Results from a 9-year Intensive Safety Surveillance Scheme (IS3) in miglustat (Zavesca®)-treated patients
Results from a 9-year Intensive Safety Surveillance Scheme (IS3) in miglustat (Zavesca®)-treated patients
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DOI：
10.1002/pds.3760
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发表时间：
2015-03-01
影响因子：
2.6
通讯作者：
Hughes, Derralynn
中科院分区：
文献类型：
--
作者：
Brand, Monika;Muller, Audrey;Hughes, Derralynn
背景在2002年在欧盟和2003年在美国获得批准后，启动了强化安全性监测计划（IS 3），以教育处方医生适当使用麦格司他治疗I型戈谢病（GD 1），并积极征集安全性相关信息。本报告总结了从2003年开始到2012年10月结束的IS 3中所有入组患者的数据。在参与研究中心接受麦格司他治疗的所有患者均根据常规医疗实践接受标准医疗护理。在患者入组、后续随访访视和治疗中止（如适用）时收集患者和疾病特征数据。使用描述性statistics.ResultsDuring9years的IS 3，407例患者在111个网站在15个欧洲国家。大约一半（n=202）患有GD 1，一半患有其他疾病（主要是C型尼曼-匹克病（NP-C），麦格司他于2009年在欧洲获批用于治疗）。总共有368例患者有至少一次随访访视的数据，其中192例为GD 1。IS 3提供了798患者-年麦格司他暴露的数据。安全性相关数据与早期发表的IS 3 5年研究结果、GD 1临床试验中麦格司他报告的安全性特征以及其他已发表的关于GD 1表现的信息一致。ConclusionsOverall，该长期安全性监测项目的结果与麦格司他众所周知的、有记录的安全性特征一致。版权所有（c）2015约翰威利父子有限公司
BackgroundFollowing approval in the EU in 2002 and the USA in 2003, an Intensive Safety Surveillance Scheme (IS3) was initiated to educate prescribers on the appropriate use of miglustat for the treatment of type I Gaucher disease (GD1), and to actively solicit safety-relevant information. This report summarises data from all patients enrolled in IS3 between its initiation in 2003 and its closure in October 2012.MethodsThe IS3 was a prospective observational drug registry with a secure internet-based data capture system. All patients receiving miglustat at participating sites received standard medical care according to routine medical practice. Data on patient and disease characteristics were collected at patient enrolment, subsequent follow-up visits and treatment discontinuation (if applicable). Data were summarised using descriptive statistics.ResultsDuring the 9years of IS3, 407 patients were enrolled at 111 sites across 15 European countries. Approximately half (n=202) had GD1, and half had other diseases (mainly Niemann-Pick disease type C (NP-C), for which miglustat was approved in Europe in 2009). In total, 368 patients had data from at least one follow-up visit, 192 of whom had GD1. IS3 provided data from 798 patient-years exposure to miglustat. Safety-relevant data were consistent with earlier published 5-year findings from IS3, the safety profile reported for miglustat in GD1 clinical trials and other published information on GD1 manifestations.ConclusionsOverall, the results of this long-term safety surveillance programme were in line with the well-known, documented safety profile of miglustat. Copyright (c) 2015 John Wiley & Sons, Ltd.