Two-year follow-up after drug desensitization in mucopolysaccharidosis.

ABSTRACT:

Background

Mucopolysaccharidosis (MPS) type 1 S and type 2 are rare lysosomal storage disorders characterized by impaired enzyme production, resulting in glycosaminoglycans accumulation within lysosomes. Enzyme Replacement Therapy (ERT) with laronidase and idursulfase are first line treatments, respectively. However, infusion-related hypersensitivity reactions (HR) may lead to ERT discontinuation. Thus, desensitization can be performed to restore ERT.

Methods

We report on a two-year follow-up after a combined desensitization approach in two MPS patients experiencing HR to ERT. This approach consists of intravenous rapid desensitization combined with the subcutaneous allergen immunotherapy-like desensitization with the culprit recombinant enzyme.

Results

The first pati

SUBMITTER: Spataro F 

PROVIDER: S-EPMC11673970 | biostudies-literature | 2024 Dec

REPOSITORIES: biostudies-literature