Abstract
BackgroundChronic wounds are a fundamental issue for patients with epidermolysis bullosa (EB). Herein, we assess the natural history of wound closure in patients with EB who were randomly assigned to the vehicle-control arm of the multicenter, randomized, double-blind, phase 3 ESSENCE (NCT02384460) trial.MethodsESSENCE was designed to assess the efficacy and safety of a topical cream formulation of 6% allantoin (SD-101 6%) vs vehicle (SD-101 0%) in patients ≥1 month old who had a diagnosis of EB (simplex, recessive dystrophic, or intermediate junctional) and a target wound 10–50 cm2 present for ≥21 days. Time to complete target wound closure and the proportion of patients with target wound closure over time were analyzed overall and by parameters including patient age and baseline body surface area index (BSAi) of total wound burden (< 5% and ≥ 5%). Changes in BSAi of lesional skin, pain, and itching were also assessed.ResultsThe vehicle-control arm included 87 patients. Mean (standard deviation [SD]) time to target wound closure within 3 months was 53.6 (28.6) days, with a range of 14 to 142 days. The proportion of patients with target wound closure increased over time from 7.1% at day 14 to 53.6% at month 3. Mean (SD) changes from baseline in BSAi of total wound burden and BSAi of lesional skin at month 3 were −2.3% (6.3) and −5.0% (13.5) of total body coverage, respectively. Reductions in pain and itching were observed at day 7 and maintained for 3 months. Faster healing times and a greater proportion of patients with wound closure were observed in patients aged 1 month to < 2 years; those with wounds < 30 days old, and in those with BSAi of total body wound burden < 5%.ConclusionsTreatment response observed in the vehicle-control arm of the ESSENCE study was unexpectedly high and may have been due to unforeseen benefits of vehicle or enhanced wound care provided by the clinical trial staff. These observations will help inform the study design of future trials in patients with EB.Trial registrationClinicalTrials.gov, NCT02384460; Date of registration: February 13, 2015; First participant enrollment: March 11, 2015.
Highlights
Epidermolysis bullosa (EB) is a genetically heterogeneous group of rare, devastating disorders characterized by fragility of skin and mucous membranes that blister in response to minor mechanical trauma [1,2,3]
Mean (SD) body surface area index (BSAi) of total body wound burden was 10.5% (9.1), and most (55 of 87) patients at baseline had a BSAi of total body wound burden ≥5% (Table 2)
Treatment response observed in the vehicle-control arm of the ESSENCE study was unexpectedly high, with overall improvements observed in target wound closure, lesional skin and total body wound burden, itch, and pain over the 3-month treatment period
Summary
Epidermolysis bullosa (EB) is a genetically heterogeneous group of rare, devastating disorders characterized by fragility of skin and mucous membranes that blister in response to minor mechanical trauma [1,2,3]. Signs and symptoms of EB, which include chronic blisters or erosions, ulcers, severe itching, pain, and recurrent wound infection, typically appear in infancy and continue throughout life [4]. Blisters can occur in mucous membranes, including the mouth, which can limit food intake and cause scarring with stricture formation [4]. EB simplex is typically the mildest form of the disease and is generally associated with less scarring and fewer internal complications compared with junctional or dystrophic EB [2]. Chronic wounds are a fundamental issue for patients with epidermolysis bullosa (EB). We assess the natural history of wound closure in patients with EB who were randomly assigned to the vehicle-control arm of the multicenter, randomized, double-blind, phase 3 ESSENCE (NCT02384460) trial
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