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高水平临床研究推动肺动脉高压危险分层内涵发展

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肺动脉高压是一种疑难危重罕见病,一直以来被视为"心血管系统的癌症",患者预后极差。对疾病进行危险分层可以帮助临床医生判断患者病情、指导治疗、评价疗效和预测预后,这需要大量的临床研究作为依据。高水平临床研究是解决临床实践问题的重要手段,我国肺动脉高压队列有着独特的特点,学习和借鉴国外肺动脉高压危险分层发展经验,对建立符合我国肺动脉高压人群的危险分层具有重要意义。本文以肺动脉高压危险分层发展为例,旨在深入剖析通过高水平临床研究解决临床问题的过程与方法。文章概述了肺动脉高压的发现与流行,明确了定义与深入开展临床研究的全过程;重点围绕靶向药治疗时代下肺动脉高压危险分层的建立与更新,总结临床研究推动肺动脉高压诊疗指南和治疗理念不断发展的方式方法;最后通过分析肺动脉高压危险分层在我国的应用现状和问题,希望国外研究肺动脉高压危险分层的思维方式对我国开展临床研究、解决临床实践中的问题有所启示。
High-level clinical research promotes the development of risk stratification for pulmonary arterial hypertension
Pulmonary arterial hypertension(PAH)is a rare and critical cardiovascular disease characterized by increased pulmonary vascular resistance,lesions in the pulmonary arterioles,and a subsequent rise in the right ventricular load,which inevitably leads to right heart failure.Unfortunately,patients with PAH have a poor prognosis,with a median survival time of 2.8 years under traditional drug therapy when there are no targeted pharmaceuticals,earning it the reputation of being a cancer of the cardiovascular system.Patients with PAH face significant challenges.They usually show nonspecific clinical manifestations in the early stages,which often leads to delayed diagnosis and treatment.The majority of the population with PAH in China are young and middle-aged females.Apart from physical difficulties,these patients may have a poor quality of life despite adequate drug treatment.Many patients experience anxiety,depression,or other psychological disorders.These burdens not only affect patients but also place immense strain on their families and society.The current 5-year survival rate for critically severe patients with PAH receiving optimal drug treatment is only 33.3%.To address these challenges,risk assessment plays a crucial role in guiding the clinicians'decision-making processes,treatment evaluation,and prognosis prediction for patients with PAH.However,establishing effective risk stratification requires extensive clinical research.High-level clinical research can serve as a means of solving clinical problems and we can gain insights into the process and methods involved.The Chinese PAH cohort represents a unique opportunity to study international experiences in the development of a risk stratification system suitable for the Chinese population.By systematically outlining the development of PAH from its first description as a disease,which garnered attention from the World Health Organization,we appreciate the importance of conducting clinical research that has helped enhance our understanding of this disease.Establishing prognostic prediction equations through clinical studies and conducting international research on risk stratification in the era of targeted drug therapy are vital steps for continuously updating risk stratification guidelines and treatment concepts.The development of high-level clinical research in China can draw inspiration from these processes.By leveraging lessons learned from international experiences,we can pave the way for advancements in PAH research within the country.This included addressing the current application status and challenges faced in the implementation of risk stratification for PAH in China.Future research should focus on refining the risk stratification methods,exploring innovative diagnostic techniques,and identifying novel treatment approaches for PAH.By continuously enhancing our understanding of this complex disease,we can improve patient outcomes and alleviate the burden on individuals,families,and the society as a whole.In conclusion,PAH presents significant challenges in diagnosis,treatment,and risk assessment.By delving into high-level clinical research to address these challenges,we can pave the way for improved management strategies and better outcomes for patients with this devastating condition.Through international collaborations and tailored approaches for the Chinese population,we can strive towards more accurate risk stratification and ultimately enhance the overall care provided to individuals with PAH.

clinical researchpulmonary arterial hypertensionrisk stratificationprognostic model

路丹、张洁馨、程春燕、杨尹鉴、刘滕飞、张苏欣、邱宝晨、李贤美、郭帆、黎婧怡、徐希奇、孙凯、荆志成

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北京大学航天临床医学院,航天中心医院,心脏医学部,北京 100049

中国医学科学院北京协和医学院北京协和医院心内科,北京 100730

南方医科大学附属广东省人民医院(广东省医学科学院),广东省心血管病研究所,广州 510180

中国医学科学院北京协和医学院北京协和医院医学科学研究中心,北京 100730

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临床研究 肺动脉高压 危险分层 预后预测模型

国家自然科学基金重点专项国家重点研发计划

822410202022YFC2703902

2024

科学通报
中国科学院国家自然科学基金委员会

科学通报

CSTPCD北大核心
影响因子:1.269
ISSN:0023-074X
年,卷(期):2024.69(13)