Developing a PROM specific to patients with NF1-associated plexiforms
Research type
Research Study
Full title
Developing a needs-based quality of life patient reported outcome measure specific to patients with NF1-associated plexiforms
IRAS ID
145609
Contact name
Stephen Patrick McKenna
Contact email
Sponsor organisation
Galen Research Ltd.
Research summary
Neurofibromatosis Type 1 (NF1) has a major impact on the lives of patients. There is currently no cure, so management involves regular monitoring and treatment for problems as and when they occur. In order to determine whether alternative treatments improve the patient’s quality of life (QoL), it is necessary to produce scientific evidence. The main purpose of the study is to give patients with plexiform neurofibromatosis (pNF) a voice in the evaluation of their treatment. This will be done by combining qualitative and quantitative methodologies to develop a disease-specific Patient-Reported Outcome Measure (PROM). The content of this measure will be derived entirely from patient interviews, thus ensuring that the issues covered are of importance and relevance to patients with pNF. In subsequent stages of the study new groups of patients will evaluate the questionnaires in terms of its ease of completion, relevance to patients, completeness, accuracy and validity.
The new measure will then be used in studies to evaluate the effectiveness of treatments and to find out how best to provide treatment services. It is also intended that the questionnaire will be used in routine assessments to see whether patients are responding to their treatments.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
14/NW/0279
Date of REC Opinion
16 May 2014
REC opinion
Favourable Opinion