Baltimore, Maryland 21231

  • Paroxysmal Hemoglobinuria, Nocturnal


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.

Study summary:

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.


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



Primary Contact:

Study Chair
Robert A. Brodsky
Johns Hopkins University

Backup Contact:


Location Contact:

Baltimore, Maryland 21231
United States

There is no listed contact information for this specific location.

Site Status: N/A

Data Source:

Date Processed: April 07, 2020

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