Published online Feb 6, 2016. doi: 10.5315/wjh.v5.i1.31
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
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.