Murakami Noriko, Asano Daiki, Tokushige Natsuko, Omura Junichi, Watanabe Megumi, Nomura Seitaro, Kitaoka Hiroaki, Tamura Yuichi
PHA Japan, 5-8-A-209 Tsukimino, Yamato-shi, Kanagawa, 242-0002, Japan.
Medical Affairs, Janssen Pharmaceutical K.K., 3-5-2 Nishikanda, Chiyoda-ku, Tokyo, 101-0065, Japan.
Pulm Ther. 2025 Jun;11(2):249-268. doi: 10.1007/s41030-025-00290-6. Epub 2025 Mar 18.
Pulmonary hypertension (PH) is a rare and severe disorder that significantly affects patients' lives. However, a comprehensive picture of the diagnosis and treatment of this condition in Japan remains unclear. This study aimed to elucidate these aspects by conducting a nationwide survey targeting patients with PH and treating physicians.
A cross-sectional survey was conducted among 160 patients with PH (119 with pulmonary arterial hypertension [PAH] and 41 with chronic thromboembolic pulmonary hypertension [CTEPH]), of whom 121 were female (75.6%), and 211 physicians across Japan. The questionnaires assessed patients' diagnostic journey, employment status, communication with physicians regarding treatment goals, health-related quality of life (HRQoL), and medication adherence.
Patients visited a mean of 2.3 medical facilities before receiving a PH diagnosis (PAH patients: 2.2 visits; CTEPH patients: 2.3 visits), with a mean time from symptom onset to diagnosis of 18.0 months (PAH: 20.2 months; CTEPH: 12.2 months). Employment and school attendance rates declined from 68.8% before diagnosis to 44.4% immediately after diagnosis, and further to 36.9% at the time of the survey. Discrepancies in communication about treatment goals were observed between patients and physicians, particularly in patients with CTEPH (82.9% of patients reported such discussions vs. 41.2% of treating physicians). Median HRQoL scores, as assessed by the emPHasis-10 questionnaire, indicated impairment (PAH: 21.5; CTEPH: 18.0), which worsened with increasing disease severity.
This nationwide study provides a comprehensive overview of the challenges faced by patients with PH in Japan. The findings suggest the essential need for earlier diagnosis, support for employment and education among patients, and improved patient-physician communication to reduce the burden of PH and enhance patient outcomes. Graphical abstract avaliable for this article.
肺动脉高压（PH）是一种罕见且严重的疾病，严重影响患者的生活。然而，日本对这种疾病的诊断和治疗的全面情况仍不清楚。本研究旨在通过针对肺动脉高压患者和治疗医生进行全国性调查来阐明这些方面。
对160例肺动脉高压患者（119例动脉性肺动脉高压[PAH]和41例慢性血栓栓塞性肺动脉高压[CTEPH]）以及日本各地的211名医生进行了横断面调查。问卷评估了患者的诊断过程、就业状况、与医生关于治疗目标的沟通、健康相关生活质量（HRQoL）和药物依从性。
患者在被诊断为肺动脉高压之前平均就诊2.3个医疗机构（PAH患者：2.2次就诊；CTEPH患者：2.3次就诊），从症状出现到诊断的平均时间为18.0个月（PAH：20.2个月；CTEPH：12.2个月）。就业和上学出勤率从诊断前的68.8%下降到诊断后立即的44.4%，在调查时进一步下降到36.9%。患者和医生之间在治疗目标沟通方面存在差异，特别是在CTEPH患者中（82.9%的患者报告进行了此类讨论，而治疗医生中这一比例为41.2%）。根据emPHasis - 10问卷评估的HRQoL中位数得分表明存在损害（PAH：21.5；CTEPH：18.0），且随着疾病严重程度的增加而恶化。
这项全国性研究全面概述了日本肺动脉高压患者面临的挑战。研究结果表明迫切需要早期诊断、支持患者就业和教育以及改善医患沟通，以减轻肺动脉高压的负担并改善患者预后。本文提供图形摘要。
