Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia
Condition: Aplastic Anemia
MeSH terms: Anemia; Anemia, Aplastic
Intervention: rabbit antithymocyte globulin
Intervention description: 1.5 vials/10kg daily for 5 days
Intervention MeSH terms: Thymoglobulin; Antilymphocyte Serum
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 35.0
Sex: All
Minimum age: 16 Years
Healthy volunteers: No
Keywords: Thymoglobuline; Rabbit ATG; Ciclosporin; Aplastic Anemia

Summary:

      To assess the tolerability and effectiveness of rabbit antithymocyte globulin (ATG,
      Thymoglobuline) with ciclosporin in the first line treatment of patients with acquired severe
      aplastic anaemia, and patients with non-severe aplastic anaemia and who are transfusion
      dependent.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Must fulfil definition of aplastic anaemia:

             There must be at least two of the following:

               -  haemoglobin < 10g/dl

               -  platelet count < 50 x 109/l

               -  neutrophil count < 1.5 x 109/l, and a hypocellular bone marrow on bone marrow
                  biopsy

             SAA as defined by a hypocellular bone marrow of <25% cellularity and two of the
             following:

               -  neutrophil count < 0.5 x 109/l

               -  platelets < 20 x 109/l

               -  reticulocytes < 20 x 109/l

             NSAA as defined by a hypocellular bone marrow and cytopenia in at least two cell lines
             and neutrophil count > 0.5 x 109/l, and red cell and/or platelet transfusion
             dependence

          2. Have acquired aplastic anaemia

          3. Time from diagnosis to study registration maximum 6 months

          4. No prior treatment except for haemopoietic growth factors given for no more than four
             weeks, and androgens

          5. Age minimum 16 years with no upper age limit

        Exclusion Criteria:

          1. Eligibility for an human leukocyte antigens (HLA)-matched sibling donor transplant for
             SAA patients

          2. Prior therapy with ATG or CSA

          3. Haematopoeitic growth factors more than 4 weeks before study enrolment

          4. Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure
             syndrome

          5. Evidence of myelodysplastic disease

          6. Paroxysmal nocturnal haemoglobinuria with evidence of significant haemolysis, history
             of Paroxysmal Nocturnal Hemoglobinuria (PNH) associated thrombosis or a PNH clone >50%
             by flow cytometry

          7. Diagnosis or previous history of carcinoma (except local cervical, basal cell,
             squamous cells, or melanoma)

          8. Subject is pregnant (e.g. positive Human Chorionic Gonadotropin (HCG) test) or is
             breast feeding

          9. Severe uncontrolled infection or unexplained fever >38 degrees Celsius

         10. Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of
             such severity that life expectancy is less than 3 months