硝呋噻肟治疗儿童恰加斯病的疗效评价

IF 0.8 4区 医学 Q4 PHARMACOLOGY & PHARMACY Expert Opinion on Orphan Drugs Pub Date : 2021-05-04 DOI:10.1080/21678707.2021.1933431
Fernanda Lascano, J. Altcheh
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引用次数: 0

摘要

摘要简介:恰加斯病(CD)是由克氏锥虫(T.cruzi)引起的一种人畜共患疾病,目前全球有600万人患病。据描述,100年前,有两种药物可用于治疗,即50多年前开发的硝呋噻肟(NFX)和苯硝唑(BZN)。尽管BZN更常用于CD,但最近发布的儿童配方和正在进行的长期随访研究对NFX进行了新的开发。涵盖领域:在这篇叙述性综述中,我们搜索了电子数据库(如PubMed、Cochrane),并从儿科的角度对NFX用于CD治疗的临床和临床前研究进行了批判性分析,重点关注该药物的药效学、药代动力学、代谢、安全性、耐受性和临床疗效。专家意见:为了实现消除CD的目标,早期诊断和及时治疗是至关重要的。在这种情况下,美国食品药品监督管理局批准NFX用于0至18岁的儿童,使该药物成为美国唯一一种适用于0至2岁和12岁以上年龄组的药物。这一批准也可能改变人们对NFX安全性和耐受性的成见。考虑到CD只有两种药理学选择,这是在儿科人群中治疗这种疾病的新时代迈出的重要一步。
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An evaluation of nifurtimox for Chagas disease in children
ABSTRACT Introduction: Chagas disease (CD) is a zoonosis disease caused by Trypanosoma cruzi (T. cruzi) and currently affects 6 million people worldwide. Described 100 years ago, two drugs are available for treatment, nifurtimox (NFX) and benznidazole (BZN), developed over 50 years ago. Though BZN has been more commonly used for CD, new development efforts on NFX has been undertaken with its recently released pediatric formulation and an ongoing study continuation with long-term follow-up. Areas covered: In this narrative review, we searched electronic databases (e.g. PubMed, Cochrane) and critically analyzed clinical and preclinical studies about NFX for CD treatment from a pediatric perspective, focusing on pharmacodynamics, pharmacokinetics, metabolism, safety, tolerance, and clinical efficacy of this drug. Expert opinion: In order to achieve CD elimination goals, early diagnosis and opportune treatment are fundamental. In this context, the approval by FDA of NFX for children 0–18 years of age has made this drug the only one available in 0 to 2 years and over 12 age groups in USA. This approval might also change preconceptions about NFX safety and tolerance. Considering CD has only two pharmacological options, this is a significant step toward a new era for the treatment of this disease in the pediatric population.
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来源期刊
Expert Opinion on Orphan Drugs
Expert Opinion on Orphan Drugs PHARMACOLOGY & PHARMACY-
CiteScore
2.30
自引率
0.00%
发文量
8
期刊介绍: Expert Opinion on Orphan Drugs is an international, peer-reviewed journal that covers all aspects of R&D on rare diseases and orphan drugs.
期刊最新文献
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