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, PhD
Past 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.
Current subgroup leader: Heather Thompson, PhD
Current domain leader: Heather Thompson, PhD
1. Wolters PL, Martin S, Merker VL, et al. Patient-reported outcomes in neurofibromatosis and schwannomatosis clinical trials. Neurology 2013 Nov 19;81(21 Suppl 1):S6-14. [PDF]
2. Wolters PL, Martin S, Merker VL, et al. Patient-reported outcomes of pain and physical functioning in neurofibromatosis clinical trials. Neurology 2016 Aug 16;87(7 Suppl 1):S4-S12. [PDF]
Presentations
REiNS PRO group poster Revised 061416.pptx
Psychosocial Survey 051320.pptx
Role-play Videos Demonstrating Administration of PROs
Clip 2_Administering Parent PROs.mp4
Clip 3_Administering Patient PROs.mp4