Glucocorticoid-resistant Evans' syndrome successfully controlled with low-dose cyclosporine

To present a patient suffering from Evans' syndrome (ES), whose bouts of severe cytopenia were prevented by low-dose cyclosporine maintenance therapy.

A boy suffering from frequent mild respiratory infections, first time evaluated in a tertiary care pediatric center at age 4, was found to have lymphadenopathy and mild splenomegaly. The thrombocytopenia was first noted at age 6. He was diagnosed to have ES at the age of 8, during another bout of thrombocytopenia, this time associated with Coombs-positive hemolytic anemia. Immunoglobulin concentration in the plasma was measured repeatedly, and was in the normal range, or even increased. Lymphocyte subpopulation numbers were in the normal range, with decreased CD4+/ CD8+ ratio (0.6). Autoimmune lymphoproliferative syndrome was excluded by the absence of CD4-CD8- T lymphocytes. Since the patient failed to respond to standard therapy with prednisolon 2 mg/kg, high dose intravenous methylprednisolone (10 mg/ kg/d for 3 days) and high dose intravenous immunoglobulin (1 g/kg/d for 2 days), cyclosporine treatment was initiated (6 mg/ kg/d) and resulted in normalization of platelet count and resolution of hemolysis. Two attempts to withdraw cyclosporine therapy resulted in life-threatening hemolytic crisis with severe thrombocytopenia, requiring the re-institution of cyclosporine. The dose of cyclosporine was eventually tapered to the present 0.5 mg/kg, corresponding to drug serum levels of 5 - 8 mg/ml. The patient is now free of manifestations of Evans' syndrome but, after 20 years of cyclosporine treatment, has slightly impaired kidney function.

Low-dose cyclosporine therapy given to our patient appears to have subdued the autoimmune process thought to underlie the manifestations of ES, albeit at the cost of some toxicity to the kidneys.