Novel therapy for idiopathic pulmonary fibrosis-How to evaluate the efficacy?

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As disease concepts have changed, the treatment of idiopathic interstitial pneumonias (IIPs) has become focused on idiopathic pulmonary fibrosis (IPF), which has the most adverse prognosis. Since 1998, large-scale multicenter trials have evolved, and clinical trials using novel therapeutic drugs such as interferon-γ in US and N-acetylcysteine in Europe and pirfenidone in Japan, have been organized. Most of these trials were evaluated using 1-year FVC or VC changes as the therapy endpoint, with significant differences obtained for non-progressive stage IPF. However, no significant differences were obtained in the progressive stages for any of the trials, and whether or not they contribute to improvement in life prognosis has been left to the post-market evaluation. A debate has begun in Europe and the US as to the value of assessment evaluation indicator done during activity since constant speed treadmill walking tests were introduced in the pirfenidone clinical trials carried out in Japan. Until now, therapy evaluations for IIPs have been compared using diagnostic imaging, pulmonary function tests, and pathology. In the future, therapeutic efficacy and prognosis will be discussed in terms not only of resting respiratory function evaluation, but also of systemic evaluation using exercise endurance, which is also recognized as an ADL evaluation indicator. In recent years, acute exacerbation of IPF has been the focus of global concern. A joint perspective by ATS/ERS was published, and standardized diagnosis and treatment have been provided. Acute exacerbation is an adverse prognosis factor, as are lung cancer complications, and treatments for improving life prognosis are being explored. This paper presents therapeutic drugs and treatments whose introduction is awaited, and discusses the relationship between indicators of therapy evaluation and disease concepts. © 2008 Elsevier Ltd. All rights reserved.




Azuma, A., & Usuki, J. (2008). Novel therapy for idiopathic pulmonary fibrosis-How to evaluate the efficacy? Respiratory Medicine CME, 1(2), 75–81.

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