Sun XS, Liu X, Xu KL, Chen A, Rybka WB, Pu JJ. Advances and perspectives on cellular therapy in acquired bone marrow failure diseases. World J Hematol 2016; 5(1): 31-36 [DOI: 10.5315/wjh.v5.i1.31]
Corresponding Author of This Article
Jeffrey J Pu, MD, PhD, Penn State Hershey Cancer Institute, Penn State College of Medicine, H064, PO Box 850, 500 University Drive, Hershey, PA 17033, United States. jeffreypu@hmc.psu.edu
Research Domain of This Article
Hematology
Article-Type of This Article
Minireviews
Open-Access Policy of This Article
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/
World J Hematol. Feb 6, 2016; 5(1): 31-36 Published online Feb 6, 2016. doi: 10.5315/wjh.v5.i1.31
Advances and perspectives on cellular therapy in acquired bone marrow failure diseases
Xiao-Shen Sun, Xin Liu, Kai-Lin Xu, Allshine Chen, Witold B Rybka, Jeffrey J Pu
Xiao-Shen Sun, Xin Liu, Allshine Chen, Witold B Rybka, Jeffrey J Pu, Penn State Hershey Cancer Institute, Penn State College of Medicine, Hershey, PA 17033, United States
Kai-Lin Xu, the Key Laboratory of Transplantation Immunity, Department of Hematology, Affiliated Hospital of Xuzhou Medical College, Xuzhou 221000, Jiangsu Province, China
Witold B Rybka, Jeffrey J Pu, Department of Medicine and Pathology, Penn State College of Medicine, Hershey, PA 17033, United States
Author contributions: Pu JJ outlined and designed the review; Sun XS and Chen A wrote the manuscript and generated the table and figure; Liu X and Pu JJ contributed to the writing of the manuscript; Liu X, Xu KL, Rybka WB and Pu JJ reviewed and edited the manuscript.
Supported by AA and MDSIF research grant to Pu JJ, No. 146818; American Cancer Society grant to Pu JJ, No. 124171-IRG-13-043-02; and a Pennsylvania State University College of Medicine research grant to Pu JJ.
Conflict-of-interest statement: The authors have no conflict–of-interest in this publication.
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: Jeffrey J Pu, MD, PhD, Penn State Hershey Cancer Institute, Penn State College of Medicine, H064, PO Box 850, 500 University Drive, Hershey, PA 17033, United States. jeffreypu@hmc.psu.edu
Telephone: +1-717-5314944 Fax: +1-717-5310647
Received: August 11, 2015 Peer-review started: August 13, 2015 First decision: September 17, 2015 Revised: December 12, 2015 Accepted: January 5, 2016 Article in press: January 7, 2016 Published online: February 6, 2016 Processing time: 169 Days and 17.7 Hours
Abstract
Acquired bone marrow failure diseases (ABMFD) are a class of hematopoietic stem cell diseases with a commonality of non-inherited disruption of hematopoiesis that results in pancytopenia. ABMFDs also are a group of heterogeneous diseases with different etiologies and treatment options. The three most common ABMFDs are aplastic anemia, myelodysplastic syndrome, and paroxysmal nocturnal hemoglobinuria. Stem cell transplantation is the only treatment that can cure these diseases. However, due to high therapy-related mortality, stem cell transplantation has rarely been used as a first line treatment in treating ABMFD. With the advance of personalized medicine and precision medicine, various novel cellular therapy strategies are in trial to increase the efficiency and efficacy of ABMFD treatment. This article aims to review current available stem cell transplantation protocols and promising cellular therapy research in treating ABMFD.
Core tip: Stem cell transplantation is the only method can cure acquired bone marrow failure diseases (ABMFD). However, due to the high mortality rate of stem cell transplantation itself, this method is not usually used as the first line treatment for ABMFD. With the advance of current cellular therapy technology, it is becoming possible to cure ABMFD without significant treatment related complications.
