Upper Airway Disease in Adults with Cystic Fibrosis in the Era of CFTR Modulators

Abstract

ObjectivesChronic rhinosinusitis (CRS) is prevalent in people with cystic fibrosis (PwCF) and is often refractory to treatments. Uncontrolled CRS might negatively impact the lower airways and the quality of life. The aim of this study is to evaluate the burden of cystic fibrosis (CF)‐related CRS in the era of CF transmembrane conductance regulator (CFTR) modulators.MethodsAdult PwCF were asked to fill in a questionnaire on sinonasal complaints, they underwent a nasal endoscopy, bacteriological sampling, and a CT scan. Afterwards, these outcome measures were compared between patients treated with and without modulators.ResultsIn the 122 included patients, CRS was present in 83%. CFTR modulators were prescribed in 48% of the patients, with a median of 10 months since the start of the treatment. Subjectively, the median SNOT‐22 score was 16/110. Objectively, a median Lund‐Kennedy score of 6/12 and modified Lund‐Mackay score of 10/24 were observed. No correlation could be found between SNOT‐22 score and other outcome measures including endoscopy and radiology. Altogether, 21% of the patients had controlled disease. When comparing patients treated with and without modulators, significantly lower CT scores (p = 0.0018) and less bacterial colonization (p = 0.0082) were observed in patients receiving modulators.ConclusionCF‐CRS is highly prevalent in our cohort and only the minority of PwCF has a well‐controlled disease. A multidisciplinary ENT‐pneumology clinic would be beneficial, as there is a high discrepancy between patient‐reported symptoms and the extent of the disease. CFTR modulators are promising, as lower CT scores and less bacterial colonization were observed in the modulator group.Level of EvidenceLevel 3 Laryngoscope, 2023

Impact of extended sinus surgery on allograft infection, allograft function and overall survival in cystic fibrosis lung transplant recipients

“…This study does not reflect on the evolving field of novel medical agents such as CFTR modulator therapies and its impact on future management of sinus disease in CF patients and improved allograft outcomes. As clinicians experience on a daily basis, the modulator therapies provide a promising conservative treatment option with great impact on symptoms and clinical signs (e.g., polyps) and are already changing current treatment protocols [ 24 ].…”

Dual and triple modulator therapy for chronic rhinosinusitis in cystic fibrosis patients

“…and was designed to accommodate paired data analysis with a two-sided test, an alpha (significance) level of 0.05, and a desired statistical power of 90%. The Modified Lund-Mackay score was used as outcome parameter to build the power calculation, as previous literature has shown that patientreported outcome measures poorly correlate with objective disease extent (22) .…”

“…Prior to this study, no (pilot)data were available for the TEZ/IVA subgroup. Consequently, the power analysis was conducted using the baseline Modified Lund-Mackay scores from our preexisting patient registry, which consisted of 122 patients with a mean score of 11.69 and a standard deviation of 6.31 (22) . To detect a MCID of >5, power analysis revealed an estimated sample size requirement of 19 participants for this specific subgroup.…”