A phase II randomized vehicle-controlled trial of intradermal allogeneic fibroblasts for recessive dystrophic epidermolysis bullosa.
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.


CONCLUSIONS
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.
Department of Dermatology, St George Hospital, Sydney, Australia; University of New South Wales, Sydney, Australia.
Supriya S
SS
Venugopal
Wenfei
W
Yan
John W
JW
Frew
Heather I
HI
Cohn
Lesley M
LM
Rhodes
Kim
K
Tran
Wei
W
Melbourne
Janis A
JA
Nelson
Marian
M
Sturm
Janice
J
Fogarty
M Peter
MP
Marinkovich
Satomi
S
Igawa
Akemi
A
Ishida-Yamamoto
Dedee F
DF
Murrell
D000328:Adult; D004311:Double-Blind Method; D016108:Epidermolysis Bullosa Dystrophica / Q000628:therapy*; D005260:Female; D005347:Fibroblasts / Q000637:transplantation*; D006801:Humans; D007271:Injections, Intradermal; D008297:Male; D014184:Transplantation, Homologous; D014945:Wound Healing; D055815:Young Adult
EB; HLA; RDEB; RDEB-GS; VAS; allogeneic fibroblasts; cell therapy; collagen VII; epidermolysis bullosa; generalized severe recessive dystrophic epidermolysis bullosa; injections; mRNA; messenger RNA; phase II; quality of life; randomized controlled trial; recessive dystrophic epidermolysis bullosa; visual analog scale; wound healing