Abstract
Sickle cell disease is an inherited hemoglobinopathy characterized by hemolytic anemia, frequent painful episodes, poor quality of life, end organ damage and a shortened lifespan. Although the seminal event is the polymerization of the abnormal hemoglobin, the downstream pathophysiology of vaso-occlusion results from heterotypic interactions between the altered, adhesive sickle cell RBCs, neutrophils, endothelium, and platelets. Ischemia reperfusion injury, hemolysis and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. These downstream targets are the focus of emerging treatments with considerable potential to ameliorate disease manifestations. This review summarizes the progress on development of these agents.
| Original language | English (US) |
|---|---|
| Journal | Seminars in Hematology |
| DOIs | |
| State | Accepted/In press - Jan 1 2018 |
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Keywords
- Adhesion pathway
- Inflammation
- Novel agents
- Sickle cell disease
ASJC Scopus subject areas
- Hematology
Cite this
Novel Sickle Cell Disease Therapies : Targeting Pathways Downstream of Sickling. / Morrone, Kerry; Mitchell, William Beau; Manwani, Deepa.
In: Seminars in Hematology, 01.01.2018.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Novel Sickle Cell Disease Therapies
T2 - Targeting Pathways Downstream of Sickling
AU - Morrone, Kerry
AU - Mitchell, William Beau
AU - Manwani, Deepa
PY - 2018/1/1
Y1 - 2018/1/1
N2 - Sickle cell disease is an inherited hemoglobinopathy characterized by hemolytic anemia, frequent painful episodes, poor quality of life, end organ damage and a shortened lifespan. Although the seminal event is the polymerization of the abnormal hemoglobin, the downstream pathophysiology of vaso-occlusion results from heterotypic interactions between the altered, adhesive sickle cell RBCs, neutrophils, endothelium, and platelets. Ischemia reperfusion injury, hemolysis and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. These downstream targets are the focus of emerging treatments with considerable potential to ameliorate disease manifestations. This review summarizes the progress on development of these agents.
AB - Sickle cell disease is an inherited hemoglobinopathy characterized by hemolytic anemia, frequent painful episodes, poor quality of life, end organ damage and a shortened lifespan. Although the seminal event is the polymerization of the abnormal hemoglobin, the downstream pathophysiology of vaso-occlusion results from heterotypic interactions between the altered, adhesive sickle cell RBCs, neutrophils, endothelium, and platelets. Ischemia reperfusion injury, hemolysis and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. These downstream targets are the focus of emerging treatments with considerable potential to ameliorate disease manifestations. This review summarizes the progress on development of these agents.
KW - Adhesion pathway
KW - Inflammation
KW - Novel agents
KW - Sickle cell disease
UR - http://www.scopus.com/inward/record.url?scp=85047370073&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85047370073&partnerID=8YFLogxK
U2 - 10.1053/j.seminhematol.2018.04.007
DO - 10.1053/j.seminhematol.2018.04.007
M3 - Article
C2 - 30616808
AN - SCOPUS:85047370073
JO - Seminars in Hematology
JF - Seminars in Hematology
SN - 0037-1963
ER -