title: Recombinant osteoprotegerin for juvenile Paget's disease.
abstract: Juvenile Paget's disease, a genetic bone disease characterized by accelerated bone turnover, results from inactivating mutations in the gene encoding osteoprotegerin--a key regulator of osteoclastogenesis. The effects of recombinant osteoprotegerin were investigated in two adult siblings with juvenile Paget's disease. Bone resorption (assessed by N-telopeptide excretion) was suppressed by once-weekly subcutaneous doses of 0.3 to 0.4 mg per kilogram of body weight. After 15 months of treatment, radial bone mass increased in one patient by 9 percent and in the other by 30 percent, skeletal bisphosphonate retention decreased by 37 percent and 55 percent, respectively, and there was radiographic improvement. Apart from mild hypocalcemia and hypophosphatemia, no apparent adverse events occurred.
authors.affiliation: Department of Medicine, Faculty of Medical and Health Sciences, University of Auckland, Auckland, New Zealand.
authors.forename: Tim
authors.initials: T
authors.lastname: Cundy
authors.forename: James
authors.initials: J
authors.lastname: Davidson
authors.forename: Michael D
authors.initials: MD
authors.lastname: Rutland
authors.forename: Carolyn
authors.initials: C
authors.lastname: Stewart
authors.forename: Alex M
authors.initials: AM
authors.lastname: DePaoli
mesh_terms: D000328:Adult; D000906:Antibodies / Q000097:blood; D015415:Biomarkers / Q000097:blood; D015519:Bone Density / Q000187:drug effects; D001862:Bone Resorption / Q000188:drug therapy; D005260:Female; D006023:Glycoproteins / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D006801:Humans; D006811:Humerus / Q000000981:diagnostic imaging; D008297:Male; D010001:Osteitis Deformans / Q000188:drug therapy* / Q000235:genetics; D053244:Osteoprotegerin; D011859:Radiography; D011884:Radius / Q000000981:diagnostic imaging / Q000187:drug effects; D018160:Receptors, Cytoplasmic and Nuclear / Q000276:immunology / Q000627:therapeutic use*; D018124:Receptors, Tumor Necrosis Factor / Q000276:immunology / Q000627:therapeutic use*; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use
chemical_list: D000906:Antibodies; D015415:Biomarkers; D006023:Glycoproteins; D053244:Osteoprotegerin; D018160:Receptors, Cytoplasmic and Nuclear; D018124:Receptors, Tumor Necrosis Factor; D011994:Recombinant Proteins; C506030:TNFRSF11B protein, human