Eligibility criteria
| Inclusion criteria |
| Males with severe F.IX deficiency |
| Age 18 y or older |
| Ability to give informed consent |
| More than 20 exposure days of treatment with F.IX protein |
| No history or presence of an inhibitor to F.IX protein |
| Able to infuse F.IX protein on a home infusion protocol |
| Subjects with F.IX missense mutations |
| Exclusion criteria |
| Active infections |
| End-stage renal disease |
| Severe liver disease defined as any of the following: |
| Bilirubin; 2.1-3.0 × normal |
| Transaminases: 5-10 × normal |
| Alkaline phosphatase: 5-10 × normal |
| Platelet count less than 50 000/μL |
| Presence of inflammatory muscle disease (eg, myositis) |
| Inclusion criteria |
| Males with severe F.IX deficiency |
| Age 18 y or older |
| Ability to give informed consent |
| More than 20 exposure days of treatment with F.IX protein |
| No history or presence of an inhibitor to F.IX protein |
| Able to infuse F.IX protein on a home infusion protocol |
| Subjects with F.IX missense mutations |
| Exclusion criteria |
| Active infections |
| End-stage renal disease |
| Severe liver disease defined as any of the following: |
| Bilirubin; 2.1-3.0 × normal |
| Transaminases: 5-10 × normal |
| Alkaline phosphatase: 5-10 × normal |
| Platelet count less than 50 000/μL |
| Presence of inflammatory muscle disease (eg, myositis) |