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

Inclusion criteria

• 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
• Time from diagnosis to study registration ≤ 6mths
• No prior treatment except for haemopoietic growth factors given for no more than 4 weeks, and androgens.
• Age ≥ 16yrs (≥ 18yrs in Germany in accordance with German law), with no upper age limit.

Exclusion criteria

• Eligibility for an HLA-matched sibling donor transplant for SAA patients
• Prior therapy with ATG or CSA
• Haematopoeitic growth factors more than 4 weeks before study enrollment
• Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome
• Evidence of myelodysplastic disease
• Paroxysmal nocturnal haemoglobinuria with evidence of significant haemolysis, history of PNH associated thrombosis or a PNH clone > 50% by flow cytometry
• Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma)
• Subject is pregnant (e.g. positive HCG test) or is breast feeding
• Severe uncontrolled infection or unexplained fever > 38oC
• Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months