对系统性右心室患者可能的药物治疗效果对结局影响的倾向评分分析。
Propensity Score Analysis of Possible Medication Effects on Outcomes in Patients With Systemic Right Ventricles.
作者信息
Misra Amrit, Rodriguez-Monserrate Carla P, Gauvreau Kimberlee, Dellborg Mikael, Fusco Flavia, Gupta Tripti, Kay Joseph, Kutty Shelby, Kauling Robert M, Nicolarsen Jeremy, Roos-Hesselink Jolien, John Anitha S, Wong Joshua, Burchill Luke J, Krieger Eric V, Lubert Adam M, Gallego Pastora, Kuo Marissa, Aboulhosn Jamil, Cramer Jonathan, Antonova Petra, Baker David, Dehghani Payam, Opotowsky Alexander R, van Dissel Alexandra, Grewal Jasmine, Yeung Elizabeth, Fernandes Susan, Ginde Salil, Khairy Paul, Han Frank, Muhll Isabelle Vonder, Wilson William M, Kay William Aaron, Pylypchuk Stephen, Sarubbi Berardo, O'Donnell Clare, Rodriguez Fred, Jayadeva Pavithra S, Celermajer David S, Shah Sangeeta, Cotts Timothy, DeZorzi Christopher, Magalski Anthony, Valente Anne Marie, Broberg Craig S
机构信息
Boston Children's Hospital and Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
出版信息
JACC Adv. 2024 Dec 13;4(1):101443. doi: 10.1016/j.jacadv.2024.101443. eCollection 2025 Jan.
BACKGROUND
Patients with systemic right ventricle (SRV), either d-transposition of the great arteries following an atrial switch procedure or congenitally corrected transposition of the great arteries, develop severe right ventricular dysfunction, prompting appropriate medical therapy. However, the efficacy of beta-blockers and angiotensin receptor blockers or angiotensin-converting enzyme inhibitors (ACEI) in SRV patients is unproven.
OBJECTIVES
The objective of this study was to determine the effects of ACEI/ARB and beta-blockers on outcomes in SRV patients after accounting for likely cofounders affecting their use.
METHODS
From a retrospective, multicenter study on heart failure-related outcome in individuals with SRV, those who were taking an ACEI/ARB, beta-blocker, or both of these medication were identified. We performed a propensity analysis to match them to those not using these medications at their initial visit. Matching was based on a propensity score, which captured co-morbidities, demographics, and baseline echocardiographic parameters. Primary outcome of death, transplant, or mechanical circulatory support, and secondary outcomes of heart failure hospitalizations/atrial arrhythmias were analyzed respectively.
RESULTS
We identified 393 patients taking ACEI/ARB or beta-blocker, or taking both a beta-blocker and ACEI/ARB (62.1% male, median age 31.3 years) and 484 patients (56.4% male, median age of 26.0 years) who were neither on a beta-blocker nor on ACEI/ARB at the time of initial clinic visit. Median follow-up was ∼8 years. After propensity matching, medication use was not associated with decreased mortality, heart failure hospitalizations, or arrhythmias. Hazard ratios remained positive for beta blockers, implying potential harm rather than benefit.
CONCLUSIONS
In this large multicenter propensity-matched observational study, patients with SRV taking beta-blockers or ACEI/ARB did not have a benefit in survival or reduced hospitalization. The likelihood of demonstrating favorable effects in larger studies appears remote.
背景
系统性右心室（SRV）患者，无论是心房调转术后的右型大动脉转位，还是先天性矫正型大动脉转位，都会出现严重的右心室功能障碍，从而需要适当的药物治疗。然而，β受体阻滞剂和血管紧张素受体阻滞剂或血管紧张素转换酶抑制剂（ACEI）在SRV患者中的疗效尚未得到证实。
目的
本研究的目的是在考虑可能影响其使用的混杂因素后，确定ACEI/ARB和β受体阻滞剂对SRV患者预后的影响。
方法
在一项关于SRV患者心力衰竭相关结局的回顾性多中心研究中，确定正在服用ACEI/ARB、β受体阻滞剂或同时服用这两种药物的患者。我们进行了倾向分析，以将他们与初次就诊时未使用这些药物的患者进行匹配。匹配基于倾向评分，该评分涵盖了合并症、人口统计学和基线超声心动图参数。分别分析死亡、移植或机械循环支持的主要结局以及心力衰竭住院/房性心律失常的次要结局。
结果
我们确定了393例服用ACEI/ARB或β受体阻滞剂，或同时服用β受体阻滞剂和ACEI/ARB的患者（男性占62.1%，中位年龄31.3岁），以及484例初次就诊时既未服用β受体阻滞剂也未服用ACEI/ARB的患者（男性占56.4%，中位年龄26.0岁）。中位随访时间约为8年。经过倾向匹配后，药物使用与死亡率降低、心力衰竭住院或心律失常无关。β受体阻滞剂的风险比仍为阳性，这意味着可能有害而非有益。
结论
在这项大型多中心倾向匹配观察性研究中，服用β受体阻滞剂或ACEI/ARB的SRV患者在生存或减少住院方面没有获益。在更大规模研究中显示出有利效果的可能性似乎很小。
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