Cytokinetics Incorporated, 350 Oyster Point Blvd, South San Francisco, CA, USA.
EVERSANA, 113-3228 South Service Road, Burlington, ON, L7N 3H8, Canada.
BMC Cardiovasc Disord. 2024 Aug 10;24(1):416. doi: 10.1186/s12872-024-04084-7.
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.
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.
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.
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.
梗阻性肥厚型心肌病（oHCM）患者由于与该疾病相关的一系列症状和并发症，存在巨大的人文、临床和经济负担。本研究旨在确定与 oHCM 相关的关键证据缺口，特别是在欧洲、北美和日本。
使用 PubMed 进行了有针对性的文献回顾，以确定 2012 年至 2022 年间在法国、德国、意大利、西班牙、英国、美国、加拿大和日本发表的评估 HCM/oHCM 患者的英语研究。感兴趣的结局包括流行病学、自然病史、病理生理学、管理以及临床、经济和人文负担。定性评估所确定的研究以描述证据缺口。
在筛选的 2262 篇摘要和 531 篇全文文章中，从 PubMed 搜索中纳入了 178 篇文章。通过最初于 2023 年 1 月进行并于 2024 年 7 月更新的补充 Google Scholar 搜索，又确定了 16 项独特的研究。全球范围内，疾病的自然病史、病理生理学和管理得到了很好的记录。在 oHCM 的流行病学、治疗和负担方面存在显著的证据缺口。尽管在美国有多项关于 oHCM 的临床、经济和人文负担的研究，在日本有一项关于临床负担的研究，但在法国、德国、意大利、西班牙、英国和加拿大却缺乏相关证据。
oHCM 的流行病学、治疗和负担方面存在重大证据缺口。未来的研究应解决这些缺口，特别关注为加拿大和欧洲国家生成真实世界证据，这将支持在这些地区评估新兴疗法。
