Core Outcome Measures in Effectiveness Trials

Patient-reported outcomes in neurofibromatosis and schwannomatosis clinical trials

General Information

Abstract:
OBJECTIVES: Neurofibromatosis (NF) is a genetic disease with multiple clinical manifestations that can significantly impact quality of life (QOL). Clinical trials should include patient-reported outcomes (PROs) as endpoints to assess treatment effects on various aspects of QOL, but there is no consensus on the selection and use of such measures in NF. This article describes the PRO Working Group of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) Collaboration, its main goals, methods for identifying appropriate PRO measures for NF clinical trials, and recommendations for assessing pain intensity.
METHODS: The REiNS PRO group selected core endpoint domains important to assess in NF. The members developed criteria to rate PRO measures, including patient characteristics, psychometric properties, and feasibility, and utilized a systematic process to evaluate PROs for NF clinical trials. Within the subdomain of pain intensity, the group reviewed the Numerical Rating Scale-11 (NRS-11), the Visual Analogue Scale, and the Faces Pain Scale-Revised using this process.
RESULTS: Based on the review criteria, each of these pain intensity scales is brief, reliable, valid, and widely used. However, the NRS-11 was given the highest rating for use in NF clinical trials due to recommendations from pain experts and other consensus groups, its extensive use in research, strong psychometric data including sensitivity to change, and excellent feasibility in ages > 8 years.
CONCLUSIONS: The systematic review criteria and process are effective for identifying appropriate PRO measures and provide information utilized by the REiNS Collaboration to achieve consensus regarding PROs in NF clinical trials.

Aim:
This article describes the PRO Working Group of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) Collaboration, its main goals, methods for identifying appropriate PRO measures for NF clinical trials, and recommendations for assessing pain intensity.

Authors:
Wolters, Pamela L. Martin, Staci Merker, Vanessa L. Gardner, Kathy L. Hingtgen, Cynthia M. Tonsgard, James H. Schorry, Elizabeth K. Baldwin, Andrea R. EiNS International Collaboration

Publication

Journal:
Neurology
Volume:
81
Issue:
21 Suppl 1
Pages:
S6 - 14
Year:
2013
DOI:
Further Study Information

Date:
Not stated
Funding source(s):
This research is supported by the Intramural Research Program of the NIH, National Cancer Institute.

Health Area

Disease Category
Genetic disorders

Disease Name
Neurofibromatoses

Target Population

Age Range
0 - 100

Sex
Either


Nature / type of Intervention
Any

Method(s)

Teleconferences

The PRO Working Group was formed as part of the REiNS subcommittees charged with identifying outcome measures to use as NF trial endpoints. The initial phone and Web conference of the PRO group was in August 2010. The members agreed that one of the main goals was to identify core endpoint domains relevant to NF clinical trials. The group researched, discussed, and generated 4 core endpoint domains important to assess in the NF population as part of a clinical trial.


Stakeholders Involved

Academic research representatives
Clinical experts
Patient/ support group representatives

Study Type

COS for clinical trials or clinical research

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