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 patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020 in a real-world setting.
This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.
Study Type
OBSERVATIONAL
Enrollment
37
Phoenix Children's Hospital
Phoenix, Arizona, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician
Time frame: Up to Month 60
Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician
Time frame: Up to Month 60
Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations
Skeletal manifestations may include changes in the angle of spine curvature.
Time frame: Up to Month 60
Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)
Time frame: Up to Month 60
Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60
Time frame: Baseline, Month 60
Change From Baseline in the Pain Interference Index (PII) Score at Month 60
Time frame: Baseline, Month 60
Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60
Time frame: Baseline, Month 60
Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60
Time frame: Baseline, Month 60
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