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Please note the correction in the following article from our current newsletter:
FROM THE TRENCHES: TWO THERAPEUTIC CONSIDERATIONS FOR INDIVIDUALS WITH FAMILIAL DYSAUTONOMIA
by Suzanne Adler, MD
BISPHOSPHONATES:
One of the observations made at the spinal surgery of some children with FD is that their bones are softer than expected. Also, individuals with FD are more prone to repeated fractures or bone breaks, some of which go unrecognized because the associated pain is very mild. Over a long period of time, this can lead to permanent bone problems. A family of commonly used medications has been used with good effect in individuals with similar problems. This class of drug, called bisphosphonates, has been studied in children with a fragile bone disease called osteogenesis imperfecta, in diabetics who have nerve and blood vessel damage (which can cause bone and joint problems in the feet), in people with osteoporosis, and in a condition called reflex sympathetic dystrophy. The effect of bisphosphonates is to preserve the integrity of bone and to reduce the risk of further fractures and bone damage.
One of the bisphosphonates is called Pamidronate or APD. Pamidronate can be given intravenously over a few hours (note: not as a daily procedure as originally written). Our experience with one child with FD suggests that this drug may be helpful in familial dysautonomia as well. This case, an eleven year old prepubertal child with FD, who had a number of stress fractures around the knees and feet, was having frequent episodes of pain on the bony areas around the body, lasting a day or so. He received intravenous Pamidronate, (initially given every three months (note: not three per month as originally written) for about one year and then less frequently, depending on his bone pains). Testing performed before and during treatment included measurement of vitamin D and basic biochemical tests. All remained normal. A DEXA bone density scan was performed as a baseline and was found to be a little low for age. Following treatment, there was a rapid disappearance of the bone pains and this benefit lasted about three months after each infusion. The child has had no further major stress fractures and the bone density is improving. Pamidronate was not used for the effect on bone density but rather for symptom relief and the hope of preventing further bone damage.
Treatment with Pamidronate may be a therapeutic approach to bone pain in FD and it requires further study. Such treatment would be best done by a pediatric endocrinologist with access to a Bone Density Unit that has the expertise to interpret children’s bone density studies. (The analysis must take into account the size of the child and the pubertal status; the standard age-matched tables are inadequate.)
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