Patient Reported Outcomes (PRO) Initiative

The NTAP Patient Reported Outcome (PRO) Initiative aims to develop reliable measures that assess the impact of plexiform neurofibromas (pNFs) on patients with neurofibromatosis type 1 (NF1).

This program is leading the creation and validation of PRO measures to be used as treatment outcomes that are the most relevant to patients, and which can be used as primary or secondary endpoints in clinical trials and product labeling applications.

There are currently no PRO measures that have been validated specifically for use in patients with pNFs.

Visit the index of funded NF1 research initiatives to learn more about the following NTAP-funded projects to establish patient reported outcome measures for patients with NF1.

  • Development of Patient Reported Outcomes System for Patients with NF1-Associated Plexiform Neurofibromas using Mixed Method Approach.
  • Development of a Child Neurofibromatosis Type 1 Health Related Quality of Life Measure
  • The development and Validation of PRO Measures to Assess Pain in Individuals with NF1 and pNF
  • Development of a needs-based quality of life Patient Reported Outcome measure specific patients with NF1-associated pNFs