Compositions and methods for diagnosis and treatment of anemia
View Patent ↗Compositions including a fatty acid, such as an odd chain fatty acid or a very long even chain fatty acid, and salts and derivatives thereof, and methods for treatment and prophylaxis of conditions related to anemic conditions, are provided, including compositions and methods for treating anemic conditions such as hemolytic anemia and anemia of chronic disease, and other related conditions.
1. A method of treatment of an anemic condition, wherein the anemic condition is selected from the group consisting of hemolytic anemia, thalassemia, hereditary spherocytosis, hereditary elliptocytosis, glucose-6-phosphate dehydrogenase deficiency, pyruvate kinase deficiency, immune hemolytic anemia, alloimmune hemolytic anemia, drug-induced hemolytic anemia, mechanical hemolytic anemia, paroxysmal nocturnal hemoglobinuria, anemia of chronic disease, anemia, aplastic anemia, congenital hypoplastic anemia, Diamond-Blackfan anemia, Fanconi anemia, iron deficiency anemia, anemia of abnormal RBC size, megaloblastic anemia, microcytic anemia, vitamin deficiency anemia, pernicious anemia, anemia of RBC mutation, sideroblastic anemia, and sickle cell anemia, comprising:
administering to a patient in need thereof, a composition comprising at least ten percent of one or more odd-chain saturated fatty acids, or pharmaceutically acceptable salts thereof, wherein the one or more odd-chain saturated fatty acids are selected from the group consisting of C15:0, C17:0, and combinations thereof.
2. The method of claim 1 , wherein the composition is a pharmaceutical composition in a unit dosage form comprising the one or more odd-chain saturated fatty acids or pharmaceutically acceptable salts thereof and a pharmaceutically acceptable carrier.
3. The method of claim 2 , wherein the unit dosage form comprises from 0.01 mg to 10000 mg of the one or more odd-chain saturated fatty acids or pharmaceutically acceptable salts thereof.
4. The method of claim 1 , wherein the one or more odd chain saturated fatty acids is heptadecanoic acid.
5. The method of claim 2 , wherein the pharmaceutical composition is substantially free from even chain saturated fatty acids.
6. The method of claim 1 , wherein the composition further comprises behenic acid.
7. The method of claim 2 , wherein the pharmaceutical composition comprises a plurality of different saturated fatty acids.
8. The method of claim 1 , wherein from 1.0 mg to 11 mg of the one or more odd-chain saturated fatty acids or pharmaceutically acceptable salts thereof is administered to the patient, per 1 kg of body weight, per day.
9. The method of claim 1 , wherein the one or more odd-chain saturated fatty acids or pharmaceutically acceptable salts thereof is administered to the patient once per day.
10. The method of claim 1 , wherein a serum concentration or a red blood cell membrane concentration of the one or more odd-chain saturated fatty acids is increased by at least about 0.001×10 −4 M above a pretreatment value.
11. The method of claim 2 , wherein the pharmaceutical composition is configured to modulate a marker of anemia or a symptom of anemia selected from the group consisting of serum or red blood cell membrane very long even chain saturated fatty acid percentage, serum concentration of a very long even chain saturated fatty acid, serum total very long even chain saturated fatty acid, serum ferritin, serum iron, transferrin saturation, red blood cell count, reticulocytes, hematocrit or packed cell volume, red blood cell distribution width, mean corpuscular hemoglobin concentration (MCHC), mean corpuscular volume (MCV), mean corpuscular hemoglobin (MCH), hemoglobin, white cell count, iron deficiency, serum iron, total iron-binding capacity, transferrin saturation, zinc protoporphyrin, and serum sTfRs.
12. The method of treatment of claim 1 , wherein the composition further comprises at least one even-chain saturated fatty acid, wherein the at least one even-chain saturated fatty acid is selected from the group consisting of C16:0, C18:0, and combinations thereof.
13. The method of treatment of claim 1 , wherein the composition further comprises at least one very long even chain saturated fatty acid, wherein the at least one very long even chain saturated fatty acid is selected from the group consisting of C20:0, C:22:0, and C24:0, and combinations thereof.