Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data

Mehta, Atul; Beck, Michael; Elliott, Perry; Giugliani, Roberto; Linhart, Aleš; Sunder‐Plassmann, Gere; Schiffmann, Raphael; Barbey, Frédéric; Ries, Markus; Clarke, Joe T.R.

doi:10.1016/s0140-6736(09)61493-8

Cited by 251 publications

(263 citation statements)

References 27 publications

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“…Mehta et al 28 reported a modest reduction in LVMI in patients from the Fabry Outcome Survey observational database who had LVH and were treated with agalsidase alfa at a dose of 0.2 mg/kg/2 weeks for 5 years. However, LVMI slopes were not calculated; data were analyzed as change from baseline after each year of treatment, with the number of patients (n) ranging from 23 to 32 after each year.…”

Section: Discussionmentioning

confidence: 99%

“…However, LVMI slopes were not calculated; data were analyzed as change from baseline after each year of treatment, with the number of patients (n) ranging from 23 to 32 after each year. 28 It is difficult to compare those findings with the current LVM slope data, because Mehta et al 28 pooled echocardiographic data from men and women and grouped patients by baseline LVH status.…”

Section: Discussionmentioning

confidence: 99%

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Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry

Germain

Weidemann

Abiose

et al. 2013

Genetics in Medicine

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Fabry disease (FD) (OMIM 301500) is a rare disorder in which globotriaosylceramide and other glycosphingolipids accumulate within lysosomes because of insufficient activity of α-galactosidase A. 1,2 Over time, the glycosphingolipid substrate progressively accumulates within blood vessels and various other cell types throughout the body. The initial signs and symptoms of FD frequently include neuropathic pain in the extremities, hypohidrosis, angiokeratomas, and gastrointestinal discomfort. 3 Over a period of decades, the accumulation of glycosphingolipids impairs vital organ function, putting patients at risk of developing renal failure, stroke, and cardiovascular dysfunction. [4][5][6] Because it is an X-linked disorder, hemizygous males typically experience the most severe manifestations of FD, 1 but heterozygous females can also develop serious complications. [4][5][6][7] The cardiovascular manifestations of FD can include leftventricular hypertrophy (LVH), atrial and left-ventricular arrhythmias, heart block, valvular dysfunction, angina, and arterial wall thickening. 4,5,[8][9][10][11][12][13] As the disease progresses, the hypertrophy becomes more severe, including the development of myocardial fibrosis and serious cardiac events including cardiac-related death. 4,5,8,11 Agalsidase-β, a recombinant form of human α-galactosidase A (Fabrazyme, Genzyme a Sanofi Company, Cambridge, MA), is approved for use as enzyme replacement therapy (ERT) for FD. In clinical studies, agalsidase-β at a dose of 1 mg/kg/2 weeks cleared microvascular endothelial glycosphingolipid deposits from the heart, as well as from kidney and skin, within 5 months. 14 It also provided long-term stabilization of renal function in patients with mild renal involvement 15 and delayed time to renal, cardiovascular, and cerebrovascular events in patients with more advanced FD. 16 Because it is a rare disorder, the randomized clinical trials of agalsidase-β included a relatively limited number of subjects with FD. 14,16 Several prospective, observational studies have reported cardiac data from patients treated with agalsidase-β, but these also involved only small numbers of patients. 17,18 Purpose: The aim of this study was to evaluate the progression of left ventricular hypertrophy in untreated men with Fabry disease and to assess the effects of agalsidase-β (recombinant human α-galactosidase A) on left ventricular hypertrophy. Methods:Longitudinal Fabry Registry data were analyzed from 115 men treated with agalsidase-β (1 mg/kg/2 weeks) and 48 untreated men. Measurements included baseline left-ventricular mass and at least one additional left-ventricular mass assessment over ≥2 years. Patients were grouped into quartiles, based on left-ventricular mass slopes. Multivariate logistic regression analyses identified factors associated with left ventricular hypertrophy progression.Results: For men in whom treatment was initiated at the age of 18 to <30 years, mean left ventricular mass slope was −3.6 g/year (n = 31) compared with +9.5 g/year in untr...

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry

Germain

Weidemann

Abiose

et al. 2013

Genetics in Medicine

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“…Numerous clinical trials, observational studies, and registry data have provided some evidence for the safety and efficacy of ERT in improving disease symptoms, cardiac mass, renal function, and quality of life Schiffmann et al 2001;Weidemann et al 2003;Wilcox et al 2004;Banikazemi et al 2007;Hughes et al 2008;Koskenvuo et al 2008;Imbriaco et al 2009;Mehta et al 2009;Feriozzi et al 2012). To date, there have been limited comparisons of the two agents, from which no firm conclusion can be drawn regarding their relative efficacy and safety (Lidove et al 2010).…”

Section: Introductionmentioning

confidence: 99%

Effects of Switching from Agalsidase Beta to Agalsidase Alfa in 10 Patients with Anderson-Fabry Disease

et al. 2012

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“…Wilcox et al indicated that QoL in symptomatic Fabry females became impaired at a later age than in males, but both genders experience significantly impaired QoL from the third decade of life onward [3]. Three studies using the EQ-5D questionnaire found an improvement in the QoL of Fabry patients receiving ERT after one year of treatment, with improvement maintained during the second year [17,18] and after 5 years of supplementation [19].…”

Section: Discussionmentioning

confidence: 99%

Quality of life among polish Fabry patients — a cross-sectional study quality of life among polish Fabry patients

Żuraw

Golicki

Jurecka³

et al. 2011

Open Medicine

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AbstractThis study sought to explore the following issues 1) health-related quality of life (HRQoL) in Fabry patients relative to the general population 2) the quality of life (QoL) level in heterozygous females as compared to hemizygous males and the general population. A prospective, cross-sectional study was performed in patients diagnosed with Fabry disease in Poland (n=33). HRQoL was assessed with two generic questionnaires: the Medical Outcomes Study Short Form-36 (SF-36) and EuroQol questionnaire (EQ-5D), which includes the EQ-5D descriptive system and the EQ-visual analogue scale (EQ VAS), as well as a disease-specific author’s questionnaire. When measured with EQ-VAS, the subjective perception of health status was significantly lower in Fabry patients than that of the general population. SF-36 norm-based scores showed that patients are disadvantaged mainly in social functioning, bodily pain, and mental health. Objective assessments of HRQoL according to the EQ-5D Index tend to be lower for males than for females. Only male patients experienced extreme problems identified by the EQ-5D descriptive system. HRQoL of Fabry patients, measured by EQ-5D and SF-36, is lower as compared with that of the general population. Fabry disease effects QoL in its physical, mental and social dimensions.

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Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data

Cited by 251 publications

References 27 publications

Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry

Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry

Effects of Switching from Agalsidase Beta to Agalsidase Alfa in 10 Patients with Anderson-Fabry Disease

Quality of life among polish Fabry patients — a cross-sectional study quality of life among polish Fabry patients

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