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Medicina & Laboratorio

2022, Number 3

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Medicina & Laboratorio 2022; 26 (3)

Hypersensitivity reactions to alglucosidase alfa in Pompe disease: two cases of successful desensitization

Ortiz-Giraldo B, Santamaría-Salazar LC, Mopán-Tique JM

Language: Spanish
References: 26
Page: 287-296
PDF size: 91.24 Kb.


ABSTRACT

Pompe disease is a low prevalence autosomal recessive neuromuscular disorder, caused by total or partial deficiency of the acid alpha-glucosidase (GAA) enzyme, and its only available enzyme replacement therapy is the recombinant alglucosidase alfa. Infusion-associated adverse reactions occur frequently. Two cases of successful desensitization with alglucosidase alfa using protocols with a target dose of 20 mg/kg administered biweekly are reported; the first was a girl who had a history of serious adverse reaction at the age of 15 months, and undergone to a scheme with an initial dilution of 1/10,000,000 with 28 steps and a total duration of 13.1 hours. In the second case, the patient had a severe adverse reaction at the age of 4 years, a 22-step protocol was used with an initial concentration of 1/1,000,000 and a total duration of 7.2 hours. In conclusion, in patients with Pompe disease who presented adverse reactions during enzyme replacement therapy with alglucosidase alfa, it is possible to perform desensitization every two weeks with the standard dose of 20 mg/kg, and progressively achieve the usual administration of the infusion.


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