Persistence and evolution of Pseudomonas aeruginosa following initiation of highly effective modulator therapy in cystic fibrosis. With the widespread availability of elexacaftor/tezacaftor/ivacaftor (ETI) and dramatic improvement in respiratory symptoms, attention is turning to the role of therapies designed to maintain lung health. Some early studies have reported a reduced bacterial burden in the lungs after ETI, though results are easily confounded by the reduction in sputum samples and the impact on chronic P. aeruginosa infection remains unknown ..read more

Cystic fibrosis (CF) is a multisystem disease caused by variants in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and resulting in deficient or dysfunctional CFTR protein [1]. F508del is the most prevalent disease-causing variant in people with CF (pwCF), occurring in 80–85 % of cases [2,3]. The prognosis has largely been determined by the pulmonary complications of the disease ..read more

For more than two decades, culture-independent microbiome analysis has been employed in efforts to improve clinical outcomes for those with CF and to gain a better fundamental understanding of polymicrobial infections. The use of these techniques began during the 1990s [1,2], in response, at least in part, to growing concerns over the emergence of new infectious syndromes. Pathogens such as Burkholderia cepacia [3] and rapidly growing nontuberculous mycobacteria [4] were increasingly associated with sudden and profound deterioration in lung function in persons with CF (PwCF) and the potential ..read more

Chronic rhinosinusitis (CRS) has been for long addressed as a hallmark of cystic fibrosis (CF). It has been reported to affect the majority of people with CF (pwCF), with almost 100 % of patients revealing pathologic findings in CT scans, which are currently replaced by MRI scans to reduce exposure to ionising radiation and to enhance differentiation of soft tissue structures [1,2]. The burden of sinonasal symptoms markedly impairs a patient´s well-being, and indeed, to a greater extent than “some rhinitis” is generally expected to bother general health ..read more

The efficacy of azithromycin as an anti-inflammatory agent in infants with CF was investigated in the COMBAT-CF study [1]. Children with CF diagnosed by newborn screening were 1:1 randomly assigned to maintenance treatment with azithromycin or placebo from diagnosis until age of three, stratified by site. The primary outcome measure was structural airway disease as assessed on chest computed tomography scans (CTs) by the manual Perth-Rotterdam Annotated Grid Morphometric for CF (PRAGMA-CF) scoring method [2 ..read more

Sexual and reproductive health (SRH) in cystic fibrosis (CF) is an expansive field that has recently gained more attention with the improved fertility and longevity of our CF population in the era of CFTR modulator therapy. People with cystic fibrosis (pwCF) – both men and women - have identified this field as a priority area of interest for clinicians to focus research and educational efforts [1]. SRH in CF is a broad topic encompassing not just fertility and sexuality, but also health outcomes associated with SRH events such as pregnancy/parenthood, and health services delivery of SRH needs ..read more

Cystic fibrosis (CF) is an autosomal recessive genetic disease that affects about 30,000 individuals in the United States [1,2]. Advances in the treatment of CF, including cystic fibrosis transmembrane conductance regulator (CFTR) modulators, have increased the life expectancy, lung function, and nutritional status in people with CF (pwCF). With increased longevity, extrapulmonary manifestations, such as CF-related liver disease (CFLD), have become increasingly relevant in the management and care of pwCF [3 ..read more

Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators are small-molecule therapeutics that target the underlying cause of CF. Ivacaftor, a CFTR potentiator, improves CFTR function by increasing channel gating activity at the cell surface [1,2]. In clinical trials and real-world studies, ivacaftor has been shown to be safe and efficacious in children as young as 4 months of age with CFTR gating pathogenic variants, with early and sustained improvements in lung function, CFTR function, and respiratory symptoms [3–6 ..read more

Racial and ethnic disparities in health, shaped by implicit and overt racism, are pervasive and substantial. Societal structures such as health care, economic and legal systems, education and housing profoundly impact health [1–3]. Experiences of racism, both direct and vicarious, also contribute to poorer health outcomes among minoritized individuals [4]. With the growing emphasis on improving health equity over the past two decades has come increased attention to the racist origins and racial biases in a range of clinical tests [5], including spirometry [6], glomerular filtration rate [7] an ..read more

Nasal nitric oxide (nNO) is known to be reduced in people with primary ciliary dyskinesia (PwPCD) and the measurement is used in PCD's diagnostic workup [1]. It was found, that nNO is not only reduced in PCD, but also in people with cystic fibrosis (PwCF), and in chronic rhinosinusitis (CRS) even without underlying CF and nasal polyps [2,3]. It is increased in people with allergic rhinitis and asthma compared to healthy controls (HC) [2]. People with reduced nNO levels and a history of lung disease should therefore not only be evaluated for PCD, but also for CF and other diseases associated wi ..read more