ABCB5
ABCB5
inflammation
mesenchymal stromal cells
recessive dystrophic epidermolysis bullosa
wound healing
Journal
Cytotherapy
ISSN: 1477-2566
Titre abrégé: Cytotherapy
Pays: England
ID NLM: 100895309
Informations de publication
Date de publication:
07 2023
07 2023
Historique:
received:
23
11
2022
revised:
30
01
2023
accepted:
31
01
2023
pmc-release:
01
07
2024
medline:
9
6
2023
pubmed:
4
3
2023
entrez:
3
3
2023
Statut:
ppublish
Résumé
Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary, rare, devastating and life-threatening skin fragility disorder with a high unmet medical need. In a recent international, single-arm clinical trial, treatment of 16 patients (aged 6-36 years) with three intravenous infusions of 2 × 10 Documentary photographs of the affected body regions taken on days 0, 17, 35 and at 12 weeks were evaluated regarding proportion, temporal course and durability of wound closure as well as development of new wounds. Of 168 baseline wounds in 14 patients, 109 (64.9%) wounds had closed at week 12, of which 63.3% (69 wounds) had closed already by day 35 or day 17. Conversely, 74.2% of the baseline wounds that had closed by day 17 or day 35 remained closed until week 12. First-closure ratio within 12 weeks was 75.6%. The median rate of newly developing wounds decreased significantly (P = 0.001) by 79.3%. Comparison of the findings with published data from placebo arms and vehicle-treated wounds in controlled clinical trials suggests potential capability of ABCB5 Clinicaltrials.gov NCT03529877; EudraCT 2018-001009-98.
Sections du résumé
BACKGROUND AND AIMS
Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary, rare, devastating and life-threatening skin fragility disorder with a high unmet medical need. In a recent international, single-arm clinical trial, treatment of 16 patients (aged 6-36 years) with three intravenous infusions of 2 × 10
METHODS
Documentary photographs of the affected body regions taken on days 0, 17, 35 and at 12 weeks were evaluated regarding proportion, temporal course and durability of wound closure as well as development of new wounds.
RESULTS
Of 168 baseline wounds in 14 patients, 109 (64.9%) wounds had closed at week 12, of which 63.3% (69 wounds) had closed already by day 35 or day 17. Conversely, 74.2% of the baseline wounds that had closed by day 17 or day 35 remained closed until week 12. First-closure ratio within 12 weeks was 75.6%. The median rate of newly developing wounds decreased significantly (P = 0.001) by 79.3%.
CONCLUSIONS
Comparison of the findings with published data from placebo arms and vehicle-treated wounds in controlled clinical trials suggests potential capability of ABCB5
TRIAL REGISTRATION
Clinicaltrials.gov NCT03529877; EudraCT 2018-001009-98.
Identifiants
pubmed: 36868990
pii: S1465-3249(23)00036-1
doi: 10.1016/j.jcyt.2023.01.015
pmc: PMC10257763
mid: NIHMS1872711
pii:
doi:
Substances chimiques
Collagen Type VII
0
ABCB5 protein, human
0
ATP Binding Cassette Transporter, Subfamily B
0
Banques de données
ClinicalTrials.gov
['NCT03529877']
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
782-788Subventions
Organisme : NEI NIH HHS
ID : R24 EY028767
Pays : United States
Organisme : NIA NIH HHS
ID : P01 AG071463
Pays : United States
Organisme : NIAMS NIH HHS
ID : R01 AR063070
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL161087
Pays : United States
Organisme : NEI NIH HHS
ID : R01 EY025794
Pays : United States
Organisme : RRD VA
ID : I01 RX000989
Pays : United States
Commentaires et corrections
Type : ErratumIn
Informations de copyright
Copyright © 2023 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.
Déclaration de conflit d'intérêts
Declaration of Competing Interest NYF and MHF are inventors or co-inventors of U.S. and international patents assigned to Brigham and Women's Hospital, Boston Children's Hospital, and/or the VA Boston Healthcare System, Boston, Massachusetts, USA, licensed to TICEBA GmbH, Heidelberg, Germany, and RHEACELL GmbH & Co. KG, Heidelberg, Germany. MHF serves as scientific advisor and holds stock in TICEBA GmbH and RHEACELL GmbH & Co. KG. DK and JT serve as scientific advisors to RHEACELL. KD, CD and SF are employees of RHEACELL. ENR is employee of TICEBA. CG is CEO, and MAK is CSO of TICEBA and RHEACELL. JAM declares that he has no conflicts of interest.
Références
J Clin Med. 2021 Dec 13;10(24):
pubmed: 34945131
J Am Acad Dermatol. 2021 Nov;85(5):1161-1167
pubmed: 32199895
N Engl J Med. 2022 Dec 15;387(24):2211-2219
pubmed: 36516090
JCI Insight. 2021 Jan 25;6(2):
pubmed: 33491668
J Am Acad Dermatol. 2020 Aug;83(2):447-454
pubmed: 31786163
Dermatopathology (Basel). 2020 Sep 14;7(2):41-47
pubmed: 32937752
JCI Insight. 2019 Oct 3;4(19):
pubmed: 31578311
Stem Cell Res Ther. 2022 Sep 5;13(1):455
pubmed: 36064604
J Am Acad Dermatol. 2021 Nov;85(5):1296-1298
pubmed: 32896597
PLoS One. 2014 Aug 13;9(8):e104479
pubmed: 25119884
Stem Cells. 2016 Sep;34(9):2393-406
pubmed: 27299700
Stem Cells. 2019 Aug;37(8):1057-1074
pubmed: 31002437
J Biol Chem. 2003 Nov 21;278(47):47156-65
pubmed: 12960149
Pediatr Dermatol. 2021 May;38(3):704-706
pubmed: 33749033
J Am Acad Dermatol. 2014 Jan;70(1):89-97.e1-13
pubmed: 24355263
Dermatol Ther. 2016 Mar-Apr;29(2):96-100
pubmed: 26439431
Br J Dermatol. 2013 Nov;169(5):1025-33
pubmed: 24032424
J Clin Invest. 2017 Aug 1;127(8):3028-3038
pubmed: 28691931
Br J Dermatol. 2023 Jan 23;188(1):12-21
pubmed: 36689495
Clin Exp Rheumatol. 2016 Sep-Oct;34(5):961-962
pubmed: 26885873
JID Innov. 2022 Jan;2(1):
pubmed: 34870260
J Invest Dermatol. 2015 Sep;135(9):2319-2321
pubmed: 25905587
Exp Dermatol. 2021 Dec;30(12):1724-1733
pubmed: 34142388
Orphanet J Rare Dis. 2020 Jan 3;15(1):1
pubmed: 31900176
JCI Insight. 2021 Nov 22;6(22):
pubmed: 34665781
Am J Pathol. 2017 Jul;187(7):1445-1453
pubmed: 28460207
Orphanet J Rare Dis. 2020 Jul 21;15(1):190
pubmed: 32693833
Orphanet J Rare Dis. 2020 Jun 23;15(1):158
pubmed: 32576219
Cytotherapy. 2019 May;21(5):546-560
pubmed: 30878384
J Dermatol Sci. 2020 Dec;100(3):209-216
pubmed: 33143962
Pediatr Dermatol. 2015 Jan-Feb;32(1):41-52
pubmed: 24650374
Stem Cells. 2021 Jul;39(7):897-903
pubmed: 33609408
Cytotherapy. 2021 Feb;23(2):165-175
pubmed: 33011075
Int J Mol Sci. 2019 Nov 14;20(22):
pubmed: 31739489
Trials. 2019 Jun 11;20(1):350
pubmed: 31186047
Stem Cell Res Ther. 2020 Nov 16;11(1):482
pubmed: 33198791
Orphanet J Rare Dis. 2016 Mar 25;11:31
pubmed: 27015660
Nat Rev Dis Primers. 2020 Sep 24;6(1):78
pubmed: 32973163
J Invest Dermatol. 2022 Jun;142(6):1725-1736.e10
pubmed: 34808236