Patient Reported Outcomes 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, PhD

Past group chair:  Pam Wolters, PhD

Click Here For A Working Group Snapshot

Current Work

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

Current subgroup leader:  Heather Thompson, PhD

Anxiety and Depression Domain

       Current domain leader:  Heather Thompson, PhD


Previous Domains and 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]

3. Wolters PL, Vranceanu AM, Thompson HL, Martin S, Merker VL, Baldwin A, Barnett C, Koetsier KS, Hingtgen CM, Funes CJ, Tonsgard JH, Schorry EK, Allen T, Smith T, Franklin B, Reeve S; REiNS International Collaboration. 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. doi: 10.1212/WNL.0000000000012421. Epub 2021 Jul 6. PMID: 34230198; PMCID: PMC8594008. [PDF]

4. Thompson HL, Blanton A, Franklin B, Merker VL, Franck KH, Welling DB; REiNS International Collaboration. Patient Report of Hearing in Neurofibromatosis Type 2: Recommendations for Clinical Trials. Neurology. 2021 Aug 17;97(7 Suppl 1):S64-S72. doi: 10.1212/WNL.0000000000012424. Epub 2021 Jul 6. PMID: 34230203; PMCID: PMC8594003. [PDF]

5. Merker VL, Thompson HL, Wolters PL, Buono FD, Hingtgen CM, Rosser T, Barton B, Barnett C, Smith T, Haberkamp D, McManus ML, Baldwin A, Moss IP, Röhl C, Martin S. Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials. Clin Trials. 2024 Feb;21(1):6-17. doi: 10.1177/17407745231205577. Epub 2023 Dec 23. PMID: 38140900; PMCID: PMC10922038. [PDF]

6. Thompson HL, Grabowski J, Franklin B, Koetsier KS, Welling DB. Patient-reported measures of tinnitus for individuals with neurofibromatosis type 2-related schwannomatosis: Recommendations for clinical trials. Clin Trials. 2024 Feb;21(1):18-28. doi: 10.1177/17407745231217279. Epub 2024 Feb 6. PMID: 38321701; PMCID: PMC10865763. [PDF]


Presentations

REiNS PRO group poster Revised 061416.pptx

Psychosocial Survey of Adults with Neurofibromatosis

Psychosocial Survey 051320.pptx

Resources

Role-play Videos Demonstrating Administration of PROs

Clip 1_Introducing PROs.mp4

Clip 2_Administering Parent PROs.mp4

Clip 3_Administering Patient PROs.mp4