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      New insights into the pathophysiology and development of novel therapies for sickle cell disease

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          Abstract

          Although the seminal event in sickle cell disease is the polymerization of abnormal hemoglobin, the downstream pathophysiology of vasoocclusion results from heterotypic interactions between the altered, adhesive sickle cell red blood cells, neutrophils, endothelium, and platelets. Ischemia reperfusion injury, hemolysis, and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system. These various pathways are the focus of emerging treatments with potential to ameliorate disease manifestations. This review summarizes the considerable progress in development of these agents despite challenges in selection of study end points and complex pathophysiology.

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          Author and article information

          Journal
          Hematology Am Soc Hematol Educ Program
          Hematology Am Soc Hematol Educ Program
          bloodbook
          bloodbook
          Hematology: the American Society of Hematology Education Program
          American Society of Hematology (Washington, DC )
          1520-4391
          1520-4383
          30 November 2018
          30 November 2019
          : 2018
          : 1
          : 493-506
          Affiliations
          [1 ]Children’s Hospital, Montefiore Medical Center, Bronx, NY; and
          [2 ]Department of Microbiology and Immunology and
          [3 ]Division of Pediatric Hematology, Oncology, Marrow and Blood Cell Transplantation, Albert Einstein College of Medicine, Bronx, NY
          Author notes
          Correspondence Deepa Manwani, Children's Hospital at Montefiore, 3415 Bainbridge Ave, Bronx, NY 10467; e-mail: dmanwani@ 123456montefiore.org .

          Conflict-of-interest disclosure: The authors declare no competing financial interests.

          Off-label drug use: None disclosed.

          Article
          PMC6245971 PMC6245971 6245971 01867
          10.1182/asheducation-2018.1.493
          6245971
          30504350
          dd9ef040-e70c-4a6f-a63d-e974e6caf086
          © 2018 by The American Society of Hematology. All rights reserved.
          History
          Page count
          Pages: 14
          Categories
          Sickle Cell Disease: New Frontiers

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