title: Quality of life in children with osteogenesis imperfecta treated with oral bisphosphonates (Olpadronate): a 2-year randomized placebo-controlled trial.
abstract: In this double-blind randomised placebo-controlled trial it was investigated during a two-year follow-up whether oral bisphosphonates (Olpadronate 10 mg/m2/day) influence quality of life in children with osteogenesis imperfecta (OI). Thirty-four children with OI (classified according to Sillence criteria), aged 3 to 18 years of age, with a restricted level of ambulation were included. Randomisation was performed using a list of computer generated random numbers to allocate patients to receive Olpadronate or placebo. Quality of life was measured using self-perception profile for children (SPPC) and health-utility index (HUI). Differences between baseline measurements and measurements at two years follow-up were analysed within the Olpadronate and placebo group using a student's t-test. Differences in HUI and SPPC regression coefficients were analysed by random-effects repeated measures analysis (SAS, Proc Mixed, version 8.2), adjusted for age, gender and type of OI. Within the Olpadronate group there was a significant decrease in pain utility; however, difference in six months' regression coefficients between the placebo and Olpadronate group were not significant. Within the placebo group there was a significant increase in scholastic competence and behavioural conduct. The item behavioural conduct showed a steeper annual regression coefficient favourable for the placebo group. In the other SPPC items none of the annual regression coefficients showed a significant difference between the Olpadronate and the placebo group.
  
  
  CONCLUSIONS
  We found only slight differences in quality of life in favour of the bisphosphonate group. A small but not significant decrease in pain was detected in the bisphosphonate group.
authors.affiliation: Department of Orthopaedic Surgery, Wilhelmina Children's Hospital, University Medical Centre Utrecht, Utrecht, The Netherlands.
authors.forename: Dieke H J
authors.initials: DH
authors.lastname: Kok
authors.forename: Ralph J B
authors.initials: RJ
authors.lastname: Sakkers
authors.forename: Arieke J
authors.initials: AJ
authors.lastname: Janse
authors.forename: Hans E H
authors.initials: HE
authors.lastname: Pruijs
authors.forename: Ab J
authors.initials: AJ
authors.lastname: Verbout
authors.forename: Rene M
authors.initials: RM
authors.lastname: Castelein
authors.forename: Raoul H H
authors.initials: RH
authors.lastname: Engelbert
mesh_terms: D000124:Achievement; D000284:Administration, Oral; D002648:Child; D004164:Diphosphonates / Q000008:administration & dosage / Q000627:therapeutic use*; D004311:Double-Blind Method; D006801:Humans; D010013:Osteogenesis Imperfecta / Q000188:drug therapy* / Q000473:pathology; D011594:Psychometrics; D011788:Quality of Life; D012649:Self Concept; D012720:Severity of Illness Index; D012963:Sociometric Techniques; D011795:Surveys and Questionnaires
chemical_list: D004164:Diphosphonates; C055774:olpadronic acid