High-dose intravenous corticosteroid for a patient with Diamond-Blackfan syndrome refractory to classical prednisone treatment.

A 2.5-year-old boy with congenital pure red cell aplasia (PRCA) became unresponsive to conventional prednisone (2 mg/kg) treatment following a febrile period related to diphtheria-pertussis-tetanus (DPT) immunization and required transfusions. Following an administration of high-dose intravenous methylprednisolone, his bone marrow and hematological findings were completely normalized and continued so for 18 months after initiation and 9.5 months after discontinuation of the treatment. When his anemia relapsed, following an upper respiratory infection, he was again found refractory to conventional prednisone administration but responded promptly to intravenous bolus methylprednisolone treatment.