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 is assessing measures in the core domains of general QOL, disease-specific QOL, pain, and physical functioning using a systematic review and rating method we developed.  To date, we have made recommendations in the domains of pain and physical functioning 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.  

Current group leader:  Pam Wolters, PhD

Current Work

Currently, we are working on the final two core domains of general and disease-specific quality of life.  We also have two smaller subgroups working on identifying outcome measures to assess hearing and communication as well as young child pain.

Quality of Life Domain

Current domain co-leaders:  Ana-Maria Vranceanu, PhD and Pam Wolters, PhD

Hearing and Communication PRO Subgroup

Current subgroup leader:  Heather Thompson, PhD

Young Child Pain PRO Subgroup

Current subgroup co-leaders:  Taryn Allen, PhD and Andrea Baldwin, CRNP

Publications

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

Resources