title: The effect of cyclical intravenous pamidronate in children and adolescents with osteogenesis imperfecta type V.
abstract: Intravenous treatment with pamidronate is beneficial in children and adolescents with moderate to severe forms of osteogenesis imperfecta (OI) types I, III and IV, but there is little information on the effects of this treatment on the newly described OI type V. Here, we describe the results of 2 years of pamidronate treatment in 11 children and adolescents with OI type V (age at start of therapy 1.8 to 15.0 years; 6 girls). Pamidronate was given in intravenous cycles at a cumulative yearly dose of 9 mg/kg. The first infusion cycle was associated with fever and mild hypocalcemia in most patients, but no other short-term side effects were noted. Two years of pamidronate treatment led to a decrease in the urinary excretion of N-terminal telopeptide of type I collagen to 50% of baseline levels. Both the size and volumetric bone mineral density of lumbar vertebrae increased compared to age- and sex-matched reference data (P < 0.05 in both cases). Histomorphometry of transiliac bone samples showed an average increase of 86% in cortical thickness (N = 7; P = 0.005). No significant changes with treatment were observed in the age-related z scores of isometric maximal grip force and height. Fracture incidence decreased from 1.5 fractures per year before treatment to 0.5 fractures per year during the fist 2 years of treatment. Ambulation status improved in four patients and remained unchanged in the others. In conclusion, the intravenous pamidronate therapy has a similar effect in OI type V as it has in the other OI types.
authors.affiliation: Genetics Unit, Shriners Hospital for Children and McGill University, 1529 Cedar Avenue, Montréal, Québec, Canada H3G 1A6.
authors.forename: Leonid
authors.initials: L
authors.lastname: Zeitlin
authors.forename: Frank
authors.initials: F
authors.lastname: Rauch
authors.forename: Rose
authors.initials: R
authors.lastname: Travers
authors.forename: Craig
authors.initials: C
authors.lastname: Munns
authors.forename: Francis H
authors.initials: FH
authors.lastname: Glorieux
mesh_terms: D000293:Adolescent; D000469:Alkaline Phosphatase / Q000097:blood; D015519:Bone Density / Q000187:drug effects; D050071:Bone Density Conservation Agents / Q000008:administration & dosage* / Q000494:pharmacology; D002118:Calcium / Q000097:blood; D002170:Canada / Q000453:epidemiology; D002648:Child; D002675:Child, Preschool; D004164:Diphosphonates / Q000008:administration & dosage* / Q000494:pharmacology; D005260:Female; D006801:Humans; D007223:Infant; D007275:Injections, Intravenous; D008297:Male; D010013:Osteogenesis Imperfecta / Q000145:classification* / Q000188:drug therapy*; D000077268:Pamidronate; D010281:Parathyroid Hormone / Q000097:blood; D010710:Phosphates / Q000097:blood; D012189:Retrospective Studies; D014807:Vitamin D / Q000031:analogs & derivatives / Q000097:blood
chemical_list: D050071:Bone Density Conservation Agents; D004164:Diphosphonates; D010281:Parathyroid Hormone; D010710:Phosphates; D014807:Vitamin D; C097949:1,25-dihydroxyvitamin D; D000469:Alkaline Phosphatase; D000077268:Pamidronate; D002118:Calcium