Long-Term Treatment of Osteopetrosis with Recombinant Human Interferon Gamma

Congenital osteopetrosis is a group of disorders resulting in decreased osteoclastic function and hence decreased bone resorption. The accumulation of sclerotic bone compromises marrow space and cranial-nerve foramina and predisposes patients to pathologic fractures. Most patients become blind or anemic before six months of age and die because of poor resistance to infection, neurologic deficits, or bone marrow failure during the first decade — often the first two years — of life. 1 – 4 Bone marrow transplantation is curative therapy, 5 but an acceptable donor can be found for only about 40 percent of patients. Overall survival 23 months after transplantation was . . .