Eculizumab in Treating Patients With Paroxysmal Nocturnal Hemoglobinuria

Inclusion Criteria

• •DISEASE CHARACTERISTICS:
• •Diagnosis of paroxysmal nocturnal hemoglobinuria
• •Must have required ≥ 4 episodes of transfusions for anemia or anemia-related symptoms within the past year
• •* Mean pre-transfusion hemoglobin ≤ 10. 5 g/dL over the past year
• •Glycosylphosphatidylinositol (GPI)-deficient red blood cell clone (type III cells) of ≥ 10% by flow cytometry
• •Must have received 1 packed red blood cell transfusion during the study observation period (within 48 hours of the hemoglobin level that precipitated the transfusion) and within 1.5 g/dL of the mean pre-transfusion hemoglobin level over the past year
• •* Pre-transfusion hemoglobin ≤ 9 g/dL with symptoms
• •* Pre-transfusion hemoglobin ≤ 7 g/dL without symptoms
• •Received Neisseria meningitidis vaccination at least 2 weeks before initiation of study therapy
• •PATIENT CHARACTERISTICS:
• •Age
• •18 and over
• •Performance status
• •Not specified
• •Life expectancy
• •Not specified
• •Hematopoietic
• •See Disease Characteristics
• •Absolute neutrophil count \> 500/mm\^3
• •Platelet count ≥ 100,000/mm\^3
• •Hepatic
• •Lactate dehydrogenase ≥ 1.5 times upper limit of normal
• •Renal
• •Not specified
• •Immunologic
• •No known or suspected active bacterial infection
• •No recurrent bacterial infections
• •No history of meningococcal disease
• •Other
• •No known or suspected hereditary complement deficiency
• •No other condition that would increase the patient's risk or confound the outcome of the study
• •Not pregnant or nursing
• •Negative pregnancy test
• •Fertile patients must use effective contraception
• •PRIOR CONCURRENT THERAPY:
• •Biologic therapy
• •See Disease Characteristics
• •No prior bone marrow transplantation
• •Concurrent epoetin alfa allowed\*
• •Chemotherapy
• •Not specified
• •Endocrine therapy
• •Concurrent corticosteroids allowed\*\*
• •Radiotherapy
• •Not specified
• •Surgery
• •Not specified
• •Other
• •More than 30 days since prior participation in another investigational drug trial
• •More than 30 days since prior investigational agents, devices, or procedures
• •Concurrent immunosuppressants allowed\*
• •Concurrent warfarin allowed provided INR level is stable for the past 4 weeks and expected to remain stable during observation and study treatment
• •Concurrent iron supplements or folic acid allowed\*\*
• •Concurrent low-molecular weight heparin allowed\*\* NOTE: \*Provided dose is stable for the past 26 weeks and during study observation and treatment
• •NOTE: \*\*Provided dose is stable for the past 4 weeks and expected to remain stable (or decrease for corticosteroids) during study observation and treatment