Phase I/II open-label trial of intravenous allogeneic mesenchymal stromal cell therapy in adults with recessive dystrophic epidermolysis bullosa

被引：55

作者：

Rashidghamat, Ellie ^{[1
]}

Kadiyirire, Tendai ^{[1
]}

Ayis, Salma ^{[2
]}

Petrof, Gabriela ^{[5
]}

Liu, Lu ^{[6
]}

Pullabhatla, Venu ^{[7
]}

Ainali, Chrysanthi ^{[1
,9
]}

Guy, Alyson ^{[6
]}

Aristodemou, Sophia ^{[6
]}

McMillan, James R. ^{[6
]}

Ozoemena, Linda ^{[6
]}

Mee, John ^{[10
]}

Pramanik, Rashida ^{[1
]}

Saxena, Alka ^{[7
]}

Nuamah, Rosamund ^{[7
]}

de Rinaldis, Emanuele ^{[11
]}

Serrano, Sonia ^{[8
]}

Maurin, Clarisse ^{[8
]}

Martinez-Queipo, Magdalena ^{[1
]}

Lwin, Su M. ^{[1
]}

Ilic, Dusko ^{[3
]}

Martinez, Anna ^{[5
]}

Dazzi, Francesco ^{[4
]}

Slaper-Cortenbach, Ineke ^{[12
]}

Westinga, Kasper ^{[12
]}

Zeddies, Sabrina ^{[12
]}

van den Broek, Marcel ^{[12
]}

Onoufriadis, Alexandros ^{[1
]}

Mellerio, Jemima E. ^{[1
]}

McGrath, John A. ^{[1
]}

机构：

[1] Kings Coll London, St Johns Inst Dermatol, Sch Basic & Med Biosci, London, England

[2] Kings Coll London, Sch Populat Hlth & Environm Sci, London, England

[3] Kings Coll London, Fac Life Sci & Med, Dept Women & Childrens Hlth, Guys Assisted Concept Unit,Stem Cell Labs, London, England

[4] Kings Coll London, Rayne Inst, Dept Haematol Med, London, England

[5] Great Ormond St Hosp Children NHS Fdn Trust, Dept Dermatol, London, England

[6] Guys Hosp, Robin Eady Natl Diagnost Epidermolysis Bullosa La, Viapath, London, England

[7] Guys & St Thomas NHS Fdn Trust, UK NIHR GSTFT KCL Comprehens Biomed Res Ctr, London, England

[8] Guys & St Thomas NHS Fdn Trust, Clin Trial Management Res Platform, NIHR Biomed Res Ctr, London, England

[9] Dignosis Ltd, London, England

[10] St Thomas Hosp, Viapath, Immunodermatol Lab, London, England

[11] Sanofi, I&I Precis Immunol, Cambridge, MA USA

[12] Univ Med Ctr Utrecht, Dept Clin Pharm, Cell Therapy Facil, Utrecht, Netherlands

来源：

JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY | 2020年 / 83卷 / 02期

关键词：

BM-MSC; epidermolysis bullosa; mesenchymal stromal cells; RDEB; IIND INTERNATIONAL-SYMPOSIUM; BONE-MARROW; STEM-CELLS; MANAGEMENT; CARCINOMA; SEVERITY; SANTIAGO; CHILE; ITCH; EB;

D O I：

10.1016/j.jaad.2019.11.038

中图分类号：

R75 [皮肤病学与性病学];

学科分类号：

100206 ;

摘要：

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 3 106 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.

引用

页码：447 / 454

页数：8

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