Over 75% of children who are in hospital because of a congenital heart defect receive at least one drug that has not been specifically tested and approved for their condition. Those who have undergone heart surgery are worst affected: they receive, on average, over three off-label medications. Children who have undergone heart transplantation receive on average more than six. The metabolic processes and the function of the organ systems change considerably from birth to infancy, and further modifications occur at kindergarten and school age. It is therefore not possible to infer pediatric pharmacokinetics and pharmacodynamics from adult data. Clinical studies of medication specifically for pediatric cardiac patients are urgently needed to fill this gap in our knowledge.

The European Medical Agency (EMA) has issued various regulations and initiated programmes to facilitate research into pediatric medication. In Freiburg we are conducting research on pharmacokinetics and pharmacodynamics and safety measurements of new cardiovascular substances in children.

We are currently involved as Principal Investigator in several industry-sponsored studies:

• Apixaban study for thromboprophylaxis (CV185-362-002)
• Entresto study for heart failure (PANORAMA)
• Selexipag study in childhood Pulmonary Arterial Hypertension
• Landiolol study in pediatric SVT (LANDI-PED)

We also have a collaboration with the Freiburg University Dermatology Center to investigate Losartan in epidermolysis bullosa (IIT).

Prof. Brigitte Stiller is member of the Conect4Children “Cardiology Expert Group”, which aims to improve European research into drug approval for children.

Coming soon!