Fabry in the older patient: Clinical consequences and possibilities for treatment.

Details

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State: Public
Version: Final published version
Serval ID
serval:BIB_4031C9ABB616
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Fabry in the older patient: Clinical consequences and possibilities for treatment.
Journal
Molecular genetics and metabolism
Author(s)
Lidove O., Barbey F., Niu D.M., Brand E., Nicholls K., Bizjajeva S., Hughes D.A.
ISSN
1096-7206 (Electronic)
ISSN-L
1096-7192
Publication state
Published
Issued date
08/2016
Peer-reviewed
Oui
Volume
118
Number
4
Pages
319-325
Language
english
Notes
Publication types: Journal Article
Publication Status: ppublish
Abstract
Baseline demographic and phenotypic characteristics of patients aged ≥50years in the Fabry Outcome Survey (Shire; data extracted June 2014) were compared with younger adults to investigate potential factors influencing treatment decisions in later life. Age groups were defined using age at treatment initiation or at FOS entry for untreated patients: 18-49 (n=1344; 49.5% male; 64.6% received agalsidase alfa enzyme replacement therapy [ERT]); 50-64 (n=537; 35.4% male; 74.3% treated); 65-74 (n=137; 32.1% male; 68.6% treated); and ≥75years (n=26; 26.9% male; 50.0% treated). Successive age groups showed higher median age at first symptom and diagnosis. Median alpha-galactosidase A activity, measured as percentage activity of the midpoint of the normal range, was much greater in females than males of all groups except ≥75years (33.4% in females; 27.8% in males). Patients aged ≥75years showed greater values than patients aged 18-49years for median left ventricular mass indexed to height (62.7 vs 42.4g/m(2.7)), mean ventricular wall thickness (15.0 vs 10.0mm) and prevalence of hypertension (57.7% vs 21.8%), and lower median estimated glomerular filtration rate (Modification of Diet in Renal Disease: 65.6 vs 98.5mL/min/1.73m(2)). Larger proportions in the groups aged ≥50 exhibited cardiac and/or cerebrovascular manifestations compared with patients aged 18-49years. The smaller proportion of patients receiving ERT aged ≥75years compared with the younger groups might reflect relatively milder disease burden or physician/patient reluctance to initiate/continue ERT at this age. Further studies are needed to increase knowledge of Fabry disease and ERT in later life.

Keywords
Adolescent, Adult, Age Factors, Aged, Aged, 80 and over, Enzyme Replacement Therapy/adverse effects, Fabry Disease/complications, Fabry Disease/drug therapy, Fabry Disease/physiopathology, Female, Heart Diseases/chemically induced, Heart Diseases/physiopathology, Humans, Kidney Diseases/chemically induced, Kidney Diseases/physiopathology, Male, Middle Aged, Sex Characteristics, alpha-Galactosidase/administration & dosage, alpha-Galactosidase/adverse effects, Agalsidase alfa, Disease burden, Elderly, Enzyme replacement therapy, Fabry disease
Pubmed
Web of science
Open Access
Yes
Create date
06/06/2016 16:12
Last modification date
15/01/2020 6:26
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