Radiography of successful bone marrow transplantation for osteopetrosis.

Hematopoietic stem cell transplantation (HSCT) is the only curative therapy for autosomal recessive infantile malignant osteopetrosis, an otherwise fatal disease. HSCT has also been utilized in patients with the less severe autosomal recessive intermediate form in an attempt to correct hematologic abnormalities and halt cranial nerve deficits caused by progressive cranial foraminal stenosis. Successful engraftment is accompanied by rapid normalization of radiographic signs of osteopetrosis.