Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?

被引：3

作者：

Lenders, Malte ^{[1
]}

Schmitz, Boris ^{[2
]}

Brand, Stefan-Martin ^{[2
]}

Brand, Eva ^{[1
]}

机构：

[1] Univ Hosp Muenster, Dept Nephrol Hypertens & Rheumatol, Internal Med D, Albert Schweitzer Campus 1, D-48149 Munster, Germany

[2] Univ Hosp Muenster, Inst Sports Med, Mol Genet Cardiovasc Dis, Munster, Germany

来源：

ORPHANET JOURNAL OF RARE DISEASES | 2018年 / 13卷

关键词：

Enzyme replacement therapy; Longitudinal; Prospective; REPLACEMENT THERAPY; MEMBRANOUS NEPHROPATHY; AGALSIDASE-ALPHA; BETA;

D O I：

10.1186/s13023-018-0916-1

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Fabry disease (FD) is a rare X-linked disorder caused by a deficiency of lysosomal a-galactosidase A activity. Treatment with recombinant enzyme replacement therapy is available since 2001 and the effects of anti-drug antibodies (ADA) on therapy efficacy and disease outcome in affected patients have been controversially reported. In this letter we discuss the importance of adequate measurements of neutralizing ADAs and appropriate longitudinal analysis to determine therapy efficiency and clinical outcome in patients with FD.

引用

页数：2

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