2018 年法国心脏分配方案实施后，罕见心力衰竭病因患者的候补者结局。
Waitlist Outcomes in Candidates With Rare Causes of Heart Failure After Implementation of the 2018 French Heart Allocation Scheme.
机构信息
Agence de la Biomédecine, Saint Denis La Plaine Cedex, France (C.L., C.C., C.J., F.K., R.D.).
Department of Cardiac and Thoracic Surgery, Cardiology Institute, Pitié Salpêtrière Hospital (G.C.), Assistance Publique-Hôpitaux de Paris (AP-HP), Sorbonne University Medical School, France.
出版信息
Circ Heart Fail. 2024 Feb;17(2):e010837. doi: 10.1161/CIRCHEARTFAILURE.123.010837. Epub 2024 Feb 1.
BACKGROUND
In 2018, an algorithm-based allocation system for heart transplantation (HT) was implemented in France. Its effect on access to HT of patients with rare causes of heart failure (HF) has not been assessed.
METHODS
In this national study, including adults listed for HT between 2018 and 2020, we analyzed waitlist and posttransplant outcomes of candidates with rare causes of HF (restrictive cardiomyopathy [RCM], hypertrophic cardiomyopathy, and congenital heart disease). The primary end point was death on the waitlist or delisting for clinical deterioration. Secondary end points included access to HT and posttransplant mortality. The cumulative incidence of waitlist mortality estimated with competing risk analysis and incidence of transplantation were compared between diagnosis groups. The association of HF cause with outcomes was determined by Fine-Gray or Cox models.
RESULTS
Overall, 1604 candidates were listed for HT. At 1 year postlisting, 175 patients met the primary end point and 1040 underwent HT. Candidates listed for rare causes of HF significantly differed in baseline characteristics and had more frequent score exceptions compared with other cardiomyopathies (31.3%, 32.0%, 36.4%, and 16.7% for patients with hypertrophic cardiomyopathy, RCM, congenital heart disease, and other cardiomyopathies). The cumulative incidence of death on the waitlist and probability of HT were similar between diagnosis groups (=0.17 and 0.40, respectively). The adjusted risk of death or delisting for clinical deterioration did not significantly differ between candidates with rare and common causes of HF (subdistribution hazard ratio (HR): hypertrophic cardiomyopathy, 0.51 [95% CI, 0.19-1.38]; =0.18; RCM, 1.04 [95% CI, 0.42-2.58]; =0.94; congenital heart disease, 1.82 [95% CI, 0.78-4.26]; =0.17). Similarly, the access to HT did not significantly differ between causes of HF (hypertrophic cardiomyopathy: HR, 1.18 [95% CI, 0.92-1.51]; =0.19; RCM: HR, 1.19 [95% CI, 0.90-1.58]; =0.23; congenital heart disease: HR, 0.76 [95% CI, 0.53-1.09]; =0.14). RCM was an independent risk factor for 1-year posttransplant mortality (HR, 2.12 [95% CI, 1.06-4.24]; =0.03).
CONCLUSIONS
Our study shows equitable waitlist outcomes among HT candidates whatever the indication for transplantation with the new French allocation scheme.
背景
2018 年，法国实施了一种基于算法的心脏移植（HT）分配系统。其对心力衰竭（HF）罕见病因患者接受 HT 的影响尚未得到评估。
方法
在这项全国性研究中，纳入了 2018 年至 2020 年期间接受 HT 名单的成年人，我们分析了 HF 罕见病因（限制型心肌病[RCM]、肥厚型心肌病和先天性心脏病）患者的候补者和移植后结局。主要终点是等待名单上的死亡或因临床恶化而被除名。次要终点包括获得 HT 和移植后的死亡率。用竞争风险分析估计等待名单死亡率的累积发生率，并比较不同诊断组之间的移植发生率。通过 Fine-Gray 或 Cox 模型确定 HF 病因与结局的关系。
结果
总体而言，有 1604 名候选者被列入 HT 名单。在列入名单后的 1 年，175 名患者达到了主要终点，1040 名患者接受了 HT。与其他心肌病相比，HF 罕见病因的候补者在基线特征和更频繁的评分异常方面存在显著差异（肥厚型心肌病、RCM、先天性心脏病和其他心肌病患者的发生率分别为 31.3%、32.0%、36.4%和 16.7%）。各组之间等待名单上的死亡率和 HT 的累积发生率相似（=0.17 和 0.40）。HF 罕见和常见病因患者的死亡或因临床恶化而被除名的调整风险比（subdistribution hazard ratio，HR）无显著差异（肥厚型心肌病，0.51[95%CI，0.19-1.38]；=0.18；RCM，1.04[95%CI，0.42-2.58]；=0.94；先天性心脏病，1.82[95%CI，0.78-4.26]；=0.17）。同样，HF 病因之间获得 HT 的情况也无显著差异（肥厚型心肌病：HR，1.18[95%CI，0.92-1.51]；=0.19；RCM：HR，1.19[95%CI，0.90-1.58]；=0.23；先天性心脏病：HR，0.76[95%CI，0.53-1.09]；=0.14）。RCM 是移植后 1 年死亡的独立危险因素（HR，2.12[95%CI，1.06-4.24]；=0.03）。
结论
我们的研究表明，在新的法国分配方案下，无论移植的适应证如何，HT 候补者的等待名单结局都是公平的。
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