Chest computed tomography: a validated surrogate endpoint of cystic fibrosis lung disease?

Abstract

Clinical trials for the treatment of cystic fibrosis (CF) lung disease are important to test and optimise new therapeutic interventions. To evaluate the effect of these interventions, sensitive and accurate outcome measures are needed. The most commonly used endpoints are spirometric variables such as the forced expiratory volume in 1 s and respiratory tract exacerbations. Unfortunately, these endpoints are relatively insensitive for monitoring progression of CF lung disease, and thus require a large number of patients when used in clinical studies. In addition, these endpoints are not suitable to study CF lung disease in young children. Chest computed tomography (CT) holds great promise for use as a sensitive surrogate endpoint in CF. A large body of evidence has been produced to validate the use of chest CT as a primary endpoint to study CF lung disease. However, before chest CT can be used in clinical trials, it has to be recognised as a validated surrogate endpoint by regulatory agencies. The aim of this review is to summarise what is currently known about the use of chest CT as surrogate endpoint in clinical trials in CF. @ERSpublications Evaluation of the use of chest CT as a sensitive surrogate endpoint in clinical trials in CF

Is Lung Clearance Index (LCI) Affected by the Severity of Lung Disease in CF?

“…However, spirometry is difficult to be performed by preschoolers and infants, not only because it requires special equipment [17,18], but also because FEV 1 is now of less value in monitoring children with CF, due to its lack to assess early or mild CF lung disease [19][20][21][22]. Structural damages may be present in High Resolution Computed Tomography (HRCT) in one third of CF children with normal spirometry [20][21][22][23][24][25].…”

Methicillin-resistant Staphylococcus aureus in cystic fibrosis patients: do we need to care? A cohort study

“…24 Several studies have shown that despite stable spirometric parameters, HRCT scores show annual progression in total CT score. 7 Despite the well-established importance of HRCT scores for CF, few studies have made reference to these scores in different microbiological groups, 11 with no published data regarding MRSA colonized patients. In this context, our study highlights the importance of HRCT scores in CF patients with chronic colonization with MRSA, thus making a significant contribution towards aiding clinicians who deal with this group of patients.…”

“…7 Imaging examinations are able to detect early disease progression, thereby contributing to treatment effectiveness and quality of life among CF patients. Furthermore, HRCT can be used both for patients who are unable to undergo spirometry and for analysis on the two tests, since HRCT shows early changes in patients with normal spirometry.…”

“…Since the first proposal for creation of a computed tomography scoring system to quantify structural abnormalities in CF patients, 9 other scoring systems have been described and been shown to be reproducible and comparable. 7 The modified Bhalla score 10 was shown to be reproducible and reliable, regardless of the severity of the lesions, which suggests that it is applicable in clinical practice for CF cases. 11…”

“…Furthermore, it has high sensitivity and specificity for diagnosing lung injury and also allows better qualitative description of respiratory impairment. 7,8 HRCT results are presented as graphical images, which may be converted to score values that are useful for evaluating disease severity. Since the first proposal for creation of a computed tomography scoring system to quantify structural abnormalities in CF patients, 9 other scoring systems have been described and been shown to be reproducible and comparable.…”