Key clinical point: Gene therapy to induce the fetal form of hemoglobin reversed the symptoms of sickle cell disease in an adult patient.
Major finding: During 6 months of follow-up the patient did not experience sickle cell disease–related pain, respiratory events, or neurologic events, and did not have anemia.
Study details: A first-in-human study in seven adults with sickle cell disease.
Disclosures: Dr. Esrick reported receiving honoraria from Bluebird Bio, maker of the short hairpin RNA construct used in the trial.
Esrick EB et al. ASH 2018, Abstract 1023.
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Gene therapy reverses sickle cell phenotype in small study, Esrick EB et al. ASH 2018, Abstract 1023
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Sickle cell: Daily hydroxyurea safe in African children, Tshilolo L et al. ASH 2018, Abstract 3