Tolerance and effectiveness on pain control of Pamidronate® intravenous infusions in children with neuromuscular disorders.

UNLABELLED: Osteoporosis is a common complication in children with motor impairments. They have a higher risk of fractures (20% during their lifetime) mostly at femoral level. Furthermore, these children have pain yet no clear relation has been established between osteoporosis and pain. The efficacy of bisphosphonates has been validated in adults and for children with osteogenesis imperfecta (OI). However, its use in children with motor impairments has not yet been validated.

PATIENTS AND

METHODS

Retrospective study on the medical charts of children presenting neurological diseases and motor impairments associated to secondary symptomatic osteoporosis. These children underwent treatment with Pamidronate(®) intravenous infusions (I.V.) in Lyon and Valence between 2002 and 2008. Data were collected on pain control, incidence and frequency of fractures and bone mass density (BMD). Data on adverse events were also collected to evaluate treatment's tolerance.

RESULTS

Twelve children's charts were studied for a total of 50 Pamidronate(®) I.V. infusions. Regarding treatment's efficacy, we observed a clear decrease and even total relief of the pain with improvement reported after 98% of perfusions. Regarding BMD, there was a real improvement after the treatment (e.g., lumbar BMD measures, -46.5% before treatment and -27% after treatment). The adverse events, flu-like syndrome, muscle pain and asymptomatic hypocalcemia, were minor and quickly reversible.

CONCLUSION

It seems quite essential to screen for osteoporosis-related pain in these children and treat them quickly to avoid a negative impact on their quality of life. Treatment with I.V. bisphosphonates has shown its relevance, yet practical modalities still need to be defined. It would be interesting but quite difficult to implement, in light of the positive effect of this study, a prospective, randomized, controlled, double-blind vs. placebo study on a large enough sample of patients.