Introduction Nivolumab, an anti-PD-1 monoclonal antibody, showed promising results as a monotherapy for advanced HCC, but lacked a statistically significant survival benefit over sorafenib. Aim To evaluate the efficacy and OS of nivolumab as monotherapy in cirrhotic patients with advanced HCC who are ineligible for other treatments. Materials and methods Our retrospective, single-centre study included 22 cirrhotic patients with advanced HCC treated off-label with nivolumab between September 2019 and April 2023. Enrolled patients had disease progression or intolerance to prior TKI therapy and were not eligible for local therapy or other systemic therapies. Outcomes measured were OS, radiological response (RR), defined as stable disease, complete or partial response, and biological response (BR), defined as a ≥ 25% decrease in AFP blood level from baseline. Results 22 patients were included: 73% male, median age 64.5 years (range: 30-80), 91% BCLC-C, 82% Child-Pugh A, 94% received prior lines of treatment. The median duration of treatment was 3.5 months (range: 0.4-36.9). OS was 7.8 months (95%CI 4.7-14.2; range 1.1-34.1). RR at 3 months was achieved in 8 patients and maintained in 6 patients at 6 months. BR at 3 months was achieved in 6 patients. OS was significantly associated with RR (p 0.002) and BR (p 0.0001) at 3 months. Median OS in patients with RR at 3 months was 27.7 months (95%CI 4.7-27.7) versus 6.4 months (95%CI 3.2-9.1) in patients with progression (p 0.0003). At baseline, better performance status (p 0.031), presence of metastatic lymph nodes (p 0.040) and lower disease burden (p 0.009) correlated with improved OS. Grade 3-4 AEs occurred in 5 patients. Conclusions Nivolumab monotherapy in HCC patients with no other therapeutic option showed acceptable efficacy, with OS exceeding 6 months in 68% of patients. RR and BR at 3 months predicted longer survival (median OS 27.7 and 27.9 months, respectively). Nivolumab showed a manageable safety profile.
Nivolumab monotherapy has proven to be effective sometimes for advanced HCC and could be a valuable treatment option for patients outside current treatment indications and reimbursement criteria for the standard of care.
Background/aims: There are limited data about optimal strategy of cardiovascular (CV) risk assessment during liver transplant (LT) workup. We evaluated CV risk profile and its correlation with early post-LT cardiac events.
Background and aims: It has been proved that complete necrosis with percutaneous thermal-ablation in early HCC and cirrhosis is related to a better survival. We compared the efficacy of RFA and MW to obtain complete response(CR) in patients with HCC.
Aims: Cytoplasmic accumulation of the nuclear protein transactive response DNA-binding protein 43 (TDP-43) is an early determinant of motor neuron degeneration in most amyotrophic lateral sclerosis (ALS) cases. We previously disclosed this accumulation in circulating lymphomonocytes (CLM) of ALS patients with mutant TARDBP, the TDP-43-coding gene, as well as of a healthy individual carrying the parental TARDBP mutation. Here, we investigate TDP-43 subcellular localization in CLM and in the constituent cells, lymphocytes and monocytes, of patients with various ALS-linked mutant genes. Methods: TDP-43 subcellular localization was analysed with western immunoblotting and immunocytofluorescence in CLM of healthy controls (n = 10), patients with mutant TARDBP (n = 4, 1 homozygous), valosin-containing protein (VCP; n = 2), fused in sarcoma/translocated in liposarcoma (FUS; n = 2), Cu/Zn superoxide dismutase 1 (SOD1; n = 6), chromosome 9 open reading frame 72 (C9ORF72; n = 4), without mutations (n = 5) and neurologically unaffected subjects with mutant TARDBP (n = 2). Results: TDP-43 cytoplasmic accumulation was found (P < 0.05 vs. controls) in CLM of patients with mutant TARDBP or VCP, but not FUS, in line with TDP-43 subcellular localization described for motor neurons of corresponding groups. Accumulation also characterized CLM of the healthy individuals with mutant TARDBP and of some patients with mutant SOD1 or C9ORF72. In 5 patients, belonging to categories described to carry TDP-43 mislocalization in motor neurons (3 C9ORF72, 1 TARDBP and 1 without mutations), TDP-43 cytoplasmic accumulation was not detected in CLM or in lymphocytes but was in monocytes. Conclusions: In ALS forms characterized by TDP-43 mislocalization in motor neurons, monocytes display this alteration, even when not manifest in CLM. Monocytes may be used to support diagnosis, as well as to identify subjects at risk, of ALS and to develop/monitor targeted treatments.
Background: Many studies, almost all in an Endoscopic Retrograde Cholangiopancreatography (ERCP) setting, have been conducted to establish if a link exists between periampullary diverticula (PADs) and biliopancreatic diseases but the issue is still debated. Aims: The objective was to clarify the link between PADs and biliopancreatic disease, for the first time using Endoscopic Ultrasound (EUS). Methods: We retrospectively reviewed our database seeking patients scheduled for EUS with an indication that entailed the exploration of the second duodenum. For each patient with a PAD enrolled in the study, 6 controls were randomly selected. Results: 2475 patients met the inclusion criteria. Among them, 185 subjects with a PAD were found (prevalence 7.5%), 1110 subjects served as controls. Patients with a PAD had more frequently a history of cholangitis (8.1 vs 2.2%; OR 3.99, p < 0.001), a higher prevalence of common bile duct (CBD) dilation (44.3 vs 28.2%; OR 2, p < 0.0001) and a higher prevalence of CBD stones (34.1 vs 19.6%; OR 2.1, p < 0.0001). No differences were found about history of jaundice, acute/recurrent pancreatitis or EUS signs of chronic pancreatitis. Conclusion: Whereas PADs were linked with history of cholangitis, CBD stones and dilation, no association was found with pancreatic diseases. (c) 2018 Editrice Gastroenterologica Italiana S.r.l. Published by Elsevier Ltd. All rights reserved.
Introduction: Liver transplantation (LT) for patients with portopulmonary hypertension (POPH) is considered acceptably safe for mean pulmonary arterial pressure (mPAP) ≤35 mmHg and pulmonary vascular resistance (PVR) ≤5 WU. Obtainment of these targets and time needed are therefore crucial, nevertheless optimal POPH treatment isn’t clearly established and data on pharmacological response are ambiguous.
Introduction: Portal vein thrombosis (PVT) is a complication of cirrhosis that may increase surgical complexities during orthotopic liver transplantation (OLT) and cause complications after surgery.
Introduction: Sorafenib and Y90-Radioembolization (TARE) are the optional therapies for cirrhotic patients with advanced mono-lobar hepatocellular carcinoma (HCC).
POSTERS group (range 2-189) and in the RFA group (range 6-111) (p = 0.99).No procedure related mortality was noted.Major complications occurred only in 1 patient for both treatment group (p = 0.87).OS rates at 1-, 3-, 5-years were 97%, 83.3% and 64.6% in PEI group, and 97.7%, 77.1% and 62.3%, in RFA group (p = 0.16).HCC-related death occurred in only 42.3% of PEI and 25.6% of RFA patients (p = 0.15).HCC recurrence rates at 1-, 3-, 5-years were 16.1%, 61.4%, 79.4% in the PEI group and 20.4%, 47.7%, and 57.4% in RFA group (p = 0.28).Furthermore, cumulative local tumor progression and distant intrahepatic recurrence rates were not different between the two populations.Local tumor progression was diagnosed mostly in the first 2 follow up years (77.7% in PEI group and 71.4% in RFA group).Multivariate analysis revealed that ascites was the only pretreatment risk factor negatively associated with OS in both groups (OR = 13.6,95% CI 2.5-73.3,p = 0.002 for PEI group and OR = 8.7, 95% CI 2.8-27, p < 0.001 for RFA group).Conclusions: PEI and RFA are equally effective in the treatment of HCC ≤2 cm in terms of OS, local tumor progression and overall HCC recurrence.