A phase II randomized vehicle-controlled trial of intradermal allogeneic fibroblasts for recessive dystrophic epidermolysis bullosa JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY Venugopal, S. S., Yan, W., Frew, J. W., Cohn, H. I., Rhodes, L. M., Kim Tran, K., Melbourne, W., Nelson, J. A., Sturm, M., Fogarty, J., Marinkovich, M. P., Igawa, S., Ishida-Yamamoto, A., Murrell, D. F. 2013; 69 (6): 898-915


Chronic wounds are a major source of morbidity and mortality in generalized severe recessive dystrophic epidermolysis bullosa (RDEB-GS).This was a phase II double-blinded randomized controlled trial of intralesional allogeneic cultured fibroblasts in suspension solution versus suspension solution alone for wound healing in RDEB-GS.Adult patients with RDEB-GS were screened for chronic ulcers and reduced collagen VII expression. Up to 6 pairs of symmetric wounds were measured and biopsied at baseline, then randomized to cultured allogeneic fibroblasts in a crystalloid suspension solution with 2% albumin or suspension solution alone. Ulcer size, collagen VII protein and messenger RNA expression, anchoring fibril numbers, morphology, and inflammatory markers were measured at 2 weeks and at 3, 6, and 12 months.All wounds healed significantly more rapidly with fibroblasts and vehicle injections, with an area decrease of 50% by 12 weeks, compared with noninjected wounds. Collagen VII expression increased to a similar degree in both study arms in wounds from 3 of 5 patients.The number of patients with RDEB-GS who met inclusion criteria was a limitation, as was 1 trial center rather than multicenter.The injection of both allogeneic fibroblasts and suspension solution alone improved wound healing in chronic nonhealing RDEB-GS wounds independently of collagen VII regeneration. This may provide feasible therapy for wound healing in patients with RDEB-GS.

View details for DOI 10.1016/j.jaad.2013.08.014

View details for Web of Science ID 000327736900023

View details for PubMedID 24075228