Are there investigational treatments for sickle cell disease (SCD)?

Updated: May 12, 2021
  • Author: Joseph E Maakaron, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Investigational treatments include nitric oxide inhalation, topical granulocyte-macrophage colony-stimulating factor (GM-CSF), butyrate, and arginine, as follows:

  • Nitric oxide inhalation has been investigated in the treatment of pulmonary hypertension.
  • Topical GM-CSF has been reported to hasten the healing of leg ulcers.
  • Butyrate was studied to decrease vaso-occlusive crisis.
  • Arginine has been proposed to use as a precursor of nitric oxide production.

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