INDUCERS OF HEMATOPOIESIS AND FETAL GLOBIN PRODUCTION FOR TREATMENT OF CYTOPENIAS AND HEMOGLOBIN DISORDERS

摘要

The present invention generally relates to compositions containing chemical compounds and compositions and pharmaceutical formulations of the compounds which increase the expression of total hemoglobin or globin protein such as embryonic or fetal globin, or the proliferation of hemoglobin expressing and other blood cells. These compositions can be used to treat or prevent the symptoms associated with anemia, sickle cell diseases, thalassemia, and other blood cell deficiencies and blood disorders. The invention also relates to methods for administering these compositions to subjects and for use as medical aids for the treatment and prevention of blood and other disorders.

主权项

1. A method for increasing the percentage or absolute amount of fetal hemoglobin in the blood of a subject, comprising administering to the subject a pharmaceutical composition comprising 2 amino-3-hydroxy-N′-(2,3,4-trihydroxybenzyl) propanehydrazide (benserazide), or 8-Chloro-6,11-dihydro-11-(4-piperidinylidene)-5H-benzo[5,6]cyclohepta[1,2-b]pyridine (desloratadine) or both, or a pharmaceutically acceptable salt or ester thereof, wherein the dose of benserazide or desloratadine is equal to or less than 2 mg/kg/day, and wherein after administration, the percentage of fetal hemoglobin in the blood of the subject increases.