Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia
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To assess the tolerability and efficacy of rabbit antithymocyte globulin (ATG, Thymoglobuline®) with ciclosporin (CSA) in the first line treatment of patients with acquired severe aplastic anaemia (SAA), and patients with non-severe aplastic anaemia (NSAA) and who are transfusion dependent. To compare the response rate of the combination of rabbit ATG (Thymoglobuline® and CSA from this pilot study with the response rate observed in a series of matched AA patients; treated after 1994 with the combination of horse ATG (Lymphoglobuline®) and CSA; obtained from the EBMT database (comparative study).
Rabbit ATG, Thymoglobuline (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours.
Ciclosporin (CSA) 5mg/kg/day orally from day +1 for a minimum of 6 months, with later tailing according to individual patient response. Aim to maintain trough whole blood CSA levels between 150 and 250 ng/ml.
Drug: Rabbit ATG, Thymoglobuline (Genzyme)
Rabbit ATG, Thymoglobuline® (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours.
Ciclosporin (CSA) 5mg/kg/day orally from day +1 for a minimum of 6 months
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Ages Eligible for Study:
16 Years to 80 Years (Child, Adult, Senior)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
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.
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