Harari Sergio, Caminati Antonella
U.O. di Pneumologia e Terapia Semi-Intensiva Respiratoria, Servizio di Fisiopatologia Respiratoria ed Emodinamica Polmonare, Ospedale San Giuseppe, MultiMedica IRCCS, Milan, Italy
U.O. di Pneumologia e Terapia Semi-Intensiva Respiratoria, Servizio di Fisiopatologia Respiratoria ed Emodinamica Polmonare, Ospedale San Giuseppe, MultiMedica IRCCS, Milan, Italy.
Eur Respir Rev. 2015 Sep;24(137):420-7. doi: 10.1183/16000617.0042-2015.
Randomised controlled clinical trials are fundamental in medicine to develop new effective drugs and new therapeutic regimens and are the strength of evidence-based medicine. These studies allow us to avoid the repetition of misleading experiences that have been reported in the past, where drugs or associations were utilised without compelling evidence and ultimately proven to be ineffective. In recent years, randomised clinical trials have been conducted and concluded for many rare diseases, including idiopathic pulmonary fibrosis. However, clinical trials do not always reflect the real-life scenario. Patients selected for clinical trials present fewer comorbidities, they fall between certain age limits, and the severity of their disease is defined; therefore, they do not always reflect the whole of the population affected by a specific disease. These are the reasons why we also need data that mirror real-life experience. The limitations that these kind of studies present are always several and the studies should be interpreted with caution, although they can fill the important gap between efficacy and effectiveness. In this article, we will review the existing clinical data on real-life treatment of idiopathic pulmonary fibrosis.
随机对照临床试验是医学领域开发新的有效药物和新治疗方案的基础,也是循证医学的优势所在。这些研究使我们能够避免重复过去报道的误导性经验,即在没有确凿证据的情况下使用药物或联合用药,最终被证明无效。近年来,针对包括特发性肺纤维化在内的许多罕见疾病开展并完成了随机临床试验。然而,临床试验并不总是反映现实生活中的情况。入选临床试验的患者合并症较少,年龄在一定范围内,且疾病严重程度有明确界定;因此,他们并不总是能反映受特定疾病影响的全部人群。这就是为什么我们还需要反映现实生活经验的数据。这类研究存在的局限性总是多方面的,尽管它们可以填补疗效和有效性之间的重要差距,但对研究结果的解读仍应谨慎。在本文中,我们将回顾关于特发性肺纤维化现实生活治疗的现有临床数据。