Eight-Year Outcomes of Cardiosphere-Derived Cells in Single Ventricle Congenital Heart Disease.

IF 5 1区医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS

Journal of the American Heart Association Pub Date : 2024-11-19 Epub Date: 2024-11-11 DOI:10.1161/JAHA.124.038137

Kenta Hirai, Ryusuke Sawada, Tomohiro Hayashi, Toru Araki, Naomi Nakagawa, Maiko Kondo, Kenji Yasuda, Takuya Hirata, Tomoyuki Sato, Yuki Nakatsuka, Michihiro Yoshida, Shingo Kasahara, Kenji Baba, Hidemasa Oh

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引用次数: 0

Abstract

Background: Cardiosphere-derived cell (CDC) infusion was associated with better clinical outcomes at 2 years in patients with single ventricle heart disease. The current study investigates time-to-event outcomes at 8 years.

Methods and results: This cohort enrolled patients with single ventricles who underwent stage 2 or stage 3 palliation from January 2011 to January 2015 at 8 centers in Japan. The primary outcomes were time-dependent CDC treatment effects on death and late complications during 8 years of follow-up, assessed by restricted mean survival time. Among 93 patients enrolled (mean age, 2.3±1.3 years; 56% men), 40 received CDC infusion. Overall survival for CDC-treated versus control patients did not differ at 8 years (hazard ratio [HR], 0.60 [95% CI, 0.21-1.77]; P=0.35). Treatment effect had nonproportional hazards for death favoring CDCs at 4 years (restricted mean survival time difference +0.33 years [95% CI, 0.01-0.66]; P=0.043). In patients with heart failure with reduced ejection fraction, CDC treatment effect on survival was greater over 8 years (restricted mean survival time difference +1.58 years [95% CI, 0.05-3.12]; P=0.043). Compared with control participants, CDC-treated patients showed lower incidences of late failure (HR, 0.45 [95% CI, 0.21-0.93]; P=0.027) and adverse events (subdistribution HR, 0.50 [95% CI, 0.27-0.94]; P=0.036) at 8 years.

Conclusions: By 8 years, CDC infusion was associated with lower hazards of late failure and adverse events in single ventricle heart disease. CDC treatment effect on survival was notable by 4 years and showed a durable clinical benefit in patients with heart failure with reduced ejection fraction over 8 years.

Registration: URL: https://www.clinicaltrials.gov; Unique identifiers: NCT01273857 and NCT01829750.

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单心室先天性心脏病中的心球衍生细胞八年后的疗效

背景：单心室心脏病患者输注心球衍生细胞（CDC）2年后可获得更好的临床疗效。本研究调查了8年后的时间到事件结果：该队列纳入了 2011 年 1 月至 2015 年 1 月期间在日本 8 个中心接受 2 期或 3 期姑息治疗的单心室患者。主要结果是随访 8 年期间 CDC 治疗对死亡和晚期并发症的时间依赖性影响，通过限制性平均生存时间进行评估。在入组的 93 名患者中（平均年龄为 2.3±1.3 岁；56% 为男性），40 人接受了 CDC 输注。经 CDC 治疗的患者与对照组患者的 8 年总生存率没有差异（危险比 [HR]，0.60 [95% CI，0.21-1.77]；P=0.35）。治疗效果在 4 年时有利于 CDC 的死亡具有非比例危害（限制性平均生存时间差 +0.33 年 [95% CI，0.01-0.66]；P=0.043）。在射血分数降低的心力衰竭患者中，CDC治疗对8年生存率的影响更大（限制性平均生存时间差+1.58年[95% CI，0.05-3.12]；P=0.043）。与对照组参与者相比，接受CDC治疗的患者在8年后的晚期失败（HR，0.45 [95% CI，0.21-0.93]；P=0.027）和不良事件（亚分布HR，0.50 [95% CI，0.27-0.94]；P=0.036）发生率较低：结论：单心室心脏病患者输注 CDC 8 年后，晚期衰竭和不良事件的发生率较低。4年前，CDC治疗对生存期的影响显著，8年后，射血分数降低的心力衰竭患者可获得持久的临床获益：URL：https://www.clinicaltrials.gov；唯一标识符：NCT01273857和NCT01829750。

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来源期刊

Journal of the American Heart Association CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

9.40

自引率

1.90%

发文量

1749

审稿时长

12 weeks

期刊介绍： As an Open Access journal, JAHA - Journal of the American Heart Association is rapidly and freely available, accelerating the translation of strong science into effective practice. JAHA is an authoritative, peer-reviewed Open Access journal focusing on cardiovascular and cerebrovascular disease. JAHA provides a global forum for basic and clinical research and timely reviews on cardiovascular disease and stroke. As an Open Access journal, its content is free on publication to read, download, and share, accelerating the translation of strong science into effective practice.