Methods: Subjects aged 1 mo to 17 yrs with ICC were enrolled into a prospective, open-label, non-comparative, multi-center, global study (NCT00761267) to receive ANID (3 mg/kg on day 1, 1.5 mg/kg daily thereafter). An interim analysis was completed in children 2-17 yrs. Subjects were to receive ANID for at least 10 d up to 35 d. A central venous catheter suspected as a site of infection was to be removed. A switch to oral fluconazole could be made after day 10. Treatment was required for at least 14 d after two negative cultures separated by 24h. Efficacy, based on a determination of global response (combination of clinical and microbiological response) was assessed at End of IV Treatment (EOIVT), End of Treatment (EOT), 2- and 6-wk follow up. Safety was assessed through 6-wk follow-up.
Results: A total of 48 subjects (18, 2-<5 yrs; 30, 5-17 yrs) received at least 1 dose of ANID (mean 11 d; range 1-35 d) and were assessed for safety. Forty-seven subjects had microbiologically confirmed ICC and were evaluated for efficacy. The most common baseline pathogens were C. albicans (38%) and C. parapsilosis (26%). Forty-four (93.6%) subjects had candidemia only. Global response success rates at EOIVT and EOT were 72.3% and 74.5%, respectively. All subjects reported at least 1 treatment emergent adverse event (AE) with diarrhoea (22.9%), vomiting (22.9%) and pyrexia (18.8%) being most frequent. Five subjects discontinued treatment due to AEs of which 4 [increased transaminases (2), vomiting, pruritus generalis] were considered related to ANID. All-cause mortality by the 2- and 6-wk follow-up visit was 12.5% and 14.6%, respectively. Of the 7 deaths during the study, 1 was considered related to ICC; 2 were related to disease progression (Ewing’s sarcoma, medulloblastoma); the remaining were related to other conditions (intracranial hemorrhage, sepsis/septic shock, respiratory failure).
Conclusion: Anidulafungin was effective with an acceptable tolerability and safety profile in children aged 2 to 17 yrs diagnosed with ICC.
H. Leister-Tebbe, Pfizer: Employee , Salary
U. Conte, Pfizer Inc: Employee , Salary
J. L. Yan, Pfizer: Employee , Salary
P. Liu, Pfizer: Employee , Salary
M. Tawadrous, Pfizer: Employee , Salary
J. Aram, Pfizer Inc: Employee , Salary
F. Queiroz-Telles, Pfizer: Grant Investigator , Research grant