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

  • Research Report
  • Chapter
  • First Online:
JIMD Reports – Case and Research Reports, 2012/6

Part of the book series: JIMD Reports ((JIMD,volume 9))

Abstract

Anderson-Fabry disease (AFD) is a multiorgan X-linked lysosomal storage disease that particularly affects the heart, kidneys, and cerebrovascular system. Current treatment is enzyme replacement therapy (ERT) with agalsidase beta (Fabrazyme®, Genzyme Corporation, Cambridge, MA, USA) or agalsidase alfa (Replagal®, Shire Human Genetic Therapies AB, Lund, Sweden). It was recommended that patients switch to agalsidase alfa due to a manufacturing shortage of agalsidase beta beginning in June 2009. This study assessed the effect of switching to agalsidase alfa on clinical outcomes in patients with AFD previously treated with agalsidase beta. Ten patients (seven male, three female) with genetically confirmed AFD and at least 48 months’ continuous data collected during treatment with agalsidase beta 1 mg/kg every other week were switched to agalsidase alfa 0.2 mg/kg every other week for at least 20 months, with prospective clinical evaluations every 6 months. Pre-switch data was collected retrospectively from patient charts. Cardiac functional parameters were assessed using magnetic resonance imaging. Results showed that renal function was normal (estimated glomerular filtration rate ≥90 mL/min/1.73 m2) in 8 of 10 patients prior to agalsidase alfa and generally remained stable after the switch. Cardiac mass decreased significantly (p < 0.05 vs pre-ERT) after agalsidase beta and remained unchanged after switching to agalsidase alfa. Symptoms of pain and health status scores did not deteriorate during agalsidase alfa therapy. Adverse events were mostly mild and infusion related. In conclusion, switching to agalsidase alfa was relatively well tolerated and associated with stable clinical status and preserved renal and cardiac function.

Competing interests: None declared

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Author information

Authors and Affiliations

  1. Department of Nephrology, University “Federico II”, Via Pansini 5, 80131, Napoli, Italy

    A. Pisani, B. Visciano, I. Capuano, M. Sabbatini & E. Riccio

  2. Department of Cardiovascular and Immunological Science, University “Federico II”, Napoli, Italy

    L. Spinelli

  3. Department of Biomorphological and Functional Science, University “Federico II”, Napoli, Italy

    G. Messalli & M. Imbriaco

Authors
  1. A. Pisani
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  2. L. Spinelli
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  3. B. Visciano
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  4. I. Capuano
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  5. M. Sabbatini
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  6. E. Riccio
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  7. G. Messalli
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  8. M. Imbriaco
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Corresponding author

Correspondence to A. Pisani .

Editor information

Editors and Affiliations

  1. Sektionen für Humangenetik und Klinische, Medizinische Universität Innsbruck, Innsbruck, Austria

    Johannes Zschocke (Editors-in-Chief) (Editors-in-Chief)

  2. Biological Sciences, Michigan Technological University, Houghton, Michigan, USA

    K. Michael Gibson (Editors-in-Chief) (Editors-in-Chief)

  3. Department of Biochemistry Genetics Unit, University of Oxford, Oxford, UK

    Garry Brown (Editors) (Editors)

  4. Medical Center Department of Pediatrics IGMD, Radboud University Nijmegen, Nijmegen, Netherlands

    Eva Morava (Editors) (Editors)

  5. Center for Child and Adolescent Medicine, Heidelberg University Hospital, Heidelberg, Germany

    Verena Peters (Managing Editor) (Managing Editor)

Additional information

Communicated by: Robin Lachmann

Appendices

Appendix Details of the Contributions of Individual Authors

AP was primarily involved in patient management, literature search, and preparation of manuscript. BV, IC, MS, and ER assisted in patient management and were involved in manuscript preparation. LS was involved in the literature search. GM performed the cMRI analysis. MI was involved in the literature search and manuscript preparation. All authors read and approved the final manuscript. The results presented in this chapter have not been published previously in whole or part, except in abstract form.

Guarantor

Dr. Antonio Pisani serves as guarantor for the chapter, accepts full responsibility for the work and/or the conduct of the study, had access to the data, and controlled the decision to publish.

Competing Interest Statement

No authors at any time received payment or services from a third party for any aspect of the submitted work and they have nothing to declare.

Provide Details of Funding

Editorial assistance for manuscript preparation was provided by Tracy Harrison and Mary Hines, in Science Communications, Springer Healthcare.

Details of Ethics Approval and Patient Consent

The study was conducted in accordance with the Declaration of Helsinki and was approved by the institutional ethics committee. All patients switched to agalsidase alfa and included in the final study population provided written informed consent.

Synopsis

Patients with Anderson-Fabry disease can be safely switched from long-term treatment with agalsidase beta to agalsidase alpha and maintain their health status, without any worsening of renal function, cardiac mass, or pain symptoms.

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Pisani, A. et al. (2012). Effects of Switching from Agalsidase Beta to Agalsidase Alfa in 10 Patients with Anderson-Fabry Disease. In: Zschocke, J., Gibson, K.M., Brown, G., Morava, E., Peters, V. (eds) JIMD Reports – Case and Research Reports, 2012/6. JIMD Reports, vol 9. Springer, Berlin, Heidelberg. https://doi.org/10.1007/8904_2012_177

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  • DOI: https://doi.org/10.1007/8904_2012_177

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  • Publisher Name: Springer, Berlin, Heidelberg

  • Print ISBN: 978-3-642-35517-2

  • Online ISBN: 978-3-642-35518-9

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