Complications and. Treatment of Acquired Aplastic Anaemia

Abstract

SUMMARYThe clinical and haematological findings in 43 cases of aplastic anaemia are reviewed. Approximately two‐thirds of the cases were considered to be secondary to toxic agents, the commonest of which were chloramphenicol and phenylbutazone, and one‐third were idiopathic. Severe neutropenia and thrombocytopenia were a feature of the series, a neutrophil count of less than 500/cu.mm. and a platelet count of less than 20,000/cu.mm. being found initially in over one‐half of cases. There was an unexplained and marked elevation of the ESR in over 90 per cent of cases. Fifty‐two episodes of infection occurred in 29 patients (67 per cent); 36 of these episodes were serious. Infection was more frequent in young patients and serious infection was more likely in the presence of severe neutropenia. The occurrence of infection, especially serious infection, was usually followed by a fall in neutrophil and platelet counts, and frequently by the onset of serious bleeding. Bleeding phenomena occurred at some stage in all but one patient and in two‐thirds of cases the bleeding was serious. Thrombocytopenia was marked in patients with serious bleeding but equally low platelet counts were found in patients in whom bleeding was not serious. The onset of serious bleeding was more likely in the presence of infection.Administration of corticosteroids or corticotrophin either with or without androgens was followed by a haematological response in about two‐thirds of cases, but in only one‐third was the response sustained; in the remainder it was transient. In those with a sustained response, the haemoglobin and neutrophil values gradually increased and ultimately returned to normal; however, the platelet count returned to normal in less than one‐half of these.The overall mortality rate was 63 per cent, bleeding and infection being the commonest causes of death. The mortality was higher in the idiopathic cases (86 per cent) than in the secondary cases (52 per cent); however, the period of survival in fatal secondary cases was shorter than that in fatal idiopathic cases. The prognosis appeared to be better in patients with secondary aplasia, in patients with an initial haemoglobin value greater than 8 g. per 100 ml., and in patients who remained free of serious bleeding. The outcome showed no relation to sex, age, the initial neutrophil and platelet counts or the cellularity of the bone marrow on admission. Seven of the 16 survivors are considered cured, eight are well but are still thrombo‐cytopenic, and one has developed paroxysmal nocturnal haemoglobinuria 8 years after diagnosis and 6 years after a complete haematological remission. Treatment with corticosteroids is still necessary to control bleeding in three of the eight survivors with thrombocytopenia.