MesenSistem-EB: systemic haploidentical mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa associated with clinical benefits and correlated with MCP1 and sCD40L dynamics
Abstract
Background: Recessive dystrophic epidermolysis bullosa (RDEB) is a devastating genodermatosis caused by biallelic COL7A1 mutations, where chronic inflammation drives disabling complications affecting quality of life and survival. Effective inflammatory control remains a major unmet need. While systemic administration of mesenchymal stromal cells (MSC) from various sources has proven safe with transient benefits yet only one recent placebo-controlled study in RDEB. Consistent type VII collagen (C7) deposition is lacking, and therapeutic mechanisms and predictive biomarkers remain undefined. The MesenSistem-EB trial evaluated for the first time the use of haploidentical BM-MSC and extensively explored their anti-inflammatory potential as a monotherapy in RDEB.
Methods: Phase I/II, single-center, open-label trial, in which nine children with severe RDEB were recruited and eight completed the study after three intravenous infusions of haploidentical BM-MSC (2-3×106 cells/kg) at 21-day intervals. Safety, clinical and patient-reported outcomes, systemic inflammatory mediators and peripheral blood immune profiles were assessed over 12 months.
Results: MSC therapy was well-tolerated without serious adverse events. Long-term pruritus, sleep and fatigue were globally reduced. In 7/8 patients at least one key domain significantly improved (disease severity, pruritus, or inflammation) with five classified as good responders. Global median CRP and fibrinogen levels remained stable…
How strong is the evidence?
Evidence Hub reads this as a clinical trial, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence. Reported size: 8 participants or records.
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