Enzyme Replacement Therapy With Elosulfase Alfa Decreases Storage of Glycosaminoglycan in White Blood Cells of Patients With Morquio A Syndrome

Fernanda Bender; Roberto Giugliani; Camila Bittar; Fabiano Poswar; Andressa Federhen; Guilherme Baldo; Rejane Gus

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Enzyme Replacement Therapy With Elosulfase Alfa Decreases Storage of Glycosaminoglycan in White Blood Cells of Patients With Morquio A Syndrome

机译：硫磺酶Alfa的酶替代治疗可减少Morquio A综合征患者白细胞中糖胺聚糖的储存

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Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is a lysosomal storage disorder caused by a deficient N-acetylgalactosamine-6-sulfate sulfatase activity, leading to cellular storage of undegraded keratan sulfate. Recently enzyme replacement therapy (ERT) was approved for MPS IVA, but some of ERT effects are still unknown. In the present study, we aimed to evaluate the efficacy of elosulfase alfa upon glycosaminoglycan (GAG) storage in peripheral blood white blood cells of patients with MPS IVA treated for 6 months, comparing samples from patients who received weekly infusions of enzyme (ERT-W) versus infusions every other week (ERT-EOW) versus placebo. A significant reduction in GAG storage was observed in both ERT-treated groups, with weekly ERT showing slightly better performance than ERT-EOW.

机译：粘多糖贮积症IVA（MPS IVA; Morquio A综合征）是一种溶酶体贮积病，由N-乙酰半乳糖胺-6-硫酸盐硫酸酯酶活性不足引起，导致未降解硫酸角质素的细胞贮存。最近，酶替代疗法（ERT）已被批准用于MPS IVA，但是一些ERT的作用仍然未知。在本研究中，我们旨在评估环磷酰胺酶对6个月接受MPS IVA治疗的MPS IVA患者外周血白细胞中糖胺聚糖（GAG）储存的疗效，比较每周接受酶输注（ERT-W ）与每两周输液（ERT-EOW）与安慰剂之比。在两个ERT治疗组中均观察到GAG储存量显着减少，每周ERT显示的性能略好于ERT-EOW。

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《Journal of Inborn Errors of Metabolism & Screening》 |2015年第12期|共页
作者
Fernanda Bender; Roberto Giugliani; Camila Bittar; Fabiano Poswar; Andressa Federhen; Guilherme Baldo; Rejane Gus;
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中图分类内科学;
关键词
Morquio Amucopolysaccharidosis IVAenzyme replacement therapyelosulfase alfa;

机译：Morquio粘多糖贮积症IVA酶替代疗法;

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1. Elosulfase alfa decreases glycosaminoglycan storage in white blood cells from Morquio syndrome type A patients undergoing enzyme replacement [J] . Baldo Guilherme, Poswar Fabiano, Federhen Andressa, Molecular genetics and metabolism . 2015,第2期

机译：Elosulfase alfa降低了接受酶置换的A型Morquio综合征患者白细胞中糖胺聚糖的储存
2. Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment [J] . Hendriksz Christian, Santra Saikat, Jones Simon A., Molecular genetics and metabolism . 2018,第4期

机译：Morquio A综合征患者的安全性，免疫原性和临床结果参与了Elosulfase Alfa酶替代疗法（Mor-002 / Mor-100）的2个顺序开放标签研究，代表了5年的治疗方法
3. Elosulfase alfa enzyme replacement therapy attenuates disease progression in a non-ambulatory Japanese patient with Morquio A syndrome (case report) [J] . Misako Hiramatsu, Kimitoshi Nakamura Molecular Genetics and Metabolism Reports . 2017,第2期

机译：Elosulfase alfa酶替代疗法可减轻非门诊日本Morquio A综合征患者的疾病进展（病例报告）
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7. Enzyme Replacement Therapy With Elosulfase Alfa Decreases Storage of Glycosaminoglycan in White Blood Cells of Patients With Morquio A Syndrome [O] . Guilherme Baldo PhD, Fabiano Poswar MD, Andressa Federhen MSc, 2015

机译：Elosulfase alfa酶替代疗法减少morquio a综合征患者白细胞中糖胺聚糖的储存

Enzyme Replacement Therapy With Elosulfase Alfa Decreases Storage of Glycosaminoglycan in White Blood Cells of Patients With Morquio A Syndrome

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