Phase I/II open-label trial of intravenous allogeneic mesenchymal stromal cell therapy in adults with recessive dystrophic epidermolysis bullosa.
J Am Acad Dermatol
; 83(2): 447-454, 2020 Aug.
Article
em En
| MEDLINE
| ID: mdl-31786163
ABSTRACT
BACKGROUND:
Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary blistering disorder due to a lack of type VII collagen. At present, treatment is mainly supportive.OBJECTIVES:
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.METHODS:
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).RESULTS:
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.LIMITATIONS:
Open-label trial with no placebo.CONCLUSIONS:
MSC infusion is safe in RDEB adults and can have clinical benefits for at least 2 months.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Prurido
/
Epidermólise Bolhosa Distrófica
/
Transplante de Células-Tronco Mesenquimais
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article