Aplastic Anemia, Paroxysmal Hemoglobinuria, Nocturnal
Conditions
Keywords
aplastic anemia, hematologic disorders, paroxysmal nocturnal hemoglobinuria, rare disease
Brief summary
OBJECTIVES: I. Confirm the efficacy demonstrated in a pilot study using high dose cyclophosphamide in patients with severe aplastic anemia. II. Determine whether the addition of filgrastim (G-CSF) to high dose cyclophosphamide shortens the time to recovery in these patients. III. Determine whether this regimen is efficacious in treating paroxysmal nocturnal hemoglobinuria.
Detailed description
PROTOCOL OUTLINE: Patients receive high dose cyclophosphamide IV on days 1-4. Beginning on day 10, patients receive filgrastim (G-CSF) until the absolute neutrophil count is greater than 1,000/mm3 for 2 consecutive days. Patients are followed every 3 months for at least 2 years and annually thereafter.
Interventions
DRUGcyclophosphamide
DRUGfilgrastim
Sponsors
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Study design
Primary purpose
TREATMENT
Eligibility
Sex/Gender
ALL
Age
No minimum to 70 Years
Healthy volunteers
No
Inclusion criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Acquired severe aplastic anemia or paroxysmal nocturnal hemoglobinuria * Not a candidate for allogeneic bone marrow transplantation * Must meet one of the following criteria: * Severe aplastic anemia * Less than 25% bone marrow cellularity and depression in two of three blood counts (reticulocytes less than 40,000/mm3 * platelet count less than 20,000/mm3 and granulocytes less than 500/mm3) * Life-threatening paroxysmal nocturnal hemoglobinuria * Absolute neutrophil count less than 500/mm3 * platelet transfusion dependent * thrombotic disease * No Fanconi anemia * No abnormal cytogenetics --Patient Characteristics-- * Renal: Creatinine no greater than 2.0 mg/dL * Cardiovascular: Cardiac ejection fraction at least 45% * Other: Not preterminal or moribund Not pregnant
Countries
United States
Outcome results
None listed