Quality of Life Evaluation in Epidermolysis Bullosa

JW Frew1,8 SS Venugopal1,8, WF Yan1,8,LK Martin1, T Nijsten2, HI Cohn1, LM Rhodes1, K Tran3,8, W Melbourne3, J Nelson4, M Sturm5, J Fogarty5, P Marinkovich6 S Igawa7, A Ishida-Yamamoto7, DF Murrell1,8

1Dept of Dermatology, St George Hospital , 2Department of Dermatology, Erasmus MC, Rotterdam, the Netherlands, 3Department of Anatomical Pathology, St George Hospital, 4Pharmacy Department, St George Hospital, Sydney, Australia, 5Cell and Tissue Therapies WA, Royal Perth Hospital, Perth, Australia, 6Department of Dermatology, Stanford University, USA, 7Asahikawa Medical College, Asahikawa, Japan, 8University of New South Wales, Sydney, Australia.

Quality of Life (QoL) is a complex concept which takes into account the physical, emotional, psychological and social aspects in which a disease may impact upon an individual. In individuals with Epidermolysis Bullosa (EB) it is especially important as it can help identify areas in which to focus treatment, help evaluate new treatments, compare the burden of disease across different types of EB and give accurate measurements of changes in QoL in EB across time.

Previous studies have used general dermatology QoL scales to measure QoL in EB which do not take into account specific problems encountered by individuals with EB. Such measurement tools can result in underestimating the QoL impact found in patients with EB.

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Due to these problems with current QoL measurement tools, we developed an EB-specific Quality of Life scale (The QOLEB Questionnaire) which was statistically proven to be valid and reliable and allowed us to stratify quality of life into very mild, mild, moderate, severe and very severe QoL impairment, as well as allowing us to monitor QoL changes during the use of new treatments in clinical trials.


One such clinical trial was a double blind randomized placebo controlled trial of allogeneic fibroblasts compared with injection of a placebo suspension solution. Our results showed dramatic wound healing with both fibroblast and placebo injections as well as increases in Collagen VII levels and overall improvements in QoL. 3 of the 5 patients in the trial showed QoL changes which were above the Minimal Clinically Important Difference (MCID) for the QOLEB Questionnaire which indicated the treatment made a significant difference to their overall Quality of Life.

Therefore, our QOLEB Questionnaire has a significant contribution in evaluating Quality of Life in EB and is also useful within clinical trials to show the effectiveness of new treatments in creating overall benefit in the QoL of patients with EB. In the future we hope to translate the QOLEB into a variety of different languages in order to help encourage the use of this questionnaire worldwide.

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