α -1抗胰蛋白酶缺乏症:目前的治疗方法和新出现的靶点。

IF 2.9 3区 医学 Q2 RESPIRATORY SYSTEM Expert Review of Respiratory Medicine Pub Date : 2023-03-01 DOI:10.1080/17476348.2023.2174973
Oisín F McElvaney, Daniel D Fraughen, Oliver J McElvaney, Tomás P Carroll, Noel G McElvaney
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引用次数: 0

摘要

Alpha1抗胰蛋白酶缺乏症(AATD)是一种常见的遗传性疾病,主要影响肺、肝和皮肤,在过去的5年里,它已经成为医学上一些最令人兴奋的治疗方法的焦点。在这篇综述中,我们讨论了目前针对AATD不同表现的治疗方法和正在开发的新疗法。涵盖领域:我们回顾了AATD个体肺、肝和皮肤表现的治疗选择,以及旨在治疗这三种疾病的方法。随着人们对AATD治疗的兴趣重新燃起,挑战也随之而来。AAT如何最好地输送到肺部?血液循环和肺中AAT的理想水平是多少,哪些治疗方法应该提供?治疗肝病会增加患肺病的可能性吗?是否有针对潜在遗传缺陷的治疗方法,可以预防aatd相关疾病的所有方面?专家意见:由于能够参与临床研究的人群相对较少,迫切需要提高对AATD的认识和诊断。更好、更敏感的临床参数将有助于为当前和新出现的治疗方法产生可接受和有力的治疗效果证据。
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Alpha-1 antitrypsin deficiency: current therapy and emerging targets.

Introduction: Alpha1 antitrypsin deficiency (AATD), a common hereditary disorder affecting mainly lungs, liver and skin has been the focus of some of the most exciting therapeutic approaches in medicine in the past 5 years. In this review, we discuss the therapies presently available for the different manifestations of AATD and new therapies in the pipeline.

Areas covered: We review therapeutic options for the individual lung, liver and skin manifestations of AATD along with approaches which aim to treat all three. Along with this renewed interest in treating AATD come challenges. How is AAT best delivered to the lung? What is the desired level of AAT in the circulation and lungs which therapeutics should aim to provide? Will treating the liver disease increase the potential for lung disease? Are there treatments to target the underlying genetic defect with the potential to prevent all aspects of AATDrelated disease?

Expert opinion: With a relatively small population able to participate in clinical studies, increased awareness and diagnosis of AATD is urgently needed. Better, more sensitive clinical parameters will assist in the generation of acceptable and robust evidence of therapeutic effect for current and emerging treatments.

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来源期刊
CiteScore
6.80
自引率
2.60%
发文量
90
期刊介绍: Coverage will include the following key areas: - Prospects for new and emerging therapeutics - Epidemiology of disease - Preventive strategies - All aspects of COPD, from patient self-management to systemic effects of the disease and comorbidities - Improved diagnostic methods, including imaging techniques, biomarkers and physiological tests. - Advances in the treatment of respiratory infections and drug resistance issues - Occupational and environmental factors - Progress in smoking intervention and cessation methods - Disease and treatment issues for defined populations, such as children and the elderly - Respiratory intensive and critical care - Updates on the status and advances of specific disease areas, including asthma, HIV/AIDS-related disease, cystic fibrosis, COPD and sleep-disordered breathing morbidity
期刊最新文献
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