Richeldi Luca, Azuma Arata, Cottin Vincent, Hesslinger Christian, Stowasser Susanne, Valenzuela Claudia, Wijsenbeek Marlies S, Zoz Donald F, Voss Florian, Maher Toby M
Unità Operativa Complessa di Pneumologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Università Cattolica del Sacro Cuore, Rome, Italy.
Pulmonary Medicine & Oncology, Nippon Medical School, Tokyo, Japan.
J Comp Eff Res. 2022 Dec 20;12(1). doi: 10.2217/cer-2022-0142.
WHAT IS THIS SUMMARY ABOUT?: This plain language summary describes the main findings from a trial in people with idiopathic pulmonary fibrosis (also called IPF) that was recently published in the . IPF is a rare disease, where the lungs become more and more scarred, with breathing and oxygen uptake becoming increasingly difficult. This trial looked at the medication BI 1015550 as a potential treatment for IPF. It compared BI 1015550 to placebo (a dummy drug that does not contain any active ingredients) to investigate the effectiveness of the drug in treating people with IPF. The study also looked at the additional medical issues (referred to as adverse events) reported during the study. Some participants took approved treatments to reduce scarring (nintedanib or pirfenidone), and some did not.
WHAT WERE THE RESULTS?: Overall, 147 people with IPF from 22 countries took part in the trial. The results showed that BI 1015550 prevented lung function from decreasing in people with IPF. There was no difference in the percentage of patients with medical issues rated as severe by the study physician with BI 1015550 or placebo. However, more people treated with BI 1015550 had diarrhoea. Among those treated with BI 1015550, 13 participants stopped their treatment due to medical issues, whereas treatment was not stopped due to medical issues for any participants treated with placebo.
WHAT DO THE RESULTS MEAN?: These results provide evidence that BI 1015550 prevents lung function from worsening in people with IPF. Further clinical studies will be conducted in the future to test BI 1015550 in a larger group of people with IPF and other forms of lung scarring that get worse over time, and for a longer time period.
本摘要的内容是什么?:这份通俗易懂的摘要描述了一项针对特发性肺纤维化(也称为IPF)患者的试验的主要研究结果,该试验最近发表于……。IPF是一种罕见疾病,肺部会逐渐形成越来越多的瘢痕,呼吸和氧气摄取变得愈发困难。该试验研究了药物BI 1015550作为IPF潜在治疗方法的效果。它将BI 1015550与安慰剂(一种不含任何活性成分的虚拟药物)进行比较,以研究该药物治疗IPF患者的有效性。该研究还观察了研究期间报告的其他医学问题(称为不良事件)。一些参与者接受了批准的治疗以减少瘢痕形成(尼达尼布或吡非尼酮),一些则没有。
结果如何?:总体而言,来自22个国家的147名IPF患者参与了该试验。结果显示,BI 1015550可防止IPF患者的肺功能下降。研究医生评定为严重医学问题的患者百分比在使用BI 1015550或安慰剂的患者中没有差异。然而,使用BI 1015550治疗的患者中腹泻的人数更多。在使用BI 1015550治疗的患者中,有13名参与者因医学问题停止了治疗,而使用安慰剂治疗的参与者中没有因医学问题停止治疗的情况。
这些结果意味着什么?:这些结果提供了证据,证明BI 1015550可防止IPF患者的肺功能恶化。未来将进行进一步的临床研究,以在更大规模的IPF患者群体以及其他随时间推移会恶化的肺瘢痕形成形式的患者中,对BI 1015550进行更长时间的测试。