Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia
Aplastic Anemia, Neutropenia, Pancytopenia
About this trial
This is an interventional treatment trial for Aplastic Anemia focused on measuring Aplastic Anemia, Immunosuppression, T-Cells, Hematopoiesis, Autoimmunity, Severe Aplastic Anemia
Eligibility Criteria
- INCLUSION CRITERIA:
Severe aplastic anemia characterized by:
Bone marrow cellularity less than 30 percent (excluding lymphocytes)
AND
At least two of the following:
Absolute neutrophil count less than 500/ microL
Platelet count less than 20,000/ microL
Absolute reticulocyte count less than 60,000/ microL
Age greater than or equal to 2 years old
Weight greater than or equal to 12 kg
EXCLUSION CRITERIA:
Diagnosis of Fanconi anemia
Cardiac ejection fraction less than 30 percent (evaluated by ECHO)
Evidence of a clonal hematologic bone marrow disorder on cytogenetics. Patients with the presence of trisomy 8, loss of Y or del(20q) will not be excluded in the absence of dysplastic changes in the marrow. Patients with very severe neutropenia (ANC less than 200 /microL) will not be excluded initially if cytogenetics are not available or pending. If evidence of a clonal disorder is later identified, the patient will go off study.
Prior immunosuppressive therapy with high dose Cy or ATG
Infection not adequately controlled with appropriate therapy
Serologic evidence of HIV infection
Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy, or that death within 30 days is likely
Subjects with cancer who are not considered cured, are on active chemotherapeutic treatment or who take drugs with hematological effects
Current pregnancy or unwillingness to take oral contraceptives or refrain from pregnancy if of childbearing potential
Not able to understand the investigational nature of the study or to give informed consent.
Sites / Locations
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Arms of the Study
Arm 1
Experimental
SAA hematologic response
Treatment-naive severe aplastic anemia patients will receive a low dose of cyclophosphamide (120mg/kg) and low dose cyclosporine ( target therapeutic level of 100-200 micrograms per liter). Cyclophosphamide will be given once daily for 4 doses. Cyclosporine will be started after cyclophosphamide completion, cyclosporine will be given twice daily. The dosing will be modified to attain the therapeutic level.