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 Ht 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 Ht 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

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

18 Scopus citations

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 (US)
Pages (from-to)	765-769.e1
Journal	Journal of Pediatrics
Volume	167
Issue number	3
DOIs	https://doi.org/10.1016/j.jpeds.2015.05.051
State	Published - Sep 1 2015
Externally published	Yes

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health

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10.1016/j.jpeds.2015.05.051

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Geyer, M. B., Radhakrishnan, K., Giller, R., Umegaki, N., Harel, S., Kiuru, M. H., 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), 765-769.e1. https://doi.org/10.1016/j.jpeds.2015.05.051

Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa. / Geyer, Mark B.; Radhakrishnan, Kavita; Giller, Roger; Umegaki, Noriko; Harel, Sivan; Kiuru, Maija Ht; Morel, Kimberly D.; Leboeuf, Nicole; Kandel, Jessica; Bruckner, Anna; Fabricatore, Sandra; Chen, Mei; Woodley, David; McGrath, John; Baxter-Lowe, Leeann; Uitto, Jouni; Christiano, Angela M.; Cairo, Mitchell S.

In: Journal of Pediatrics, Vol. 167, No. 3, 01.09.2015, p. 765-769.e1.

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

Geyer, MB, Radhakrishnan, K, Giller, R, Umegaki, N, Harel, S, Kiuru, MH, 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. 765-769.e1. https://doi.org/10.1016/j.jpeds.2015.05.051

Geyer, Mark B. ; Radhakrishnan, Kavita ; Giller, Roger ; Umegaki, Noriko ; Harel, Sivan ; Kiuru, Maija Ht ; Morel, Kimberly D. ; Leboeuf, Nicole ; Kandel, Jessica ; Bruckner, Anna ; Fabricatore, Sandra ; Chen, Mei ; Woodley, David ; McGrath, John ; Baxter-Lowe, Leeann ; Uitto, Jouni ; Christiano, Angela M. ; Cairo, Mitchell S. / Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa. In: Journal of Pediatrics. 2015 ; Vol. 167, No. 3. pp. 765-769.e1.

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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.",

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

Abstract

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