Quality of life in children with osteogenesis imperfecta treated with oral bisphosphonates (Olpadronate): a 2-year randomized placebo-controlled trial.
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.
Kok
Dieke H J
DH
Department of Orthopaedic Surgery, Wilhelmina Children's Hospital, University Medical Centre Utrecht, Utrecht, The Netherlands.
Sakkers
Ralph J B
RJ
Janse
Arieke J
AJ
Pruijs
Hans E H
HE
Verbout
Ab J
AJ
Castelein
Rene M
RM
Engelbert
Raoul H H
RH
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
D004164:Diphosphonates; C055774:olpadronic acid