Journal
WORLD JOURNAL OF STEM CELLS
Volume 9, Issue 12, Pages 219-226Publisher
BAISHIDENG PUBLISHING GROUP INC
DOI: 10.4252/wjsc.v9.i12.219
Keywords
Hematopoiesis; Targeted therapies; Stem cells; Hematopoietic stem cell transplantation; Aplastic anemia; Mesenchymal stem cells
Categories
Funding
- National Center for Advancing Translational Sciences, National Institutes of Health [UL1TR001436, 1TL1TR001437]
- MACC Fund
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Acquired aplastic anemia (AA) is a bone marrow failure syndrome characterized by peripheral cytopenias and bone marrow hypoplasia. It is ultimately fatal with-out treatment, most commonly from infection or hemorrhage. Current treatments focus on suppressing immune-mediated destruction of bone marrow stem cells or replacing hematopoietic stem cells (HSCs) by transplantation. Our incomplete understanding of the pathogenesis of AA has limited development of targeted treatment options. Mesenchymal stem cells (MSCs) play a vital role in HSC proliferation; they also modulate immune responses and maintain an environment supportive of hematopoiesis. Some of the observed clinical mani-festations of AA can be explained by mesenchymal dysfunction. MSC infusions have been shown to be safe and may offer new approaches for the treatment of this disorder. Indeed, infusions of MSCs may help suppress auto-reactive, T-cell mediated HSC destruction and help restore an environment that supports hematopoiesis. Small pilot studies using MSCs as monotherapy or as adjuncts to HSC transplantation have been attempted as treatments for AA. Here we review the current understanding of the pathogenesis of AA and the function of MSCs, and suggest that MSCs should be a target for further research and clinical trials in this disorder.
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