Mesenchymal stromal cell infusions of umbilical cord-derived mesenchymal stromal cells in children with…

StudyOrphanet journal of rare diseases· Jun 2026

Mesenchymal stromal cell infusions of umbilical cord-derived mesenchymal stromal cells in children with Recessive Dystrophic Epidermolysis Bullosa (MissionEB): a qualitative sub study of a randomised, double-blind, placebo controlled, crossover, phase 3 trial.

Katie Biggs, Shamila Ditta, Maria L Bageta … Anna E Martinez

Pediatric DermatologyStudyTreatmentQ1IF 3.5

Blinded participants could distinguish active mesenchymal stromal cell therapy from placebo by symptoms

Mesenchymal stromal cell infusions of umbilical cord-derived mesenchymal stromal cells in children with Recessive Dystrophic Epidermolysis Bullosa (MissionEB): a qualitative sub study of a randomised, double-blind, placebo controlled, crossover, phase 3 trial.

Katie Biggs … Anna E Martinez

Orphanet journal of rare diseases · Jun 2026 · Q1 in General Medicine

Background

Recessive Dystrophic Epidermolysis Bullosa (Recessive Dystrophic Epidermolysis Bullosa) is a rare genetic skin condition causing fragile skin, blistering, and scarring.

Methods

This study used a qualitative research design with semi-structured interviews and thematic analysis, to explore Mission EB trial treatment impact on the quality of life of patients and parents.

n = 13 individuals

10

Results

10

of 13

some children and parents could tell active therapy from placebo by symptom changes

n = 13 individuals

More results

Recessive Dystrophic Epidermolysis Bullosa significantly impacted daily life, marked by pain and itch.

UC-MSC infusions led to reduced pain/itchiness, improved wound healing, and resulted in fewer self-reported dressing changes.

These benefits often translated to increased energy, improved eating, and greater daily activity participation.

More results

Benefits were more pronounced with active treatment.

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Conclusion · 1 of 2

This qualitative research provides valuable insights into perceived benefits of UC-MSC treatment for children with Recessive Dystrophic Epidermolysis Bullosa.

Conclusion · 2 of 2

Interview findings regarding symptom improvement and participants' ability to discern active treatment shed light on its perceived benefit in Recessive Dystrophic Epidermolysis Bullosa, especially in milder Recessive Dystrophic Epidermolysis Bullosa patients.

Mesenchymal stromal cell infusions of umbilical cord-derived mesenchymal stromal cells in children with Recessive Dystrophic Epidermolysis Bullosa (MissionEB): a qualitative sub study of a randomised, double-blind, placebo controlled, crossover, phase 3 trial.

Mesenchymal stromal cell infusions of umbilical cord-derived mesenchymal stromal cells in children with Recessive Dystrophic Epidermolysis Bullosa (MissionEB): a qualitative sub study of a randomised, double-blind, placebo controlled, crossover, phase 3 trial.

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