Cystic fibrosis (CF) is the most common life-limiting genetic disease in Caucasian patients. Continued advances have led to improved survival, and adults with CF now outnumber children. As our understanding of the disease improves, new therapies have emerged that improve the basic defect, enabling patient-specific treatment and improved outcomes. However, recurrent exacerbations continue to lead to morbidity and mortality, and new pathogens have been identified that may lead to worse outcomes. In addition, new complications, such as CF-related diabetes and increased risk of gastrointestinal cancers, are creating new challenges in management. For patients with end-stage disease, lung transplantation has remained one of the few treatment options, but challenges in identifying the most appropriate patients remain.

中文翻译：

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囊性纤维化：新兴的理解和疗法。

囊性纤维化（CF）是白种人患者中最常见的限制生命的遗传疾病。持续的进步导致了存活率的提高，患有CF的成年人现在已经超过了儿童。随着我们对疾病的了解的提高，出现了新的疗法，这些疗法可以改善基本缺陷，实现针对患者的治疗并改善治疗效果。然而，反复发作继续导致发病率和死亡率，并且已经鉴定出可能导致更糟结果的新病原体。另外，新的并发症，例如与CF相关的糖尿病和胃肠道癌的风险增加，也给管理带来了新的挑战。对于患有终末期疾病的患者，肺移植仍是为数不多的治疗选择之一，但在确定最合适的患者方面仍然存在挑战。