When should ERT be started in fabry disease

When should ERT be started in fabry disease and what are the risks?

Published guidelines suggest that ERT be initiated at the time of diagnosis in males older than 16 years and at the onset of significant symptoms in males younger than 16 years, and should be considered in asymptomatic males between the ages of 10 and 13 years.

ERT is considered safe for use in children based on a pediatric trial. ERT should be started in heterozygous females with clinical symptoms or when there is evidence of organ involvement (i.e., proteinuria, cardiac dysfunction).

ERT has been associated with infusion-related reactions for which pre-medication with an antipyretic and antihistamine is recommended.

Type 1 Classically affected males typically develop IgG antibodies to ERT, and the titers tend to decrease with continued treatment.

These antibodies may be neutralizing but have not affected efficacy based on kidney biopsies after 5 years of treatment.

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