Abstract
Enzyme-replacement therapy (ERT) with alglucosidase alfa has improved the lifespan of patients with classic infantile Pompe disease, although ERT is not effective in a subset of patients who mount an immune response to the exogenous enzyme. We studied the development of antibodies in response to ERT and its effect on clinical outcomes in 11 patients with classic infantile Pompe disease treated with ERT since 1999 for a median of 4.2 years (range 3 months to 12 years).
Highlights
Enzyme-replacement therapy (ERT) with alglucosidase alfa has improved the lifespan of patients with classic infantile Pompe disease, ERT is not effective in a subset of patients who mount an immune response to the exogenous enzyme
We studied the development of antibodies in response to ERT and its effect on clinical outcomes in patients with classic infantile Pompe disease treated with ERT since 1999 for a median of 4.2 years
Earlier initiation of ERT may prevent the formation of a severe immune response
Summary
Enzyme-replacement therapy (ERT) with alglucosidase alfa has improved the lifespan of patients with classic infantile Pompe disease, ERT is not effective in a subset of patients who mount an immune response to the exogenous enzyme. Antibody formation to enzyme therapy in classic infantile Pompe disease: implications of patient age C van Gelder1*, M Kroos2, L Özkan2, I Plug1, A Reuser2, A van der Ploeg1 From Proceedings of the 6th European Symposium: Steps Forward in Pompe Disease Berlin, Germany.
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