scholarly journals Differential Toxicity Profile of Busulfan and Treosulfan on Endothelial Cells - Relevance to Hematopoietic Stem Cell Transplantation

2019 ◽  
Vol 25 (3) ◽  
pp. S190-S191
Author(s):  
Balaji Balakrishnan ◽  
Raveen Stephen Stallon Illangeswaran ◽  
Bharathi Murugan Rajamani ◽  
Arun kumar A ◽  
Ezhilpavai Mohanan ◽  
...  
Keyword(s):  
Endothelial Cells ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Toxicity Profile ◽  
Hematopoietic Stem ◽  
Differential Toxicity

scholarly journals β-Galactosylceramidase Deficiency Causes Bone Marrow Vascular Defects in an Animal Model of Krabbe Disease

2019 ◽  
Vol 21 (1) ◽  
pp. 251
Author(s):  
Mirella Belleri ◽  
Daniela Coltrini ◽  
Marco Righi ◽  
Cosetta Ravelli ◽  
Sara Taranto ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Endothelial Cells ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Krabbe Disease ◽  
Microscopy Imaging ◽  
Hematopoietic Stem

Krabbe disease (KD) is an autosomal recessive sphingolipidosis caused by the deficiency of the lysosomal hydrolase β-galactosylceramidase (GALC). Oligodendroglia degeneration and demyelination of the nervous system lead to neurological dysfunctions which are usually lethal by two years of age. At present, the only clinical treatment with any proven efficacy is hematopoietic stem-cell transplantation, which is more effective when administered in the neonatal period to presymptomatic recipients. Bone marrow (BM) sinusoidal endothelial cells (SECs) play a pivotal role in stem cell engraftment and reconstitution of hematopoiesis. Previous observations had shown significant alterations of microvascular endothelial cells in the brain of KD patients and in Galc mutant twitcher mice, an authentic model of the disease. In the present study, we investigated the vascular component of the BM in the femurs of symptomatic homozygous twitcher mice at postnatal day P36. Histological, immunohistochemical, and two-photon microscopy imaging analyses revealed the presence of significant alterations of the diaphyseal BM vasculature, characterized by enlarged, discontinuous, and hemorrhagic SECs that express the endothelial marker vascular endothelial growth factor receptor-2 (VEGFR2) but lack platelet/endothelial cell adhesion molecule-1 (CD31) expression. In addition, computer-aided image analysis indicates that twitcher CD31−/VEGFR2+ SECs show a significant increase in lumen size and in the number and size of endothelial gaps compared to BM SECs of wild type littermates. These results suggest that morphofunctional defects in the BM vascular niche may contribute to the limited therapeutic efficacy of hematopoietic stem-cell transplantation in KD patients at symptomatic stages of the disease.

Caregiver perspectives on psychosocial care in pediatric hematopoietic stem cell transplantation (HCT).

2020 ◽  
Vol 8 (1) ◽  
pp. 67-78 ◽  
Author(s):  
Anne E. Kazak ◽  
Avi Madan Swain ◽  
Ahna L. H. Pai ◽  
Kimberly Canter ◽  
Olivia Carlson ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Psychosocial Care ◽  
Hematopoietic Stem
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