Development of high sustained anti-drug antibody titers and corresponding clinical decline in a late-onset Pompe disease patient after 11+years on enzyme replacement therapy

被引：4
|
作者：
Kim, Katherine H. [1 ,4 ]
Desai, Ankit K. [2 ]
Vucko, Erika R. [1 ]
Boggs, Tracy [3 ]
Kishnani, Priya S. [2 ]
Burton, Barbara K. [1 ]
机构：
[1] Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Dept Pediat, Div Genet Genom & Metab,Feinberg Sch Med, 225 E Chicago Ave Box 59, Chicago, IL 60611 USA
[2] Duke Univ, Dept Pediat, Div Med Genet, Med Ctr, Box 103856 DUM, Durham, NC 27710 USA
[3] Duke Univ Hlth Syst, Dept Rehabil Serv, 234 Crooked Creek Pkwy,Suite 310, Durham, NC 27713 USA
[4] Ann & Robert H Lurie Childrens Hosp Chicago, 225 E Chicago Ave,Box 59, Chicago, IL 60611 USA
来源：
MOLECULAR GENETICS AND METABOLISM REPORTS | 2023年 / 36卷
关键词：
Pompe disease; Anti -drug antibody titers; Enzyme replacement therapy; Immune modulation therapy; Urine glucose tetrasaccharide (Glc 4 ); ALGLUCOSIDASE ALPHA; MOTOR FUNCTION;
D O I：
10.1016/j.ymgmr.2023.100981
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
摘要：
A late-onset Pompe disease patient developed high sustained antibody titers (HSAT) of & GE;51,200 after 11+ years on alglucosidase alfa and previous tolerance. There was a corresponding worsening of motor function and rise in urinary glucose tetrasaccharide (Glc4). Following immunomodulation therapy, HSAT were eliminated with improved clinical outcomes and biomarker trends. This report highlights the importance of continued surveillance of antibody titers and biomarkers, the negative impact of HSAT, and improved outcomes with immunomodulation therapy.
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页数：4
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