Creating and Testing New Questionnaires to Measure Pain and Daily Life Impact in People with Neurofibromatosis 1 NF1 and Plexiform Neurofibromas pNFs

What this Trial Is About

People with neurofibromatosis 1 NF1 who have plexiform neurofibromas pNFs often experience pain that can impact their daily lives. This research aims to improve questionnaires that measure pain, daily functioning, and physical abilities specifically for people with NF1 and pNFs. It is an observational study involving individuals aged 5 years and older, including parents of children with NF1 and pNFs. The study is divided into two phases. Phase 1 involved group discussions or one-on-one interviews with participants to review and improve existing pain assessment questionnaires and how pNFs affect life. Phase 2, now complete, involved participants completing the revised questionnaires either on paper or electronically, with some completing forms twice to check consistency and some who started new pain treatments completing forms before and after treatment changes. Participants will provide information through group discussions, interviews, and by completing questionnaires that assess pain intensity and interference as well as physical function. The study includes measures of reliability, feasibility, and collection of normative data. Some participants will complete forms twice to evaluate consistency, and small groups will be monitored around treatment changes. The study aims to provide better tools to assess patient-reported outcomes in NF1 with pNFs over several months.

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)