[Romiplostim:特发性血小板减少性紫癜的治疗进展]。

M E Mingot Castellano
{"title":"[Romiplostim:特发性血小板减少性紫癜的治疗进展]。","authors":"M E Mingot Castellano","doi":"","DOIUrl":null,"url":null,"abstract":"<p><p>Primary Immune thrombocytopenia or idiopathic thrombocytopenic purpura (ITP) is an acquired immune disorder presenting with abnormal hemorrhagic symptoms resulting from a decrease in the number of platelets. The disorder used to be attributed to increased destruction of platelets mediated by antibodies. In the past few years, the description of its etiopathology has changed. A deficiency in the marrow production of thrombocytes has been demonstrated; because it is associated with increased peripheral platelet destruction, the deficiency cannot be compensated. These findings have justified the realization of studies assessing the utility of second generation thrombopoietin analogues for the treatment of these patients. These drugs include romiplostim or AMG 537 (Nplate), a peptidic analogue that stimulates the thrombopoietin receptor and induces an increase In the production and differentiation of megakaryocytes. Data obtained from the clinical trials that led to the authorization and subsequent follow-up describe romiplostin as an effective and safe drug for adult patients with chronic ITR The overall response rate is 94%; despite variations in the levels of platelets throughout treatment, 50% of patients maintain the response 95% of the time, and 78% of patients discontinue or significantly reduce the use of rescue treatment. The most common adverse event is headache. Reticulin fibrosis has been described, which is reversible after treatment discontinuation.</p>","PeriodicalId":18443,"journal":{"name":"Methods and findings in experimental and clinical pharmacology","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2010-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"[Romiplostim: an advance in the treatment of idiopathic thrombocytopenic purpura].\",\"authors\":\"M E Mingot Castellano\",\"doi\":\"\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Primary Immune thrombocytopenia or idiopathic thrombocytopenic purpura (ITP) is an acquired immune disorder presenting with abnormal hemorrhagic symptoms resulting from a decrease in the number of platelets. The disorder used to be attributed to increased destruction of platelets mediated by antibodies. In the past few years, the description of its etiopathology has changed. A deficiency in the marrow production of thrombocytes has been demonstrated; because it is associated with increased peripheral platelet destruction, the deficiency cannot be compensated. These findings have justified the realization of studies assessing the utility of second generation thrombopoietin analogues for the treatment of these patients. These drugs include romiplostim or AMG 537 (Nplate), a peptidic analogue that stimulates the thrombopoietin receptor and induces an increase In the production and differentiation of megakaryocytes. Data obtained from the clinical trials that led to the authorization and subsequent follow-up describe romiplostin as an effective and safe drug for adult patients with chronic ITR The overall response rate is 94%; despite variations in the levels of platelets throughout treatment, 50% of patients maintain the response 95% of the time, and 78% of patients discontinue or significantly reduce the use of rescue treatment. The most common adverse event is headache. Reticulin fibrosis has been described, which is reversible after treatment discontinuation.</p>\",\"PeriodicalId\":18443,\"journal\":{\"name\":\"Methods and findings in experimental and clinical pharmacology\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2010-12-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Methods and findings in experimental and clinical pharmacology\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Methods and findings in experimental and clinical pharmacology","FirstCategoryId":"1085","ListUrlMain":"","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0

摘要

原发性免疫性血小板减少症或特发性血小板减少性紫癜(ITP)是一种获得性免疫疾病,表现为血小板数量减少引起的异常出血症状。这种疾病过去被认为是由于抗体介导的血小板破坏增加。在过去的几年中,对其病因的描述发生了变化。骨髓中产生血小板的缺陷已被证实;因为它与外周血小板破坏增加有关,这种缺陷不能得到补偿。这些发现证明了评估第二代血小板生成素类似物治疗这些患者的效用的研究的实现是合理的。这些药物包括romiplostim或AMG 537 (Nplate),这是一种肽类似物,可刺激血小板生成素受体并诱导巨核细胞的产生和分化增加。从导致批准的临床试验和随后的随访中获得的数据表明,romiplostin是一种有效和安全的药物,用于成人慢性ITR患者,总有效率为94%;尽管在整个治疗过程中血小板水平会发生变化,但50%的患者在95%的时间内保持反应,78%的患者停止或显著减少抢救治疗的使用。最常见的不良反应是头痛。网状蛋白纤维化已被描述,在停止治疗后是可逆的。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
[Romiplostim: an advance in the treatment of idiopathic thrombocytopenic purpura].

Primary Immune thrombocytopenia or idiopathic thrombocytopenic purpura (ITP) is an acquired immune disorder presenting with abnormal hemorrhagic symptoms resulting from a decrease in the number of platelets. The disorder used to be attributed to increased destruction of platelets mediated by antibodies. In the past few years, the description of its etiopathology has changed. A deficiency in the marrow production of thrombocytes has been demonstrated; because it is associated with increased peripheral platelet destruction, the deficiency cannot be compensated. These findings have justified the realization of studies assessing the utility of second generation thrombopoietin analogues for the treatment of these patients. These drugs include romiplostim or AMG 537 (Nplate), a peptidic analogue that stimulates the thrombopoietin receptor and induces an increase In the production and differentiation of megakaryocytes. Data obtained from the clinical trials that led to the authorization and subsequent follow-up describe romiplostin as an effective and safe drug for adult patients with chronic ITR The overall response rate is 94%; despite variations in the levels of platelets throughout treatment, 50% of patients maintain the response 95% of the time, and 78% of patients discontinue or significantly reduce the use of rescue treatment. The most common adverse event is headache. Reticulin fibrosis has been described, which is reversible after treatment discontinuation.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
自引率
0.00%
发文量
0
审稿时长
>12 weeks
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信