A phase II randomized vehicle-controlled trial of intradermal allogeneic
fibroblasts for recessive dystrophic epidermolysis bullosa.
Author(s): Venugopal SS(1), Yan W, Frew JW, Cohn HI, Rhodes LM, Tran K, Melbourne W, Nelson
JA, Sturm M, Fogarty J, Marinkovich MP, Igawa S, Ishida-Yamamoto A, Murrell DF.
Affiliation(s): Author information:
(1)Department of Dermatology, St George Hospital, Sydney, Australia; University of
New South Wales, Sydney, Australia.
Publication date & source: 2013, J Am Acad Dermatol. , 69(6):898-908
BACKGROUND: Chronic wounds are a major source of morbidity and mortality in
generalized severe recessive dystrophic epidermolysis bullosa (RDEB-GS).
OBJECTIVE: 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.
METHODS: 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.
RESULTS: 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.
LIMITATIONS: The number of patients with RDEB-GS who met inclusion criteria was a
limitation, as was 1 trial center rather than multicenter.
CONCLUSIONS: 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.
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