Off-label drug use evaluation in paediatrics--applied to ciprofloxacin when used as treatment of cholangitis.

Purpose: To evaluate drug and indication specific off-label use in paediatrics, applied to ciprofloxacin (CPFX) in cholangitis.

Methods: We collected four different sets of data for an off-label drug use evaluation. (1) Literature review from medical journals, (2) the use and safety profile from the whole Swedish paediatric population by extracting data from national registers, (3) locally performed retrospective drug chart reviews, and (4) interviews regarding paediatric patients with CPFX treated cholangitis.

Results: The literature reviews show a lack of information for paediatric use of CPFX in cholangitis. The prescribing of CPFX to Swedish children has grown over the last decade and generated a small number of reports for adverse drug reactions. In our local biliary atresia population 32 patients had suffered from at least one episode of cholangitis and 13 patients had been prescribed CPFX. The dosing strategy had an empirical prescribing approach, since monitoring of bacterial resistance and efficacy is difficult in the biliary ducts. No clear relationship was seen between dosing and age/weight. Reports of suspected side effects could not be found in the retrospective chart reviews. The interviews show that the existing dosage forms are well accepted.

Conclusions: This drug use evaluation creates awareness of the off-label situation. The international and national data are sparse for the paediatric use of CPFX in cholangitis. Locally we have highlighted a heterogeneous dosing strategy of CPFX, drug/drug interactions, and the need to monitor and report the risk of short- and long-term adverse drug reactions.