AMIODARONE THERAPY IN PATIENTS WITH HCM AND REFRACTORY CARDIAC SYMPTOMS

胺碘酮治疗 HCM 和难治性心脏症状患者

• 批准号: 3966723
• 负责人: M B LEON
• 金额: --
• 依托单位: NATIONAL HEART, LUNG, AND BLOOD INSTITUTE
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3966723
• 关键词: cardiotonic agents; drug administration routes; heart disorder chemotherapy; heart function; heart output disorder; human subject; human therapy evaluation; hypertrophic myocardiopathy

An ongoing clinical trial was formulated to assess therapeutic efficacy of high dose oral amiodarone in patients with hypertrophic cardiomyopathy and refractory cardiac symptoms compared with standard pharmacologic agents and to characterize hemodynamic and left ventricular functional responses associated with amiodarone to help define mechanisms of action. Thusfar, 40 patients with refractory cardiac symptoms on standard medical therapies have been studied as inpatients on the Cardiology Service and have been seen as outpatients at 2 months, 6 months, and 1 year after instituting oral amiodarone treatment. After stopping medications for several days in the hospital, high dose oral amiodarone resulted in beneficial clinical responses (defined as at least a 50% improvement in exercise performance compared with no medication values and a decrease in cardiac symptoms by at least one functional class) in 68% of the patient group. During chronic lower dose outpatient amiodarone treatment, improvement was generally maintained (63% clinical responders); some patients manifested further symptom benefit, and in others symptoms were worse compared with higher dose acute treatment. As might be expected in this group of severely symptomatic patients unresponsive to standard pharmacologic therapy, many patients failed to experience maintained clinical benefit. Nonetheless, 45% of the total patient cohort are still receiving amiodarone for an average follow-up of 14 months and have continued to describe persistent improvement in symptoms. One half of all patients with left ventricular outflow tract obstruction who were deemed operative candidates prior to initiating amiodarone have experienced sufficient symptomatic improvement so that surgery could be deferred. From this ongoing clinical trial we conclude that high dose oral amiodarone improves exercise capacity and reduces cardiac symptoms in the majority of patients with hypertrophic cardiomyopathy who have been refractory to standard medical therapy and long-term clinical follow-up indicates a modest diminution in clinical efficacy although almost one half of all patients have continued treatment after the first year with continued symptomatic improvement.

一项正在进行的临床试验旨在评估 大剂量口服胺碘酮治疗肥厚型心肌病 与标准药物相比，难治性心脏症状， 表征血流动力学和左心室功能反应 与胺碘酮相关，以帮助确定作用机制。 到目前为止， 40例接受标准药物治疗的难治性心脏症状患者 作为心脏病服务的住院患者进行了研究， 在开始治疗后2个月、6个月和1年时作为门诊患者观察 口服胺碘酮治疗 在停止药物治疗几天后， 医院，大剂量口服胺碘酮产生有益的临床 反应（定义为运动表现至少改善50%） 与未用药值相比， 至少有一个功能分类），占68%。 在慢性 低剂量门诊胺碘酮治疗，改善一般 维持（63%临床应答者）;一些患者表现出进一步的 症状受益，而在其他症状更差相比，更高 剂量急性治疗。 正如在这组严重的 对标准药物治疗无反应的症状性患者，许多 患者未能获得持续的临床获益。 尽管如此， 45%的患者队列仍在接受胺碘酮治疗， 平均随访14个月，并继续描述持续性 症状改善。 一半的左心室患者 流出道梗阻，在手术前被认为是手术候选人， 开始胺碘酮治疗的患者症状得到充分改善 这样手术就可以推迟了 从这项正在进行的临床试验中， 得出结论，高剂量口服胺碘酮可提高运动能力， 减少大多数肥厚型心肌病患者的心脏症状 对标准药物治疗无效的心肌病患者， 长期临床随访表明， 尽管几乎一半的患者继续接受治疗， 第一年后，症状持续改善。