Chemokine levels predict progressive liver disease in Down syndrome patients with transient abnormal myelopoiesis

The objective is to search for biomarkers predicting the development of hepatic failure in DS infants with TAM.
Source: Pediatrics and Neonatology - Category: Perinatology & Neonatology Authors: Tags: Original Article Source Type: research

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Publication date: 17 September 2019Source: Cell Reports, Volume 28, Issue 12Author(s): Anetta Ptasinska, Anna Pickin, Salam A. Assi, Paulynn Suyin Chin, Luke Ames, Roberto Avellino, Stephan Gröschel, Ruud Delwel, Peter N. Cockerill, Cameron S. Osborne, Constanze BoniferSummaryAcute myeloid leukemia (AML) is associated with mutations in transcriptional and epigenetic regulator genes impairing myeloid differentiation. The t(8;21)(q22;q22) translocation generates the RUNX1-ETO fusion protein, which interferes with the hematopoietic master regulator RUNX1. We previously showed that the maintenance of t(8;21) AML is depend...
Source: Cell Reports - Category: Cytology Source Type: research
Authors: Petrakis I, Kontogiorgis C, Nena E, Athanasakis K, Gougoula V, Kotsianidis I, Constantinidis TC Abstract Introduction: Real-world effectiveness of hemato-oncology pharmaceuticals may not necessarily mimic clinical trial efficacy results, mainly due to demographic and clinical practice variability. The aim of this review was to systematically assess the availability of real-world evidence (RWE) and the transferability of clinical trial (CT) efficacy results to real life, for novel agents recently approved to manage lymphomas, leukemias and multiple myeloma. This is the largest cross-indication review compar...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Authors: Johnston MP, Patel J, Byrne CD Abstract Introduction The pandemic of obesity over the last two decades has triggered a rise in prevalence of non-alcoholic fatty liver disease (NAFLD). NAFLD is associated with liver-related- and cardiovascular complications. Despite this, the first licensed drug for NAFLD is yet to be approved. Given the scale of the problem and unmet needs, there is a myriad of agents in the development pipeline. Areas covered We discuss promising agents in early phase clinical trials and categorize these agents based on their action on steatosis, steatohepatitis and fibrosis. Furthermore,...
Source: Expert Opinion on Investigational Drugs - Category: Drugs & Pharmacology Tags: Expert Opin Investig Drugs Source Type: research
Authors: Westerouen van Meeteren MJ, Drenth JPH, Tjwa ETTL Abstract Introduction: Non-alcoholic fatty liver disease (NAFLD) encompasses a progressive disease phenotype starting from simple steatosis, which can progress to nonalcoholic steatohepatitis (NASH). It is component of the metabolic syndrome with a large impact on mortality in these patients. PPARs are nuclear receptors that regulate lipid and insulin metabolism, two key components in pathophysiology of NAFLD and NASH. Elafibranor acts as an agonist of PPAR-α and PPAR-δ and is currently under development for the treatment of NAFLD. Areas covered...
Source: Expert Opinion on Investigational Drugs - Category: Drugs & Pharmacology Tags: Expert Opin Investig Drugs Source Type: research
Condition:   Acute Myeloid Leukemia Interventions:   Drug: Gemtuzumab Ozogamicin 147;   Drug: Gemtuzumab Ozogamicin 1;   Drug: Glasdegib;   Drug: Placebo Oral Tablet Sponsor:   University Hospital Heidelberg Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Conditions:   Acute Myeloid Leukemia;   Chronic Myelomonocytic Leukemia;   Myelodysplastic Syndromes Intervention:   Drug: ASTX727 Sponsor:   Astex Pharmaceuticals, Inc. Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Conditions:   Chronic Lymphocytic Leukemia;   Small Lymphocytic Lymphoma Intervention:   Drug: Ibrutinib Sponsors:   The First Affiliated Hospital with Nanjing Medical University;   Xian-Janssen Pharmaceutical Ltd. Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Conditions:   B-cell Acute Lymphoblastic Leukemia;   Diffuse Large B-cell Lymphoma Intervention:   Biological: Tisagenlecleucel Sponsor:   Novartis Pharmaceuticals Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
We report an infant with DS and TAM who developed end-stage liver failure. Liver dysfunction progressed even after blast cells disappeared from the circulation. He underwent a living-donor liver transplantation at 56 days of life without surgical complications. The explanted liver showed atrophy and severe fibrosis without leukemic cell infiltration. The posttransplant course was favorable with no hematological abnormality. He is doing well 8 months after transplantation. To the best of our knowledge, this report is the first showing that liver transplantation might be a treatment option for TAM-related liver failure. ...
Source: Neonatology - Category: Perinatology & Neonatology Authors: Tags: Neonatology Source Type: research
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