Efficacy of low dose schedule pamidronate infusion in children with osteogenesis imperfecta.
BACKGROUND
Osteogenesis imperfecta (OI) is a rare condition in which bones are abnormally brittle with frequent fractures. A variety of therapeutic agents has been used with low efficacy. In this study, we present three patients treated for 4 years with i.v. pamidronate.


METHODS
Three prepubertal patients, aged 9 (M), 9 (F) and 11 (F) years old, with OI, were treated with 30-60 mg i.v. pamidronate every 6 months over four years. Determinations were made of plasma 1,25-dihydroxycholecalciferol, 25-hydroxycholecalciferol, insulin-like growth factor-I (IGF-I) and its transport protein (IGFBP3), osteocalcin, total alkaline phosphatase and its osseous fraction, and parathormone (PTH) at baseline and after every pamidronate infusion, Densitometry and X-ray of the vertebral column were performed at the same intervals.


RESULTS
Significant reductions of number of bone fractures and pain were observed in all patients, despite lack of any modification in biochemical parameters. Lumbar X-ray and densitometry showed a striking improvement by the end of the treatment period.


CONCLUSIONS
Pamidronate seems to be useful in the treatment of patients with osteogenesis imperfecta.
González
E
E
Department of Pediatrics, Unidad Integrada Hospital Sant Joan de Déu-Hospital Clínic, Universidad de Barcelona, Spain. 7129cps@comb.es
Pavía
C
C
Ros
J
J
Villaronga
M
M
Valls
C
C
Escolá
J
J
D015502:Absorptiometry, Photon; D015519:Bone Density / Q000187:drug effects; D002648:Child; D004164:Diphosphonates / Q000008:administration & dosage* / Q000627:therapeutic use; D004305:Dose-Response Relationship, Drug; D005260:Female; D050723:Fractures, Bone / Q000517:prevention & control; D006801:Humans; D007262:Infusions, Intravenous; D008159:Lumbar Vertebrae / Q000378:metabolism; D008297:Male; D010013:Osteogenesis Imperfecta / Q000188:drug therapy* / Q000378:metabolism; D010166:Palliative Care; D000077268:Pamidronate
D004164:Diphosphonates; D000077268:Pamidronate