Oct 24th, 2021 - The 2019 European guidelines (ESC/EAS) for the treatment of dyslipidaemias recommend more aggressive targets for low-density lipoprotein cholesterol (LDL-C) in patients with familial hypercholesterolemia (FH). Current lipid-lowering treatment is often inadequate to achieve these targets. Data from the HELLAS-FH registry were analysed to assess achievement of LDL-C targets in adults with FH base...

Oct 24th, 2021 - To investigate the pathogenesis of a congenital form of hepatic fibrosis, human hepatic organoids were engineered to express the most common causative mutation for Autosomal Recessive Polycystic Kidney Disease (ARPKD). Here we show that these hepatic organoids develop the key features of ARPKD liver pathology (abnormal bile ducts and fibrosis) in only 21 days. The ARPKD mutation increases colla...

Oct 24th, 2021 - Around three out of one hundred thousand people are diagnosed with glioblastoma multiforme, simply called glioblastoma, which is the most common primary brain tumor in adults. With a dismal prognosis of a little over a year, receiving a glioblastoma diagnosis is oftentimes fatal. A major advancement in its treatment was made almost two decades ago when the alkylating chemotherapeutic agent temo...

Oct 24th, 2021 - IgA nephropathy (IgAN) is one of the most prevalent primary glomerulopathies in children. There are various studies investigating the efficacy of angiotensin-converting enzyme inhibitors (ACEI) and angiotensin II receptor blockers (ARB) in adults with IgAN. However, only few studies evaluated the efficacy of these medications in pediatric patients. To evaluate the efficacy and safety of ACEI/AR...

Oct 24th, 2021 - Immune checkpoint inhibitors (ICIs) have led to a revolution in cancer management, mainly due to lasting long-term durable responses in a subset of patients with metastatic solid tumours (Gettinger et al. in JCO 36(17):1675-1684, 2018). As immunotherapy is gradually being applied for the treatment of a large range of solid tumours, the incidence of neurological immune-related adverse events (ir...

Oct 24th, 2021 - Relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) is associated with a poor prognosis despite the availability of multiple treatment options. Preliminary evidence suggests that DLBCL may be responsive to programmed death ligand 1 (PD-L1)/programmed death 1 inhibitors. The JAVELIN DLBCL study was conducted to assess whether a combination of agents could augment and sustain the anti...

Oct 24th, 2021 - Immune checkpoint inhibitors (ICIs) treatment among cancer patients has been shown to have antiviral effects by reactivating exhausted T cells. However, they could also trigger inflammatory storm. Therefore, prior exposure to ICIs may influence the risk of SARS-CoV2 infection and subsequent mortality. Recent results from studies of ICIs treatment on incidence and mortality of COVID-19 are contr...

Oct 24th, 2021 - Poly (ADP)-ribose polymerase (PARP) inhibitors have entered routine clinical practice for the treatment of high-grade serous ovarian cancer (HGSOC), yet the molecular mechanisms underlying treatment response to PARP1 inhibition (PARP1i) are not fully understood. Here, we used unbiased mass spectrometry based proteomics with data-driven protein network analysis to systematically characterize how...

Oct 24th, 2021 - Polycythemia vera (PV) is a common type of Philadelphia chromosome-negative chronic myeloproliferative disorder. PV-associated kidney disease is rarely reported and remains poorly understood. It has been observed that chronic kidney disease could be a risk factor for poor prognosis in PV. We retrospectively analyzed the clinicopathological features of renal presentations in eight patients with ...

Oct 23rd, 2021 - Despite advance in pharmacotherapy of lipid disorders, many heterozygous Familial Hypercholesterolemia patients do not achieve a desirable lipid target to significantly reduce the risk of atherosclerotic cardiovascular disease. The aim of the present work is to evaluate the interaction between Lipoprotein apheresis (LA) and PCSK9i in a small FH cohort in which the guidelines therapeutic target ...

Oct 23rd, 2021 - Bing-Neel syndrome (BNS) is a rare syndrome that occurs in patients with Waldenstrom macroglobulinemia and is characterized by lymphoplasmacytic infiltration into the leptomeningeal tissue and/or the central nervous system. It represents an extramedullary manifestation which may translate into various neurological symptoms. Accurate diagnosis of BNS can be established via histologic sampling an...

Oct 23rd, 2021 - Results of early trials led to FDA approval of immune checkpoint inhibitors (ICIs) for advanced and recurrent/metastatic (R/M) cutaneous squamous cell carcinoma (CSCC). Updated data from these trials are pending and extent of survival outcomes is undetermined. The aim of this study was to assess the efficacy of ICIs in advanced CSCC, comprising locally advanced (LA), locoregionally advanced (LR...

Oct 23rd, 2021 - The pressing need for SARS-CoV-2 controls has led to a reassessment of strategies to identify and develop natural product inhibitors of zoonotic, highly virulent, and rapidly emerging viruses. This review article addresses how contemporary approaches involving computational chemistry, natural product (NP) and protein databases, and mass spectrometry (MS) derived target-ligand interaction analys...

Oct 23rd, 2021 - Small molecule inhibitors have previously been investigated in different studies as possible therapeutics in the treatment of SARS-CoV-2. In the current drug repurposing study, we identified the leukotriene (D4) receptor antagonist Montelukast as a novel agent that simultaneously targets two important drug targets of SARS-CoV-2. We initially demonstrated the dual inhibition profile of Monteluka...

Oct 23rd, 2021 - Mantle cell lymphoma (MCL) is an uncommon subtype of non-Hodgkin lymphoma in which immunochemotherapy +/- high dose therapy and autologous stem cell transplantation remains standard front-line therapy. Despite clear efficacy, patients envitably relapse requiring subsequent therapy. Within this review, we discuss the key therapeutic approaches in the management of patients with relapsed MCL, cov...

Oct 22nd, 2021 - Background: Among the new cases of bile tract carcinoma (BTC) that are diagnosed every year in the United States, there are approximately 6,500 cases of gallbladder carcinoma, 3,000 cases of extrahepatic cholangiocarcinoma, and 3,000 cases of intrahepatic cholangiocarcinoma. Current treatment options for patients with cholangiocarcinoma are limited and take no account of the known biological an...

Oct 22nd, 2021 - Background: Genetic and sporadic mutations on one allele of the GATA2 gene lead to a syndrome termed MonoMAC. MonoMAC is characterized by: 1) infections with Mycobacterium avium complex (MAC) and other opportunistic infections, 2) deficiency of monocytes, B-lymphocytes, and Natural Killer (NK) cells in the peripheral blood, and 3) progression to myelodysplastic syndrome (MDS), chronic myelomono...

Oct 22nd, 2021 - Auto-immune hemolytic anemia (AIHA) is a heterogeneous syndrome in adults. This disease is associated with significant morbidity-mortality. First-line treatment with prolonged corticosteroid is well identified but there is not enough data about cyclosporine treatment in case of resistance or dependence on steroids. the cyclosporine showed efficacy in many immune cytopenic diseases in the light ...

Oct 22nd, 2021 - This is a single arm, single institution study, which aims to evaluate the efficacy and safety of combination therapy with radiosurgery nivolumab and ipilimumab as a maintenance regimen following first line induction chemotherapy in patients with metastatic pancreatic cancer. Chemotherapy will be administrated for a minimum of 4 cycles. Patients who have not progressed on chemotherapy will rece...

Oct 22nd, 2021 - There is an increased risk of Venous and Arterial thromboembolism in COVID-19 infection. The main reason being that the novel coronavirus SARS-CoV-2 induces hyperinflammation and subsequently immunothrombolism which is wide spread in the body3. The angiotensin converting enzyme 2 (ACE2) receptor, which is utilized by the virus to infect the host, is present throughout the human body organs and ...