OBJECTIVES

: The approval of propranolol in infantile hemangioma (IH) by the EMA was conditional upon the submission of a Risk Management Plan (RMP) designed to monitor and minimise safety concerns. This Drug Utilization Study aimed to evaluate the off-label use of propranolol and the effectiveness of the risk minimisation measures (RMM) to mitigate the important risks characterized in the RMP.

METHODS

: A retrospective multicentre hospital cohort study of patients treated with propranolol was conducted in France and Germany. Off-label criteria were assessed by an independent adjudication committee. Data from the French Système National d’Information Inter-Régimes de l’Assurance Maladie (SNIIRAM) and IQVIA® longitudinal Pharmacy prescription database (LRx) in Germany were also used.

RESULTS

: A total of 273 patients from 37 French centres and 247 patients from 18 German centres were included. Patients with IH requiring systemic treatment were ≥85% in both countries, in line with the Summary of Product Characteristics (SmPC). The reason for treatment was adjudicated as off-label in 6% of cases in both countries. Age at treatment initiation (corrected for prematurity) was in line with the SmPC (<5 weeks or >5 months) for ≥70% of patients. The mean treatment duration (including temporary discontinuation) was more than the recommended 6 months in both France (8.7 months) and Germany (8.4 months). Overall, there was a low incidence of adverse events (<4%).

CONCLUSIONS

: Overall conditions of usage of propranolol in IH comply to the SmPC and identified off-label conditions of use that could jeopardize the safety of infants were considered as very limited. Propranolol usage in infants with IH, when used along with adequate RMM, has demonstrated no new safety concern and no new signal that could impact the benefit/risk of the product.