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Investigational therapies for sickle cell disease

Griffin P Rodgers, MD
Section Editors
Stanley L Schrier, MD
Donald H Mahoney, Jr, MD
Deputy Editor
Jennifer S Tirnauer, MD


The major causes of morbidity and mortality in sickle cell disease (SCD) are the acute and long-term consequences of vaso-occlusion, many of which involve irreversible tissue injury or infarction, coupled with the effects of chronic hemolytic anemia.

The major interventions to reduce vaso-occlusive episodes include long-term hydroxyurea administration or regular blood transfusions (or exchange transfusions). However, hydroxyurea is not completely effective in preventing vaso-occlusive episodes, and chronic transfusions place a high burden on the patient, and it often is not feasible to administer transfusions indefinitely.

This topic reviews potential therapies for SCD that are under development or clinical investigation, including their rationale, preclinical data, and information from early clinical trials of their use.

Separate topic reviews discuss existing management options including comprehensive care, administration of hydroxyurea, red blood cell (RBC) transfusion, and hematopoietic cell transplantation (HCT) in SCD:

Management overview – (see "Overview of the management and prognosis of sickle cell disease")

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Literature review current through: Nov 2017. | This topic last updated: Oct 18, 2017.
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