High dose intravenous glucocorticoid in the treatment of childhood acquired aplastic anaemia.

31 children with acute acquired aplastic anaemia were treated with very high doses of i.v. bolus methylprednisolone. In 3 of them, paroxysmal nocturnal haemoglobinuria was diagnosed. Therefore the responses of 28 patients have been evaluated. Normoblastaemia and reticulocytes were observed on about the 6th d and leucocyte and granulocyte response around the 11th d of treatment. The first haemoglobin (greater than or equal to 0.5 g/dl) and haematocrit elevations were documented on about the 16th d and the initial platelet response (average greater than or equal to 34 X 10(9)/l) took more than a month. At least 64% of the patients responded to this treatment including 2 cases in whom aplasia was observed following hepatitis. Although 10 episodes of recurrences occurred in 8 patients (with the exception of 3 patients' in whom 5 recurrences were observed), response to the same regimen was obtained. With 1 exception the side-effects of this treatment could be managed by decreasing the dose. With this treatment, acquired aplastic anaemia should no longer be considered a fatal disease, at least in children.