Fibroblast cell therapy enhances initial healing in recessive dystrophic epidermolysis bullosa wounds: results of a randomized, vehicle-controlled trial

TY - JOUR

T1 - Fibroblast cell therapy enhances initial healing in recessive dystrophic epidermolysis bullosa wounds

T2 - results of a randomized, vehicle-controlled trial

AU - Petrof, G.

AU - Martinez-Queipo, M.

AU - Mellerio, J. E.

AU - Kemp, P.

AU - McGrath, J. A.

PY - 2013/11

Y1 - 2013/11

N2 - Background Fibroblast cell therapy can modify disease biology in recessive dystrophic epidermolysis bullosa (RDEB) although whether it improves wound healing is not clear. Objective To assess the effects of injecting of allogeneic fibroblasts into the margins of chronic erosions in individuals with RDEB in a prospective, double-blind, randomized, vehicle-controlled phase II trial. Methods Erosions were randomized 1:1, to either a single treatment of 5x10(6) fibroblasts per linear cm of erosion margin or vehicle. All subjects continued standard wound care. The trial sponsor, participants and study outcome assessor were masked to treatment allocation. A hierarchy of endpoints germane to erosion closure was assessed. Results Twenty-six erosions in 11 subjects with RDEB were injected; 14 erosions received fibroblasts and 12 vehicle alone. A single series of injections was given at day 0 and all follow-up visits were completed. Treatment difference between fibroblasts and vehicle was -235% [95% confidence interval (CI) -35 to -435, P=0025] at day 7, -1915% (95% CI 336 to -4166, P=0089) at day 14 and -2883% (95% CI 797 to -6563, P=011) at day 28. Beyond day 28, however, changes in mean erosion area did not differ significantly between the two groups. Conclusion A single intradermal injection of allogeneic fibroblasts increases the initial rate of erosion healing in subjects with RDEB within the first 28days but not thereafter. Further studies are needed to address the potential benefits of retreatment as well as optimal cell delivery.

AB - Background Fibroblast cell therapy can modify disease biology in recessive dystrophic epidermolysis bullosa (RDEB) although whether it improves wound healing is not clear. Objective To assess the effects of injecting of allogeneic fibroblasts into the margins of chronic erosions in individuals with RDEB in a prospective, double-blind, randomized, vehicle-controlled phase II trial. Methods Erosions were randomized 1:1, to either a single treatment of 5x10(6) fibroblasts per linear cm of erosion margin or vehicle. All subjects continued standard wound care. The trial sponsor, participants and study outcome assessor were masked to treatment allocation. A hierarchy of endpoints germane to erosion closure was assessed. Results Twenty-six erosions in 11 subjects with RDEB were injected; 14 erosions received fibroblasts and 12 vehicle alone. A single series of injections was given at day 0 and all follow-up visits were completed. Treatment difference between fibroblasts and vehicle was -235% [95% confidence interval (CI) -35 to -435, P=0025] at day 7, -1915% (95% CI 336 to -4166, P=0089) at day 14 and -2883% (95% CI 797 to -6563, P=011) at day 28. Beyond day 28, however, changes in mean erosion area did not differ significantly between the two groups. Conclusion A single intradermal injection of allogeneic fibroblasts increases the initial rate of erosion healing in subjects with RDEB within the first 28days but not thereafter. Further studies are needed to address the potential benefits of retreatment as well as optimal cell delivery.

KW - VII COLLAGEN

KW - EXTRACUTANEOUS MANIFESTATIONS

KW - SKIN

KW - KERATINOCYTES

KW - COMPLICATIONS

KW - ULCERS

KW - TISSUE

KW - COL7A1

U2 - 10.1111/bjd.12599

DO - 10.1111/bjd.12599

M3 - Article

SN - 0007-0963

VL - 169

SP - 1025

EP - 1033

JO - British Journal of Dermatology

JF - British Journal of Dermatology

IS - 5

M1 - N/A

ER -