Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia

Inclusion Criteria:

1. To define aplastic anaemia there must be at least two of the following: (1) haemoglobin < 10g/dl; (2) platelet count < 50 x 109/l; (3) neutrophil count < 1.5 x 109/l, and a hypocellular bone marrow on bone marrow biopsy
2. Time from diagnosis to study registration ≤ 6mths
3. No prior treatment except for haemopoietic growth factors given for no more than 4 weeks, and androgens.
4. Age ≥ 16yrs (≥ 18yrs in Germany in accordance with German law), with no upper age limit.

Exclusion criteria:

1. Eligibility for an 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 enrollment
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 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 HCG test) or is breast feeding
9. Severe uncontrolled infection or unexplained fever > 38oC
10. Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months