Advanced

Transplantation of Haploidentical TcRaß-Depleted Hematopoietic Cells Allows for Optimal Timing and Sustained Correction of the Metabolic Defect in Children With Infantile Osteopetrosis

Pronk, Kees-Jan LU ; Turkiewicz, Dominik; Vult von Steyern, Kristina LU ; Ehinger, Mats LU ; Dykes, Josefina LU and Toporski, Jacek LU (2016) In Journal of Bone and Mineral Research p.1-4
Abstract

In osteopetrosis, osteoclast dysfunction can lead to deafness, blindness, bone marrow failure, and death. Hematopoietic cell transplantation (HCT) is currently the only curative treatment, but outcome remains disappointing. Although a rapid progression toward HCT is detrimental to prevent further progress of disease manifestations, 70% of cases lack an HLA-matched sibling and require alternative stem cell sources. We present two cases of osteopetrosis that successfully received an HCT with haploidentical TcRαβ-depleted cells from one of the parents. These cases showed no further disease progression, had restoration of functional osteoclasts, and illustrate this approach to enable prompt HCT with ready available parental donors and rapid... (More)

In osteopetrosis, osteoclast dysfunction can lead to deafness, blindness, bone marrow failure, and death. Hematopoietic cell transplantation (HCT) is currently the only curative treatment, but outcome remains disappointing. Although a rapid progression toward HCT is detrimental to prevent further progress of disease manifestations, 70% of cases lack an HLA-matched sibling and require alternative stem cell sources. We present two cases of osteopetrosis that successfully received an HCT with haploidentical TcRαβ-depleted cells from one of the parents. These cases showed no further disease progression, had restoration of functional osteoclasts, and illustrate this approach to enable prompt HCT with ready available parental donors and rapid and sustained hematological, including osteoclast, recovery.

(Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
in press
subject
keywords
HAPLOIDENTICAL, HEMATOPOIETIC CELL TRANSPLANTATION, OSTEOPETROSIS, TcRαβ-DEPLETION
in
Journal of Bone and Mineral Research
pages
1 - 4
publisher
AMBMR
external identifiers
  • Scopus:84987681409
ISSN
0884-0431
DOI
10.1002/jbmr.2921
language
English
LU publication?
yes
id
579ca972-de2c-4d42-856c-f0e6fbbe783b
date added to LUP
2016-10-07 13:31:57
date last changed
2016-10-20 10:59:35
@misc{579ca972-de2c-4d42-856c-f0e6fbbe783b,
  abstract     = {<p>In osteopetrosis, osteoclast dysfunction can lead to deafness, blindness, bone marrow failure, and death. Hematopoietic cell transplantation (HCT) is currently the only curative treatment, but outcome remains disappointing. Although a rapid progression toward HCT is detrimental to prevent further progress of disease manifestations, 70% of cases lack an HLA-matched sibling and require alternative stem cell sources. We present two cases of osteopetrosis that successfully received an HCT with haploidentical TcRαβ-depleted cells from one of the parents. These cases showed no further disease progression, had restoration of functional osteoclasts, and illustrate this approach to enable prompt HCT with ready available parental donors and rapid and sustained hematological, including osteoclast, recovery.</p>},
  author       = {Pronk, Kees-Jan and Turkiewicz, Dominik and Vult von Steyern, Kristina and Ehinger, Mats and Dykes, Josefina and Toporski, Jacek},
  issn         = {0884-0431},
  keyword      = {HAPLOIDENTICAL,HEMATOPOIETIC CELL TRANSPLANTATION,OSTEOPETROSIS,TcRαβ-DEPLETION},
  language     = {eng},
  pages        = {1--4},
  publisher    = {ARRAY(0xbc312d0)},
  series       = {Journal of Bone and Mineral Research},
  title        = {Transplantation of Haploidentical TcRaß-Depleted Hematopoietic Cells Allows for Optimal Timing and Sustained Correction of the Metabolic Defect in Children With Infantile Osteopetrosis},
  url          = {http://dx.doi.org/10.1002/jbmr.2921},
  year         = {2016},
}