Department of Medicine, Royal College of Surgeons in Ireland, Education and Research Centre, Beaumont Hospital, Dublin 9, Ireland; Tissue Engineering Research Group, Royal College of Surgeons in Ireland, Dublin 2, Ireland.
Department of Medicine, Royal College of Surgeons in Ireland, Education and Research Centre, Beaumont Hospital, Dublin 9, Ireland; Tissue Engineering Research Group, Royal College of Surgeons in Ireland, Dublin 2, Ireland.
EBioMedicine. 2024 Jun;104:105135. doi: 10.1016/j.ebiom.2024.105135. Epub 2024 May 7.
Interstitial lung diseases (ILDs) in adults and children (chILD) are a heterogeneous group of lung disorders leading to inflammation, abnormal tissue repair and scarring of the lung parenchyma often resulting in respiratory failure and death. Inherited factors directly cause, or contribute significantly to the risk of developing ILD, so called familial pulmonary fibrosis (FPF), and monogenic forms may have a poor prognosis and respond poorly to current treatments. Specific, variant-targeted or precision treatments are lacking. Clinical trials of repurposed drugs, anti-fibrotic medications and specific treatments are emerging but for many patients no interventions exist. We convened an expert working group to develop an overarching framework to address the existing research gaps in basic, translational, and clinical research and identified areas for future development of preclinical models, candidate medications and innovative clinical trials. In this Position Paper, we summarise working group discussions, recommendations, and unresolved questions concerning precision treatments for FPF.
成人和儿童的间质性肺疾病（ILDs）是一组异质性肺部疾病，导致肺部炎症、异常组织修复和瘢痕形成，常导致呼吸衰竭和死亡。遗传因素直接导致ILD，即所谓的家族性肺纤维化（FPF）的发生，或显著增加ILD 的发病风险，某些单基因形式可能预后较差，对现有治疗反应不佳。缺乏特定的、针对变异的或精准的治疗方法。重新利用药物、抗纤维化药物和特定治疗方法的临床试验正在出现，但对许多患者来说，尚无干预措施。我们召集了一个专家工作组，制定了一个总体框架，以解决基础、转化和临床研究中存在的研究空白，并确定了未来开发临床前模型、候选药物和创新临床试验的领域。在这份立场文件中，我们总结了工作组关于 FPF 精准治疗的讨论、建议和未解决的问题。
