Alexion's Ongoing Research on Plexiform Neurofibromas (NF1-PNs)

Author: Alexion

Published On: 10/29/2023

Alexion is conducting two ongoing studies focusing on adults with NF1 with plexiform neurofibromas (NF1-PNs). Each study seeks to understand a different element regarding plexiform neurofibromas and potential treatment options. The first focuses on pediatric patients taking selumetinib, and the second focuses on challenges in treatment and pain management. 

The first study, Qualitative Research to Understand the NF1 PN QoL Treatment Journey, is a self-reported, cross-sectional study of parents/caregivers and their
children.

The primary objectives of this study are:

• Understand patient and parent/caregiver experience and the impact of selumetinib on patient quality of life since initiating treatment with selumetinib
• Understand the patient and parent/caregiver reason for starting selumetinib
• Understand patient and parent/caregiver expectations of selumetinib therapy

The second study, Perspectives on Managing NF1 PN in Adults, is a study to Better understand patient, HCP and payer perspectives, experiences, approaches and
challenges to managing and treating adult patients with NF1 PN in anticipation of Koselugo’s adult indication. 

The patient research objectives are: 

• Patient / physician dynamics 
• Symptoms / disease progression
• Disease management similarities and differences in childhood vs adulthood
• Unmet needs in the treatment of NF1 PN
• Impact of insurance coverage / treatment costs

Learn more about Alexion and their work in neurofibromatosis type 1 by visiting their website.