Treatment of Poorly Developed Bones (Osteopenia) and Prevention of Fractures, 4/2003

Children with cerebral palsy can have poorly calcified bones due to a developmental defect (e.g. hormone deficiency), nutritional inadequacy (calcium; vitamin D), insufficient use (e.g. non-ambulatory), or a medical drug effect (e.g. anticonvulsant). As a result, non-ambulatory children with CP often have poor bone density (osteopenia) and many will have bone fractures secondary to minor injuries. Fractures diminish the quality of life of the children and add to their care requirements.

A drug (bisphosphonates) has been approved by the FDA to treat loss of minerals in elderly patients who have suffered bone mineral loss. Will the same drug help enrich the mineral content of bone in children with 'thin bones' due to cerebral palsy?

With funds from the UCP Research and Educational Foundation, Dr. Richard E. Henderson and his colleagues¹ have completed a pilot study to address this question. Six pairs of children with quadriplegic cerebral palsy were the subjects of the study. One child in each pair received the drug (IV Pamidronate); the other a placebo. They were treated at three month intervals for one year and then followed for an additional six months. All children also received supplements of calcium, multi-vitamins and vitamin D. Bone mineral density was measured in two bone sites on each child.

The treatment was found to be 'a safe and very effective agent to increase bone mineral density in non-ambulatory children with quadriplegic cerebral palsy' as compared to little change in the children not receiving the drug.

Comment:
This is a very exciting funding in that bone fractures are a common and debilitating occurrence in non-ambulatory children with cerebral palsy. The pilot study demonstrates that the use of a safe and available drug may increase bone density and prevent fractures in these children. However, although very well done, this was a small study and now needs to be done in a larger group of children in order to make certain of its results. The NIH has provided Dr. Henderson with the funds to plan this larger study.

The Foundation is pleased to have made the 'risk investment.' It will probably result in a much needed and relatively easily provided method for preventing bone fractures in non-ambulatory children with disabilities due to cerebral palsy.

¹ Henderson, R.C. et. al. Bisphosphonates to Treat Osteopenia in Children with Quadriplegic Cerebral Palsy: A Randomized Placebo-controlled Clinical Trial. J. Pediatrics 2002; 141:644-651