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Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary blistering disorder due to a lack of type VII collagen. At present, treatment is mainly supportive.
To determine whether intravenous allogeneic bone marrow–derived mesenchymal stromal/stem cells (BM-MSCs) are safe in RDEB adults and if the cells improve wound healing and quality of life.
We conducted a prospective, phase I/II, open-label study recruiting 10 RDEB adults to receive 2 intravenous infusions of BM-MSCs (on day 0 and day 14; each dose 2-4 × 106 cells/kg).
BM-MSCs were well tolerated with no serious adverse events to 12 months. Regarding efficacy, there was a transient reduction in disease activity scores (8/10 subjects) and a significant reduction in itch. One individual showed a transient increase in type VII collagen.
Open-label trial with no placebo.
MSC infusion is safe in RDEB adults and can have clinical benefits for at least 2 months.Le texte complet de cet article est disponible en PDF.
Key words : BM-MSC, epidermolysis bullosa, mesenchymal stromal cells, RDEB
Abbreviations used : BM, C7, CI, EB, HMGB-1, MSC, RDEB, SCC, SD, SEM
| Funding sources: Supported by Debra UK and the Sohana Research Fund (now known as Cure EB). The research was funded/supported by the National Institute for Health Research Biomedical Research Centre based at Guy's and St Thomas' NHS Foundation Trust and King's College London, with the trial undertaken in the Clinical Research Facility at Guy's Hospital.
| Conflicts of interest: None disclosed.
| Reprints not available from the authors.