2020
DOI: 10.1016/j.ymgmr.2020.100650
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Anti-drug antibody formation in Japanese Fabry patients following enzyme replacement therapy

Abstract: Enzyme replacement therapy (ERT) for Fabry disease (deficiency of α-galactosidase A, α-Gal) with recombinant α-Gals (agalsidase alfa and agalsidase beta) is widely available and improves some of the clinical manifestations and biochemical findings. However, recent reports suggest that recurrent administration of recombinant enzymes often induces the formation of anti-drug antibodies, which may have a negative impact on the outcome of the therapy. We examined the formation of anti-drug antibodies using blood sa… Show more

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Cited by 6 publications
(8 citation statements)
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“…Using serial dilutions of sera from FD patients, Linthorst and colleagues provided relative ADA titers and demonstrated cross-reactivity of ADAs with respect to agalsidase-alfa and agalsidase-beta [4]. In general, relative titers can be expressed by using sera from healthy controls, from patients who ultimately did not develop ADAs, or from patients who are ERT-naïve [2,4,49,65]. Some other studies used animal-derived anti-AGAL antibodies as positive controls [59].…”
Section: Methods For Determination Of Adasmentioning
confidence: 99%
“…Using serial dilutions of sera from FD patients, Linthorst and colleagues provided relative ADA titers and demonstrated cross-reactivity of ADAs with respect to agalsidase-alfa and agalsidase-beta [4]. In general, relative titers can be expressed by using sera from healthy controls, from patients who ultimately did not develop ADAs, or from patients who are ERT-naïve [2,4,49,65]. Some other studies used animal-derived anti-AGAL antibodies as positive controls [59].…”
Section: Methods For Determination Of Adasmentioning
confidence: 99%
“…The patient was treated with 1.0 mg/kg body weight agalsidase beta biweekly, and monitoring of the anti-drug antibody titer, serum-mediated α-Gal inhibition rate, plasma Lyso-Gb3 concentration, and urinary Gb3 excretion was performed according to the methods previously described [ 6 8 ]. No increase in the anti-drug antibody titer (Fig.…”
Section: Case Presentationmentioning
confidence: 99%
“…No increase in the anti-drug antibody titer (Fig. 1 a, green line; the cut-off value indicating antibody-positive: ∆OD 490 nm > 0.250 [ 6 ]) or serum-mediated inhibition rate (Fig. 1 b, green line; the cut-off value indicating inhibition-positive: > 49% [ 6 ]) was found in the patient during ERT.…”
Section: Case Presentationmentioning
confidence: 99%
“…The presence of the blood-brain barrier (BBB) hinders current commercial recombinant enzymes from reaching the most critical site for clinical manifestation 9 , the central nervous system, leaving disorders with neurodegenerative pathology untreatable except for a few enzymes that can be intracerebroventricularly administered 10 , 11 . Another issue is the generation of neutralizing antibodies to therapeutic biologics, especially when infused into patients with a null mutation in question 12 . Solutions for these concerns are to modulate biologics with the addition of peptides to pass over the BBB 13 and to provide the enzyme activity in question to native enzymes through the replacement of amino acids in the binding pocket for the substrate 14 .…”
Section: Introductionmentioning
confidence: 99%
“…A concern is a dose of alpha-galactosidase (aGLA) with a mannose-6-phosphate (M6P) moiety to be supplied by exogenous genetically modified hematological cells. In a previous study, we calculated a naïve cell number of 1.7 × 10 12 , which releases aGLA equivalent to clinically used biologics 20 . When patients with Fabry disease received kidney transplantation, cross-correction in other organs, such as the heart, that suffered from aGLA dysfunction was not recognized 21 .…”
Section: Introductionmentioning
confidence: 99%