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首页> 外文期刊>Drugs of today: Medicamentos de actualidad >Velaglucerase alfa: a new option for Gaucher disease treatment.
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Velaglucerase alfa: a new option for Gaucher disease treatment.

机译：Velaglucerase alfa：一种用于治疗高雪氏病的新方法。

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摘要

Type 1 Gaucher disease (GD) results from inherited beta-glucocerebrosidase gene mutations, leading to anemia, thrombocytopenia, splenomegaly, hepatomegaly and skeletal disease. Velaglucerase alfa is a beta-glucocerebrosidase produced by gene activation in a human cell line, and indicated for type 1 GD. A phase I/II clinical trial (TKT025; N = 12), its ongoing extension (TKT025EXT) and three phase III trials (total N = 82), showed that velaglucerase alfa is generally well tolerated in adult and pediatric patients. Many disease-related parameters improved significantly in two phase III trials in treatment-naive patients, and were successfully maintained in imiglucerase-experienced patients in a phase II/III switch study. Ten adults in TKT025EXT sustained improvements through 5 years, including bone mineral density. Comparison with imiglucerase shows that velaglucerase alfa is an effective, generally well-tolerated alternative enzyme replacement therapy. In vitro data suggest velaglucerase alfa may be internalized into cells more efficiently and have a lower rate of seroconversion. However, these results do not necessarily correlate with clinical efficacy.
机译：1型高雪氏病（GD）是由遗传的β-葡萄糖脑苷脂酶基因突变导致的，导致贫血，血小板减少，脾肿大，肝肿大和骨骼疾病。 Velaglucerase alfa是一种在人细胞系中通过基因激活产生的β-葡萄糖脑苷脂酶，适用于1型GD。 I / II期临床试验（TKT025; N = 12），正在进行的扩展试验（TKT025EXT）和三期III期试验（总N = 82）表明，成人和儿童患者对维拉苷酶α的耐受性普遍良好。在未经治疗的两名患者的两项III期临床试验中，许多与疾病相关的参数均得到了显着改善，并且在经历过伊米苷酶治疗的患者中，II / III期转换研究成功地维持了这些疾病参数。 TKT025EXT的10位成年人在5年中持续改善，包括骨矿物质密度。与伊米苷酶的比较表明，维拉苷酶α是一种有效的，一般耐受性良好的替代酶替代疗法。体外数据表明，维拉苷酶α可以更有效地内化到细胞中，并具有较低的血清转化率。但是，这些结果不一定与临床疗效相关。

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