Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

Inclusion Criteria:

Must have required at least 4 transfusions in the past 12 months
PNH type III red blood cell (RBC) clone by flow cytometry of >10%
Lactate dehydrogenase (LDH) level > 1.5 x upper limit of normal
Platelet count > 100,000/mm3
Patient taking erythropoietin must be on a stable dose for at least 26 weeks
Patient taking immunosuppressants must be on a stable dose for at least 26 weeks
Patient taking corticosteroids must be on a stable dose for at least 4 weeks
Patient taking coumadin must be at a stable INR for at least 4 weeks
Patient taking iron supplements or folic acid must be on a stable dose for 4 weeks
Willing and able to give written informed consent
Must avoid conception

Exclusion Criteria:

Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl
Absolute neutrophil count <500/ul
Active bacterial infection
Hereditary complement deficiency
Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures within 30 days
Pregnant, breast-feeding, or intending to conceive
History of meningococcal disease
History of bone marrow transplantation