Abstract
Mucopolysaccharidosis type VI (MPS VI) is a progressive, chronic, and multisystem lysosomal storage disease. Enzyme replacement therapy (ERT) with the recombinant human arylsulfatase B enzyme (galsulfase [Naglazyme]) is recommended as first-line therapy. It is generally reported as safe and well tolerated. Frequently observed mild to moderate infusion-related reactions which can be easily handled by reducing or interrupting the infusion and/or administering additional antihistamines, antipyretics, and corticosteroids are mostly mediated by non-IgE mechanisms. Here we report two children with MPS VI who experienced IgE-mediated reactions with galsulfase at the second year of the therapy. One child had anaphylaxis and the other had urticarial eruptions. They could receive ERT after successful rapid desensitization. To our knowledge, this is the second report on galsulfase allergy with IgE-mediated reaction. It is important to recognize IgE-mediated reactions since they can be life-threatening and do not respond to the standard therapies. We recommend allergy skin tests in the evaluation of infusion-related reactions unresponsive to standard therapies, so that continuation of ERT will be feasible after successful desensitization.
Competing interests: None declared
This is a preview of subscription content, log in via an institution to check access.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
Abbreviations
- BWH:
-
Brigham and Women’s Hospital
- ERT:
-
Enzyme replacement therapy
- GAG:
-
Glycosaminoglycan
- MPS VI:
-
Mucopolysaccharidosis type VI
- N-acetylgalactosamine-4-sulfatase:
-
Arylsulfatase B
References
Begin P, Chapdelaine H, Lemyre E, Paradis L, Roches A (2013) Successful desensitization in a type VI mucopolysaccharidosis patient with probable IgE-mediated allergy to galsulfase [Naglazyme]. Ann Allergy Asthma Immunol 110:55–56
Brennan P, Bouza RT, Hsu FI, Sloane DE, Castells M (2009) Hypersensitivity reactions to mAbs: 105 desensitizations in 23 patients, from evaluation to treatment. J Allergy Clin Immunol 124:1259–1266
Castells M (2006a) Desensitization for drug allergy. Curr Opin Allergy Clin Immunol 6:476–481
Castells M (2006b) Rapid desensitization for hypersensitivity reactions to chemotherapy agents. Curr Opin Allergy Clin Immunol 6:271–277
Castells M, Sancho-Serra Mdel C, Simarro M (2012) Hypersensitivity to antineoplastic agents: mechanisms and treatment with rapid desensitization. Cancer Immunol Immunother 61:1575–1584
Harmatz P, Giugliani R, Schwartz I et al (2006) Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr 148:533–539
Harmatz P, Giugliani R, Schwartz IV et al (2008) Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol Genet Metab 94:469–475
Harmatz PR, Garcia P, Guffon N et al (2013) Galsulfase (NaglazymeR) therapy in infants with mucopolysaccharidosis VI. J Inherit Metab Dis. doi:10.1007/s10545-013-9654-7 [Epub ahead of print]
Horovitz D, Magalhaes T, Acosta A et al (2013) Enzyme replacement therapy with galsulfase in 34 children younger than five years of age with MPS VI. Mol Genet Metab 109:62–69
Kim KH, Decker C, Burton BK (2008) Successful management of difficult infusion-associated reactions in a young patient with mucopolysaccharidosis type VI receiving recombinant human arylsulfatase B (galsulfase [Naglazyme]). Pediatrics 3:714–717
Liu A, Fanning L, Chong H et al (2011) Desensitization regimens for drug allergy: state of the art in the 21st century. Clin Exp Allergy 41:1679–1689
Miebach E (2009) Management of infusion-related reactions to enzyme replacement therapy in a cohort of patients with mucopolysaccharidosis disorders. Int J Clin Pharmacol Ther 47:S100–S106
Valayannopoulos V, Nicely H, Harmatz P, Turbeville S (2010) Mucopolysaccharidosis VI. Orphanet J Rare Dis 5:5
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Additional information
Communicated by: Olaf Bodamer, MD
Appendices
Take-Home Message
Allergy skin tests should be performed in the evaluation of infusion-related reactions unresponsive to standard therapies, so that continuation of ERT will be feasible after successful desensitization.
Compliance with Ethics Guidelines
Conflict of Interest
Zeynep Tamay, Gulden Gökçay, Fatih Dilek, Mehmet C Balci, Deniz Ozceker, Mubeccel Demirkol, and Nermin Guler declare that they have no conflict of interest.
Competing Interests
All procedures followed were in accordance with the ethical standards of the responsible committee on human experimentation (institutional and national) and with the Helsinki Declaration of 1975, as revised in 2000. Informed consent was obtained from all patients for being included in the study.
This article does not contain any animal subjects performed by the any of the authors.
Details of the Contributions of Individual Authors
Zeynep Tamay designed and reported the cases; Fatih Dilek, Mehmet C Balci, and Deniz Ozceker conducted the rapid desensitization procedures; Gulden Gokcay revised the language and also revised the intellectual content with Mubeccel Demirkol and Nermin Guler.
Rights and permissions
Copyright information
© 2016 SSIEM and Springer-Verlag Berlin Heidelberg
About this chapter
Cite this chapter
Tamay, Z. et al. (2016). Rapid Desensitization for Immediate Hypersensitivity to Galsulfase Therapy in Patients with MPS VI. In: Morava, E., Baumgartner, M., Patterson, M., Rahman, S., Zschocke, J., Peters, V. (eds) JIMD Reports, Volume 30. JIMD Reports, vol 30. Springer, Berlin, Heidelberg. https://doi.org/10.1007/8904_2016_542
Download citation
DOI: https://doi.org/10.1007/8904_2016_542
Received:
Revised:
Accepted:
Published:
Publisher Name: Springer, Berlin, Heidelberg
Print ISBN: 978-3-662-53680-3
Online ISBN: 978-3-662-53681-0
eBook Packages: Biomedical and Life SciencesBiomedical and Life Sciences (R0)