TY - JOUR
T1 - Primary myelofibrosis and its targeted therapy
AU - Shantzer, Lindsey
AU - Berger, Kristin
AU - Pu, Jeffrey J.
N1 - Funding Information:
The authors thank the patients and their family for contributing invaluable knowledge and experience for this study and also would like to thank Dr. Jerry Spivak for providing sincere comments on this manuscript. This study was supported by: AA&MDSIF research grant to JJP (146818), American Cancer Society grant to JJP (124171-IRG-13-043-02), and A Pennsylvania State University College of Medicine research grant to JJP.
Publisher Copyright:
© 2016, Springer-Verlag Berlin Heidelberg.
PY - 2017/4/1
Y1 - 2017/4/1
N2 - Primary myelofibrosis is a unique entity among BCR-ABL-negative myeloproliferative diseases, manifesting as bone marrow fibrosis and pancytopenia. Considerable evidence indicates that genetic and epigenetic abnormalities can result in defective clonal hematopoietic stem cell proliferation in addition to bone marrow microenvironment alteration. The “bad seeds in bad soil” theory illustrates the orchestrating efforts of hematopoietic stem cells, stromal cells, and their surrounding signaling molecules in myelofibrosis progression and malignancy transformation, though the exact mechanism of myelofibrosis is still not clear. This study reviews current concepts and questions regarding the pathogenesis of primary myelofibrosis and discusses the emerging targeted therapy aimed at restoring normal bone marrow environment and halting bone marrow fibrotic deterioration.
AB - Primary myelofibrosis is a unique entity among BCR-ABL-negative myeloproliferative diseases, manifesting as bone marrow fibrosis and pancytopenia. Considerable evidence indicates that genetic and epigenetic abnormalities can result in defective clonal hematopoietic stem cell proliferation in addition to bone marrow microenvironment alteration. The “bad seeds in bad soil” theory illustrates the orchestrating efforts of hematopoietic stem cells, stromal cells, and their surrounding signaling molecules in myelofibrosis progression and malignancy transformation, though the exact mechanism of myelofibrosis is still not clear. This study reviews current concepts and questions regarding the pathogenesis of primary myelofibrosis and discusses the emerging targeted therapy aimed at restoring normal bone marrow environment and halting bone marrow fibrotic deterioration.
KW - Bone marrow microenvironments
KW - Genetic and epigenetic abnormality
KW - Hematopoietic bone marrow stem cell
KW - Primary myelofibrosis
KW - Target therapy
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U2 - 10.1007/s00277-016-2785-9
DO - 10.1007/s00277-016-2785-9
M3 - Review article
C2 - 27539616
AN - SCOPUS:84982305867
VL - 96
SP - 531
EP - 535
JO - Annals of Hematology
JF - Annals of Hematology
SN - 0939-5555
IS - 4
ER -