Survey and online discussion groups to develop a patient-rated outcome measure on acceptability of treatment response in vitiligo

Annals of Occupational and Environmental Medicine

Selina K Tour²  Kim S Thomas²  Dawn-Marie Walker³  Paul Leighton³  Adrian SW Yong¹  Jonathan M Batchelor² 

[1] Norfolk and Norwich University Hospitals NHS Foundation Trust, Norwich, UK

[2] Centre of Evidence Based Dermatology, The University of Nottingham, Nottingham, UK

[3] Faculty of Medicine and Health Sciences, University of Nottingham, Nottingham, UK

关键词: Randomised controlled trial;    Patient-reported outcome;    Outcome measure;    Vitiligo;   

Others  :  814022
DOI  :  10.1186/1471-5945-14-10

 received in 2014-02-10, accepted in 2014-06-04,  发布年份 2014

【 摘 要 】

Background

Vitiligo is a chronic depigmenting skin disorder which affects around 0.5-1% of the world’s population. The outcome measures used most commonly in trials to judge treatment success focus on repigmentation. Patient-reported outcome measures of treatment success are rarely used, although recommendations have been made for their inclusion in vitiligo trials. This study aimed to evaluate the face validity of a new patient-reported outcome measure of treatment response, for use in future trials and clinical practice.

Method

An online survey to gather initial views on what constitutes treatment success for people with vitiligo or their parents/carers, followed by online discussion groups with patients to reach consensus on what constitutes treatment success for individuals with vitiligo, and how this can be assessed in the context of trials. Participants were recruited from an existing database of vitiligo patients and through posts on the social network sites Facebook and Twitter.

Results

A total of 202 survey responses were received, of which 37 were excluded and 165 analysed. Three main themes emerged as important in assessing treatment response: a) the match between vitiligo and normal skin (how well it blends in); b) how noticeable the vitiligo is and c) a reduction in the size of the white patches. The majority of respondents said they would consider 80% or more repigmentation to be a worthwhile treatment response after 9 months of treatment. Three online discussion groups involving 12 participants led to consensus that treatment success is best measured by asking patients how noticeable their vitiligo is after treatment. This was judged to be best answered using a 5-point Likert scale, on which a score of 4 or 5 represents treatment success.

Conclusions

This study represents the first step in developing a patient reported measure of treatment success in vitiligo trials. Further work is now needed to assess its construct validity and responsiveness to change.

【 授权许可】

   
2014 Tour et al.; licensee BioMed Central Ltd.

【 图 表 】

Figure 1.

Figure 2.

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