Immunosuppressive treatment of aplastic anemia with antilymphocyte globulin, methylprednisolone, and cyclosporine appears to be more effective than a regimen of antilymphocyte globulin and methylprednisolone without cyclosporine and may thus represent a treatment of choice for patients who are not eligible for bone marrow transplantation.

Paroxysmal nocturnal haemoglobinuria (PNH) is now generally accepted as a disease in which bone marrow derived cells are deficient in phosphatidylinositolglycan (PIG)-anchored surface molecules. A series of new monoclonal antibodies detecting PIG-anchored surface structures on human leucocytes (CD48, CD55, CD59) has recently been described. In the present study 12 patients with the diagnosis PNH and a positive Ham test were examined for PIG-anchored surface antigen expression on various cell lineages using immunofluorescence. In all patients deficient cells were detected in erythrocyte, granulocyte and monocyte analysis. A deficient lymphocyte subset was also observed in all but one of these patients. Using two-colour analysis, all lymphocyte subpopulations such as T, B and NK cells were found to be affected. In addition, peripheral blood cells of 22 patients with severe aplastic anaemia (SAA) were tested for the PIG-anchoring defect. In five of these patients the defect was detected, and in four of the five the lack of PIG-anchored molecules was confined to the granulocyte and monocyte lineages apparently without affecting the erythrocytes. The results of these studies demonstrate that cytofluorographic testing of peripheral blood cells provides a simple and reliable method for establishing the diagnosis of PNH. Furthermore, especially in the case of aplastic anaemia patients, the sensitivity of immunophenotyping might be superior to conventional laboratory tests.

The introduction of immunosuppressive therapy for treatment of aplastic anemia has led to a considerable improvement in the prognosis of this disease. However, long-term follow-up of these patients showed a high incidence of “late” hematologic complications such as myelodysplasia and paroxysmal nocturnal hemoglobinuria (PNH). The detection of the glycosylphosphatitylinositol (GPI)-anchoring defect on peripheral blood cells of patients with aplastic anemia is now available as a new tool for early specific detection of PNH and is more sensitive than the Ham- test. Granulocytes appear to be the first cells affected in 11 patients with a GPI-anchoring defect of 29 suffering from aplastic anemia investigated in the present study. The later involvement of erythrocytes and a positive Ham test was observed in 1 patient. From our data it can be concluded that the rate of PNH resulting from aplastic anemia might be higher than reported in the literature when the Ham test alone was used for follow-up. Furthermore, our results suggest the clinical response to immunosuppressive therapy appears to be worse in the group developing the GPI-anchoring defect than in the group without this deficiency.

CASUS derivatives including δ CASUS have a good prognostic value for cardiac surgery patients with regard to the prediction of mortality and survival during ICU stay, with the exception of total CASUS which was not informative.