Vaso-occlusion in sickle cell disease: pathophysiology and novel targeted therapies

D Manwani, PS Frenette - Blood, The Journal of the American …, 2013 - ashpublications.org
D Manwani, PS Frenette
Blood, The Journal of the American Society of Hematology, 2013ashpublications.org
Recurrent and unpredictable episodes of vaso-occlusion are the hallmark of sickle cell
disease. Symptomatic management and prevention of these events using the fetal
hemoglobin–reactivating agent hydroxyurea are currently the mainstay of treatment.
Discoveries over the past 2 decades have highlighted the important contributions of various
cellular and soluble participants in the vaso-occlusive cascade. The role of these elements
and the opportunities for therapeutic intervention are summarized in this review.
Abstract
Recurrent and unpredictable episodes of vaso-occlusion are the hallmark of sickle cell disease. Symptomatic management and prevention of these events using the fetal hemoglobin–reactivating agent hydroxyurea are currently the mainstay of treatment. Discoveries over the past 2 decades have highlighted the important contributions of various cellular and soluble participants in the vaso-occlusive cascade. The role of these elements and the opportunities for therapeutic intervention are summarized in this review.
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