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World J Stem Cells. Dec 26, 2017; 9(12): 219-226
Published online Dec 26, 2017. doi: 10.4252/wjsc.v9.i12.219
Yin and Yang of mesenchymal stem cells and aplastic anemia
Larisa Broglie, David Margolis, Jeffrey A Medin
Larisa Broglie, David Margolis, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI 53226, United States
Jeffrey A Medin, Departments of Pediatrics and Biochemistry, Medical College of Wisconsin, Milwaukee, WI 53226, United States
Author contributions: All authors contributed to the conception and design of the study; Broglie L performed the literature review and wrote the initial draft; Margolis D and Medin JA were involved in critical revision and editing; all authors approved the final version of the manuscript.
Supported by National Center for Advancing Translational Sciences, National Institutes of Health, through Grant Nos. UL1TR001436 and 1TL1TR001437 (to Broglie L); and MACC Fund (to Margolis D and Medin JA). Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the NIH.
Conflict-of-interest statement: The authors declare that they have no conflicts of interest with this work.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Correspondence to: Larisa Broglie, MD, Academic Fellow, Instructor, Department of Pediatrics, Medical College of Wisconsin, 8701 Watertown Plank Rd, MFRC 3018, Milwaukee, WI 53226, United States. lbroglie@mcw.edu
Telephone: +1-414-2664122 Fax: +1-414-9556543
Received: August 1, 2017
Peer-review started: August 3, 2017
First decision: September 4, 2017
Revised: September 14, 2017
Accepted: October 17, 2017
Article in press: October 17, 2017
Published online: December 26, 2017
Processing time: 146 Days and 7.1 Hours
Abstract

Acquired aplastic anemia (AA) is a bone marrow failure syndrome characterized by peripheral cytopenias and bone marrow hypoplasia. It is ultimately fatal without 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 manifestations 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.

Keywords: Hematopoiesis; Targeted therapies; Stem cells; Hematopoietic stem cell transplantation; Aplastic anemia; Mesenchymal stem cells

Core tip: Acquired aplastic anemia (AA) is a bone marrow failure syndrome characterized by peripheral cytopenia and bone marrow hypoplasia and is ultimately fatal without treatment. Our incomplete understanding of the pathogenesis of AA has limited development of targeted treatment options. Here we review the current understanding of the pathogenesis of AA and the function of mesenchymal stem cells (MSCs), and suggest that MSCs should be a target for further trials in AA.