背景:梗阻性肥厚型心肌病(oHCM)患者具有实质性的人文,临床,以及与疾病相关的一系列症状和并发症造成的经济负担。本综述的目的是确定与oHCM相关的关键证据差距,特别是在欧洲,北美,和日本。
方法:使用PubMed进行了针对性的文献综述,以确定2012年至2022年间发表的评估法国HCM/oHCM患者的英语研究。德国,意大利,西班牙,英国(UK),美国(US),加拿大,和日本。感兴趣的结果是流行病学,自然史,病理生理学,管理,临床,经济,和人文负担。定性评估已确定的研究,以表征证据差距。
结果:在筛选的2,262篇摘要和531篇全文文章中,178篇文章来自PubMed搜索。通过最初于2023年1月进行并于2024年7月更新的补充Google学者搜索,确定了另外16项独特的研究。疾病自然史,病理生理学,和管理在全球范围内都有据可查。在流行病学方面注意到了显著的证据差距,治疗,和OHCM的负担。尽管在临床上发现了多项美国研究,经济,和oHCM的人文负担,在日本发现了一项临床负担研究,法国缺乏证据,德国,意大利,西班牙,英国,和加拿大。
结论:流行病学存在主要证据空白,治疗,和OHCM的负担。未来的研究应该解决这些差距,特别着重于为加拿大和欧洲国家提供真实世界的证据,以支持对这些地区新兴疗法的评估。
BACKGROUND: Patients with obstructive hypertrophic cardiomyopathy (oHCM) have a substantial humanistic, clinical, and economic burden due to the array of symptoms and complications associated with the disease. The objective of this review was to identify key evidence gaps related to oHCM, specifically in Europe, North America, and Japan.
METHODS: A targeted literature review was conducted using PubMed to identify English-language studies published between 2012 and 2022 assessing patients with HCM/oHCM in France, Germany, Italy, Spain, the United Kingdom (UK), the United States (US), Canada, and Japan. Outcomes of interest were epidemiology, natural history, pathophysiology, management, and clinical, economic, and humanistic burden. Identified studies were assessed qualitatively to characterize evidence gaps.
RESULTS: Among 2,262 abstracts and 531 full-text articles screened, 178 articles were included from PubMed searches. An additional 16 unique studies were identified via a supplemental Google Scholar search initially conducted in January 2023 and updated in July 2024. Disease natural history, pathophysiology, and management were well documented globally. Significant evidence gaps were noted for the epidemiology, treatment, and burden of oHCM. Although multiple US studies were identified on the clinical, economic, and humanistic burden of oHCM, and one clinical burden study was found for Japan, there was a lack of evidence for France, Germany, Italy, Spain, the UK, and Canada.
CONCLUSIONS: Major evidentiary gaps exist for the epidemiology, treatment, and burden of oHCM. Future research should address these gaps, with a specific focus on generating real-world evidence for Canada and European countries that will support the evaluation of emerging therapies in these regions.