Is premedication a necessity before galsulfase replacement therapy?

Abstract

Mucopolysaccharidosis (MPS) type VI (Maroteaux-Lamy syndrome) is an autosomal recessive disorder that results in accumulation of dermatan sulfate in tissues. [1] Harmatz P. Giugliani R. Schwartz I. et al. MPS VI Phase 3 Study GroupEnzyme 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. 2006; 148: 533-539 Abstract Full Text Full Text PDF PubMed Scopus (322) Google Scholar The only known treatment strategy for this life-threatening condition is weekly enzyme (N-acetylgalactosamine 4-sulfatase; galsulfase, Biomarin Pharmaceutical Inc, Novato, California) replacement therapy. During enzyme replacement, half of the patients being studied developed infusion-related allergic reactions, and after repeated infusions, 16% of them had anaphylactoid-type symptoms. [2] Harmatz P. Giugliani R. Schwartz I.V. et al. MPS VI Study GroupLong-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. 2008; 94: 469-475 Abstract Full Text Full Text PDF PubMed Scopus (192) Google Scholar Such symptoms mostly involved flushing but also included urticarial, itching, and other severe allergic reactions. Although these reactions are frequently assumed to be non–IgE-mediated, IgE-mediated mechanisms are still not ruled out. 3 Tamay Z, Gokcay G, Dilek F, et al. Rapid desensitization for ımmediate hypersensitivity to galsulfase therapy in patients with MPS VI [published online March 8, 2016]. JIMD Rep. http://dx.doi.org/10.1007/8904_2016_542. Google Scholar , 4 Bégin P. Chapdelaine H. Lemyre E. Paradis L. Des Roches A. Successful desensitization in a type VI mucopolysaccharidosis patient with probable IgE-mediated allergy to galsulfase [Naglazyme]. Ann Allergy Asthma Immunol. 2013; 110: 55-56 Abstract Full Text Full Text PDF PubMed Scopus (9) Google Scholar In this article, we describe a galsulfase desensitization protocol without premedication compared with premedication in a child with MPS type VI.