Bisphosphonates to treat osteopenia in children with quadriplegic cerebral palsy: a randomized, placebo-controlled clinical trial.

OBJECTIVE

To evaluate in a double-blind, placebo-controlled clinical trial the safety and efficacy of intravenous pamidronate to treat osteopenia in nonambulatory children with cerebral palsy.

STUDY DESIGN

Six pairs of subjects generally matched within each pair for age, sex, and race completed the protocol. One member of each pair randomly received plain saline placebo, the other pamidronate. Drug/placebo was administered intravenously daily for 3 consecutive days, and this 3-day dosing session was repeated at 3-month intervals for one year. Evaluations were continued for 6 months after the year of treatment. Bone mineral density (BMD) was measured in the distal femur, a site specifically developed for use in this contracted population, and the lumbar spine.

RESULTS

In the metaphyseal region of the distal femur, BMD increased 89% +/- 21% (mean +/- SEM) over the 18-month study period in the pamidronate group compared with 9% +/- 6% in the control group. Age-normalized z scores increased from -4.0 +/- 0.6 to -1.8 +/- 1.0 in the pamidronate group and did not significantly change in the control group (-4.2 +/- 0.3 to -4.0 +/- 0.3). The first dosing with pamidronate caused a transient drop in serum calcium that was asymptomatic and not treated. No other potentially adverse effects were noted.

CONCLUSIONS

In this small controlled clinical trial, pamidronate was found to be a safe and very effective agent to increase BMD in nonambulatory children with cerebral palsy.