Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia
Condition: Aplastic Anemia
MeSH terms: Anemia; Anemia, Aplastic
Intervention: cyclosporine; anti-thymocyte globulin
Intervention description: Given orally; Given IV
Intervention MeSH terms: Cyclosporine; Cyclosporins; Thymoglobulin; Antilymphocyte Serum; Immunoglobulins
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 20.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No

Summary:

      RATIONALE: Immunosuppressive therapies, such as anti-thymocyte globulin and cyclosporine, may
      improve bone marrow function and increase blood cell counts. PURPOSE: This phase II trial is
      studying how well giving anti-thymocyte globulin together with cyclosporine as first-line
      therapy works in treating patients with severe aplastic anemia.
    

Eligibility criteria:

        Inclusion Criteria:

          -  All patients with sAA as defined by Camitta who are candidates for IS therapy; these
             criteria include bone marrow cellularity < 25% or 25-50% with < 30% of hematopoietic
             cells; it should also have two of the following three parameters: peripheral blood
             neutrophils < 0.5 x 10^9/L, platelets < 20 x 10^9/L and reticulocytes < 60 x 10^9/L in
             anemic patients

          -  If cytogenetic testing has been done, it should show normal karyotype or be not
             informative

          -  Patients should be either unwilling or otherwise ineligible (age, comorbidities, lack
             of donor) for bone marrow transplantation as a therapeutic modality

          -  Not previously treated with ATG for sAA

          -  Patients must have ECOG performance status of 0, 1, or 2

          -  Vitamin B12 and folic acid deficiency must be ruled out by measurement of serum levels

          -  Patients must have had a bone marrow biopsy examination in the three months prior to
             enrolling in the study

          -  Must be able to provide informed consent

          -  Systemic and other hematologic causes of pancytopenia, based on clinical presentation,
             must have been ruled out

        Exclusion Criteria:

          -  Patients with clinically evident congestive heart failure, serious cardiac
             arrhythmias; symptoms of coronary artery disease must be cleared by cardiology prior
             to therapy

          -  Patients who have had chemotherapy, radiotherapy, or immunotherapy or other
             investigational drug use within 3 weeks prior to study entry

          -  Pregnant women

          -  All females of childbearing potential must have a blood test or urine study within two
             weeks prior to induction registration to rule out pregnancy

          -  Women of childbearing potential are strongly advised to use an accepted and effective
             method of contraception

          -  Patients who have medical, psychological, or social conditions that may interfere with
             the patient's participation in the study or evaluation of the study results