Methods and low dose regimens for treating red blood cell disorders
AssigneesTrustees of Boston University, Hemaquest Pharmaceuticals, Inc.
InventorsSusan P Perrine, Douglas V Faller, Ronald J Berenson
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Abstract
Disclosed herein are methods and low dose regimens for increasing fetal hemoglobin levels in patients with red blood cell disorders, such as beta thalassemia, sickle cell disease, other anemias, or blood loss. Fetal and total hemoglobin levels and red blood cell counts are increased by administering 2,2-dimethylbutyrate (DMB) alone or in combination with hydroxyurea, decitabine or an HDAC inhibitor. Treatment can be continued for at least two weeks.
Claims29
A method for increasing the percentage of fetal hemoglobin in the blood of a subject, comprising administering to said subject 2,2-dimethylbutyrate as the free acid, a pharmaceutically acceptable salt, or ester thereof;
- wherein a total daily dose of 2,2-dimethylbutyrate is more than 10 mg/kg and not more than 40 mg/kg;
- and wherein after the administration the percentage of fetal hemoglobin in the blood of the subject increases.
A method for increasing the percentage of fetal hemoglobin in the blood of a subject comprising administering to said subject:
- a) 2,2-dimethylbutyrate as the free acid, a pharmaceutically acceptable salt, or ester thereof, wherein a total daily dose of 2,2-dimethylbutyrate is more than 10 mg/kg and not more than 40 mg/kg;
- and
- b) one or more agents selected from the group consisting of hydroxyurea, decitabine, and an HDAC inhibitor;
- wherein after the administration the percentage of fetal hemoglobin in the blood of the subject increases.
Dependencies are inferred from the claim text.