Effect of agalsidase alfa replacement therapy on fabry disease—related hypertrophic cardiomyopathy: A 12- to 36-month, retrospective, blinded echocardiographic pooled analysis
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Cited by (37)
The Role of Cardiac Imaging in the Diagnosis and Management of Anderson-Fabry Disease
2019, JACC: Cardiovascular ImagingCitation Excerpt :Similarly, in 2 separate studies, Kampmann et al. (76) and Kampmann et al. (77) found that an increased LV mass index at baseline (in both males and females) predicted a reduction in LV mass index with ERT. However, those without LVH at baseline did not demonstrate any significant change in LV mass (76,77). These findings were also confirmed by Mehta et al. (78) in a registry study of the FOS (Fabry Outcome Survey; NCT03289065) (78,79) and by Motwani et al. (79).
Echocardiographic Assessment of Patients with Fabry Disease
2018, Journal of the American Society of EchocardiographyLong-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis
2015, Molecular Genetics and Metabolism ReportsCitation Excerpt :Risk of morbidity was also reduced in treated patients from FOS and survival was improved. Our findings of beneficial effects of agalsidase alfa on the progression of renal impairment and cardiomyopathy outcomes concur broadly with those from previous analyses of treated patients from FOS and from other studies [11,13,15–17,24–26]. In treated patients from the FOS with a mean follow-up period of 7.4 years, progression of renal impairment was stabilized in women and showed a small decline in men [16].
Fabry's disease
2014, Journal of the Neurological SciencesCitation Excerpt :Agalsidase alfa treatment caused a significant decrease in left ventricular mass (LVM) (p = 0.008). The reduction in mass occurred in patients with the greatest degree of hypertrophy at baseline [243]. There was reduction of heart rate variability in boys and QRS interval in women (p = 0.003) with FD.
Enzyme replacement therapy in two Japanese siblings with Fabry disease, and its effectiveness on angiokeratoma and neuropathic pain
2013, Molecular Genetics and MetabolismCitation Excerpt :Two forms of ERT are available for the treatment of Fabry disease: agalsidase alfa, which is manufactured in a human cell-line by gene activation [7,8] and agalsidase beta, which is manufactured in Chinese hamster ovary cells [9]. In clinical studies involving adult patients, ERT is associated with clinical benefits, including improvement in neuropathic pain [10,11], stabilization of kidney function [12,13], and improvement of cardiac structure and/or function [11,14,15]. Although clinical trial experience with ERT for the treatment of pediatric patients is limited, recent studies evaluating long-term treatment (up to 4 years of follow-up) with ERT in children with Fabry disease demonstrate that agalsidase alfa is well tolerated, with an efficacy profile consistent with that reported in adults [16–18].
Enzyme replacement therapy in patients with Fabry disease: State of the art and review of the literature
2012, Molecular Genetics and MetabolismCitation Excerpt :Recent studies, however, outline that treatment must be started early enough, to effectively reduce symptoms and improve heart function and morphology [67,69]. A small number of studies and observational data have provided evidence showing that 0.6–4.0 years of treatment with agalsidase alfa results in a progressive decrease in interventricular septal thickness and a reduction or stabilization of left ventricular mass index [14,23,70–73]. Long-term ERT (up to 5 years) in Fabry patients has been reported in observational database [51].
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Currently affiliated with the Baylor Institute of Metabolic Disease, Dallas, Texas.