Department of Respiratory Medicine, Nagasaki University Graduate School of Biomedical Sciences, Nagasaki, Japan
Research Institute for Diseases of the Chest, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
BMJ Open. 2021 Jun 29;11(6):e047249. doi: 10.1136/bmjopen-2020-047249.
Idiopathic pulmonary fibrosis (IPF) is a fibrotic disease of unknown aetiology with a poor prognosis. Several clinical trials of nintedanib in patients with IPF have reported its inhibitory effect on reduced lung function, incidence of acute exacerbation of IPF and worsened health-related quality of life. Although nintedanib has a manageable safety and tolerability profile over long-term use, it was discontinued in over 20% of patients because of adverse events such as diarrhoea and liver dysfunction. This might explain why nintedanib use in patients with IPF is not widespread, especially among patients with early-stage IPF. In the present study, we aimed to clarify the efficacy, safety and tolerability of nintedanib in patients with stage I/II IPF, based on the Japanese IPF disease severity staging classification system.
This is an ongoing, prospective, multicentre observational cohort study of patients with stage I/II IPF who will start receiving nintedanib. Totally, 215 patients at 35 sites in Kyushu and Okinawa, Japan will be enrolled and followed up for 3 years. Nintedanib therapy would be initiated at the discretion of the investigator. The primary endpoint, change in forced vital capacity (FVC) at 156 weeks, will be shown as the mean change in FVC from baseline to week 156 with 95% CIs estimated using the Wald method. The safety endpoint-occurrence of adverse events-will be assessed in each system organ class/preferred term.
The study protocol and informed consent documents were approved by the Institutional Review Board at Nagasaki University Hospital (approval number 19102146) and each participating site. Written informed consent was obtained from all participants. Patient recruitment has begun. The results will be disseminated through scientific peer-reviewed publications and national and international conferences.
UMIN000038192.
特发性肺纤维化（IPF）是一种病因不明的纤维化疾病，预后较差。几项尼达尼布治疗 IPF 患者的临床试验报告了其对肺功能下降、IPF 急性加重发生率和健康相关生活质量恶化的抑制作用。尽管尼达尼布在长期使用中具有可管理的安全性和耐受性，但由于腹泻和肝功能障碍等不良事件，超过 20%的患者停止了使用。这可能解释了为什么尼达尼布在 IPF 患者中的使用并不广泛，尤其是在早期 IPF 患者中。在本研究中，我们旨在根据日本特发性肺纤维化疾病严重程度分期分类系统，阐明尼达尼布在 I/II 期特发性肺纤维化患者中的疗效、安全性和耐受性。
这是一项正在进行的、前瞻性、多中心观察性队列研究，研究对象为开始接受尼达尼布治疗的 I/II 期特发性肺纤维化患者。日本九州和冲绳的 35 个地点共招募 215 名患者，随访 3 年。尼达尼布治疗将由研究者自行决定开始。主要终点为 156 周时用力肺活量（FVC）的变化，将使用 Wald 法估计从基线到第 156 周时 FVC 的平均变化，并给出 95%CI。安全性终点——不良事件的发生——将根据每个系统器官类别/首选术语进行评估。
该研究方案和知情同意文件已获得长崎大学医院机构审查委员会（批准号 19102146）和每个参与地点的批准。所有参与者均签署了书面知情同意书。患者招募工作已经开始。研究结果将通过科学同行评审的出版物和国家及国际会议进行传播。
UMIN000038192。
