R1 resection, including four with R0 resection. Most patients demonstrated reduced tumor burden and surgical complexity, as reflected by improvements in the MMM score and the Invasive Thyroid Class. The objective response rate was 47% and the 12-month progression-free survival rate was 58.2%. Conclusion: Neoadjuvant treatment may improve tumor downstaging and surgical feasibility in selected patients with initially unresectable thyroid cancer. Further prospective studies are required to validate these findings. Full article

Imaging techniques used for the care of cutaneous melanoma patients have greatly changed over the past century, from symptom-driven radiography toward a multimodality framework integrated for staging, directing surgery, and systemic therapy, and surveillance. Historically, clinical evaluation and skin exams have been the tenets of melanoma diagnosis and staging. In recent years, noninvasive imaging, such as dermoscopy, total-body photography and reflectance confocal microscopy, has expanded the diagnostic toolset for primary melanoma detection. Concurrently, several imaging techniques have been developed to detect metastases and follow disease progression, including computed tomography (CT), magnetic resonance imaging (MRI), fluorodeoxyglucose positron emission tomography/computed tomography (FDG PET/CT), lymphoscintigraphy, and single-photon emission computed tomography/computed tomography (SPECT/CT). The use of immune checkpoint inhibitors has also altered imaging interpretation by introducing atypical response patterns, including pseudoprogression, requiring immune-adapted assessment frameworks such as Immune Response Evaluation Criteria in Solid Tumors (iRECIST). While there is a strong consensus for high-risk patients, imaging techniques and surveillance schedules for low-risk patients (stage I/II) remain controversial due to limited supporting evidence and conflicting data on costs and patient benefit. The development of new technologies, including image-guided surgery, non-FDG PET tracers, phone apps, artificial intelligence-assisted image analysis, and radiomics, may further change melanoma imaging. The aim of this review is to detail the historical evolution of melanoma imaging, the development of new imaging techniques, and their role and future in clinical practice. Full article

Objective: The objective of this study was to construct a large language model (LLM) for the Response Evaluation Criteria in Solid Tumors (RECIST) assessment using exclusively longitudinal radiology report text. Methods: This study included 258 patients with solid tumors, encompassing 2065 longitudinal CT/MRI examination time points. We developed WiNGPT-32B, an open-source and locally deployable LLM, by infusing it with domain-specific medical knowledge and optimizing it via knowledge distillation, using GPT-4 as the teacher model. Central to its architecture is the Chained Task Execution (CTE) framework, which structures RECIST assessment into four modular components: lesion diameter extraction, sum of longest diameter computation, tumor response classification, and report generation. Model performance (accuracy, recall, precision, and F1 score) was benchmarked against GPT-4 and a single radiologist, utilizing the consensus of three independent radiologists as the reference standard. Results: The number of patients with imaging time points was 212 (82.2%) with 4–10, 36 (13.9%) with 11–20, and 10 (3.9%) with >20 time points. For target lesions, the successful extraction rate of WiNGPT-32B was 0.934 (95% CI: 0.922–0.944), which was slightly higher than that of GPT-4 0.920 (0.907–0.931; p = 0.083). In five-category RECIST classification (complete response, partial response, stable disease, progressive disease, and not evaluable), WiNGPT-32B achieved an overall accuracy of 0.805 (0.786–0.823), significantly higher than GPT-4 (0.699, 0.678–0.720; p < 0.001) but lower than the radiologist (0.915, 0.901–0.928; p < 0.001). For progressive disease, WiNGPT-32B had an F1 score of 0.841 (0.813–0.870), significantly outperforming GPT-4’s 0.755 (0.720–0.790), and approaching the radiologist’s 0.922 (0.902–0.942). Conclusions: WiNGPT-32B demonstrates the feasibility of a text-only, open-source LLM with the CTE framework for longitudinal RECIST assessment, with promising performance in detecting disease progression. Full article

Background/Objectives: Trabectedin is a standard-of-care chemotherapy for recurrent/metastatic liposarcomas, including myxoid/round cell liposarcoma (MRCLS). This pooled analysis was performed to evaluate trabectedin efficacy in MRCLS across multiple trials to establish a standard-of-care efficacy baseline. Methods: The Yale University Open Data Access (YODA) Project was queried for prospective trabectedin trials. Data on patients with unresectable, locally advanced/metastatic MRCLS who received ≥ 1 trabectedin dose(s) (1.0–1.5 mg/m² administered as a 24-h continuous infusion every 3 weeks) were extracted from three trials: NCT00060944, NCT00210665, and NCT01343277. The primary outcome was the objective response rate as per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Secondary outcomes included disease control rate and overall survival, defined as the time from first trabectedin administration to death. Responses were assessed using RECIST by investigators in NCT01343277 and by independent review in NCT00060944. In NCT00210665, tumor assessment followed institutional standards, so only overall survival was analyzed. Results: Sixty-three patients were included (42 from NCT01343277, 13 from NCT00060944, 8 from NCT00210665): 32% were female, the median age was 50 years, and 79% were White. The objective response rate was 16.3% (8/49) of patients (37 from NCT01343277, 12 from NCT00060944). The disease control rate was 77.6% (38/49). Overall survival data were available for 63 patients: the median overall survival was 22.51 months (95% CI: 16.99–34.33). Conclusions: This analysis represents one of the largest pooled populations to date of patients with advanced MRCLS treated with trabectedin in prospective clinical trials to be reported. Whilst limited by typical factors affecting pooled analyses, including cross-study heterogeneity, nevertheless the results estimate a trabectedin efficacy baseline against which to inform other therapies for MRCLS. Full article

Objective: Pembrolizumab monotherapy is an anti-PD-1 immunotherapy that is approved as a first-line treatment for non-small cell lung cancer (NSCLC) patients with high PD-L1 expression (≥50%). However, approximately 55% of these patients do not respond. Early identification of likely non-responders is critical to enable timely transition to alternative treatments. Materials: This study analyzed a retrospective cohort of NSCLC patients treated with first-line PD-L1 monotherapy, divided into a discovery training set (n: 97; 27 non-responders) and a preliminary test set (n: 17; 9 non-responders). Treatment response was assessed using baseline and follow-up CT scans in accordance with the response evaluation criteria in solid tumors (RECIST v1.1). Methods: Our objective was to extract deep learning (DL) features from the two groups of patients and apply transfer learning techniques to identify patients at risk of progression on pembrolizumab monotherapy. A nonparametric statistical test (Mann–Whitney U) was employed to rank the discriminative power of the 128 features from these training groups. Two types of support vector machine (SVM-RBF and SVM-Polynomial) classifiers were employed to investigate the discriminating power of the highest-ranked features as measured by F1 score and AUC values over ROC curves at the three levels of the data (slice, lesion, and patient) with and without clinical descriptors. Results: SVM-RBF performed best when trained on the 10 highest-ranked DL features and five clinical descriptors, achieving AUC of 0.742 (CI 95% 0.47–1.00), SN of 88.9%, SP of 75% and F1 score of 84.2% on preliminary test set patients, whereas an AUC of 0.902 ± 0.031, SN of 81.5%, SP of 81.4% and F1 score of 71% were observed for the discovery training set. Conclusions: Integrating CT-based DL features with clinical descriptors demonstrated balanced performance, offering a promising tool to identify patients at risk of progression on pembrolizumab monotherapy to support first-line treatment decisions in PD-L1-high NSCLC. Full article

Background/Objectives: Efficacy endpoint selection in adult primary central nervous system (CNS) tumor trials remains challenging because conventional solid tumor frameworks do not adequately capture the anatomical, radiographic, and biological complexity of brain tumors. In particular, postoperative irregular residual lesions, non-enhancing tumor components, and treatment-related imaging changes complicate the interpretation of objective response and progression. This narrative review examines the current landscape of endpoint selection in adult primary CNS tumor trials and discusses strategies for standardization in the Response Assessment in Neuro-Oncology (RANO) 2.0 era from integrated regulatory science and clinical perspectives. Methods: This study was conducted as a narrative review intended to provide a regulatory science-oriented synthesis of efficacy endpoint evaluation in adult primary CNS tumor trials. The literature search primarily utilized PubMed and ClinicalTrials.gov, supplemented by major consensus guidelines, pivotal clinical trials, and regulatory documents from the major regulatory authorities, including those in the United States, Europe, and Japan, published over the past two decades. Search terms included combinations of keywords such as “brain tumor,” “glioblastoma,” “meningioma,” “Phase I,” “Phase II,” “efficacy endpoint,” and “RANO”. In addition to the literature synthesis, this review incorporates findings from our previously published empirical analyses regarding endpoint selection in Phase II glioblastoma trials, Phase II meningioma trials, and Phase I brain tumor trials. Results: Response Evaluation Criteria in Solid Tumors-based response assessment remains fundamentally limited in neuro-oncology. Across recent early-phase trials, substantial heterogeneity persists in endpoint selection and in the operational definitions of objective response rate, progression-free survival, and progression. RANO 2.0 is intended to provide a more unified and implementation-oriented framework by refining baseline definition, progression confirmation, non-enhancing lesion interpretation, and the role of minor response, while improving compatibility with contemporary molecular classification and immunotherapy-era trial design. However, important implementation challenges remain, including potential reproducibility concerns in complex imaging assessments, operational complexity, imaging standardization, and the need for independent central review. Conclusions: Standardization of endpoint strategies in adult primary CNS tumor trials should move beyond simple adoption of conventional solid tumor metrics toward a disease-specific, harmonized framework integrating imaging, clinical context, tumor biology, and regulatory interpretability. This review provides a regulatory science-oriented synthesis linking response assessment criteria, early-phase adult primary CNS tumor trial endpoint trends, empirical analyses, and emerging endpoint frameworks. RANO 2.0 represents an important step toward this goal, but it should be regarded as an evolving framework that requires continued validation, international collaboration, and implementation-focused refinement. Full article

Background: To evaluate tumor response, local control (LC), survival, and toxicity after radiotherapy for adrenal metastases from hepatocellular carcinoma (HCC), we conducted a retrospective, bi-institutional study spanning two decades. Methods: Twenty patients received radiotherapy for adrenal metastases (2005–2025). Techniques included three-dimensional conformal radiotherapy, intensity-modulated radiotherapy, stereotactic body radiotherapy, and MR-guided radiotherapy. Tumor response was assessed using Response Evaluation Criteria in Solid Tumors version 1.1. LC, overall survival (OS), and progression-free survival (PFS) were estimated using Kaplan–Meier; the association between biologically effective dose (BED₁₀) and local recurrence was assessed using Fisher’s exact test. Results: The objective response rate was 55.0%, and the disease control rate was 95.0%. The 6- and 12-month adrenal LC rates were 94.4% and 87.2%; local recurrence occurred in three patients (15.0%). No local recurrence was observed at BED₁₀ ≥ 75 Gy (0/8) versus 25.0% (3/12) at BED₁₀ < 75 Gy. However, this difference did not reach statistical significance (p = 0.242, Fisher’s exact test) and should be interpreted as exploratory and hypothesis-generating given the limited sample size. Median OS was 13.4 months, and median PFS was 6.3 months. Systemic progression was the predominant failure pattern. No grade ≥3 radiotherapy-related toxicity was observed. Conclusions: Radiotherapy achieved favorable LC with acceptable toxicity across a 20-year bi-institutional experience, supporting its role as an effective local treatment modality for adrenal metastases from HCC. Full article

Background/Objectives: Patients with unresectable hepatocellular carcinoma (uHCC) refractory or intolerant to immune checkpoint inhibitor (ICI)-based regimens represent a growing yet therapeutically underserved population with limited treatment options. We investigated the efficacy, safety, and mechanistic underpinnings of lenvatinib combined with New FP hepatic arterial infusion chemotherapy (LEN–New FP) in this challenging clinical setting. Methods: We retrospectively analyzed 14 consecutive patients with uHCC treated with LEN–New FP between April 2022 and March 2025. Tumor response was assessed by the modified Response Evaluation Criteria in Solid Tumors (mRECIST). Proper hepatic artery (PHA) diameter was serially measured on angiography as an exploratory assessment of vascular remodeling, and tumor vascularity was semi-quantitatively evaluated using a 4-point angiographic scoring system (Tumor Vascularity Score [TVS]). Results: The cohort comprised BCLC stage B/C (7/7), mALBI grade 1–2b, and 13 of 14 patients with prior ICI-containing therapy. The objective response rate and disease control rate were 85.7% and 100%, including two complete responses. Median overall survival was 22.8 months from LEN–New FP initiation (median follow-up: 15.1 months) and 36.2 months from first-line initiation; median intrahepatic progression-free survival was 10.4 months. A total of 11 of 14 patients (78.6%) transitioned to subsequent therapies, including four curative-intent conversions. PHA narrowing was observed in 10 of 13 evaluable patients (76.9%), with no clear association with hepatic function deterioration. TVS decreased in 10 of 12 evaluable patients (83.3%), with reduction observed in 90.0% of PR/CR cases. Conclusions: LEN–New FP achieved sustained intrahepatic tumor control and encouraging survival in aggressive uHCC, including ICI-refractory or -intolerant disease. The concordant reduction in PHA diameter and tumor vascularity score provides angiographic evidence of VEGFR inhibition-mediated vascular remodeling, offering mechanistic insight into the synergistic antitumor effects of this regimen and supporting LEN–New FP as a promising multimodal strategy within the evolving landscape of HCC treatment. Full article

Reactive oxygen species contribute to the cytotoxic effects of anticancer drugs; however, the clinical relevance of systemic antioxidant capacity in metastatic colorectal cancer (CRC) remains unclear. In this original single-center observational cohort study, we examined the association of baseline blood antioxidant capacity with chemotherapy response and prognosis in 84 patients with stage IV CRC who underwent primary tumor resection followed by systemic chemotherapy between 2015 and 2020. Baseline antioxidant capacity was assessed preoperatively using biological antioxidant potential (BAP) assays. Chemotherapy response was evaluated using contrast-enhanced computed tomography at 4 months using Response Evaluation Criteria in Solid Tumors v1.1. Three-year disease-specific survival (DSS) was assessed. Associations with treatment response were analyzed using linear regression. Survival outcomes were evaluated using Kaplan–Meier and Cox proportional hazards models. Baseline BAP was significantly associated with poorer chemotherapy response; higher BAP levels predicted greater treatment resistance in multivariable analysis (p = 0.009). Kaplan–Meier analysis demonstrated significantly worse 3-year DSS in the high-BAP group than in the low-BAP group (35.6% vs. 55.5%, log-rank p = 0.019). In multivariate Cox regression analysis, high BAP independently predicted poor DSS (hazard ratio 2.174, 95% confidence interval 1.103–4.283, p = 0.009). Elevated baseline systemic antioxidant capacity was associated with reduced chemotherapy effectiveness and poorer DSS in patients with stage IV CRC. Full article

To investigate whether pretreatment serum endocan, platelet-derived growth factor (PDGF)-CC, and -DD levels are elevated in metastatic colorectal cancer (mCRC) patients compared to healthy controls, and to assess their independent associations with chemotherapy response and survival, along with their comparative predictive performance. Adult patients with mCRC receiving systemic chemotherapy combined with an anti-angiogenic agent (bevacizumab or aflibercept) were prospectively enrolled, along with healthy controls. Pretreatment serum samples were collected prior to therapy initiation. Endocan, PDGF-CC, and PDGF-DD levels were measured in duplicate using enzyme-linked immunosorbent assay. Treatment response was evaluated according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 and categorized as responders (complete or partial response) and non-responders (stable or progressive disease). Progression-free survival (PFS) and overall survival (OS) were recorded. Median serum levels of endocan (405.5 vs. 269.0 pg/mL), PDGF-DD (499.9 vs. 315.1 pg/mL), and PDGF-CC (2330 vs. 1118 pg/mL) were significantly higher in the mCRC group compared to controls (p < 0.001). In multivariable logistic regression, all three biomarkers were independently associated with non-response to chemotherapy [Odds ratio (ORs): 1.10 for endocan, 1.05 for PDGF-DD, 1.05 for PDGF-CC; all p < 0.05]. For disease progression, Cox regression showed that higher levels of endocan [Hazard ratio (HR) = 1.04], PDGF-DD (HR = 1.03), and PDGF-CC (HR = 1.04) were significant predictors (all p < 0.01). Similar associations were observed for overall mortality (HR = 1.04, 1.02, and 1.02, respectively; all p < 0.05). Endocan, PDGF-DD, and PDGF-CC are elevated in mCRC and independently predict poor treatment response and adverse survival outcomes, highlighting their potential as prognostic biomarkers. Full article

Background/Objectives: Immune checkpoint inhibitor (ICI)-based combinations have significantly improved outcomes in metastatic clear cell renal cell carcinoma (mccRCC). However, a substantial number of patients fail to derive clinical benefit, highlighting the need for reliable predictive biomarkers. Circulating biomarkers represent an attractive, non-invasive alternative to tissue-based assays. This study aimed to evaluate the immunity-related genes ERVFRD-1 and MFSD2A as potential blood-based candidate biomarkers associated with response to ICI-based therapy in mccRCC. Methods: Peripheral blood samples were collected prior to treatment initiation from 34 patients with mccRCC receiving PD-1-based therapy. Gene expression levels of ERVFRD-1 and MFSD2A were quantified using real-time PCR. Treatment response was assessed according to the Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. Peripheral blood samples from healthy individuals were included as controls. Results: Both ERVFRD-1 and MFSD2A were significantly dysregulated in mccRCC patients compared with healthy controls. Their expression differed between patients with clinical benefit and those with progressive disease. Specifically, patients with progressive disease exhibited reduced ERVFRD-1 expression and increased MFSD2A expression compared with patients showing clinical benefit. Conclusions: ERVFRD-1 and MFSD2A were associated with treatment response in this pilot cohort and may represent promising blood-based biomarker candidates, requiring validation in larger prospective multicenter studies. Full article

Objectives: To compare the therapeutic efficacy and safety of balloon-assisted transarterial chemoembolization (B-TACE) versus conventional TACE (C-TACE) in hepatocellular carcinoma (HCC) and to evaluate the potential predictive value of intraoperative balloon-occluded arterial stump pressure (Boasp). Methods: In this prospective, single-centre, randomized controlled study, 60 patients with hepatocellular carcinoma were allocated to either the B-TACE group (n = 30) or the C-TACE group (n = 30). One patient in the B-TACE group was lost to follow-up after allocation. The primary analyses were conducted according to the intention-to-treat (ITT) principle, including all randomized patients, with conservative handling of missing data. Sensitivity analyses were performed to assess the robustness of the results. Tumor response and survival outcomes were evaluated using the modified Response Evaluation Criteria in Solid Tumors (mRECIST) and Cox proportional hazards regression models. Intraoperative balloon-occluded arterial stump pressure (BOASP) was measured as an exploratory parameter to quantify embolization adequacy. Adverse events (AEs) were systematically assessed and graded according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Results: TACE achieved a higher 3-month ORR (63.3% vs. 10.0%, p < 0.001) and 6-month disease control rates (80.0% vs. 36.7%, p < 0.001), with PFS (HR = 0.30, 95% CI 0.148–0.608) and procedures within 6 months (1 vs. 3, p < 0.001). The 6-month surgical conversion rate was higher (34.5% vs. 6.7%, p = 0.009). Changes in Boasp correlated with efficacy (AUC = 0.825, p = 0.0398). Severe infections were lower in B-TACE (17.2% vs. 76.7%, p < 0.001). Conclusions: B-TACE offers superior efficacy, survival, and surgical conversion versus C-TACE with favorable safety. Boasp provides a quantitative biomarker for predicting treatment response. Full article

Background/Objectives: Portal vein tumor thrombus (PVTT) is a severe complication of hepatocellular carcinoma (HCC) and is associated with poor outcomes. This study aimed to describe the imaging and clinical characteristics observed among HCC patients with PVTT who survived longer than one year following combined systemic therapy and radiotherapy. Methods: This retrospective, single-center study included 26 consecutive HCC patients with PVTT who survived more than one year after combined treatment. Baseline characteristics included PVTT extent classified according to the Liver Cancer Study Group of Japan—VP1 (segmental portal vein invasion), VP2 (second-order portal vein invasion), VP3 (first-order portal vein invasion), and VP4 (main portal trunk or contralateral PV invasion) and liver function assessed by Child–Pugh class and ALBI grade. Contrast-enhanced CT or MRI was evaluated at baseline and 6 months after treatment using RECIST 1.1 criteria. Results: The cohort was predominantly male (69%), and most patients had extensive PVTT (VP3–VP4, n = 19). Preserved liver function was common at baseline (Child–Pugh class A, n = 24; ALBI grade I, n = 14). Tumor response was observed in 23 patients (88%) during follow-up. Frequently observed post-treatment imaging findings included portal vein recanalization (n = 12), collateral circulation (present in 7 patients at baseline and 6 at follow-up), and compensatory liver hypertrophy (n = 6). Conclusions: Among HCC patients with PVTT who survived longer than one year after combined therapy, portal vein recanalization, collateral circulation, and compensatory liver hypertrophy were commonly observed imaging features. Given the retrospective design and survivor-selection nature of the study, these findings should be interpreted as descriptive observations rather than evidence of treatment efficacy or prognostic determinants. Full article

Background: Glypican-3 (GPC3) is overexpressed in most hepatocellular carcinoma (HCC) tissues but is absent in normal adult liver. We evaluated whether tumor GPC3 expression is associated with clinical outcomes in patients with advanced HCC treated with atezolizumab–bevacizumab (AB). Methods: We conducted a single-center retrospective cohort study of 139 patients with Barcelona Clinic Liver Cancer (BCLC) stage C HCC who received AB between January 2022 and August 2025. Tumor GPC3 expression was assessed by immunohistochemistry. The primary endpoints were overall survival (OS) and progression-free survival (PFS), and the secondary endpoint was objective response rate (ORR) according to modified Response Evaluation Criteria in Solid Tumors (mRECIST). Results: Baseline characteristics were largely balanced between GPC3-positive (n = 87) and GPC3-negative (n = 52) groups. Median OS was significantly shorter in patients with GPC3-positive tumors than in those with GPC3-negative tumors (p = 0.006). In multivariable analysis, GPC3 positivity remained independently associated with higher mortality (hazard ratio [HR] 1.77, 95% confidence interval [CI] 1.05–3.00; p = 0.033), along with Child–Pugh class B. PFS did not differ significantly between the groups (p = 0.712). ORR was lower in GPC3-positive tumors than in GPC3-negative tumors (approximately 17–18% vs. ~32%; p = 0.023). Membranous GPC3 localization was associated with inferior OS compared with cytoplasmic or absent expression (p = 0.025). Conclusions: Tumor GPC3 expression was associated with decreased OS and lower ORR among AB-treated patients with advanced HCC, suggesting potential clinical relevance and may help in risk stratification. Full article