Children's National Hospital, Washington, DC, USA.
Children's National Research Institute, Washington, DC, USA.
J Investig Med High Impact Case Rep. 2023 Jan-Dec;11:23247096231188243. doi: 10.1177/23247096231188243.
Cystic fibrosis (CF) is an important monogenic disease that affects more than 70 000 people worldwide. Defects of the CF transmembrane conductance regulator gene lead to dehydrated viscous secretions that result in chronic bacterial colonization. This leads to frequent recurrent lung infections called pulmonary exacerbations, lung inflammation, and resulting structural lung damage called bronchiectasis. in particular is a common pathogen in persons with CF associated with increased pulmonary exacerbations, long-term lung function decline, and reduced survival. In addition, commonly develops antibiotic resistance and forms biofilms, making it difficult to treat. Here, we report the details of two patients with CF with pan-drug-resistant who were treated with a novel therapeutic strategy, bacteriophages. These cases highlight the need for further research and development of this treatment modality, including pediatric clinical trials.
囊性纤维化(CF)是一种重要的单基因疾病,影响全球超过 70000 人。CF 跨膜电导调节基因的缺陷导致脱水粘性分泌物,导致慢性细菌定植。这导致频繁发生的复发性肺部感染,称为肺部恶化、肺部炎症和由此产生的称为支气管扩张的结构性肺部损伤。特别是 CF 相关的常见病原体与增加的肺部恶化、长期肺功能下降和降低的存活率有关。此外,通常会产生抗生素耐药性并形成生物膜,使其难以治疗。在这里,我们报告了两名患有泛耐药 的 CF 患者的详细信息,他们接受了一种新型治疗策略,噬菌体的治疗。这些病例突出表明需要进一步研究和开发这种治疗方式,包括儿科临床试验。