A phase II randomized vehicle-controlled trial of intradermal allogeneic fibroblasts for recessive dystrophic epidermolysis bullosa - 14/11/13
Abstract |
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.
Le texte complet de cet article est disponible en PDF.Key words : allogeneic fibroblasts, cell therapy, collagen VII, epidermolysis bullosa, HLA, injections, phase II, quality of life, randomized controlled trial, wound healing
Abbreviations used : EB, mRNA, RDEB, RDEB-GS, VAS
Plan
| Drs Venugopal and Yan contributed equally to this article. |
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| Funding for this study was provided by Australian Blistering Diseases Foundation; Research Infrastructure Support Services, Australia; Caroline Quinn Trust Fund; Australian Postgraduate Awards for PhD studies (Drs Venugopal and Yan); DebRA Australia, Ireland, and New Zealand; Premier Specialists, Sydney, Australia; Office of Research Administration, Palo Alto Department of Veterans Affairs Medical Center; the Epidermolysis Bullosa Medical Research Fund; and American Australian Association. |
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| Conflicts of interest: None declared. |
Vol 69 - N° 6
P. 898 - décembre 2013 Retour au numéro
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