BACKGROUND: Relevant and reliable outcomes play a crucial role in the correct interpretation and comparison of the results of clinical trials. There is a lack of consensus around methods of assessment and outcome measures for vitiligo, which makes it difficult to compare results of randomised controlled trials and perform meta-analysis.
OBJECTIVE: The objective of this study was to describe the heterogeneity in outcome measures used in published randomised controlled trials of vitiligo treatments, and to report the most desirable outcomes from patients’ and clinicians’ perspectives.
METHODS: We conducted a systematic review of outcome measures used in randomised controlled trials (RCTs) as well as a survey of the most desirable outcomes identified by patients and clinicians as part of a vitiligo priority setting partnership.
RESULTS: Outcomes from 54 eligible trials were analysed and compared to outcomes suggested by patients and clinicians.
In the systematic review, 25 different outcomes were reported. Only 22% of trials had clearly stated primary outcome measures. Repigmentation was the most frequently reported outcome in 96% of trials and was measured using 48 different scales. Only 9% of trials assessed quality of life. Thirteen percent measured cessation of spreading of the disease and 17% of studies reported patients’ opinions and satisfaction with the treatment.
In contrast, out of 438 suggestions made by patients and clinicians, cosmetically acceptable repigmentation (rather than percentage of repigmentation) was the most desirable outcome (68%), followed by cessation of spread of vitiligo (15%), quality of life (6%) and maintenance of repigmentation (4%).
CONCLUSIONS: We propose that future vitiligo trials should include: repigmentation, cosmetic acceptability of results, global assessment of the disease, quality of life, maintenance of repigmentation, stabilisation of vitiligo and side effects.
International consensus amongst clinicians, researchers and patients is needed to establish an agreed core outcome set for future vitiligo trials.
V.Eleftheriadou, K.S. Thomas, M.E. Whitton, J.M Batchelor, J.C. Ravenscroft
Disease Category: Skin
Disease Name: Vitiligo
Age Range: 18 - 100
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (patients)
- Systematic review of outcomes measured in trials
- COS for clinical trials or clinical research
- Systematic review
- Survey
Systematic review of outcome measures in randomised controlled trials of treatments for vitiligo - A review was conducted of the outcome measures used in RCTs published in English and included in the updated Cochrane systematic review “Interventions for vitiligo” published in 2010. The review included RCTs published up to November 2009. All the outcomes reported in the eligible RCTs were retrieved and the scales and measures used to monitor these were collated.
Survey of the most desirable outcomes for vitiligo identified by patients and clinicians - A survey was conducted as part of the Vitiligo Priority Setting Partnership (Vitiligo PSP). This was co-ordinated from the Centre of Evidence Based Dermatology at the University of Nottingham, in collaboration with the James Lind Alliance and the Vitiligo Society UK. Patients and healthcare professionals were asked in an open-ended questionnaire to submit details of their uncertainties regarding vitiligo treatment, along with their views on what outcomes should be measured in future vitiligo trials. The survey was open for submissions between January and March 2009.