Background:
Major advances have been made in the pathogenesis and treatment of both genetic and autoimmune blistering diseases in the last 20 years, perhaps more than in any other field of dermatology. However, compared with other fields in dermatology, randomized controlled trials of treatment have been few, in part owing to a lack of validated outcome measures required to demonstrate responsiveness for clinical trials.
Objective:
To review the stages of validation for the objective disease severity scores in autoimmune (AIBD) and genetic blistering diseases as well as subjective quality of life scores.
Results:
Three objective disease severity scores have been tested for validity in epidermolysis bullosa (EB): the Birmingham EB score, the EBDASI and the iSCOREB. The latter combines a function questionnaire and laboratory values. One subjective score, the QOLEB, has been validated and shown responsiveness in clinical trials of new therapies for EB, and revalidated in Spanish, Dutch and Portuguese so far.
In AIBD, uniform consensus definitions have been published for pemphigus, pemphigoid and mucous membrane pemphigoid. The objective scoring systems, PDAI, BPDAI and MMPDAI have been developed and the former two have been validated by several independent groups. Another one, termed ABSIS, combines objective and subjective measures, with a weighting towards oral involvement. This score is less accurate at lower degrees of severity as it cannot score areas less than 1% of body surface area.
Two specific quality of life (QOL) measures for AIBD have been validated, ABQOL and TABQOL. These are being re-validated in various foreign languages.
Conclusion:
There are now sufficient disease-specific validated tools in blistering diseases to enable randomized controlled trials to progress in this orphan population.