Patterns and organ treatment response of Erdheim-Chester disease with cardiac involvement.

IF 5.1 2区 医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS
Heart Pub Date : 2024-06-17 DOI:10.1136/heartjnl-2024-323867
Hui-Lei Miao, Long Chang, He Lin, Zheng-Zheng Liu, Wei Wu, Na Niu, Xin-Xin Cao
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引用次数: 0

Abstract

Objective: To evaluate the heart response of Erdheim-Chester disease (ECD) through continuous follow-up within our large cohort, for which there is a lack of understanding.

Methods: We conducted a retrospective analysis of clinical data from patients with ECD with cardiac involvement diagnosed at our centre between January 2010 and August 2023. We assessed the heart response by integrating pericardial effusion and metabolic responses.

Results: A total of 40 patients were included, with a median age of 51.5 years (range: 29-66) and a BRAFV600E mutation rate of 56%. The most common imaging manifestations observed were pericardial effusion (73%), right atrium (70%) and right atrioventricular sulcus infiltration (58%). Among 21 evaluable patients, 18 (86%) achieved a heart response including 5 (24%) complete response (CR) and 13 (62%) partial response (PR). The CR rate of pericardial effusion response was 33%, while the PR rate was 56%. Regarding the cardiac mass response, 33% of patients showed PR. For cardiac metabolic response, 32% and 53% of patients achieved complete and partial metabolic response, respectively. There was a correlation between pericardial effusion response and cardiac metabolic response (r=0.73 (95% CI 0.12 to 0.83), p<0.001). The median follow-up was 50.2 months (range: 1.0-102.8 months). The estimated 5-year overall survival was 78.9%. The median progression-free survival was 59.4 months (95% CI 26.2 to 92.7 months). Patients who received BRAF inhibitors achieved better heart response (p=0.037) regardless of treatment lines.

Conclusion: We pioneered the evaluation of heart response of ECD considering both pericardial effusion and cardiac metabolic response within our cohort, revealing a correlation between these two indicators. BRAF inhibitors may improve heart response, regardless of the treatment lines.

心脏受累的埃尔德海姆-切斯特病的模式和器官治疗反应。
目的方法:我们对 2010 年 1 月至 2023 年 8 月期间在本中心确诊的埃尔德海姆-切斯特病(ECD)心脏受累患者的临床数据进行了回顾性分析:我们对 2010 年 1 月至 2023 年 8 月期间在本中心确诊的心脏受累的 ECD 患者的临床数据进行了回顾性分析。我们通过整合心包积液和代谢反应来评估心脏反应:共纳入 40 例患者,中位年龄为 51.5 岁(范围:29-66 岁),BRAFV600E 基因突变率为 56%。最常见的影像学表现为心包积液(73%)、右心房(70%)和右房室沟浸润(58%)。在 21 例可评估的患者中,18 例(86%)获得了心脏反应,包括 5 例(24%)完全反应(CR)和 13 例(62%)部分反应(PR)。心包积液反应的 CR 率为 33%,而 PR 率为 56%。在心脏肿块反应方面,33% 的患者表现为 PR。在心脏代谢反应方面,分别有 32% 和 53% 的患者获得了完全和部分代谢反应。心包积液反应与心脏代谢反应之间存在相关性(r=0.73 (95% CI 0.12 to 0.83),pBRAF抑制剂可获得更好的心脏反应(p=0.037),与治疗方案无关:我们开创性地在队列中同时考虑心包积液和心脏代谢反应来评估 ECD 的心脏反应,结果显示这两个指标之间存在相关性。无论采用哪种治疗方法,BRAF抑制剂都能改善心脏反应。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Heart
Heart 医学-心血管系统
CiteScore
10.30
自引率
5.30%
发文量
320
审稿时长
3-6 weeks
期刊介绍: Heart is an international peer reviewed journal that keeps cardiologists up to date with important research advances in cardiovascular disease. New scientific developments are highlighted in editorials and put in context with concise review articles. There is one free Editor’s Choice article in each issue, with open access options available to authors for all articles. Education in Heart articles provide a comprehensive, continuously updated, cardiology curriculum.
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