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Enzyme replacement therapy (ERT) with alglucosidase alfa has improved clinical outcomes and prolonged survival for patients with infantile Pompe disease (IPD). However, patients characterised as Cross-Reactive Immunological Material (CRIM)negative (CN) mount an immune response against ERT resulting in clinical decline and, ultimately, death. A prophylactic immune tolerance induction (ITI) protocol has been successful in preventing the development of antibody titres for these patients. A subset of (CP) patients also mount an immune response with poor clinical outcomes, these at-risk patients are diffi cult to identify. With the advent of newborn screening for Pompe disease, preventative treatment to circumvent an immune response is warranted; therefore, our study aimed to assess the safety and effi cacy of prophylactic ITI in the naive setting for both CP and CN IPD patients.

Immunological Factors in Pompe Disease Management: Clinical Experience and Implications for Newborn Screening