Overview
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.
Description
This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.
Eligibility
Inclusion Criteria:
- Participants 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 participants who:
- Discontinued selumetinib before enrollment; or
- Initiated treatment before enrollment and are currently on selumetinib; or
- Intend to initiate selumetinib within approximately 3 months after enrollment
- Able to give written informed consent. Participant or participant's parent/legal guardian must be willing and able to give written informed consent and the participant (if minor) must be willing to give written informed assent.
Exclusion Criteria:
- Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.