Alpha-1-antitrypsin deficiency (AATD)
Objectives
- To enroll patients in the Spanish Registry of AATD
- To collaborate with the initiatives of the Alpha One International registry (A.I.R.)
- To identify the best regimens of administration of AAT based on PK/PD models
- To identify the role of AAT as a protective antiinflammatory mediator in exacerbations of Chronic Obstructive Pulmonary Disease (COPD)
Projects in Progress
- Development of the Spanish registry of patients with AATD
- Detection and identification of rare variants of AAT
- Role of AAT in exacerbations of COPD
Milestones achieved
- Consolidation of the Spanish registry of AATD
- Collaboration with A.I.R.
- Identification of some rare mutations of AAT (Ybarcelona)
- Development of a model to estimate the best therapeutic regimen of intravenous AAT for augmentation therapy
Future challenges
- To ascertain the role of AAT as antiinflammatory mediator in smoking-related COPD
- To participate in new clinical trials of inhaled AAT and new drugs for patients with the deficiency
Publications
Augmentation therapy for emphysema due to alpha-1-antitrypsin deficiency
Gema Tirado-Conde, Beatriz Lara, Marc Miravitlles.
Therapeutic Advances in Respiratory Diseases 2008; 2 (1): 13-21.
Laboratory testing of individuals with severe AAT deficiency in three European centres.
Miravitlles M, Herr C, Ferrarotti I, Jardi R, Rodriguez-Frias F, Luisetti M, Bals R
Eur Respir J 2010; 35: 960-968.
Lines list
- Animal Model
- Bronchiectasis non associated to Cystic Fibrosis (BQ-noFQ), Cystic Fibrosis (CF) and immune deficiencies
- Community-Acquired Pneumonia (CAP)
- Exacerbations of Chronic Obstructive Pulmonary Disease (COPD)
- Networks, European Projects and Collaborations
- Noninvasive Ventilation (NIV)
- Pulmonary complications in immunocompromised (IC) patients
- Translational research laboratory for advanced microbiology diagnosis and pathogen-host response in respiratory infections
- Ventilator-Associated Pneumonia (VAP)
