Bis-choline tetrathiomolybdate for treating Wilson disease
Methods for treating Wilson Disease with bis-choline tetrathiomolybdate therapy are provided. The methods may include administering 15 mg or between 30 and 90 mg of bis-choline tetrathiomolybdate once daily to a patient exhibiting NCC corrected, alamine aminotransferase (ALT), hemoglobin, platelets, or neutrophils levels meeting specified criteria. The methods may include modifying treatment by decreasing or increasing the daily dose of bis-choline tetrathiomolybdate or discontinuing treatment for a period of time.
1. A method of treating Wilson Disease in a patient in need thereof, comprising administering 15 mg of bis-choline tetrathiomolybdate once daily.
2. The method of claim 1 , wherein the 15 mg of bis-choline tetrathiomolybdate is administered as a delayed-release dosage form.
3. The method of claim 2 , wherein the dosage form is a tablet or capsule.
4. The method of claim 3 , wherein the dosage form is a tablet.
5. The method of claim 1 , wherein the patient exhibits a reduction in NCC corrected as measured after 24 weeks of administration as compared to the patient's NCC corrected as measured prior to administration.
6. The method of claim 5 , wherein the patient exhibits at least a 20% reduction in NCC corrected as measured after 24 weeks of administration as compared to the patient's NCC corrected as measured prior to administration.
7. The method of claim 1 , wherein the patient exhibits a reduction in NCC corrected as measured after 48 weeks of administration as compared to the patient's NCC corrected as measured prior to administration.
8. The method of claim 7 , wherein the patient exhibits at least a 20% reduction in NCC corrected as measured after 48 weeks of administration as compared to the patient's NCC corrected as measured prior to administration.
9. A method of treating Wilson Disease in a patient in need thereof, comprising administering from 30 to 90 mg of bis-choline tetrathiomolybdate daily, wherein the patient has one or more of the following:
a) an NCC corrected greater than 2.3 μm/L;
b) alanine aminotransferase (ALT) level less than 80 IU/mL;
c) hemoglobin of greater than 8 g/dL;
d) platelets greater than 30,000/μL; and
e) neutrophils greater than 10 3 /μL.
10. The method of claim 5 , further comprising measuring at least one of said patient's:
a) NCC corrected level;
b) alanine aminotransferase (ALT) level;
c) hemoglobin level;
d) platelets level; and
e) neutrophils level.
11. The method of claim 1 , wherein bis-choline tetrathiomolybdate is administered in the fasted state.
12. The method of claim 4 , wherein the tablet is an enterically coated tablet.
13. The method of claim 1 , wherein the patient has cirrhosis.
14. The method of claim 1 , wherein the patient does not have cirrhosis.
15. A method of treating Wilson Disease in a patient in need thereof, comprising administering 15 mg of bis-choline tetrathiomolybdate to the patient once every other day.
16. The method of claim 15 , wherein the 15 mg of bis-choline tetrathiomolybdate is administered as a delayed-release dosage form.
17. The method of claim 16 , wherein the dosage form is an enterically coated tablet.
18. The method of claim 15 , wherein bis-choline tetrathiomolybdate is administered in the fasted state.
19. The method of claim 16 , wherein the dosage form is a capsule.
20. The method of claim 15 , wherein the patient has cirrhosis.
21. The method of claim 15 , wherein the patient does not have cirrhosis.