Study of Cellutome System for Treatment of Individual Lesions in EB Pts

Not Applicable

Completed

Brief Summary: Few but persistent wounds often remain even after successful hematopoietic cell transplantation for systemic genodermatosis epidermolysis bullosa (EB). The investigators propose local wound therapy using epidermal skin grafting from the same donor that provided the hematopoietic graft, or from the same EB individual with a mosaic (naturally gene corrected) skin. In both cases permissive immune system and skin chimerism is expected to enable long-term epidermal engraftment and wound healing. The investigators will use FDA approved vacuum device (CelluTome®, Regulation number 878.4820) that enables scar-free harvesting of epidermis and its transfer on a non-adherent silicone dressing (Adaptic) to the recipient as a wound dressing.

Recruitment & Eligibility

Inclusion Criteria

Patient (Recipient)

Diagnosis of Dystrophic Epidermolysis Bullosa (DEB) or Junctional Epidermolysis Bullosa (JEB) with at least one wound, visibly free from infection (or previously treated) and meets the eligibility for Arm A or Arm B based on the skin graft source:
Cell harvest from previous hematopoietic cell transplantation (HCT) donor (Arm A) - not applicable if Arm B
- At least 6 months after hematopoietic cell transplantation with donor chimerism
  - Peripheral blood donor chimerism should be measured within 21 days of grafting and be >/= 5% and stable. Stability of chimerism will be determined by the protocol team and based on 3 peripheral blood chimerism values at least 1 month apart.
- No history of pre-BMT autoimmune cytopenias
- Off immune suppressive therapy
- Original transplant donor is available and willing to be the epidermis donor
Self-donation (Arm B) - not applicable if Arm A
- Proven somatic reversion
- Site for skin grafting free of cellulitis and any other clinically evident abnormalities
- Meets donor eligibility
Insurance pre-authorization for procedure, if applicable
Voluntary written consent (patient or parent/guardian for minors with assent) prior to any research related procedures or treatment.

Skin Graft Donor (either hematopoietic cell transplantation donor for the EB patient [Arm A] or EB patient herself/himself [Arm B])

Age > 2 years (based on prior safety testing of the device)
Healthy on physical examination in the opinion of the evaluating provider
Negativity for Hepatitis B and C, HIV, and HTLV1/2 within 30 days of donation
Voluntary written consent (donor or parent/guardian for minors with assent) prior to any research related procedures

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Graft from HCT donor	Cellutome Epidermal Harvesting System	Cells are harvested from a donor using Cellutome, then transferred via Adaptic dressing to the recipient's wound with up to 3 donor harvest sites/treated wound sites on day 0.
Self donor from intact skin patch	Cellutome Epidermal Harvesting System	Cells are harvested from the subject using Cellutome, then transferred via Adaptic dressing to that subject's wound with up to 3 harvest sites/treated wound sites on day 0.

Primary Outcome Measures

Name	Time	Method
Percentage of Grafts Successfully Treated	12 weeks after grafting	If the body surface area affected by the wound is at least 50% lower at 12 weeks relative to baseline, the graft will be considered successful.

Secondary Outcome Measures

Name	Time	Method
Participants With Lesion Free Skin	1 year after grafting	Participants without Squamous cell carcinoma (SCC) at graft site. Wound reassessments will be performed via photographs and follow-up visits.
Percentage Change of a Patient's IScoreEB Assessment Score	Baseline and 12 weeks	Measure percent of changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life.
Scar-free Healing of the Body Sites of the Donor	1 year after grafting	Percentage of donors with no evidence of non-healed skin. Wound reassessments will be performed via photographs and follow-up visits.
Longevity of Grafted Skin	1 year after grafting	Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting. Wound reassessments will be performed via photographs and follow-up visits.
Percentage Change of a Patient's IScorEB Assessment Score	Baseline and 6 weeks	Measure percent of changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life.