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

Autor: Rashidghamat E; St John's Institute of Dermatology, School of Basic and Medical Biosciences, King's College London, London, UK., Kadiyirire T; St John's Institute of Dermatology, School of Basic and Medical Biosciences, King's College London, London, UK., Ayis S; School of Population Health and Environmental Sciences, King's College London, London, UK., Petrof G; Department of Dermatology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Liu L; The Robin Eady National Diagnostic Epidermolysis Bullosa Laboratory, Viapath, Guy's Hospital, London, UK., Pullabhatla V; UK NIHR GSTFT/KCL Comprehensive Biomedical Research Centre, Guy's & St Thomas' NHS Foundation Trust, London, UK., Ainali C; St John's Institute of Dermatology, School of Basic and Medical Biosciences, King's College London, London, UK; Dignosis Ltd, London, UK., Guy A; The Robin Eady National Diagnostic Epidermolysis Bullosa Laboratory, Viapath, Guy's Hospital, London, UK., Aristodemou S; The Robin Eady National Diagnostic Epidermolysis Bullosa Laboratory, Viapath, Guy's Hospital, London, UK., McMillan JR; The Robin Eady National Diagnostic Epidermolysis Bullosa Laboratory, Viapath, Guy's Hospital, London, UK., Ozoemena L; The Robin Eady National Diagnostic Epidermolysis Bullosa Laboratory, Viapath, Guy's Hospital, London, UK., Mee J; Immunodermatology Laboratory, Viapath, St Thomas' Hospital, London, UK., Pramanik R; St John's Institute of Dermatology, School of Basic and Medical Biosciences, King's College London, London, UK., Saxena A; UK NIHR GSTFT/KCL Comprehensive Biomedical Research Centre, Guy's & St Thomas' NHS Foundation Trust, London, UK., Nuamah R; UK NIHR GSTFT/KCL Comprehensive Biomedical Research Centre, Guy's & St Thomas' NHS Foundation Trust, London, UK., de Rinaldis E; I&I Precision Immunology, Sanofi, Cambridge, Massachusetts., Serrano S; Clinical Trial Management Research Platform, NIHR Biomedical Research Centre, Guy's & St Thomas' NHS Foundation Trust, London, UK., Maurin C; Clinical Trial Management Research Platform, NIHR Biomedical Research Centre, Guy's & St Thomas' NHS Foundation Trust, London, UK., Martinez-Queipo M; St John's Institute of Dermatology, School of Basic and Medical Biosciences, King's College London, London, UK., Lwin SM; St John's Institute of Dermatology, School of Basic and Medical Biosciences, King's College London, London, UK., Ilic D; Stem Cell Laboratories, Guy's Assisted Conception Unit, Department of Women & Children's Health, Faculty of Life Sciences & Medicine, King's College London, London, UK., Martinez A; Department of Dermatology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Dazzi F; Department of Haematological Medicine, The Rayne Institute, King's College London, London, UK., Slaper-Cortenbach I; Cell Therapy Facility, Department of Clinical Pharmacy, University Medical Center Utrecht, Utrecht, The Netherlands., Westinga K; Cell Therapy Facility, Department of Clinical Pharmacy, University Medical Center Utrecht, Utrecht, The Netherlands., Zeddies S; Cell Therapy Facility, Department of Clinical Pharmacy, University Medical Center Utrecht, Utrecht, The Netherlands., van den Broek M; Cell Therapy Facility, Department of Clinical Pharmacy, University Medical Center Utrecht, Utrecht, The Netherlands., Onoufriadis A; St John's Institute of Dermatology, School of Basic and Medical Biosciences, King's College London, London, UK., Mellerio JE; St John's Institute of Dermatology, School of Basic and Medical Biosciences, King's College London, London, UK., McGrath JA; St John's Institute of Dermatology, School of Basic and Medical Biosciences, King's College London, London, UK. Electronic address: john.mcgrath@kcl.ac.uk.
Jazyk: angličtina
Zdroj: Journal of the American Academy of Dermatology [J Am Acad Dermatol] 2020 Aug; Vol. 83 (2), pp. 447-454. Date of Electronic Publication: 2019 Nov 28.
DOI: 10.1016/j.jaad.2019.11.038
Abstrakt: 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 × 10 6  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.
(Copyright © 2019 American Academy of Dermatology, Inc. Published by Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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