儿童先天性心脏病术后肺动脉高压与特发性肺动脉高压的临床特点及预后

Clinical characteristics and mid to long term outcomes in children with pulmonary arterial hypertension after repair of congenital heart disease and idiopathic pulmonary arterial hypertension

目的:因特发性肺动脉高压(IPAH)及先天性心脏病术后肺动脉高压(CD)患者均不存在心内分流,解剖、病生理及血流动力学方面存在相似性,描述分析两类患者的临床特征、功能分级、中远期结局,并分析影响预后的因素。方法:回顾性分析2005年6月至2018年7月,于北京安贞医院小儿心脏中心住院的儿童患者的预后转归情况。结果:共入组儿童222例,包括123例IPAH和99例CD患者。诊断中位年龄为11.2(7.4, 14.2)岁。末次随访时共160例(72.1%)患者接受PAH靶向药物治疗,68例(30.1%)患者应用联合治疗。CD患者的心功能及运动能力优于IPAH患者。两组患者血流动力学参数相似,IPAH的肺血管阻力指数较低,心指数稍高。随访中位时间为3.9(1.9, 6.2)年,随访期间44例患者死亡(IPAH 39例,CD 5例),右心衰竭和心源性猝死为最常见死亡原因。IPAH和CD患儿1年、3年及5年的生存率分别为89.2%,74.7%,63.2%及95.7%,95.7%,93.7%(HR=4.2,95%CI:2.3~7.6,P<0.0001)。结论:儿童IPAH和CD患者中远期预后有显著差异。死亡率在IPAH患儿中较高,且与较晚的诊断年龄有关。CD患儿的预后情况与基线状态下心功能分级相关。诊断后早期积极应用靶向药物治疗对预后有益。

Objective: Pulmonary arterial hypertension after repair of congenital heart disease(CD) and idiopathic pulmonary arterial hypertension(IPAH) both do not have cardiac shunts. They have similarities in anatomy, pathophysiology, and hemodynamics. To analysis the clinical, functional, mid to long term outcomes and analysis risk factors affected prognosis. Methods: Pediatric patients were included from Jun 2005 to Jul 2018 who hospitalized in pediatric cardiology department in Beijing Anzhen Hospital. Clinical characteristics and outcomes were analyzed retrospectively. Results: A total of 222 patients were enrolled including 123 IPAH and 99 CD patients. Age at diagnosis was 11.2(7.4, 14.2) years. 160 patients(72.1%) were treated with PAH-targeted medications at last follow-up time. 68(30.1%) were treated by combination therapies. Cardiac function and exercise capacity of CD patients are superior to those of IPAH patients while hemodynamic parameters of the two groups were similar. The pulmonary vascular resistance index(PVRI) of IPAH was lower and the cardiac index(CI) was slightly higher. Over the follow-up time of 3.9(1.9, 6.2) years, 44 patients died(39 in IPAH and 5 in CD). Right heart failure and cardiac sudden death were the most common cause of death. Survival at 1-, 3-, and 5-year was 95.7%, 95.7% and 93.7% for CD and 89.2%, 74.7% and 63.2% for IPAH(HR=4.2, 95% CI: 2.3-7.6, P<0.0001). Conclusions: Mid to long term outcomes in children with IPAH and CD were significantly different. Mortality was higher in children with IPAH and was related to later age at diagnosis. Prognosis of children with CD was related to functional class at baseline. Our findings support such an aggressive treatment approach in both IPAH and CD patients.