The impact of expanded access programs for systemic anticancer therapy in an Irish cancer centre.

Background: Expanded access programs (EAPs) allow cancer patients with unmet clinical need to obtain access to pre-authorisation treatments. There is no standardised process for implementing these programs nationally, and real-world data on their impact is lacking.

Aims: This study aimed to evaluate the prevalence of such EAPs and their impact in a cancer centre.

Optimising oncology drug expenditure in Ireland.

A combination of improvements in patient survival, increasing treatment duration, and the development of more expensive agents has led to a doubling of per-capita spending on cancer medicines in Ireland (2008-2018). Despite this, access to new drugs is poor in comparison to other EU countries. We examine methods to optimise oncology drug spending to facilitate access to newer anticancer agents.

C3 Glomerulopathy and Related Disorders in Children: Etiology-Phenotype Correlation and Outcomes.

Background And Objectives: Membranoproliferative GN and C3 glomerulopathy are rare and overlapping disorders associated with dysregulation of the alternative complement pathway. Specific etiologic data for pediatric membranoproliferative GN/C3 glomerulopathy are lacking, and outcome data are based on retrospective studies without etiologic data.

Design, Setting, Participants, & Measurements: A total of 80 prevalent pediatric patients with membranoproliferative GN/C3 glomerulopathy underwent detailed phenotyping and long-term follow-up within the National Registry of Rare Kidney Diseases (RaDaR).

MiR-126-3p Is Dynamically Regulated in Endothelial-to-Mesenchymal Transition during Fibrosis.

In fibrotic diseases, myofibroblasts derive from a range of cell types including endothelial-to-mesenchymal transition (EndMT). Increasing evidence suggests that miRNAs are key regulators in biological processes but their profile is relatively understudied in EndMT. In human umbilical vein endothelial cells (HUVEC), EndMT was induced by treatment with TGFβ2 and IL1β.

Contribution of Heparan Sulphate Binding in CCL21-Mediated Migration of Breast Cancer Cells.

Chemokine receptor CCR7 is implicated in the metastasis of breast cancer to the lymph nodes. Chemokine function is dependent upon their binding to both cell-surface heparan sulphate (HS) and to their specific receptors; thus, the role of HS in CCR7-mediated lymph node metastasis was investigated by creating a non-HS binding chemokine CCL21 (mut-CCL21). Mut-CCL21 (Δ103-134) induced leukocyte chemotaxis in diffusion gradients but did not stimulate trans-endothelial migration of PBMCs ( < 0.

Prominent members of the human gut microbiota express endo-acting O-glycanases to initiate mucin breakdown.

The thick mucus layer of the gut provides a barrier to infiltration of the underlying epithelia by both the normal microbiota and enteric pathogens. Some members of the microbiota utilise mucin glycoproteins as a nutrient source, but a detailed understanding of the mechanisms used to breakdown these complex macromolecules is lacking. Here we describe the discovery and characterisation of endo-acting enzymes from prominent mucin-degrading bacteria that target the polyLacNAc structures within oligosaccharide side chains of both animal and human mucins.

The Role of the Clinical Pharmacist in an Irish University Teaching Hospital: A Mixed-Methods Study.

Medication review (MR) is a vital part of the pharmacist's role in hospital. However, in the South Infirmary Victoria University Hospital (SIVUH), Cork, Ireland, this has not been fully implemented due to resource issues. In addition, the cost of providing this service has not been evaluated.

Cost Minimization Analysis of Intravenous or Subcutaneous Trastuzumab Treatment in Patients With HER2-Positive Breast Cancer in Ireland.

Background: Two large acute Irish University teaching hospitals changed the manner in which they treated human epidermal growth factor receptor (HER)2-positive breast cancer patients by implementing the administration of trastuzumab via the subcutaneous (SC) route into their clinical practice. The study objective is to compare the trastuzumab SC and trastuzuamb intravenous (IV) treatment pathways in both hospitals and assess which route is more cost-effective and time saving in relation to active health care professional (HCP) time.

Materials And Methods: A prospective observational study in the form of cost minimization analysis constituted the study design.

Heparan sulfate in chronic kidney diseases: Exploring the role of 3-O-sulfation.

One of the main feature of chronic kidney disease is the development of renal fibrosis. Heparan Sulfate (HS) is involved in disease development by modifying the function of growth factors and cytokines and creating chemokine gradients. In this context, we aimed to understand the function of HS sulfation in renal fibrosis.

Hyperfunctional complement C3 promotes C5-dependent atypical hemolytic uremic syndrome in mice.

Atypical hemolytic uremic syndrome (aHUS) is frequently associated in humans with loss-of-function mutations in complement-regulating proteins or gain-of-function mutations in complement-activating proteins. Thus, aHUS provides an archetypal complement-mediated disease with which to model new therapeutic strategies and treatments. Herein, we show that, when transferred to mice, an aHUS-associated gain-of-function change (D1115N) to the complement-activation protein C3 results in aHUS.

The Most Important Therapy Outcomes for School-Aged Children Who Stutter: An Exploratory Study.

Purpose: The aim of this article was to identify what school-aged children who stutter consider to be the most important outcomes from therapy.

Method: A Delphi approach was employed for the study. Eighteen participants aged 9-13 years completed a survey, generating 90 statements that would constitute successful therapy outcomes.