Researchers assessed the use of an oral sickle hemoglobin polymerization inhibitor, voxelotor, in a patient with sickle cell disease (SCD) to treat anemia resulting from a delayed hemolytic transfusion reaction (DHTR), and published their results in the European Journal of Haematology.

They observed pretransfusion hemoglobin levels in the patient within 10 days of initiation of voxelotor, and improvement continued for 27 days.

“Voxelotor is a first-in-class oral [sickle hemoglobin]-polymerization inhibitor shown to increase hemoglobin (Hb) and reduce incidences of anemia and hemolysis in patients with SCD,” the authors wrote. “Here we report a patient with SCD whose anemia was successfully treated with voxelotor following a severe DHTR, which possibly occurred due to hyperhemolysis.”

The authors report a case of a 59-year-old African American woman with SCD who presented ankle pain and ankle ulcers, along with pulmonary fibrosis. She was receiving transfusions with accompanying edema, pain, and weight gain. Given she was on transfusion treatment, she was ineligible for the phase 3 HOPE trial, which evaluated voxelotor in patients with SCD.

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Her condition quickly became critical with undetectable Hb, and no appropriate units of red blood cells for transfusion were available at that time. Voxelotor was administered as a last attempt to save the patient’s life. The next day, she had 3.8 g/dl Hb with no side effects, and voxelotor dosing was continued.

After 4 days, Hb continued to improve and the pain was reduced, and after 10 days her Hb level had returned to pretransfusion levels. After 27 days, her Hb levels were 8.1 g/dl and she was discharged to rehabilitation.

Based on the rapid improvements in this case, the authors suggest that voxelator might be a potentially life-saving treatment for patients with SCD in whom other methods of restoring Hb levels are unsafe or unavailable.

Reference

Ferlis M, Lipato T, Roseff SD, et al. Urgent use of voxelotor in sickle cell disease when immediate transfusion is not safe. Eur J Haematol. Published online July 18, 2022. doi:10.1111/ejh.13830