US Selumetinib Registry

The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in participants with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who were aged 2 to 18 years at the time selumetinib was started in a real-world setting.

Inclusion Criteria:Patients diagnosed with NF1 and PN who were or will be aged 2 to 18 years at the time of selumetinib initiation on or after 10 April 2020 including patients who:Discontinued selumetinib before enrollment; orInitiated treatment before enrollment and are currently on selumetinib; orIntend to initiate selumetinib within approximately 3 months after enrollmentAble to give written informed consent. Patient or patient's parent/legal guardian must be willing and able to give written informed consent and the patient (if minor) must be willing to give written informed assent.Exclusion Criteria:Patients currently participating in any clinical trials at time of enrollment or initiation of selumetinib.