HIGH-DOSE INTRAVENOUS METHYLPREDNISOLONE THERAPY FOR PATIENTS WITH DIAMOND-BLACKFAN ANEMIA REFRACTORY TO CONVENTIONAL DOSES OF PREDNISONE

Objectives: To assess the efficacy and toxicity of very high doses of glucocorticoids in patients with congenital pure red cell aplasia (Dia mond-Blackfan anemia) who did not respond to standard doses of prednis one. Study designs: We prospectively treated eight patients with trans fusion-dependent Diamond-Blackfan anemia with high intravenous doses o f methylprednisolone, All patients had previously not responded to one or more oral courses of prednisone in standard doses and were depende nt on erythrocyte transfusions, Every patient initially received methy lprednisolone at a dose of 30 mg/kg per day, followed by slow tapering for 4 weeks, but none responded, All patients then received a second treatment course starting at 100 mg of methylprednisolone per kilogram per day, again followed by slow tapering of the dosage. Results: Thre e patients had a complete response that has been sustained for 21+, 31 +, and 41+ months, respectively. One patient had a partial response, T oxic effects included a rise in serum alanine aminotransferase activit y in all patients, transient diabetes mellitus in one child, and three episodes of bacteremia in two patients with intravenous access device s. Conclusions: We conclude that very high doses of methylprednisolone may induce sustained remission in some patients with transfusion-depe ndent Diamond-Blackfan anemia refractory to standard-dose prednisone t herapy.