Impact of lumacaftor/ivacaftor on the bacterial and fungal respiratory pathogens in cystic fibrosis: a prospective multicenter cohort study in Sweden.

IF 3.3 3区医学 Q2 RESPIRATORY SYSTEM Therapeutic Advances in Respiratory Disease Pub Date : 2024-01-01 DOI:10.1177/17534666241254090

Mahasin Al Shakirchi, Kimmo Sorjonen, Lena Hjelte, Lena Klingspor, Peter Bergman, Petrea Ericson, Marcus Svedberg, Ulrika Lindberg, Christine Hansen, Isabelle de Monestrol

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Abstract

Background: A significant decline in pulmonary exacerbation rates has been reported in CF patients homozygous for F508del treated with lumacaftor/ivacaftor. However, it is still unclear whether this reduction reflects a diminished microbiological burden.

Objectives: The aim of this study was to determine the impact of lumacaftor/ivacaftor on the bacterial and fungal burden.

Design: The study is a prospective multicenter cohort study including 132 CF patients homozygous for F508del treated with lumacaftor/ivacaftor.

Methods: Clinical parameters as well as bacterial and fungal outcomes 1 year after initiation of lumacaftor/ivacaftor were compared to data from 2 years prior to initiation of the treatment. Changes in the slope of the outcomes before and after the onset of treatment were assessed.

Results: Lung function measured as ppFEV1 (p < 0.001), body mass index (BMI) in adults (p < 0.001), and BMI z-score in children (p = 0.007) were improved after initiation of lumacaftor/ivacaftor. In addition, the slope of the prevalence of Streptococcus pneumoniae (p = 0.007) and Stenotrophomonas maltophilia (p < 0.001) shifted from positive to negative, that is, became less prevalent, 1 year after treatment, while the slope for Candida albicans (p = 0.009), Penicillium spp (p = 0.026), and Scedosporium apiospermum (p < 0.001) shifted from negative to positive.

Conclusion: The current study showed a significant improvement in clinical parameters and a reduction of some of CF respiratory microorganisms 1 year after starting with lumacaftor/ivacaftor. However, no significant changes were observed for Pseudomonas aeruginosa, Staphylococcus aureus, or Aspergillus fumigatus, key pathogens in the CF context.

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Lumacaftor/ivacaftor 对囊性纤维化患者呼吸道细菌和真菌病原体的影响：瑞典一项前瞻性多中心队列研究。

背景：有报道称，接受鲁马卡夫托/伊伐卡夫托治疗的F508del基因CF患者的肺部恶化率明显下降。然而，目前仍不清楚这种下降是否反映了微生物负担的减轻：本研究旨在确定 lumacaftor/ivacaftor 对细菌和真菌负担的影响：该研究是一项前瞻性多中心队列研究，纳入了132名接受lumacaftor/ivacaftor治疗的F508del同源CF患者：将开始使用鲁马卡夫托/伊伐卡夫托一年后的临床参数以及细菌和真菌结果与开始治疗前两年的数据进行比较。评估了治疗开始前后结果斜率的变化：结果：开始使用lumacaftor/ivacaftor治疗后，以儿童ppFEV1（p p z-score）衡量的肺功能有所改善（p = 0.007）。此外，肺炎链球菌（p = 0.007）和嗜麦芽血单胞菌（p 白色念珠菌（p = 0.009）、青霉属（p = 0.026）和杏孢子菌（p 结论：本研究显示，儿童肺功能在使用鲁马卡夫托/伊伐卡夫托后得到显著改善：目前的研究表明，在开始使用鲁马卡夫托/伊伐卡夫托 1 年后，临床参数有了明显改善，部分 CF 呼吸道微生物也有所减少。然而，对于铜绿假单胞菌、金黄色葡萄球菌或曲霉菌（CF中的主要病原体），未观察到明显变化。

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Therapeutic Advances in Respiratory Disease RESPIRATORY SYSTEM-

CiteScore

6.90

自引率

0.00%

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审稿时长

15 weeks

期刊介绍： Therapeutic Advances in Respiratory Disease delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of respiratory disease.