• 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>Drug Target Insights >A Comprehensive Review of Pegvaliase, an Enzyme Substitution Therapy for the Treatment of Phenylketonuria
【24h】

A Comprehensive Review of Pegvaliase, an Enzyme Substitution Therapy for the Treatment of Phenylketonuria

机译：Pegvaliase，酶替代疗法治疗苯丙酮尿症的综述。

获取原文
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

Objective: To review the pharmacology, pharmacokinetics, efficacy, safety, and place in therapy of a phenylalanine-metabolizing enzyme indicated to reduce blood phenylalanine concentrations, pegvaliase injection. Data Sources: Searches of MEDLINE (1946-September 1, 2018) were conducted using the terms pegvaliase and phenylalanine ammonia lyase (PAL). Additional data were obtained from the prescribing information, the product dossier obtained from the manufacturer, and Clinicaltrials.gov. Study Selection and Data Extraction: All English language articles related to pharmacology, pharmacokinetics, efficacy, or safety of the combination therapy in human subjects were reviewed. Data Synthesis: Pegvaliase is a pegylated PAL enzyme that converts phenylalanine to ammonia and trans-cinnamic acid. Blood phenylalanine levels were reduced by approximately 50% to 70% in patients receiving therapeutic doses of pegvaliase. However, most patients experienced adverse events. Conclusions and Relevance: The mainstay of therapy in phenylketonuria (PKU) has historically consisted of dietary restriction of phenylalanine. Pegvaliase injection is the first Food and Drug Administration (FDA)–approved enzyme substitution therapy for patients with PKU. The therapy may be a viable option for patients with documented blood phenylalanine &600?μmol/L who have failed existing management strategies.
机译：目的：综述苯丙氨酸代谢酶的药理学，药代动力学，疗效，安全性和在治疗中的作用，该酶可降低血中苯丙氨酸的浓度，聚乙二醇戊二酸注射。数据来源：MEDLINE（1946-September 1，2018）的搜索使用术语聚乙二醇戊二酸酶和苯丙氨酸氨裂合酶（PAL）。从处方信息，从制造商处获得的产品资料以及Clinicaltrials.gov获得了其他数据。研究选择和数据提取：复习了与人类受试者中联合疗法的药理学，药代动力学，功效或安全性相关的所有英语文章。数据综合：Pegvaliase是一种聚乙二醇化的PAL酶，可将苯丙氨酸转化为氨和反式肉桂酸。在接受pegvaliase治疗剂量的患者中，血液苯丙氨酸水平降低了约50％至70％。然而，大多数患者经历了不良事件。结论和相关性：苯丙酮尿症（PKU）的治疗的主要历史是饮食中限制苯丙氨酸。哌加戊酸酶注射液是美国食品药品监督管理局（FDA）首次批准的用于PKU患者的酶替代疗法。对于已记录的血液苯丙氨酸> 600？μmol/ L的患者，现有治疗策略无效的患者，该疗法可能是可行的选择。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号