METHODS AND COMPOSITIONS FOR TREATMENT OF BLEEDING PHENOMENON IN HEMOPHILIA PATIENTS Russian patent published in 2023 - IPC A61K31/713 C12N15/113 C07H21/04 A61P7/04 

FIELD: medicine; hematology; hereditary diseases.

SUBSTANCE: group of inventions is intended for preventive treatment of patients with hemophilia. The following is described: a method of treating a bleeding episode in a patient with hemophilia A or B with inhibitors undergoing routine hemophilia prophylaxis using a double-stranded ribonucleic acid (dsRNA) molecule, where the dsRNA molecule contains a sense strand and an antisense strand; where the sense strand contains the nucleotide sequence 5'-GfsgsUfuAfaCfaCfCfAfuUfuAfcUfuCfaAf-3' (SEQ ID NO: 13) and the antisense strand contains the nucleotide sequence 5'-usUfsgAfaGfuAfaAfuggUfgUfuAfaCfcsasg-3' (SEQ ID NO: 14), where a, c, g and u are 2'-O-methyl (2'-OMe) A, C, G and U, respectively; Af, Cf, Gf and Uf are 2'-fluoro A, C, G and U, respectively; and s is a phosphorothioate bond; and where the ligand is conjugated to the 3' end of the sense strand as shown in the following diagram:

the method comprises the step of administering to a patient a therapeutically effective amount of an activated prothrombin complex concentrate (aPCC), wherein the therapeutically effective amount of aPCC is not more than about 50 U/kg per dose. Besides, alternative methods of the prophylactic treatment of a patient with hemophilia A or hemophilia B with inhibitors are described.

EFFECT: group of inventions provides an increase in the efficiency of prophylactic treatment of patients with hemophilia.

36 cl, 29 dwg, 5 tbl, 6 ex