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Percutaneous Transluminal Septal Reduction for Hypertrophic Obstructive Cardiomyopathy: Report from an International Pilot Study

机译:经皮腔内减压术治疗肥厚性梗阻性心肌病:一项国际试验研究报告

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摘要

Assessing the effectiveness of newer treatments for rare diseases can be challenging because of the small number of patients treated at individual centers. We enrolled patients undergoing percutaneous transluminal septal myocardial ablation (PTSMA) for hypertrophic obstructive cardiomyopathy (HOCM) at five international centers (1 Japan, 2 United Kingdom, and 2 United States). Our study group developed standard data definitions regarding clinical symptom severity, previous HOCM treatment, procedure status, and outcome, and entered patient data directly into a shared, web-based registry system. In the first 10 months of 1998, 51 patients were enrolled in our registry, with 47 ultimately receiving the PTSMA procedure. Although HOCM is consider a single disease, there were significant differences among centers in patient characteristics (age, gender, and family history of HOCM), symptom severity, diagnostic techniques (measurements taken after provocation), and treatment (amount of alcohol used, timing of injection, and number of branches attempted).
机译:由于在各个中心接受治疗的患者人数很少,因此评估针对罕见疾病的新疗法的有效性可能具有挑战性。我们在五个国际中心(1个日本,2个英国和2个美国)对因肥厚性梗阻性心肌病(HOCM)接受经皮经皮腔内心肌切除术(PTSMA)的患者进行了登记。我们的研究小组开发了有关临床症状严重程度,以前的HOCM治疗,手术状态和结果的标准数据定义,并将患者数据直接输入到基于Web的共享注册表系统中。在1998年的前10个月中,有51位患者参加了我们的注册研究,其中47位最终接受了PTSMA手术。尽管HOCM被认为是单一疾病,但各中心在患者特征(年龄,性别和HOCM家族史),症状严重程度,诊断技术(挑衅后采取的措施)和治疗(所用酒精量,时机)方面存在显着差异数量和尝试的分支数)。

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