Successful desensitization protocol to alglucosidase and avalglucosidase alfa in a patient with infantile-onset Pompe disease

Abstract

Infantile-onset Pompe disease is a lysosomal disease characterized by cardiomyopathy and muscle weakness that, without specific treatment, is fatal within the first two years of life. We present the case of an infant who developed anaphylaxia to enzyme replacement therapy with alglucosidase-alfa. We provide a desensitization protocol to alglucosidase-alfa and, for the first time, a desensitization protocol to avalglucosidase-alfa, both delivered in a reasonable time of <6 h, and without any further reactions in the patient.

Keywords: Alglucosidase alfa; Anaphylaxia; Avalglucosidase alfa; Desensitization; Infantile-onset Pompe disease; Infusion associated reaction; Pompe disease.