Advances and perspectives on cellular therapy in acquired bone marrow failure diseases  

作　　者：Xiao-Shen Sun Xin Liu Kai-Lin Xu Allshine Chen Witold B Rybka Jeffrey J Pu 

机构地区：[1]Penn State Hershey Cancer Institute, Penn State College of Medicine [2]the Key Laboratory of Transplantation Immunity, Department of Hematology, Affiliated Hospital of Xuzhou Medical College [3]Department of Medicine and Pathology, Penn State College of Medicine

出　　处：《World Journal of Hematology》2016年第1期31-36,共6页世界血液学杂志

基　　金：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;a Pennsylvania State University College of Medicine research grant to Pu JJ

摘　　要：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.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.

关 键 词：Bone marrow failure DISEASES APLASTIC anemia Cellular therapy Stem cell transplantation PAROXYSMAL NOCTURNAL HEMOGLOBINURIA MYELODYSPLASTIC syndrome 

分 类 号：R[医药卫生]