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Osteogenesis imperfecta (OI) is a chronic, disabling condition in which treatment with cyclical intravenous treatment with pamidronate can be useful for symptom relief,1 despite questions about long term safety.2 A recent study3 in this journal showed a decrease in bone turnover and gradual increase in bone density measurements without significant side effects, following such treatment in children affected with OI.
We wish to report our own experience in 10 children with OI who have received cyclical intravenous pamidronate (1 mg/kg/day for three days every three months). The median (range) age at the start of treatment was 9.1 (1.3–12.7) years. Treatment was initiated in the context of symptoms having an adverse effect on the quality of life, associated with evidence of decreased bone density assessed by dual energy x ray absorptiometry (DXA scan, QDR1000/W, Hologic systems, Boston, Massachusetts).4
Five of these children were at the severe end of the symptom spectrum (recurring pain, multiple fractures, and impaired mobility). Four children were treated for pain and fractures, whereas one received pamidronate for pain only. After 1.8 (0.9 to 3.0) years of treatment, nine children were pain free. Four children had had no further fractures and one child had improved mobility. The initial infusion of pamidronate was associated with flu like symptoms, fever, rigors, abdominal pain, or vomiting in six children. Serum calcium levels were low (<2.2 mmol/l) following therapy in six subjects, and three required treatment with calcium and vitamin D supplements.
Repeat DXA scans showed an increase in lumbar spine bone mineral content (BMC) standard deviation score (SDS) (fig 1), from −3.44 (−6.6 to −1.39) to −0.96 (−3.10 to 3.13) SDS following 1.3 (0.7 to 2.7) years of pamidronate treatment. This beneficial response to treatment was similar to that reported elsewhere.3
In conclusion, our findings add to those of others that intravenous pamidronate infusion improves bone mineral density and reduces symptoms of severe pain, recurrent fractures, and impaired mobility in children with OI. There are minor acute side effects to the treatment, but long term safety needs to be determined.
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