Immune response to enzyme replacement therapy in lysosomal storage disorder patients and animal models

The lysosomal storage disorders (LSD) are a group of severe multiple pathol ogy disorders characterized by enzyme deficiencies which cause the lysosoma l accumulation of undegraded or partially degraded macromolecules. Enzyme r eplacement therapy (ERT) has been developed as a therapy for LSD patients. However, immune responses to ERT have been reported in some individuals fro m LSD animal model and LSD human patient studies. Antibodies can have adver se effects during ERT, which include hypersensitivity/anaphylactic react io ns, enzyme inactivation, altered targeting, and increased enzyme turnover. The monitoring of antibody production during replacement therapy is an impo rtant consideration for patient management, as high-titer antibodies can af fect the safety and efficacy of the therapy. (C) 1999 Academic Press.