Journal
PEDIATRIC ALLERGY AND IMMUNOLOGY
Volume 34, Issue 6, Pages -Publisher
WILEY
DOI: 10.1111/pai.13981
Keywords
desensitization; drug allergy; enzyme replacement therapy; hypersensitivity reaction; lysosomal storage diseases
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Lysosomal storage diseases (LSDs) are rare genetic metabolic disorders characterized by the accumulation of glycosaminoglycans in lysosomes due to enzyme deficiency or reduced function. Enzyme replacement therapy (ERT) is the gold standard treatment, but hypersensitivity reactions can occur. Desensitization procedures have been successfully performed to restore ERT in patients with previous hypersensitivity reactions. Standardized testing is necessary to estimate the risk of the procedure and find the safest desensitization protocol.
Lysosomal storage diseases (LSDs) are rare genetic metabolic disorders that cause the accumulation of glycosaminoglycans in lysosomes due to enzyme deficiency or reduced function. Enzyme replacement therapy (ERT) represents the gold standard treatment, but hypersensitivity reaction can occur resulting in treatment discontinuation. Thus, desensitization procedures for different culprit recombinant enzymes can be performed to restore ERT. We searched desensitization procedures performed in LSDs and focused on skin test results, protocols and premedication performed, and breakthrough reactions occurred during infusions. Fifty-two patients have been subjected to desensitization procedures successfully. Skin tests, with the culprit recombinant enzyme, deemed positive in 29 cases, doubtful in two cases, and not performed in four patients. Moreover, 29 of the 52 desensitization protocols used at the first infusion were breakthrough reaction free. Different desensitization strategies have proved safe and effective in restoring ERT in patients with previous hypersensitivity reactions. Most of these events seem to be Type I hypersensitivity reactions (IgE-mediated). Standardized in vivo and in vitro testing is necessary to better estimate the risk of the procedure and find the safest individualized desensitization protocol.
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