Chest computed tomography: a validated surrogate endpoint of cystic fibrosis lung disease?
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Chest computed tomography : a validated surrogate endpoint of cystic fibrosis lung disease? / Loeve, Martine; Krestin, Gabriel P.; Rosenfeld, Margaret; de Bruijne, Marleen; Stick, Stephen M.; Tiddens, Harm A.
In: European Respiratory Journal, Vol. 42, No. 3, 2013, p. 844-857.Research output: Contribution to journal › Journal article › Research › peer-review
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TY - JOUR
T1 - Chest computed tomography
T2 - a validated surrogate endpoint of cystic fibrosis lung disease?
AU - Loeve, Martine
AU - Krestin, Gabriel P.
AU - Rosenfeld, Margaret
AU - de Bruijne, Marleen
AU - Stick, Stephen M.
AU - Tiddens, Harm A.
PY - 2013
Y1 - 2013
N2 - Clinical trials for the treatment of cystic fibrosis (CF) lung disease are important to test and optimize 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 one second (FEV1) and respiratory tract exacerbations (RTE). Unfortunately, these endpoints are relatively insensitive to monitor 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 primary endpoint to study CF lung disease. However, before chest CT can be used in clinical trials, it has to be recognized as a validated surrogate endpoint by regulatory agencies. The aim of this review is to summarize what is currently known about the use of chest CT as surrogate endpoint in clinical trials in CF.
AB - Clinical trials for the treatment of cystic fibrosis (CF) lung disease are important to test and optimize 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 one second (FEV1) and respiratory tract exacerbations (RTE). Unfortunately, these endpoints are relatively insensitive to monitor 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 primary endpoint to study CF lung disease. However, before chest CT can be used in clinical trials, it has to be recognized as a validated surrogate endpoint by regulatory agencies. The aim of this review is to summarize what is currently known about the use of chest CT as surrogate endpoint in clinical trials in CF.
U2 - 10.1183/09031936.00051512
DO - 10.1183/09031936.00051512
M3 - Journal article
C2 - 23258780
VL - 42
SP - 844
EP - 857
JO - The European Respiratory Journal
JF - The European Respiratory Journal
SN - 0903-1936
IS - 3
ER -
ID: 44489856