Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
Condition: Myelodysplastic Syndrome; Aplastic Anemia
MeSH terms: Preleukemia; Anemia; Myelodysplastic Syndromes; Anemia, Aplastic; Syndrome
Intervention: Thymoglobulin; Cyclosporine; Methylprednisolone; G-CSF
Intervention description: 3.5 or 2.5 mg/kg/day IV for 5 days
Aplastic anemia patients receive 3.5 mg/kg/day for 5 days
MDS patients <55 years receive 3.5 mg/kg/day for 5 days
MDS patients >55 years receive 2.5 mg/kg/day for 5 days; 5 mg/kg orally for 6 months; start after completing thymoglobulin.; 1 mg/kg/day IV for 5 days, given before each dose of thymoglobulin.; 5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin.
Intervention MeSH terms: Cyclosporine; Methylprednisolone; Methylprednisolone Acetate; Methylprednisolone Hemisuccinate; Prednisolone; Prednisolone acetate; Cyclosporins; Thymoglobulin; Antilymphocyte Serum; Prednisolone hemisuccinate; Prednisolone phosphate
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 53.0
Sex: All
Minimum age: 15 Years
Healthy volunteers: No
Keywords: Myelodysplastic Syndrome; Aplastic Anemia; Thymoglobulin; Cyclosporine; Methylprednisolone; G-CSF

Summary:

      The goal of this clinical research study is to learn if combining the drugs thymoglobulin,
      methylprednisolone, cyclosporine, and G-CSF (NeupogenTM or NeulastaTM ) can help to control
      severe aplastic anemia (AA) or hypoplastic myelodysplastic syndrome (MDS). The safety of this
      combination therapy will also be studied.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of severe aplastic anemia (bone marrow cellularity < 30%, with two of three
             peripheral counts at the time of initial presentation or currently low with absolute
             neutrophil count (ANC) < 500/mL, pre-transfusion platelet (PLT) < 20,000/mL, or
             pre-transfusion hemoglobin < 8 g/dL and presence of no other underlying disorder.

          -  Diagnosis of MDS (World Health Organization) with bone marrow cellularity < 30%, with
             two of three peripheral counts at the time of initial presentation or currently low
             with ANC < 500/mL, pre-transfusion PLT < 20,000/mL, or pre-transfusion hemoglobin < 8
             g/dL.

          -  Patients with MDS who have received prior biological therapy (not chemotherapy) are
             eligible. Hypomethylating agents and histone deacetylase inhibitors are considered as
             biological therapy.

          -  Age 15 or greater

          -  Adequate renal function (creatinine less than or equal to 2.0 mg/dL) unless related to
             the disease

          -  Adequate hepatic function (bilirubin less than or equal to 3.5 mg/dL) unless related
             to the disease

          -  No other investigational therapy in the past 14 days

          -  Able to sign consent form

          -  Able to comply with the need for contraception (abstinence, condom, birth control
             pill, or other acceptable form of contraception) during the entire study period

          -  Diagnosis of MDS (WHO) with bone marrow cellularity greater than 30%, with low or
             intermediate-1 risk by the International Prognostic Scoring System (IPSS) score, and
             requiring treatment (i.e. transfusion-dependent)

        Exclusion Criteria:

          -  Active and uncontrolled infection

          -  HIV positive test

          -  Pregnant or breast feeding

          -  Active and uncontrolled medical illness (pulmonary, cardiac, neurological, or other)
             that in the opinion of treating physician would likely interfere with study treatment