Paroxysmale nächtliche Hämoglobinurie: Klinische Erfahrungen bei 40 Patienten in einem Zentrum über 25 Jahre

In one centre (the medical department of the University of Ulm), over a period of 25 years, the diagnosis of paroxysmal nocturnal haemoglobinuria (PNH) had been made in 40 patients (27 women, 13 men). The data on these patients were analysed and the findings compared with those reported in the literature. Criteria for inclusion were a positive sucrose haemolysis and acid-serum test. Leading clinical symptoms were anaemia, tendency towards bleeding and infections, haemolysis, abdominal pain and thromboembolism. The age at which main symptoms first appeared was most often in the third and fourth decade of life. The sex ratio of 2:1 (female to male) was unusual. PNH was frequently (in 22 patients) associated with aplastic anaemia. If bone marrow transplantation, at present the only curative procedure, was not possible and there was PNH alone, treatment was supportive and directed at relief of symptoms. The course of the disease was highly variable. Lack fo prognostic criteria made it difficult to define indications for intensive therapeutic measures. While haemolytic crisis and thromboembolism are frequent complications in isolated PNH haematopoetic insufficiency defines clinical outcome in PNH combined with aplastic anaemia.