Benefit evaluation in vitiligo treatment: Development and validation of a patient-defined outcome questionnaire

Background: In addition to clinical efficacy, patient-defined benefit assessment has become an important outcome parameter. Thus far, no such instrument has been developed for vitiligo. Objectives: Development and validation of a patient-defined benefit questionnaire in the therapy of vitiligo. Methods: Open questioning of 50 vitiligo patients generated 110 benefit items, which were converted into a 26-item questionnaire by a panel of dermatologists, psychologists and patients. This was used to record patient-defined needs prior to therapy and to evaluate patient benefit attained after therapy. A 'patient benefit index' ( PBI) was formed on the basis of both. Validation was based on data of n=1,023 vitiligo patients. Results: The questionnaires were feasible in clinical practice, Cronbach's alpha ( patient need questionnaire) was 0.94. The PBI showed convergent and discriminant validity with respect to quality of life and patient satisfaction. Conclusion: The PBI for vitiligo is a valid instrument, which is highly accepted in practice for recording patient-reported benefit. Copyright (c) 2008 S. Karger AG, Basel.