Risk of hemoptysis in cystic fibrosis clinical trials: A retrospective cohort study

Abstract

Background Cystic fibrosis (CF) is characterized by airways infection and inflammation resulting in respiratory complications including hemoptysis. The objectives of this study were to characterize risk of hemoptysis attributable to the underlying disease and in the presence of standard of care therapy. Methods This retrospective cohort study estimated hemoptysis rates overall and by relevant risk factors utilizing adverse event data from longitudinal prospective CF clinical trials. Results Of the 1008 participants, 73% were ≤18 years old; of 929 with available spirometry, 27% had an FEV1 < 70% predicted. During the average 8.2 months of follow-up, 8% experienced ≥1 hemoptysis events (95% CI: 6%, 10%). Of the 125 events, 76% were mild in severity and only 9% were serious. Hemoptysis rates were greater among adults than children, those with FEV1 < 70% predicted, and participants infected with P. aeruginosa but not with S. aureus. Conclusions Hemoptysis is a common adverse event among CF clinical trial participants, and particularly in adults with more severe lung disease. These results can be used to predict event occurrence in future clinical trials.

Standardized Treatment of Pulmonary Exacerbations (STOP) study: Observations at the initiation of intravenous antibiotics for cystic fibrosis pulmonary exacerbations

“…Hemoptysis is often thought to be a manifestation of a PEx (21). Although we specifically excluded patients with massive hemoptysis, the prevalence of milder hemoptysis in our study was comparable to a recent report (22). …”

Pulmonary Complications of Cystic Fibrosis

“…4,5,17 Using placebo groups from prior randomized control trials, a rate of 8% of patients reported hemoptysis during an average period of 8 months. 18 Risk factors for the development of hemoptysis overlap with pneumothorax and includes worse lung function, older age, as well as the presence of Pseudomonas in sputum cultures.…”

Predictors of massive haemoptysis after a first episode of mild-to-moderate haemoptysis in patients with cystic fibrosis

“…THOMPSON et al [3] reported that haemoptysis is more common in CF individuals with advanced lung disease (FEV 1 <70% predicted). Although we did not detect an association between FEV 1 and MH in our cohort, consistent with a report by BARBEN et al [4], our study was limited by its retrospective design, the small number of patients, the high proportion of excluded patients, and the difficulty in precisely quantifying haemoptysis-associated blood loss.…”

“…In a study of 129 French CF patients who died between 2007 and 2010 without receiving lung transplantation, 8.5% of deaths were due to haemoptysis [ 2 ]. Factors associated with an increased risk of haemoptysis in CF patients include older age, advanced lung disease (forced expiratory volume in 1 s (FEV 1 ) <70% predicted), airway colonisation by Pseudomonas aeruginosa [ 3 ], CF-related diabetes, portal hypertension, and liver cirrhosis [ 4 ]. To our knowledge, there have been no studies of the risk factors for the development of massive haemoptysis (MH) in CF patients who have previously experienced an episode of mild-to-moderate haemoptysis.…”

“…T hompson et al . [ 3 ] reported that haemoptysis is more common in CF individuals with advanced lung disease (FEV 1 <70% predicted). Although we did not detect an association between FEV 1 and MH in our cohort, consistent with a report by B arben et al .…”