Fabry disease: overall effects of agalsidase alfa treatment

Beck, Michael; Ricci, Riccardo; Widmer, U; Dehout, F.; Lorenzo, A. García de; Kampmann, Christoph; Linhart, Aleš; Sunder‐Plassmann, Gere; Houge, Gunnar; Ramaswami, Uma; Gal, Andreas; Mehta, A

doi:10.1111/j.1365-2362.2004.01424.x

Cited by 200 publications

(181 citation statements)

References 21 publications

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“…Most, but not all (Koskenvuo et al 2008;KovacevicPreradovic et al 2008), prior studies that investigated the effects of ERT (agalsidase beta or agalsidase alfa) on cardiac functional parameters reported improvements in cardiac parameters after initiation of ERT (Weidemann et al 2003;Beck et al 2004;Hughes et al 2008;Vedder et al 2008;Imbriaco et al 2009). Results of the current study were in agreement with the majority of these studies, i.e., LV mass and wall thickness decreased significantly from pre-ERT values after initiation of ERT, which in this case was with agalsidase beta (retrospective data; see Table 1).…”

Section: Discussionmentioning

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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Section: Discussionmentioning

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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“…Enzyme replacement therapy (ERT) with agalsidase alfa (agala) alleviates many of the renal and cardiac signs and symptoms of FD, decreases pain and gastrointestinal symptoms, and improves overall quality of life (Dehout et al 2003;Beck et al 2004;Schwarting et al 2006;Choi et al 2008;Mehta et al 2009). In December 2009, because of a worldwide supply shortage of agalsidase beta (agalb), the only ERT then marketed in the United States, the US Food and Drug Administration approved HGT-REP-059, a protocol to allow access to agala for FD patients who were left without a therapeutic option.…”

Section: Introductionmentioning

confidence: 99%

Effect and Tolerability of Agalsidase Alfa in Patients with Fabry Disease Who Were Treatment Naïve or Formerly Treated with Agalsidase Beta or Agalsidase Alfa

et al. 2015

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Objectives: In a multicenter, open-label, treatment protocol (HGT-REP-059; NCT01031173), clinical effects and tolerability of agalsidase alfa (agala; 0.2 mg/ kg every other week) were evaluated in patients with Fabry disease who were treatment naïve or switched from agalsidase beta (switch). Over 24 months, data were collected on the safety profile; renal and cardiac parameters were assessed using estimated glomerular filtration rate (eGFR), left ventricular mass index (LVMI), and midwall fractional shortening (MFS).Results (AEs) were consistent with the known safety profile of agala. Two switch patients had hospitalization due to possibly/probably drug-related serious AEs (one with transient ischemic attack, one with infusion-related AEs). One switch and two treatment-naïve patients discontinued treatment because of AEs. Three patients (one each switch, treatment naïve, and previous agala) died; no deaths were considered drug-related. There was no significant change from baseline in LVMI or MFS in either group. Similarly, eGFR remained stable; mean AE standard error annualized change in eGFR (mL/min/ 1.73 m 2 ) was À2.40 AE 1.04 in switch and À1.68 AE 2.21 in treatment-naïve patients.Conclusions: This is the largest cohort of patients with Fabry disease who were started on or switched to agala in an FDA-accepted protocol during a worldwide supply shortage of agalsidase beta. Because this protocol was primarily designed to provide access to agala, there were limitations, including not having stringent selection criteria and the lack of a placebo group.

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“…Many studies show that enzyme replacement therapy (ERT) with human α-Gal A safely reverses the pathogenesis of several major clinical manifestations, specially those due to renal, cardiovascular, peripheral nerve and hearing involvement [5][6][7][8] . The effect of ERT on CNS involvement is still not clear.…”

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White matter lesions in Fabry disease before and after enzyme replacement therapy: a 2-year follow-up

Jardim

Aesse

Vedolin

et al. 2006

Arq. Neuro-Psiquiatr.

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-Purpose: To report the clinical and neuroimaging, central nervous system (CNS) findings of patients with Fabry disease (FD) during 24 months of enzyme replacement therapy (ERT) with agalsidasealpha. Method: Eight patients were included. Six completed 24 months of ERT. Clinical and magnetic resonance imaging (MRI) data were obtained at 0, 12 and 24 months of ERT. White matter lesions (WML) were evaluated as well as their relation to age, symptoms and neurological examination (CNS score). Results: MRI was stable in 3 patients. WML and CNS score worsened in one patient, fluctuated in another, and improved in the sixth patient. In the whole series, there were 15 WML at baseline, and 19 at the 24 th month. In two years, 4 lesions disappeared, whereas 8 appeared. Conclusion: A widespread pattern of silent WML in FD was seen. In two years, some WML appeared, and some disappeared. If these phenomena were related to the natural history, remains to be demonstrated.KEY WORDS: Fabry disease, enzyme-replacement therapy, magnetic resonance imaging, alpha-galactosidase A, white matter lesion.Lesões da substância branca na doença de Fabry antes e depois da terapia de reposição enzimática : um seguimento de 2 anos RESUMO -Objetivo: Relatar os achados neurológicos e de imagem do sistema nervoso central (SNC), observados durante 24 meses de tratamento de reposição enzimática (ERT) com agalsidase-alfa, em pacientes com a doença de Fabry (FD). Método: 8 pacientes foram incluídos; 6 completaram 24 meses de ERT. Os dados foram obtidos aos 0, 12 e 24 meses de ERT. Lesões de substância branca (WML) foram avaliadas assim como sua relação com a idade e o exame neurológico (escore SNC). Resultados: Os achados de ressonân-cia nuclear magnética foram estáveis em 3 pacientes. As WML e o escore SNC pioraram em um caso; flutuaram em um outro caso; e melhoraram no sexto paciente. No todo, havia 15 WML antes da ERT e 19 WML depois de 24 meses de ERT. Em dois anos, 4 lesões desapareceram e 8 novas surgiram. Conclusões: Viu-se um padrão difuso de WML assintomáticas, na FD. Em dois anos, algumas WML surgiram, enquanto outras desapareceram. Resta demonstrar se esses fenômenos fazem parte da história natural da doença. PALAVRAS-CHAVE: doença de Fabry, terapia de reposição enzimática, ressonância nuclear magnética, alfagalactosidase A, lesões de substância branca.Fabry disease (FD) is an X-linked disorder resulting from a mutation of the α-galactosidase A gene at Xq22. The gene defect causes a very low specific activity of the enzyme α-galactosidase (α-Gal A) (NM_000169), which leads to a progressive lysosomal deposition of globotriaosylceramide (GL-3) in vascular endothelium and smooth muscle cells, myocardium, renal epithelium, and the central nervous sys-

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Fabry disease: overall effects of agalsidase alfa treatment

Abstract: Enzyme replacement therapy with agalsidase alfa leads to significant clinical benefits in patients with Fabry disease, and treatment is likely to alter the natural history of this disorder.

Cited by 200 publications

References 21 publications

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

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

Effect and Tolerability of Agalsidase Alfa in Patients with Fabry Disease Who Were Treatment Naïve or Formerly Treated with Agalsidase Beta or Agalsidase Alfa

White matter lesions in Fabry disease before and after enzyme replacement therapy: a 2-year follow-up

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