Mega Dose Intravenous Methylprednisolone for Treatment of Malignant Osteopetrosis

Two infants with recessive (Malignant) osteopetrosis were treated with intravenous methylprednisolone (daily, 30 mg/kg for 3 days, 20 mg/kg for 4 days, then subsequently 10,5 and 2 mg/kg a week) followed by 1/mg/kg till the hemoglobin level reached 11 g/dl for 34 and 62 days. Treatment was continued with oral prednison (2.5 or 5 mg) for 37 and 34 months duration. The liver and spleen in both patients became almost normal in size with the elevation of the platelet count and hemoglobin level to normal with a decrease of reticulocyte count and normoblastemia, and needle aspiration showed their bone marrow to be normocellular. Plasma hemoglobin and alkaline phosphatase decreased to normal with the elevation of haptoglobin levels. Growth and development became fairly appropriate for their ages though both had macrocrania and there was exophtalmus in one. With the exception of Cushingoid appearance during mega dose administration they did not show any important side effects of corticosteroid such as hyperglycemia and growth retardation.