¹Drug Safety Center, Leipzig University and Leipzig University Hospital, Leipzig, Germany
²Clinical Pharmacy, Institute of Pharmacy, Medical Faculty, Leipzig University, Leipzig, Germany
³Department of Neuropediatrics, Hospital for Children and Adolescents, University Medicine Greifswald, Greifswald, Germany
⁴Center for Pediatric Research, University Hospital for Children and Adolescents, Leipzig University Hospital, Leipzig, Germany

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Background: Pediatric patients with inborn errors of metabolism (IEM) are chronically ill and particularly susceptible to developing drug-related problems (DRPs) due to long-term medication use. The risk is further increased when additional medication is used, for example to treat acute conditions. The aim of this study was to systematically assess the occurrence of DRPs in a cohort of pediatric patients with IEM [1].

Materials and Methods: The medications of 114 pediatric patients with IEM were analyzed, including medications for both chronic and acute conditions. Structured telephone interviews with their parents were used to record all medications available for the children at home, resulting in a total of 884 medications. Identified DRPs were classified according to type, clinical relevance, and preventability using the Pharmaceutical Care Network Europe (PCNE) classification system [1].

Results: Overall, 83 medications (9%) were associated with DRPs, affecting 50 patients (44%). Clinically relevant DRPs without safe therapeutic alternatives were found in 26 medications (3%) in 15 patients (13%), including 12 medications (1%) related to IEM. DRPs classified as clinically relevant and preventable due to the availability of safe therapeutic alternatives were identified in 20 medications (2%) in 24 patients (21%), with one medication (0.1%) related to IEM. DRPs classified as not clinically relevant in the context of these patients were found in 37 medications (4%) in 26 patients (23%), although they could be relevant in other populations. Of these, 3 medications (0.3%) were associated with IEM.

Conclusion: Nearly half of the pediatric patients were affected by DRPs. Notably, most preventable DRPs were not related to IEM or its specific therapy but rather occurred because the patients were children. These findings highlight the need for careful medication selection to improve pediatric medication safety.

References

[1] Harings T, Neininger MP, Eisenhofer S, Thiele AG, Kiess W, Bertsche A, Beblo S, Bertsche T. Parents’ Perceptions Regarding Their Children's Medications and Expert-Assessed Drug-Related Problems in Pediatric Patients with Inborn Errors of Metabolism. Children (Basel). 2023;10(12):1873.