Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease : A Fabry Outcome Survey analysis
Beck, Michael (University Medical Center, University of Mainz, Department of Paediatrics)
Hughes, Derralynn A (Royal Free London NHS Foundation Trust, University College of London)
Kampmann, Christoph (University Medical Center, University of Mainz, Department of Paediatrics)
Larroque, Sylvain (Shire, Zug)
Mehta, Atul (Royal Free London NHS Foundation Trust, University College of London)
Pintos-Morellell, Guillem (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol)
Ramaswami, Uma (Royal Free London NHS Foundation Trust, University College of London)
West, Michael (Department of Medicine, Dalhousie University)
Wijatyk, Anna (Shire)
Giugliani, Roberto (Medical Genetics Service HCPA/Dep Genet UFRGS and INAGEMP, Porto Alegre)
Universitat Autònoma de Barcelona

Data:	2015
Resum:	Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry disease in patients enrolled in the Fabry Outcome Survey (FOS) were compared with published findings for untreated patients with Fabry disease. Data were extracted from FOS, a Shire-sponsored database, for comparison with data from three published studies. Outcomes evaluated were the annualized rate of change in estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) as well as time to and ages at a composite morbidity endpoint and at death. FOS data were extracted for 740 treated patients who were followed for a median of ~ 5 years. Compared with no treatment, patients treated with agalsidase alfa demonstrated slower decline in renal function and slower progression of left ventricular hypertrophy. Treated male patients with baseline eGFR < 60 mL/min/1. 73 m 2 had a mean (standard error of the mean [SEM]) annualized change in eGFR of − 2. 86 (0. 53) mL/min/1. 73 m 2 /y compared with − 6. 8 (1. 5) in the published untreated cohort. The mean (SEM) rate of LVMI increase with treatment was 0. 33 (0. 10) g/m 2. 7 /y in males and 0. 48 (0. 09) in females, compared with 4. 07 (1. 03) in untreated males and 2. 31 (0. 81) in untreated females. Morbidity occurred later in treated patients, with ~ 16% risk of a composite morbidity event (26% in males) after 24 months with ERT versus ~ 45% without treatment, with first events and deaths also occurring at older ages in patients administered ERT (e. g. , estimated median survival in treated males was 77. 5 years versus 60 years in untreated males). Findings from these retrospective comparisons of observational data and published literature support the long-term benefits of ERT with agalsidase alfa for Fabry disease in slowing the progression of renal impairment and cardiomyopathy. Treatment also appeared to delay the onset of morbidity and mortality. Interpretation of these findings should take into account that they are based on retrospective comparisons with previously published data.
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Matèria:	ACEI, Angiotensin-converting enzyme inhibitor ; ARB, Angiotensin receptor blocker ; CI, Confidence interval ; Egfr, Estimated glomerular filtration rate ; ERT, Enzyme replacement therapy ; FOS, Fabry Outcome Survey ; LVH, Left ventricular hypertrophy ; LVMI, Left ventricular mass indexed to height ; MDRD, Modification of Diet in Renal Disease ; SE, Standard error ; SEM, Standard error of the mean ; Fabry disease ; Enzyme replacement therapy ; Agalsidase alfa ; Long-term effectiveness
Publicat a:	Molecular Genetics and Metabolism Reports, Vol. 3 (march 2015) , p. 21-27, ISSN 2214-4269

DOI: 10.1016/j.ymgmr.2015.02.002
PMID: 26937390

7 p, 412.7 KB

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Documents de recerca > Documents dels grups de recerca de la UAB > Centres i grups de recerca (producció científica) > Ciències de la salut i biociències > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP)
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