Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins69 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Purpose

TREATMENT

Enrollment

Conditions

Aplastic Anemia Paroxysmal Hemoglobinuria, Nocturnal

Interventions

cyclophosphamideDRUG

filgrastimDRUG

Eligibility

Sex: ALLMax age: 70 Years

Medical Language ↔ Plain English

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

Locations (1)

Johns Hopkins Oncology Center

Baltimore, Maryland, United States

Data from ClinicalTrials.gov

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