Our patient with mucopolysaccharidosis type VI received enzyme replacement therapy with recombinant human arylsulfatase B (galsulfase [Naglazyme, BioMarin Pharmaceutical Inc, Novato, CA]) shortly after approval by the US Food and Drug Administration. After 1 month of weekly infusions, the patient developed significant infusion-associated reactions and could not tolerate therapy at the recommended infusion rate. We were able to continue treatment successfully by the addition of steroids to the premedication regimen and by significantly reducing the rate of drug infusion. Over the next several months, the patient's infusion rate was slowly increased and the premedications were weaned. We demonstrate that by significantly reducing the rate of infusions and adjusting the premedication regimen, galsulfase infusions can continue with no additional observance of infusion-associated reactions.
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March 2008
Experience & Reason|
March 01 2008
Successful Management of Difficult Infusion-Associated Reactions in a Young Patient With Mucopolysaccharidosis Type VI Receiving Recombinant Human Arylsulfatase B (Galsulfase [Naglazyme])
Katherine H. Kim, MS;
Katherine H. Kim, MS
aDivision of Genetics, Birth Defects, and Metabolism, Children's Memorial Hospital, Chicago, Illinois
bDepartment of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
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Celeste Decker, MD;
Celeste Decker, MD
cBioMarin Pharmaceutical Inc, Novato, California
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Barbara K. Burton, MD
Barbara K. Burton, MD
aDivision of Genetics, Birth Defects, and Metabolism, Children's Memorial Hospital, Chicago, Illinois
bDepartment of Pediatrics, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
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Address correspondence to Katherine H. Kim, MS, Children's Memorial Hospital, 2300 Children's Plaza, Box 59, Chicago, IL 60614. E-mail: kkim@childrensmemorial.org
Pediatrics (2008) 121 (3): e714–e717.
Article history
Accepted:
July 27 2007
Citation
Katherine H. Kim, Celeste Decker, Barbara K. Burton; Successful Management of Difficult Infusion-Associated Reactions in a Young Patient With Mucopolysaccharidosis Type VI Receiving Recombinant Human Arylsulfatase B (Galsulfase [Naglazyme]). Pediatrics March 2008; 121 (3): e714–e717. 10.1542/peds.2007-0665
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