Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa

Mark B Geyer; Kavita Radhakrishnan; Roger Giller; Noriko Umegaki; Sivan Harel; Maija Kiuru; Kimberly D Morel; Nicole LeBoeuf; Jessica Kandel; Anna Bruckner; Sandra Fabricatore; Mei Chen; David Woodley; John McGrath; LeeAnn Baxter-Lowe; Jouni Uitto; Angela M Christiano; Mitchell S Cairo

doi:10.1016/j.jpeds.2015.05.051

Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa

Mark B Geyer, Kavita Radhakrishnan, Roger Giller, Noriko Umegaki, Sivan Harel, Maija Kiuru, Kimberly D Morel, Nicole LeBoeuf, Jessica Kandel, Anna Bruckner, Sandra Fabricatore, Mei Chen, David Woodley, John McGrath, LeeAnn Baxter-Lowe, Jouni Uitto, Angela M Christiano, Mitchell S Cairo

Dermatology

Research output: Contribution to journal › Article › peer-review

26 Citations (Scopus)

Abstract

Recessive dystrophic epidermolysis bullosa is a severe, incurable, inherited blistering disease caused by COL7A1 mutations. Emerging evidence suggests hematopoietic progenitor cells (HPCs) can be reprogrammed into skin; HPC-derived cells can restore COL7 expression in COL7-deficient mice. We report two children with recessive dystrophic epidermolysis bullosa treated with reduced-toxicity conditioning and HLA-matched HPC transplantation.

Original language	English
Pages (from-to)	765-9.e1
Journal	Journal of pediatrics
Volume	167
Issue number	3
DOIs	https://doi.org/10.1016/j.jpeds.2015.05.051
Publication status	Published - Sept 2015

Keywords

Antibodies, Monoclonal, Humanized
Busulfan
Child
Collagen Type VII
Epidermolysis Bullosa Dystrophica
Epithelial Cells
Fibroblasts
Hematopoietic Stem Cell Transplantation
Humans
Immunosuppressive Agents
Infant
Male
Mutation
Myeloablative Agonists
RNA, Messenger
Transplantation Conditioning
Transplantation, Homologous
Vidarabine

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Geyer, M. B., Radhakrishnan, K., Giller, R., Umegaki, N., Harel, S., Kiuru, M., Morel, K. D., LeBoeuf, N., Kandel, J., Bruckner, A., Fabricatore, S., Chen, M., Woodley, D., McGrath, J., Baxter-Lowe, L., Uitto, J., Christiano, A. M., & Cairo, M. S. (2015). Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa. Journal of pediatrics, 167(3), 765-9.e1. https://doi.org/10.1016/j.jpeds.2015.05.051

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title = "Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa",

abstract = "Recessive dystrophic epidermolysis bullosa is a severe, incurable, inherited blistering disease caused by COL7A1 mutations. Emerging evidence suggests hematopoietic progenitor cells (HPCs) can be reprogrammed into skin; HPC-derived cells can restore COL7 expression in COL7-deficient mice. We report two children with recessive dystrophic epidermolysis bullosa treated with reduced-toxicity conditioning and HLA-matched HPC transplantation.",

keywords = "Antibodies, Monoclonal, Humanized, Busulfan, Child, Collagen Type VII, Epidermolysis Bullosa Dystrophica, Epithelial Cells, Fibroblasts, Hematopoietic Stem Cell Transplantation, Humans, Immunosuppressive Agents, Infant, Male, Mutation, Myeloablative Agonists, RNA, Messenger, Transplantation Conditioning, Transplantation, Homologous, Vidarabine",

author = "Geyer, {Mark B} and Kavita Radhakrishnan and Roger Giller and Noriko Umegaki and Sivan Harel and Maija Kiuru and Morel, {Kimberly D} and Nicole LeBoeuf and Jessica Kandel and Anna Bruckner and Sandra Fabricatore and Mei Chen and David Woodley and John McGrath and LeeAnn Baxter-Lowe and Jouni Uitto and Christiano, {Angela M} and Cairo, {Mitchell S}",

year = "2015",

month = sep,

doi = "10.1016/j.jpeds.2015.05.051",

language = "English",

volume = "167",

pages = "765--9.e1",

journal = "Journal of pediatrics",

issn = "0022-3476",

publisher = "Mosby Inc.",

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Geyer, MB, Radhakrishnan, K, Giller, R, Umegaki, N, Harel, S, Kiuru, M, Morel, KD, LeBoeuf, N, Kandel, J, Bruckner, A, Fabricatore, S, Chen, M, Woodley, D, McGrath, J, Baxter-Lowe, L, Uitto, J, Christiano, AM & Cairo, MS 2015, 'Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa', Journal of pediatrics, vol. 167, no. 3, pp. 765-9.e1. https://doi.org/10.1016/j.jpeds.2015.05.051

TY - JOUR

T1 - Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa

AU - Geyer, Mark B

AU - Radhakrishnan, Kavita

AU - Giller, Roger

AU - Umegaki, Noriko

AU - Harel, Sivan

AU - Kiuru, Maija

AU - Morel, Kimberly D

AU - LeBoeuf, Nicole

AU - Kandel, Jessica

AU - Bruckner, Anna

AU - Fabricatore, Sandra

AU - Chen, Mei

AU - Woodley, David

AU - McGrath, John

AU - Baxter-Lowe, LeeAnn

AU - Uitto, Jouni

AU - Christiano, Angela M

AU - Cairo, Mitchell S

PY - 2015/9

Y1 - 2015/9

N2 - Recessive dystrophic epidermolysis bullosa is a severe, incurable, inherited blistering disease caused by COL7A1 mutations. Emerging evidence suggests hematopoietic progenitor cells (HPCs) can be reprogrammed into skin; HPC-derived cells can restore COL7 expression in COL7-deficient mice. We report two children with recessive dystrophic epidermolysis bullosa treated with reduced-toxicity conditioning and HLA-matched HPC transplantation.

AB - Recessive dystrophic epidermolysis bullosa is a severe, incurable, inherited blistering disease caused by COL7A1 mutations. Emerging evidence suggests hematopoietic progenitor cells (HPCs) can be reprogrammed into skin; HPC-derived cells can restore COL7 expression in COL7-deficient mice. We report two children with recessive dystrophic epidermolysis bullosa treated with reduced-toxicity conditioning and HLA-matched HPC transplantation.

KW - Antibodies, Monoclonal, Humanized

KW - Busulfan

KW - Child

KW - Collagen Type VII

KW - Epidermolysis Bullosa Dystrophica

KW - Epithelial Cells

KW - Fibroblasts

KW - Hematopoietic Stem Cell Transplantation

KW - Humans

KW - Immunosuppressive Agents

KW - Infant

KW - Male

KW - Mutation

KW - Myeloablative Agonists

KW - RNA, Messenger

KW - Transplantation Conditioning

KW - Transplantation, Homologous

KW - Vidarabine

U2 - 10.1016/j.jpeds.2015.05.051

DO - 10.1016/j.jpeds.2015.05.051

M3 - Article

C2 - 26148662

SN - 0022-3476

VL - 167

SP - 765-9.e1

JO - Journal of pediatrics

JF - Journal of pediatrics

IS - 3

ER -

Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa

Abstract

Keywords

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