Gu Li, Li Yuan Yuan, Gu Ling, Xie Liang, Liu Han Min
Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China.
Key Laboratory of Birth Defects and Related Diseases of Women and Children (Sichuan University), Ministry of Education, West China Second University Hospital, Sichuan University, Chengdu, China.
Front Pediatr. 2020 Mar 31;8:106. doi: 10.3389/fped.2020.00106. eCollection 2020.
As the most common types of pulmonary arterial hypertension (PAH) in childhood, the similarities and differences in clinical characteristics and prognosis between idiopathic PAH (IPAH) and PAH associated with congenital heart disease (PAH-CHD) are not well-known. This study describes and compares clinical features of pediatric IPAH and PAH-CHD in a single center of China during an 11-year period and explores the prognostic factors. Twenty-five children with IPAH and 60 children with PAH-CHD, diagnosed in West China Second Hospital of Sichuan University from January 2008 to December 2018, were chosen as study objects. The follow-up deadline was June 2019, and the end-point was all-cause death. The baseline data, results of auxiliary examinations, treatment strategies, and follow-up outcomes were recorded and compared between IPAH and PAH-CHD patients to explore the similarities, differences, and prognostic factors. The median diagnostic age for PAH-CHD patients was 2.3 years, which was younger than IPAH patients (7.3 years; = 0.009). Sixty-eight percent of the IPAH patients presented with exercise-induced symptoms at initial diagnosis, whereas 58.3% of the PAH-CHD patients were asymptomatic ( < 0.001). Sixty percent of the IPAH patients were in World Health Organization-functional class (WHO-FC) III or IV, which was significantly worse than those of the PAH-CHD patients ( = 0.002). The incidence of ST-segment and T-wave (ST-T) change in children with IPAH (76.0%) was significantly higher than that (28.3%) in children with PAH-CHD ( < 0.001). Mean corpuscular volume (MCV), mean platelet volume (MPV), and platelet distribution width were larger in IPAH patients than those in PAH-CHD patients ( < 0.01). The 1-, 3-, and 5-year survival rates of IPAH and PAH-CHD patients were 53.5, 46.5, and 31.2% and 96.5, 93.1, and 77.6%, respectively ( < 0.05). WHO-FC III-IV [relative risk (RR) = 2.750, = 0.008] and higher MPV (RR = 1.657, = 0.006) predicted poor prognosis for pediatric PAH. We showed that there are more differences than similarities between IPAH and PAH-CHD patients in clinical characteristics. PAH-CHD patients have a better prognosis than IPAH patients. WHO-FC III-IV and higher MPV at initial diagnosis are independent risk factors for poor prognosis.
作为儿童期最常见的肺动脉高压（PAH）类型，特发性PAH（IPAH）与先天性心脏病相关PAH（PAH-CHD）在临床特征和预后方面的异同尚不清楚。本研究描述并比较了中国某单一中心11年间儿童IPAH和PAH-CHD的临床特征，并探讨了预后因素。选取2008年1月至2018年12月在四川大学华西第二医院诊断的25例IPAH患儿和60例PAH-CHD患儿作为研究对象。随访截止时间为2019年6月，终点为全因死亡。记录并比较IPAH和PAH-CHD患者的基线数据、辅助检查结果、治疗策略及随访结局，以探讨其异同及预后因素。PAH-CHD患者的中位诊断年龄为2.3岁，低于IPAH患者（7.3岁；P = 0.009）。68%的IPAH患者在初诊时出现运动诱发症状，而58.3%的PAH-CHD患者无症状（P < 0.001）。60%的IPAH患者处于世界卫生组织功能分级（WHO-FC）III级或IV级，明显差于PAH-CHD患者（P = 0.002）。IPAH患儿ST段和T波（ST-T）改变的发生率（76.0%）显著高于PAH-CHD患儿（28.3%）（P < 0.001）。IPAH患者的平均红细胞体积（MCV）、平均血小板体积（MPV）和血小板分布宽度大于PAH-CHD患者（P < 0.01）。IPAH和PAH-CHD患者1年、3年和5年生存率分别为53.5%、46.5%和31.2%以及96.5%、93.1%和77.6%（P < 0.05）。WHO-FC III-IV级[相对危险度（RR）= 2.750，P = 0.008]和较高的MPV（RR = 1.657，P = 0.006）预示儿童PAH预后不良。我们发现，IPAH和PAH-CHD患者在临床特征上的差异多于相似之处。PAH-CHD患者的预后优于IPAH患者。初诊时WHO-FC III-IV级和较高的MPV是预后不良的独立危险因素。
