IRANIAN JOURNAL OF PEDIATRICS, cilt.22, sa.3, ss.421-424, 2012 (SCI-Expanded)
Background: Malignant infantile osteopetrosis (MIOP) presents early in life with extreme sclerosis of the reduction of bone marrow spaces. Since there is a defect in the bone marrow, the disease can cause anemia, extramedullary hematopoiesis secondary to anemia leading to hepatosplenomegaly, cranial nerves compression and severe growth failure. This disorder is often lethal within the first decade of life because of secondary infections. Stem cell transplantation (SCT) remains the only curative therapy.