title: [Medical treatment of children with hypophosphataemic rickets].
abstract: Hypophosphataemic rickets is a rare, genetic disorder resulting in defect bone mineralisation and rickets. The current medical treatment consists of phosphate supplementation and alfacalcidol, but side effects such as secondary hyperparat-hyroidism and nephrocalcinosis are common. This treatment regimen often fails to prevent bone deformity and reduced final height. The rarity and complexity of these diseases call for centralised specialist care and international collaboration. Future medical treatment may be improved by addition of new promising experimental treatments.
authors.affiliation: H.C. Andersen Børnehospital, Odense Universitetshospital, Sdr. Boulevard 29, 5000 Odense C. henrik.christesen@rsyd.dk.
authors.forename: Elise Torp
authors.initials: ET
authors.lastname: Rahbek
authors.forename: Line Hougaard
authors.initials: LH
authors.lastname: Nielsen
authors.forename: Signe Sparre
authors.initials: SS
authors.lastname: Beck-Nielsen
authors.forename: Henrik Thybo
authors.initials: HT
authors.lastname: Christesen
mesh_terms: D050071:Bone Density Conservation Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D002648:Child; D000069449:Cinacalcet / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D000089703:Fibroblast Growth Factor-23; D005346:Fibroblast Growth Factors / Q000037:antagonists & inhibitors / Q000097:blood / Q000378:metabolism; D040181:Genetic Diseases, X-Linked; D006801:Humans; D006887:Hydroxycholecalciferols / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D009842:Oligopeptides / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D010710:Phosphates / Q000008:administration & dosage / Q000009:adverse effects / Q000097:blood / Q000627:therapeutic use; D011480:Protease Inhibitors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D011859:Radiography; D063730:Rickets, Hypophosphatemic / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000503:physiopathology
chemical_list: D050071:Bone Density Conservation Agents; D006887:Hydroxycholecalciferols; D009842:Oligopeptides; D010710:Phosphates; D011480:Protease Inhibitors; C000597783:hexa-D-arginine; D005346:Fibroblast Growth Factors; D000089703:Fibroblast Growth Factor-23; D000069449:Cinacalcet; C008088:alfacalcidol