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Is there experience/reports of using voxelotor in sickle cell patients who are Jehovah Witnesses with few crises but who have fatigue and/or dyspnea?

Also is there value in using voxelotor for the purpose of reducing hemolysis, and if so what parameters do you use to determine when to initiate voxelotor with that goal in mind?
1 Answer
Mednet MemberInvited Expert
Hematology · Boston University School of Medicine
Answered on

I would not hesitate to use voxelotor in most patients with sickle cell anemia, including Jehovah's Witnesses, if they have not responded to hydroxyurea with near cessation of acute vasoocclusive events and have continued hemolytic anemia. Voxelotor usually reduces hemolysis that results in an increase of hemoglobin levels. It does not seem to increase the incidence of acute vasoocclusive episodes. Some data published in abstract form and presented at ASH 2020 suggested that the best responders had the most reduction in acute episodes.

Hemolysis is associated with many complications of sickle cell disease, including pulmonary hypertension and nephropathy. Reducing hemolysis over the long term might reduce the incidence of these, and other, vasculopathic complications that are associated with mortality.