6 patients with pure red‐cell aplasia were treated with Ciclosporin (Cyclosporine A; CS) alone or combined with prednisolone for a period of 9–46 (median 27) months. Prior to study, 5 cases had refractory disease, steroids were contraindicated in 1, and 4/6 patients, including 2 cases with congenital disease, had a disease duration exceeding 11 years. A complete haematological response was obtained in 5/6 subjects, and a partial response in 1. When the pre‐treatment Hb levels (mean±S.D. = 64±13 g/l, range 41–80) for all 6 PRCA patients were compared with the Hb levels after 6 months of CS therapy (104±17 g/1, 80–125), a significant improvement was registered (p<0.005). In half of the patients, remission is maintained with CS as single drug in a dose‐dependent manner. We also treated 5 patients with refractory severe aplastic anaemia with CS (1 case) or CS plus prednisolone (4 cases) for 3–27 (median 10) months. Only 1 patient responded. In this case, a complete haematological remission was induced with CS alone, and remission has been maintained for 27 months. Side effects of CS therapy were common but were dose‐dependent and reversible, with the exception of persistent nephrotoxicity in 1 patient with pure red‐cell aplasia. Based on our present results and a survey of the literature, we conclude that CS therapy is effective and indicated in refractory pure red‐cell aplasia. In severe aplastic anaemia resistant to conventional immunosuppression, the response rate is lower, but a small proportion (around 15) of patients may benefit from CS therapy. Longer treatment periods may, however, be needed to evaluate the role of
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