Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease.
Harmke A van KootenImke A M DittersMarianne Hoogeveen-WesterveldEdwin H JacobsJohanna M P van den HoutPieter A van DoornW W M Pim PijnappelAns T van der PloegNadine A M E van der BeekPublished in: Orphanet journal of rare diseases (2022)
Ninety-one percent of childhood onset Pompe patients developed anti-rhGAA antibodies (above background level), a minority of whom had high antibody titers at repeated time points, which do not seem to interfere with clinical outcome. High antibody titers may be associated with the occurrence of IARs. Although the majority of patients does not develop high titers; antibody titers should be determined in case of clinical deterioration.