[Bisphosphonates and other new therapeutic agents for the treatmednt of osteogenesis imperfecta].
Osteogenesis imperfecta (OI) is a genetic disorder characterized by fragile bone and reduced bone mineral density. Cyclic intravenous pamidronate is now the standard treatment for moderate to severe forms of OI, however clinical studies are not yet sufficient to conclude appropriate annual doze and ideal duration of therapy at present time. Oral alendronate is also effective in milder forms of OI. Zoledronic acid has undergone international multicentric clinical trials to examine efficiency and long-term side effects including osteonecrosis of the jaw. Teriparatide (rhPTH1-34) and denosumab (monoclonal antibody against RANK ligand) have the potential for management of OI. Stem cell and gene therapy are currently being actively investigated and may become clinically applicable in the near future.
Yamashita
Sumie
S
Osaka Koseinenkin Hospital, Department of Pediatrics.
D019386:Alendronate / Q000008:administration & dosage*; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D061067:Antibodies, Monoclonal, Humanized; D050071:Bone Density Conservation Agents / Q000008:administration & dosage*; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D000069448:Denosumab; D004164:Diphosphonates / Q000008:administration & dosage* / Q000009:adverse effects; D004334:Drug Administration Schedule; D015316:Genetic Therapy; D006801:Humans; D007093:Imidazoles / Q000008:administration & dosage* / Q000009:adverse effects; D007262:Infusions, Intravenous; D010013:Osteogenesis Imperfecta / Q000145:classification / Q000235:genetics / Q000628:therapy*; D000077268:Pamidronate; D053245:RANK Ligand / Q000627:therapeutic use; D012720:Severity of Illness Index; D033581:Stem Cell Transplantation; D019379:Teriparatide / Q000627:therapeutic use; D000077211:Zoledronic Acid
D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D050071:Bone Density Conservation Agents; D004164:Diphosphonates; D007093:Imidazoles; D053245:RANK Ligand; D019379:Teriparatide; D000069448:Denosumab; D000077211:Zoledronic Acid; D000077268:Pamidronate; D019386:Alendronate