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 randomized controlled trials (RCTs) and perform meta-analysis. OBJECTIVES: To describe the heterogeneity in outcome measures used in published RCTs 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 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 with 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 per cent 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 (8%) 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, stabilization of vitiligo and side-effects. International consensus among clinicians, researchers and patients is needed to establish an agreed core outcome set for future vitiligo trials.
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 randomized controlled trials (RCTs) and perform meta-analysis. OBJECTIVES: To describe the heterogeneity in outcome measures used in published RCTs 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 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 with 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 per cent 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 (8%) 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, stabilization of vitiligo and side-effects. International consensus among clinicians, researchers and patients is needed to establish an agreed core outcome set for future vitiligo trials.
Authors: Paula R Williamson; Douglas G Altman; Heather Bagley; Karen L Barnes; Jane M Blazeby; Sara T Brookes; Mike Clarke; Elizabeth Gargon; Sarah Gorst; Nicola Harman; Jamie J Kirkham; Angus McNair; Cecilia A C Prinsen; Jochen Schmitt; Caroline B Terwee; Bridget Young Journal: Trials Date: 2017-06-20 Impact factor: 2.279
Authors: Laura P Forsythe; Victoria Szydlowski; Mohammad Hassan Murad; Stanley Ip; Zhen Wang; Tarig A Elraiyah; Rachael Fleurence; David H Hickam Journal: J Gen Intern Med Date: 2014-08 Impact factor: 5.128
Authors: Jonathan M Batchelor; Kim S Thomas; Perways Akram; Jaskiran Azad; Anthony Bewley; Joanne R Chalmers; Seau Tak Cheung; Lelia Duley; Viktoria Eleftheriadou; Robert Ellis; Adam Ferguson; Jonathan Mr Goulding; Rachel H Haines; Hamdi Hamad; John R Ingram; Bisola Laguda; Paul Leighton; Nick Levell; Areti Makrygeorgou; Garry D Meakin; Adam Millington; Malobi Ogboli; Amirtha Rajasekaran; Jane C Ravenscroft; Andrew Rogers; Tracey H Sach; Miriam Santer; Julia Stainforth; Wei Tan; Shyamal Wahie; Jennifer White; Maxine E Whitton; Hywel C Williams; Andrew Wright; Alan A Montgomery Journal: Health Technol Assess Date: 2020-11 Impact factor: 4.014