TY - JOUR
T1 - New insights into sickle cell disease
T2 - Mechanisms and investigational therapies
AU - Kato, Gregory J.
N1 - Funding Information:
G.J.K. gratefully acknowledges funding from the Patient Centered Outcomes Institute, the National Institutes of Health (1 R01 HL121386, 1 R01 MD009162), the Institute for Transfusion Medicine and the Pittsburgh Heart, Lung and Blood Vascular Medicine Institute. Disclosures: Hydroxyurea is not approved for use in children. IVIG is not approved for use in sickle cell disease. Rivipansel is an investigational agent. DEANO is a research reagent. G.J.K. has served as a paid consultant or received research funding from AesRx, LLC, Baxalta, Biogen Idec, CSL Behring, and Bayer.
Publisher Copyright:
Copyright © 2016 Wolters Kluwer Health, Inc. All rights reserved.
PY - 2016
Y1 - 2016
N2 - Purpose of review: Sickle cell disease (SCD) afflicts millions worldwide. The simplicity of its single nucleotide mutation belies the biological and psychosocial complexity of the disease. Despite only a single approved drug specifically for the treatment of SCD, new findings reviewed from 2015 provide the direction forward. Recent findings: The last year has provided a wealth of support for mechanisms affecting the red cell, hemolysis and vasculopathy, the innate immune system activation, blood cell and endothelial adhesiveness, central sensitization to pain, and chronic brain injury. The evidence supporting expanded use of hydroxyurea continues to mount. Many promising therapies are reaching clinical trial, including curative therapies, with more on the horizon. Summary: Evidence is compelling that the use of hydroxyurea must be expanded by clinicians to gain the full pleiotropic benefits of this approved drug. Clinicians must become aware that severe acute and chronic pain has a biological and neurologic basis, and the understanding of this basis is growing. Researchers are testing investigational therapies at an unprecedented pace in SCD, and partnership between patients, researchers, and the private sector provides the most rapid and productive way forward.
AB - Purpose of review: Sickle cell disease (SCD) afflicts millions worldwide. The simplicity of its single nucleotide mutation belies the biological and psychosocial complexity of the disease. Despite only a single approved drug specifically for the treatment of SCD, new findings reviewed from 2015 provide the direction forward. Recent findings: The last year has provided a wealth of support for mechanisms affecting the red cell, hemolysis and vasculopathy, the innate immune system activation, blood cell and endothelial adhesiveness, central sensitization to pain, and chronic brain injury. The evidence supporting expanded use of hydroxyurea continues to mount. Many promising therapies are reaching clinical trial, including curative therapies, with more on the horizon. Summary: Evidence is compelling that the use of hydroxyurea must be expanded by clinicians to gain the full pleiotropic benefits of this approved drug. Clinicians must become aware that severe acute and chronic pain has a biological and neurologic basis, and the understanding of this basis is growing. Researchers are testing investigational therapies at an unprecedented pace in SCD, and partnership between patients, researchers, and the private sector provides the most rapid and productive way forward.
KW - Hemolysis
KW - Hydroxyurea
KW - Inflammation
KW - Sickle cell
KW - Vasculopathy
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U2 - 10.1097/MOH.0000000000000241
DO - 10.1097/MOH.0000000000000241
M3 - Review article
C2 - 27055046
AN - SCOPUS:84963636078
SN - 1065-6251
VL - 23
SP - 224
EP - 232
JO - Current opinion in hematology
JF - Current opinion in hematology
IS - 3
ER -