High-dose intravenous methylprednisolone for constitutional pure red cell aplasia.

I read with interest the recent article by Lenarsky et al’ about bone marrow transplantation (BMT) for Diamond-Blackfan syndrome. I reported a case of constitutional pure red cell aplasia (CPRCA) refractory to conventional prednisone (2 mg/kg) administration treated with high-dose intravenous (IV) methylprednisolone (HIVMP) (30 mg/kg for 3 days, 20 mg/kg for 4 days, then subsequently 10, 5, and 2 mg/kg for a week each, followed by 1 mg/kg until hemoglobin level reached 12 g/dL; each dose was given for 2 to 5 minutes).2 Eight other patients with Diamond-Blackfan syndrome resistant to conventional prednisone administration have also been treated with HIVMP successfully.3’4 Three of the infants died of causes not related to their disease (one died with a viral infection when he had been off treatment >2 months, and another died of pneumonia when his treatment was discontinued by the parents at home; the third infant died of pneumonia in the hospital); others are hematologically normal and do not require transfusion for