Kaemmerer H, Gorenflo M, Hoeper M, Huscher D, Ewert P, Pittrow D
Klinik für Kinderkardiologie und angeborene Herzfehler, Deutsches Herzzentrum München, Klinik an der Technischen Universität München.
Dtsch Med Wochenschr. 2013 Jun;138(23):1247-52. doi: 10.1055/s-0033-1343189. Epub 2013 May 29.
Defects of the heart and associated large vessels (CHD) are among the most frequent congenital anomalies. Owing to improved interdisciplinary management, about 90% of CHD patients reach adulthood. Up to 10% maintain or newly develop pulmonary arterial hypertension (PAH) over time, which impairs exercise tolerance and prognosis. Data on the health care situation of patients with PAH-CHD are limited. The ongoing Prospective Registry of Newly Initiated Therapies for Pulmonary Hypertension (COMPERA, ClinTrials.gov Identifier NCT01347216) prospectively documents adult patients with all forms of pulmonary hypertension, if treated with PAH drugs (mean follow-up 40 months). As of 16 November 2012, 8% of the 3642 patients in the database had PAH-CHD. Of the latter, 104 were documented in great detail in specific CHD report forms. These patients were on average 39 years old, men in 39%, had a mean 6-minute walk distance of 370 ± 102 meters, and were in NYHA functional class I/II in 39%, III in 59%, und IV in 3%. Mean quality of life on the 100-point visual analogue scale (EQ-5 D) was 51. PAH-CHD patients received monotherapy in 80%, combination therapy in 9%, and no PAH drugs in 11%. Only 20% were on oral anticoagulation (OAC). Mean 4-year survival in incident patients (PAH-CHD diagnosis after start of the registry in 2007) was 79%, compared with 72% in patients with idiopathic PAH (IPAH). According to these registry data, patients with PAH-CHD have impaired exercise capacity, and substantially reduced quality of life. They receive combination therapy or OAC, respectively, less frequently than IPAH patients, however, their survival rate is higher.
心脏及相关大血管缺陷（CHD）是最常见的先天性异常之一。由于跨学科管理的改善，约90%的CHD患者能够成年。随着时间的推移，高达10%的患者会维持或新发生肺动脉高压（PAH），这会损害运动耐量和预后。关于PAH-CHD患者的医疗状况的数据有限。正在进行的肺动脉高压新启动治疗前瞻性注册研究（COMPERA，临床试验.gov标识符NCT01347216）前瞻性记录了所有形式肺动脉高压的成年患者，前提是接受PAH药物治疗（平均随访40个月）。截至2012年11月16日，数据库中3642例患者中有8%患有PAH-CHD。其中，104例在特定的CHD报告表中有详细记录。这些患者平均年龄为39岁，男性占39%，平均6分钟步行距离为370±102米，纽约心脏协会（NYHA）功能分级为I/II级的占39%，III级的占59%，IV级的占3%。100分视觉模拟量表（EQ-5 D）上的平均生活质量为51分。PAH-CHD患者80%接受单一疗法，9%接受联合疗法，11%未接受PAH药物治疗。只有20%的患者接受口服抗凝治疗（OAC）。发病患者（2007年注册研究开始后诊断为PAH-CHD）的4年平均生存率为79%，而特发性PAH（IPAH）患者为72%。根据这些注册研究数据，PAH-CHD患者运动能力受损，生活质量大幅降低。他们接受联合疗法或OAC的频率分别低于IPAH患者，然而，他们的生存率更高。
