title: Osteopetrosis. The pharmaco-physiologic basis of therapy.
abstract: Medical treatments of osteopetrosis have attempted to improve hematologic function, reduce the osteosclerotic condition, and/or improve immune function. Prednisone therapy has improved hematologic function in some patients, but has not resulted in a reduction in bone mass. Calcium deficient diets have limited further sclerosis in some patients. High-dose calcitriol and parathormone infusions have stimulated osteoclastic activity. In some patients, high-dose calcitriol has resulted in clinical improvement. Newer treatments, such as interferon gamma and macrophage colony stimulating factor, may alter the osteoclastic and immune defects by stimulating cellular formation and function. These therapies, alone or in combination, ameliorate but do not cure the osteopetrotic condition.
authors.affiliation: Department of Pediatrics, Brenner Children's Hospital, Bowman Gray School of Medicine, Wake Forest University, Winston-Salem, North Carolina.
authors.forename: L L
authors.initials: LL
authors.lastname: Key
authors.forename: W L
authors.initials: WL
authors.lastname: Ries
mesh_terms: D000276:Adjuvants, Immunologic / Q000627:therapeutic use; D002117:Calcitriol / Q000627:therapeutic use; D002136:Calcium, Dietary; D003131:Combined Modality Therapy; D004359:Drug Therapy, Combination; D006801:Humans; D016173:Macrophage Colony-Stimulating Factor / Q000627:therapeutic use; D010010:Osteoclasts / Q000502:physiology; D010022:Osteopetrosis / Q000188:drug therapy* / Q000276:immunology / Q000503:physiopathology; D011241:Prednisone / Q000627:therapeutic use
chemical_list: D000276:Adjuvants, Immunologic; D002136:Calcium, Dietary; D016173:Macrophage Colony-Stimulating Factor; D002117:Calcitriol; D011241:Prednisone