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328 50% wound healing correlates with RDEB patient reported outcomes in pain, itch and skin durability

2018; Elsevier BV; Volume: 138; Issue: 5 Linguagem: Inglês

10.1016/j.jid.2018.03.334

1523-1747

Y. Dutt-Singkh, M. Barriga, Jaron Nazaroff, D. Solis, S. Li, M. Peter Marinkovich, Jean Y. Tang,

Tendon Structure and Treatment

RDEB is caused by mutations in the COL7A1 gene encoding type VII collagen (C7). We report on wound healing and high correlation to patient reported outcomes in our Phase I/IIA clinical trial of ex vivo C7 gene therapy (LEAES grafts, EB-101) for treatment of chronic wounds in severe RDEB (N=7 adults, 36 wounds assessed). There is limited information on measuring dynamic wounds in RDEB and the amount of wound healing that is clinically meaningful in patient reported outcomes. We compared 2 methods to calculate wound surface area: quantitative 3D imaging with high-resolution Canfield camera (Vectra H1) vs. Investigator global score as assessed by 2 physicians in real time on 36 wounds followed for 6 months. We found a high degree of agreement between Canfield 3D and Investigator global score (ICC=0.92, P<0.0001). Prior to grafting, subjects reported pain, itch and blistering in each chronic wound (N=36). After application of EB-101 grafts, subjects reported no pain (91% of wounds), absence of itch (87.5%), higher durability of skin (74.0%), and more difficulty blistering (81.7%) after 3 and 6 months. Using the Investigator global score, RDEB wounds that were ≥50% healed showed improvements in pain, itch, skin durability and ease of blistering (P<0.0001). By comparison, grafts with ≤49% of wound healing had more pain (60%), itch (67%), low durability (82%), and easy blistering (65%) after 3 and 6 months. In summary, we found that 3D wound assessment and investigator global score were well correlated and reliable, and that ≥50% wound healing correlated with statistically significant improvements in patient reported outcomes.