Medizinische Universität Graz Austria/Österreich - Forschungsportal - Medical University of Graz
Gewählte Publikation:
SHR Neuro Krebs Kardio Lipid Stoffw Microb
Odler, B; Cseh, Á; Constantin, T; Fekete, G; Losonczy, G; Tamási, L; Benke, K; Szilveszter, B; Müller, V.
Long time enzyme replacement therapy stabilizes obstructive lung disease and alters peripheral immune cell subsets in Fabry patients.
Clin Respir J. 2017; 11(6): 942-950.
Doi: 10.1111/crj.12446
Web of Science
PubMed
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- Führende Autor*innen der Med Uni Graz
- Odler Balazs
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- Abstract:
- Fabry disease is an X-linked lysosomal storage disorder, causing accumulation of globotriaosylceramid in different organs. Glycolipids are activators of different immune cell subsets the resulting inflammation is responsible for organ damage. Pulmonary involvement leads to airway inflammation; however, data on severity, as well as the effect of enzyme replacement therapy on lung function parameters and changes in peripheral immune cell subsets on lung involvement are sparse. Seven Fabry patients and four carriers underwent detailed clinical examinations screening for pulmonary manifestations. Repetitive measurements were performed on five patients on ERT (average follow-up 5 years). Patients with Fabry disease and control volunteers were included into peripheral blood cell measurements. Lung involvement was present in all patients. Symptoms suggestive for lung disease were mild, however, obstructive ventilatory disorder, dominantly affecting small airways accompanied by hyperinflation was demonstrated in all affected patients. ERT resulted in small improvement of FEV1 in most treated patients. Decreased ratio of myeloid DC, Th17 cells while increase in T helper (Th)1 cells, and no change in Th2 and regulatory T (Treg) cells were detected in Fabry patients. Fabry disease results mainly in mild symptoms related to lung involvement, characterized by moderate non-reversible obstructive ventilatory disorder. Stabilization of airway obstruction during follow-up was observed using ERT in most patients, emphasizing the importance of this treatment in respect of pulmonary manifestations. Changes of immune cell subsets in the peripheral blood might play a role in inflammatory process, including small airways in Fabry patient's lung. © 2016 John Wiley & Sons Ltd.
- Find related publications in this database (using NLM MeSH Indexing)
- Adult -
- Enzyme Replacement Therapy - methods
- Fabry Disease - blood
- Fabry Disease - complications
- Fabry Disease - drug therapy
- Fabry Disease - enzymology
- Fabry Disease - physiopathology
- Female -
- Forced Expiratory Volume - drug effects
- Humans -
- Immunity, Cellular - drug effects
- Lung - enzymology
- Lung - pathology
- Lung - physiopathology
- Lung Diseases, Obstructive - blood
- Lung Diseases, Obstructive - drug therapy
- Lung Diseases, Obstructive - enzymology
- Lung Diseases, Obstructive - physiopathology
- Male -
- Middle Aged -
- Respiratory Function Tests - methods
- Sphingolipids - metabolism
- Treatment Outcome -
- Vital Capacity - drug effects
- Find related publications in this database (Keywords)
- Fabry disease
- lung function
- pulmonary
- obstructive
- dendritic cell
- immune cell