The page below is a sample from the LabCE course Hemoglobinopathies: Hemoglobin S Disorders. Access the complete course and earn ASCLS P.A.C.E.-approved continuing education credits by subscribing online.

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Emerging New Treatments

There is no widely available cure for SCD. Several emerging new treatments are currently being evaluated. In young patients with severe SCD, a stem cell transplant may be a treatment option. This involves the transplant of bone marrow of a patient with SCD with healthy bone marrow from a donor, ideally from a sibling without SCD. However, although bone marrow transplant can cure SCD in small number of patients, it can have life-threatening side-effects in other patients.

Other emerging new treatments include gene therapy to induce the formation of normal red blood cells by the bone marrow, and new medications to stimulate production of fetal hemoglobin, or to prevent the red blood cells from sickling.