Participatory co-design of patient-reported outcome indicators and N-of-1 evaluation of a dressing glove for Epidermolysis bullosa.

Abstract

In autosomal recessive dystrophic Epidermolysis bullosa, repeat blistering results in finger webbing and severe contractures of the hands. The aim of this study was to codesign patient-reported outcome indicators for hand therapy with patients, carers and clinicians, and use these to proof-of-concept test a novel dressing glove for recessive dystrophic Epidermolysis bullosa, with cost analysis. Qualitative interviews and focus groups with patients and carers generated content for the indicators. Validity and reliability were established through expert review, piloting and consensus between patients, carers and clinicians. The indicators were self-reported by patients before and while wearing the dressing glove in an N-of-1 study. Time for dressing changes and use of conventional products were also self-reported. A total of 11 indicators were initially generated from the thematic analysis. Expert review, piloting and consensus involved six patients, five carers and eight clinicians (total n=19). Participants agreed 14 indicators, covering hand skin condition (n=4), webbing between the digits (n=4), experiences of wearing and changing dressings (n=2), hand function (n=2), wrist function (n=1) and hand pain (n=1). In Phase 3, 12 patients scored indicators before wearing the gloves and four patients completed scoring while wearing the gloves. Statistically significant improvements between pre-glove and with-glove periods were found for most participants' experience scores. Skin appearance also improved for most participants. The indicators generated useful data, differentiation between scores and participants demonstrating proof-of-concept for patients with recessive dystrophic Epidermolysis bullosa who could wear the dressing gloves. The indicators are being used in routine practice, supporting clinical follow up, commercialisation and regulatory governance of the dressing glove.