Patient Reported Outcomes
Identifying and recommending PRO measures appropriate for use as endpoints in NF clinical trials.
Leadership
About This Group
The goal of the REiNS patient-reported outcomes (PRO) working group is to identify and recommend a pool of PRO measures appropriate for use as endpoints in NF clinical trials. The PRO group recently completed their recommendations for measures in the core domains of general quality of life (QOL), disease-specific QOL, pain, and physical functioning using a systematic review and rating method we developed. To date, we have provided recommendations in the domains of pain and physical functioning as well as multidimensional QOL and disfigurement for NF clinical trials as reported in our publications below. Our group also assists other working groups in the REiNS committee to identify PRO measures in other areas such as hearing and vision, and we are available to consult with researchers who are considering using PRO measures as NF clinical trial endpoints. Several members also are developing and validating PRO measures specifically for use in NF clinical trials.
Current group chair: Heather Thompson, PhD, CCC-SLP Past group chair: Vanessa Merker, PhDPast group chair: Pam Wolters, PhD
We recently completed our review and recommendations for patient-reported outcomes to evaluate disfigurement for NF clinical trials (see Publications below). Currently, we are exploring patient-reported outcomes for assessing anxiety and depression. We also have a subgroup working on identifying outcome measures to assess hearing and communication.
Hearing and Communication PRO Subgroup
CurrentCurrent subgroup leader: Heather Thompson, PhD
Anxiety and Depression Domain
CurrentCurrent domain leader: Heather Thompson, PhD
Working Group Snapshot & Focus Updates
Currently exploring patient-reported outcomes for assessing anxiety and depression. A subgroup is also working on identifying outcome measures to assess hearing and communication. We recently completed recommendations for PRO measures to evaluate disfigurement in NF clinical trials.
Publications & References
Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in NF1 clinical trials
Clin Trials. 2024 Feb;21(1):6-17.
Patient-reported measures of tinnitus for individuals with NF2-related schwannomatosis
Clin Trials. 2024 Feb;21(1):18-28.
Current recommendations for patient-reported outcome measures assessing domains of quality of life in neurofibromatosis clinical trials
Neurology 2021 Aug 17;97(7 Suppl 1):S50-S63.
Patient report of hearing in neurofibromatosis type 2: Recommendations for clinical trials
Neurology 2021 Aug 17;97(7 Suppl 1):S64-S72.
Patient-reported outcomes of pain and physical functioning in neurofibromatosis clinical trials
Neurology 2016 Aug 16;87(7 Suppl 1):S4-S12.
Patient-reported outcomes in neurofibromatosis and schwannomatosis clinical trials
Neurology 2013 Nov 19;81(21 Suppl 1):S6-S14.
Documents & Resources
Meeting Schedule & Contact
The REiNS PRO Working Group meets once a month on a rotating schedule to accommodate different members' schedules. All monthly meetings have CART captioning. To be added to the email list, contact: