Prospective validation and clinical evaluation of a new dosage of posaconazole for children and adolescents with cystic fibrosis and Aspergillus infection.

Cystic fibrosis (CF) is the most common hereditary disabling disease in the Northern European population, affecting 90,000 people worldwide and with approximately 45,000 registered in the European Cystic Fibrosis Society (ECFS) Patient Registry. Progressive lung damage caused by recurrent infection and persistent inflammation is the major determinant of survival, with a median age of death of 29 years. Approximately 60% of CF patients are infected with A. fumigatus, a ubiquitous environmental fungus, and its presence is associated with an accelerated decline in lung function. Half of patients with Aspergillus infection are under 18 years of age. No specific evidence is available to guide the clinical management of CF-related Aspergillus disease. A recent survey showed considerable variability in clinical practice among CF consultants.

The primary objective of the study is to define the dose of posaconazole for children and adolescents with CF and Aspergillus infection in terms of clearance of Aspergillus from the airways after three months.

Approximately a year into the study, the C4C CPS reviewed the cASPerCF study and decided to withdraw funding because there was no prospect of completing the study, as initially agreed Since the study was funded, the standard of care for the target population has changed significantly. This change led to a marked improvement in the quality of life of potential study participants and reduced the sites’ ability to recruit participants.

At the time of study completion, 29 patients were included in the large initial screening phase and 1 patient progressed to the intervention phase (the latter was assigned to the control arm).