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

Lidove, O; Barbey, F; Niu, DM; Brand, E; Nicholls, K; Bizjajeva, S; Hughes, DA; (2016) Fabry in the older patient: Clinical consequences and possibilities for treatment. Molecular Genetics and Metabolism , 118 (4) pp. 319-325. 10.1016/j.ymgme.2016.05.009. Green open access

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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.

Type: Article
Title: Fabry in the older patient: Clinical consequences and possibilities for treatment
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.ymgme.2016.05.009
Publisher version: http://dx.doi.org/10.1016/j.ymgme.2016.05.009
Language: English
Additional information: Copyright © 2016 Published by Elsevier Inc. This manuscript version is made available under the CC-BY 4.0 licence (https://creativecommons.org/licenses/by/4.0/). The published article can be found on the journal website at http://dx.doi.org/10.1016/j.ymgme.2016.05.009
Keywords: Fabry disease; Elderly; Agalsidase alfa; Enzyme replacement therapy; Disease burden
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: https://discovery.ucl.ac.uk/id/eprint/1492892
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