Post-authorization Safety Study (PASS) of Pediatric Patients Initiating Selumetinib Treatment for Symptomatic Inoperable Plexiform Neurofibromas (PNs) Associated with Neurofibromatosis Type 1 (NF1).

Autor: Rosenbaum, T., Passos, J., Martin, L., Moiseenko, A., Obradovic, V., Amar, R., Dobrinsky, C., Brecht, I.
Předmět:
Zdroj: Neuropediatrics; 2023 Supplement 1, Vol. 54, pS1-S32, 32p
Abstrakt: This article discusses a post-authorization safety study (PASS) being conducted in the European Union and the United Kingdom to evaluate the safety profile of selumetinib, a MEK1/2 inhibitor, in pediatric patients with symptomatic inoperable plexiform neurofibromas (PNs) associated with neurofibromatosis type 1 (NF1). The study aims to characterize the long-term safety of selumetinib, including potential developmental effects, in children with NF1-associated PNs. The study will enroll a total of 125 patients and will collect data on safety outcomes such as cardiac function, bone development, muscle function, liver function, eye health, and pubertal development. The results of this study will provide valuable information on the safety of selumetinib in this patient population. [Extracted from the article]
Databáze: Complementary Index