Alpha-1 antitrypsin deficiency: clarifying the role of the putative protective threshold

AN Franciosi, D Fraughen, TP Carroll… - European …, 2022 - Eur Respiratory Soc
Alpha-1 antitrypsin deficiency (AATD) is the only readily identifiable monogenic cause of
COPD. To date the only condition-specific treatment for AATD-associated COPD is weekly …

Augmentation Therapy for Severe Alpha-1 Antitrypsin Deficiency Improves Survival and Is Decoupled from Spirometric Decline—A Multinational Registry Analysis

DD Fraughen, AJ Ghosh, BD Hobbs… - American Journal of …, 2023 - atsjournals.org
Rationale: Intravenous plasma-purified alpha-1 antitrypsin (IV-AAT) has been used as therapy
for alpha-1 antitrypsin deficiency (AATD) since 1987. Previous trials (RAPID and RAPID-…

[PDF][PDF] A randomized, double-blind, placebo-controlled trial of intravenous alpha-1 antitrypsin for ARDS secondary to COVID-19

…, K Donnelly, O Friel, E Browne, DD Fraughen… - Med, 2022 - cell.com
Background Patients with severe coronavirus disease 2019 (COVID-19) develop a febrile
pro-inflammatory cytokinemia with accelerated progression to acute respiratory distress …

Alpha-1 antitrypsin deficiency: current therapy and emerging targets

OF McElvaney, DD Fraughen… - Expert Review of …, 2023 - Taylor & Francis
Introduction Alpha1 antitrypsin deficiency (AATD), a common hereditary disorder affecting
mainly lungs, liver and skin has been the focus of some of the most exciting therapeutic …

[HTML][HTML] A randomised, double-blind, placebo-controlled, pilot trial of intravenous plasma purified alpha-1 antitrypsin for SARS-CoV-2-induced acute respiratory …

…, K Donnelly, O Friel, A Cullen, AM Collins, D Fraughen… - Trials, 2021 - Springer
Objectives The primary objective is to demonstrate that, in patients with PCR-confirmed
SARS-CoV-2 resulting in Acute Respiratory Distress Syndrome (ARDS), administration of 120mg/…

Alpha-1 Antitrypsin Augmentation and the Liver Phenotype of Adults With Alpha-1 Antitrypsin Deficiency (Genotype Pi∗ ZZ)

…, L Maia, C Simões, R Mahadeva, DD Fraughen… - Clinical …, 2024 - Elsevier
Background & Aims α1-Antitrypsin (AAT) is a major protease inhibitor produced by hepatocytes.
The most relevant AAT mutation giving rise to AAT deficiency (AATD), the ‘Pi∗Z’ variant, …

[HTML][HTML] Attitudes towards vaccination for coronavirus disease 2019 in patients with severe alpha-1 antitrypsin deficiency

OJ McElvaney, B Cleary, DD Fraughen… - … Diseases: Journal of …, 2022 - ncbi.nlm.nih.gov
Patients with severe alpha-1 antitrypsin deficiency (AATD) are at increased risk for the
development of chronic obstructive pulmonary disease (COPD), particularly if they smoke. This, …

[HTML][HTML] Safety and Reactogenicity of COVID-19 Vaccination in Severe Alpha-1 Antitrypsin Deficiency

OJ McElvaney, B Cleary, DD Fraughen… - … Diseases: Journal of …, 2024 - ncbi.nlm.nih.gov
Background: Patients with alpha-1 antitrypsin deficiency (AATD) exhibit dysregulated
inflammatory responses and a predilection for autoimmunity. While the adverse event (AE) profiles …

Long-term follow-up of Pi* ZZ alpha-1 antitrypsin deficient patients receiving intravenous alpha-1 antitrypsin augmentation therapy

S Roche, DD Fraughen, M Herron, R Heeney… - 2023 - Eur Respiratory Soc
Background: From March 2006 to October 2010, 140 patients with alpha-1 antitrypsin (AAT)
deficiency who received either intravenous AAT augmentation or placebo in the RAPID …

[HTML][HTML] Examining the approach to medical remediation programmes—an observational study

S Maher, S Ryan, C O'Brien, D Fraughen… - Irish Journal of Medical …, 2024 - Springer
Background Remediation of underperforming students is recognised as an important tool in
medical education; however, there is no universally agreed approach. Aims This study …