Clinical observations on enzyme replacement therapy in patients with Fabry disease and the switch from agalsidase beta to agalsidase alfa

Lin, Hui-Chen; Huang, Yu-Ren; Liao, Hsuan Chieh; Liu, Hao Chuan; Hsu, Ting-Rong; Shen, Chia I.; Li, Shao Tzu; Li, Cheng Fang; Lee, Li Hong; Lee, Pi Chang; Huang, Chun Kai; Chiang, Chuan Chi; Lin, Shuan Pei; Niu, Dau Ming

doi:10.1016/j.jcma.2013.11.006

Cited by 15 publications

(31 citation statements)

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“…The follow-up time was 13–46 months (median: 20 months). Four of the FD patients (case 1, 14, 15, and 24) had received agalsidase beta before June 2009 and then changed to agalsidase alfa due to the shortage of agalsidase beta supply worldwide [17]. All of other patients only received agalsidase alfa treatment.…”

Section: Resultsmentioning

confidence: 99%

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Globotriaosylsphingosine (lyso-Gb3) might not be a reliable marker for monitoring the long-term therapeutic outcomes of enzyme replacement therapy for late-onset Fabry patients with the Chinese hotspot mutation (IVS4+919G>A)

Liu

Lin

Yang

et al. 2014

Orphanet J Rare Dis

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BackgroundIn Taiwan, DNA-based newborn screening showed a surprisingly high incidence (1/875 in males and 1/399 in females) of a cardiac Fabry mutation (IVS4 + 919G > A). However, the natural course, long-term treatment outcomes and suitable biomarkers for monitoring the therapeutic outcomes of these patients are largely unknown.MethodsFabry disease (FD) patients who had received enzyme replacement therapy (ERT) for more than 1 year were enrolled in this study from December 2008 to April 2013. Periodic echocardiography and serum globotriaosylsphingosine (lyso-Gb3) analysis were carried out. Before and after ERT, left ventricular mass index (LVMI) and serum lyso-Gb3 level were compared and the correlation between the change of LVMI and the change of serum lyso-Gb3 were also analyzed.ResultsThirty-six patients, in four patient groups, were enrolled: (1) 16 males with IVS4 + 919G > A mutation; (2) 7 females with IVS4 + 919G > A mutation; (3) 2 males with classical mutations; and (4) 11 females with classical mutations. The follow-up period was 13–46 months. There were significant LVMI reductions after ERT in all four groups after excluding confounding factors. However, interestingly, serum lyso-Gb3 decreased significantly in the early period after ERT in all groups, but increased gradually after an average of 11.1 months after ERT in late-onset male and female Fabry groups, even when their LVMI still decreased or remained stable. Furthermore, there was no correlation between the change of serum lyso-Gb3 and the change of LVMI in both classical and IVS4 + 919G > A FD patients.ConclusionAlthough lyso-Gb3 has a high diagnostic sensitivity in late-onset Fabry patients and has a good response to ERT during the early stages, it might not be a reliable marker for monitoring the long-term therapeutic outcomes of ERT for late-onset Fabry patients with the Chinese hotspot mutation (IVS4 + 919G > A).

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Section: Resultsmentioning

confidence: 99%

“…All patients received ERT every 2 weeks with agalsidase beta (1 mg/kg) before 2010 and agalsidase alfa (0.2 mg/kg) since 2010, due to the shortage of agalsidase beta in Taiwan [17]. A comprehensive physical examination was carried out, echocardiography and serum lyso-Gb3 were evaluated prior to and during the follow-up.…”

Section: Methodsmentioning

confidence: 99%

Globotriaosylsphingosine (lyso-Gb3) might not be a reliable marker for monitoring the long-term therapeutic outcomes of enzyme replacement therapy for late-onset Fabry patients with the Chinese hotspot mutation (IVS4+919G>A)

Liu

Lin

Yang

et al. 2014

Orphanet J Rare Dis

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“…A short time comparison of three different doses of agalsidase alfa 0.2 mg/kg every other week, 0.1 mg/kg weekly and 0.2 mg/kg weekly each given for four weeks in a crossover study in 18 patients showed no statistically significant difference between the doses (Hughes et al 2013). Other studies have shown no significant change when switching from agalsidase beta 1 mg/kg every other week to agalsidase alfa 0.2 mg/kg every other week (Pisani et al 2013;Lin et al 2014;Tsuboi and Yamamoto 2014). One study with nonrandomized switch from agalsidase beta 1 mg/kg every other week to agalsidase alfa 0.2 mg/kg every other week or agalsidase beta 0.3 -0.5 mg/kg every other week was associated with progression in microalbuminuria and Fabry related symptoms after 1 year (Weidemann et al 2014).…”

Section: Discussionmentioning

confidence: 98%

A prospective 10‐year study of individualized, intensified enzyme replacement therapy in advanced Fabry disease

Schiffmann¹,

Swift²,

Wang

et al. 2015

J of Inher Metab Disea

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“…Standardization of these methods would allow comparison of lyso-GL-3 data from different studies. Moreover, discussing our results in the context of results of recently published agalsidase beta -agalsidase alfa switch studies is hampered by the fact that these studies did not report plasma lyso-GL-3 or GL-3 data (Pisani et al 2013), included Fabry-variant patients with normal or low lyso-GL-3 levels at baseline (six out of nine patients) (Lin et al 2014), and included many female patients with low lyso-GL-3 at baseline (seven out of 11 patients) (Tsuboi and Yamamoto 2014).…”

Section: Discussionmentioning

confidence: 99%

Reduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry Disease

Göker-Alpan

Gambello

Maegawa³

et al. 2015

JIMD Reports

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Introduction: Agalsidase alfa and agalsidase beta, recombinant enzyme preparations for treatment of Fabry disease (FD), have different approved dosing schedules: 0.2 mg/kg and 1.0 mg/kg every other week (EOW), respectively.Methods: This open-label, multicenter, exploratory phase 4 study evaluated plasma globotriaosylsphingosine (lyso-GL-3) and plasma and urine globotriaosylceramide (GL-3) levels at baseline and 2, 4, and 6 months after the switch from agalsidase alfa (0.2 mg/kg EOW for !12 months) to agalsidase beta (1.0 mg/kg EOW) in 15 male patients with FD. Immunoglobulin (Ig)G antidrug antibody titers were assessed, and safety was monitored throughout the study.Results: Plasma lyso-GL-3 concentrations decreased significantly within 2 months after switch and reductions continued through month 6 (mean absolute changes, À12.8, À16.1, and À16.7 ng/mL at 2, 4, and 6 months, respectively; all P < 0.001). The mean percentage reduction from baseline was 39.5% (P < 0.001) at month 6. For plasma GL-3, the mean absolute change from baseline (À0.9 mg/mL) and percentage reduction (17.9%) at month 6 were both significant (P < 0.05). Urine GL-3 measurements showed intra-patient variability and changes from baseline were not significant. No clinical outcomes were assessed in this 6-month study, and, therefore, no conclusions can be drawn regarding the correlation of observed reductions in glycosphingolipid concentrations with clinically relevant outcomes. There were no differences in IgG antidrug antibody titers between the two enzymes. The switch from agalsidase alfa to agalsidase beta was well tolerated.Conclusion: Plasma lyso-GL-3 and GL-3 levels reduced after switching from agalsidase alfa to agalsidase beta, indicating that agalsidase beta has a greater pharmacodynamic effect on these markers at the recommended dose. These data further support the use of agalsidase beta 1.0 mg/kg EOW as enzyme replacement therapy in FD.

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Clinical observations on enzyme replacement therapy in patients with Fabry disease and the switch from agalsidase beta to agalsidase alfa

Abstract: The switch of ERT from agalsidase beta to agalsidase alfa appears to be safe after 1 year of follow-up for Taiwanese patients with Fabry disease.

Cited by 15 publications

References 38 publications

Globotriaosylsphingosine (lyso-Gb3) might not be a reliable marker for monitoring the long-term therapeutic outcomes of enzyme replacement therapy for late-onset Fabry patients with the Chinese hotspot mutation (IVS4+919G>A)

Globotriaosylsphingosine (lyso-Gb3) might not be a reliable marker for monitoring the long-term therapeutic outcomes of enzyme replacement therapy for late-onset Fabry patients with the Chinese hotspot mutation (IVS4+919G>A)

A prospective 10‐year study of individualized, intensified enzyme replacement therapy in advanced Fabry disease

Reduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry Disease

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