Dataset Information

Clinical course of patients with Fabry disease who were switched from agalsidase-? to agalsidase-?.

ABSTRACT:

Background

Between 2009 and 2012, there was a worldwide shortage of agalsidase-? for the treatment of Fabry disease. Therefore, alternative treatments were needed, including switching to a different enzyme-replacement therapy.

Purpose

This is an ongoing observational study assessing the effects of switching from agalsidase-? (1.0?mg/kg every other week) to agalsidase-? (0.2?mg/kg every other week) in 11 patients with Fabry disease.

Methods

Clinical data were collected for 5 years-2 years before switching and 3 years after switching.

Results

Measures of renal function such as estimated glomerular filtration rate remained stable during the 3 years after switching to agalsidase-?. Improvements in cardiac mass were recorded in both male and female patients 12 months after switching to agalsidase-?, and the benefit was maintained during 36 months of follow-up. There was no significant difference in the severity of pain experienced by patients before and after switching enzyme-replacement therapy, and no difference in quality-of-life parameters. Agalsidase-? was generally well tolerated, and no patients experienced allergy or developed antibodies to agalsidase-?.

Conclusion

This observational study supports the safety of switching from agalsidase-? to agalsidase-? at the approved doses, with no loss of efficacy. It also suggests that if an infusion-related allergic reaction occurs in a patient receiving agalsidase-?, switching to agalsidase-? may be a viable option.

SUBMITTER: Tsuboi K

PROVIDER: S-EPMC4189383 | biostudies-literature | 2014 Oct

REPOSITORIES: biostudies-literature

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Publications

Clinical course of patients with Fabry disease who were switched from agalsidase-β to agalsidase-α.

Tsuboi Kazuya K Yamamoto Hiroshi H

Genetics in medicine : official journal of the American College of Medical Genetics 20140320 10

<h4>Background</h4>Between 2009 and 2012, there was a worldwide shortage of agalsidase-β for the treatment of Fabry disease. Therefore, alternative treatments were needed, including switching to a different enzyme-replacement therapy.<h4>Purpose</h4>This is an ongoing observational study assessing the effects of switching from agalsidase-β (1.0 mg/kg every other week) to agalsidase-α (0.2 mg/kg every other week) in 11 patients with Fabry disease.<h4>Methods</h4>Clinical data were collected for 5 ...[more]

PMID: 24651606

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Project description:BackgroundPatients with Fabry disease (FD, α-galactosidase A deficiency or absence) accumulate glycosphingolipids, leading to progressive dysfunction of kidneys, heart and nervous system. Generalizable real-world outcomes following agalsidase beta treatment initiation outside trials are limited. We investigated the associations of long-term agalsidase beta treatment with estimated glomerular filtration rate (eGFR) changes over time and the risk of developing a composite clinical event in a matched analysis of treated and untreated patients with FD.MethodsAgalsidase beta-treated adult patients (aged ≥16 years) from the Fabry Registry and adult untreated patients from a natural history cohort were matched 1:1 and X:X (with one occurrence and multiple occurrences of each untreated patient, respectively) by sex, phenotype, age and (for eGFR slope analysis) baseline eGFR. Outcomes included eGFR slope over 5 years and composite clinical event risk (cardiovascular, cerebrovascular or renal event, or death) over 10+ years. As a surrogate indicator of therapeutic response in paediatric patients, the percentage experiencing normalization in plasma globotriaosylceramide (GL-3) from treatment initiation was assessed in patients aged 2 to <16 years.ResultsOverall, eGFR slopes for 1:1-matched untreated and treated adult patients [122 pairs (72.1% male)] were -3.19 and -1.47 mL/min/1.73 m2/year, respectively (reduction in rate of decline = 53.9%, P = .007), and for X:X-matched [122 untreated/950 treated (59.4% male)] were -3.29 and -1.56 mL/min/1.73 m2/year, respectively (reduction in rate of decline = 52.6%, P < .001). Agalsidase beta treatment was associated with lower risk of clinical events, with hazard ratios of 0.41 (P = .003) and 0.67 (P = .008) for 1:1-matched and X:X-matched analyses, respectively. Plasma GL-3 declined markedly in paediatric patients and normalized in most within 6 months of treatment initiation.ConclusionAgalsidase beta treatment preserves kidney function and delays progression to severe clinical events among adult patients with FD. Plasma GL-3 levels analysed in paediatric patients showed normalization of elevated pre-treatment levels in most patients.