Toxic Epidermal Necrolysis in Recessive Dystrophic Epidermolysis Bullosa following Bone Marrow Transplantation

Boull, Christina; Hylwa, Sara; Sajic, Dusan; Wagner, John E.; Tolar, Jakub; Hook, Kristen P.

doi:10.1016/j.jpeds.2016.02.037

Cited by 12 publications

(21 citation statements)

References 24 publications

(22 reference statements)

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“…Treatments for severe EB remain difficult, especially in terms of technology and cost effectiveness, although various epoch-making approaches have been reported (Uitto et al, 2018). Allogeneic stem cell transplantation has been performed in the US and a few other countries with remarkable results for RDEB and JEB; however, several issues remain to be solved, such as fostering close collaboration between professional dermatologists and experienced pediatric hematologists, preparing donors, and reducing costs (Boull et al, 2016;Geyer et al, 2015;Tolar and Wagner, 2013;Wagner et al, 2010). The local injection or intravenous infusion of allogeneic mesenchymal stromal cells has also been reported with promising results, although these mesenchymal stromal cells can disappear rapidly after infusion, which has the potential to render the therapy effective only for the short term (Conget et al, 2010;El-Darouti et al, 2016;Petrof et al, 2015).…”

Section: Discussionmentioning

confidence: 99%

“…Developing fundamental regenerative medical treatments is challenging for severe inherited cutaneous disorders including EB, because allogeneic cells are prone to elimination by the host's immune systems (Dixit et al, 2017), and the use of autologous cells is limited unless the patient's own gene abnormalities are corrected. Several cell therapies for EB have been reported, such as allogeneic stem cell transplantation, allogeneic mesenchymal stromal cell injection, allogeneic fibroblast injection, and gene-corrected keratinocyte sheet transplantation (Bauer et al, 2017;Boull et al, 2016;Conget et al, 2010;El-Darouti et al, 2016;Geyer et al, 2015;Hirsch et al, 2017;Mavilio et al, 2006;Petrof et al, 2015;Siprashvili et al, 2016;Tolar and Wagner, 2013;Uitto et al, 2018;Wagner et al, 2010;Wong et al, 2008). However, these novel therapies should be carefully approached because of sublethal premedication and unexpected risks of gene modification, which are potential hurdles (Boull et al, 2016;Tolar and Wagner, 2013;Uitto et al, 2018;Wagner et al, 2010).…”

Section: Introductionmentioning

confidence: 99%

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Cultured Epidermal Autografts from Clinically Revertant Skin as a Potential Wound Treatment for Recessive Dystrophic Epidermolysis Bullosa

Matsumura

Fujita

Shinkuma

et al. 2019

Journal of Investigative Dermatology

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Inherited skin disorders have been reported recently to have sporadic normal-looking areas, where a portion of the keratinocytes have recovered from causative gene mutations (revertant mosaicism). We observed a case of recessive dystrophic epidermolysis bullosa treated with cultured epidermal autografts (CEAs), whose CEAgrafted site remained epithelized for 16 years. We proved that the CEA product and the grafted area included cells with revertant mosaicism. Based on these findings, we conducted an investigator-initiated clinical trial of CEAs from clinically revertant skin for recessive dystrophic epidermolysis bullosa. The donor sites were analyzed by genetic analysis, immunofluorescence, electron microscopy, and quantification of the reverted mRNA with deep sequencing. The primary endpoint was the ulcer epithelization rate per patient at 4 weeks after the last CEA application. Three patients with recessive dystrophic epidermolysis bullosa with 8 ulcers were enrolled, and the epithelization rate for each patient at the primary endpoint was 87.7%, 100%, and 57.0%, respectively. The clinical effects were found to persist for at least 76 weeks after CEA transplantation. One of the three patients had apparent revertant mosaicism in the donor skin and in the post-transplanted area. CEAs from clinically normal skin are a potentially well-tolerated treatment for recessive dystrophic epidermolysis bullosa.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Cultured Epidermal Autografts from Clinically Revertant Skin as a Potential Wound Treatment for Recessive Dystrophic Epidermolysis Bullosa

Matsumura

Fujita

Shinkuma

et al. 2019

Journal of Investigative Dermatology

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“…Therefore, both TEN and GVHD share partially similar pathogenic mechanisms; hence, accurate clinical observations and the overall disease course are important to differentiate between these two entities 6 . A recent study 7 showed histological differences between TEN and GVHD (i.e.…”

Section: Discussionmentioning

confidence: 99%

Case of toxic epidermal necrolysis occurring after bone marrow transplantation accompanied by engraftment failure

Asai

Watanabe

Mukaijo

et al. 2019

The Journal of Dermatology

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Toxic epidermal necrolysis (TEN) is a rare condition, causing life‐threatening adverse cutaneous reactions. TEN occurrence after bone marrow transplantation (BMT) is a well‐known phenomenon; however, to date, only a few cases have been reported in the published work. Here, we describe the case of a 53‐year‐old woman who experienced TEN after undergoing allogenic BMT for malignant lymphoma. Skin erosion spread across a maximum of 70% of the body surface area and severe mucosal lesions developed. Steroid pulse therapy, plasma apheresis and immunoglobulin therapy were administrated, which resulted in the complete resolution of TEN. However, she developed hemophagocytic lymphohistiocytosis and died 38 days after BMT, owing to rupture of the lower digestive tract complicated by multi‐organ failure. In our case, engraftment failure occurred, and the peripheral white blood cell count was less than 100/μL during the TEN course, suggesting that the presence of only a few immune cells could cause TEN. Our findings showed that high mortality rates and widespread skin erosion could be regarded as the most important characteristics of TEN occurring after BMT.

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“…The successful use of hematopoietic cell transplantation (HCT) has previously been shown to treat pediatric patients with RDEB [ 70 , 71 ], an intractable genetic blistering skin disease caused by mutations to the COL7A1 gene that deactivated the production of functional type VII collagen protein (C7) essential for skin integrity [ 72 , 73 ]. Several lines of evidence have demonstrated that MSCs enhanced therapeutic effects of HCT graft on pediatric RDEB partly due to their intrinsic immunomodulatory, trophic properties and restorative effects on C7 production [ 71 , 74 – 78 ].…”

Section: Applications Of Mscs In Cutaneous Diseasesmentioning

confidence: 99%

An Update on the Potential of Mesenchymal Stem Cell Therapy for Cutaneous Diseases

Yang

et al. 2021

Stem Cells International

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Mesenchymal stem or stromal cells (MSCs) are nonhematopoietic postnatal stem cells with self-renewal, multipotent differentiation, and potent immunomodulatory and anti-inflammatory capabilities, thus playing an important role in tissue repair and regeneration. Numerous clinical and preclinical studies have demonstrated the potential application of MSCs in the treatment of tissue inflammation and immune diseases, including inflammatory skin diseases. Therefore, understanding the biological and immunological characteristics of MSCs is important to standardize and optimize MSC-based regenerative therapy. In this review, we highlight the mechanisms underlying MSC-mediated immunomodulation and tissue repair/regeneration and present the latest development of MSC-based clinical trials on cutaneous diseases.

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Toxic Epidermal Necrolysis in Recessive Dystrophic Epidermolysis Bullosa following Bone Marrow Transplantation

Cited by 12 publications

References 24 publications

Cultured Epidermal Autografts from Clinically Revertant Skin as a Potential Wound Treatment for Recessive Dystrophic Epidermolysis Bullosa

Cultured Epidermal Autografts from Clinically Revertant Skin as a Potential Wound Treatment for Recessive Dystrophic Epidermolysis Bullosa

Case of toxic epidermal necrolysis occurring after bone marrow transplantation accompanied by engraftment failure

An Update on the Potential of Mesenchymal Stem Cell Therapy for Cutaneous Diseases

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