Background: Bisphosphonate treatment for bone fragility has expanded beyond the children with osteogenesis imperfecta (OI) to those with other causes of low bone mass. Pamidronate is effective such as Pagets disease, hypercalcaemia of malignancy, osteolytic bone metastasis, steroid-induced osteoporosis and idiopathic osteoporosis.
Objective and hypotheses: The experience with bisphosphonates treatment other than OI in children is limited although there are a growing number of publications showing their usefulness in several bone and metabolic diseases. Our objective is to demonstrate the efficacy of pamidronate other than OI.
Methods: The i.v. administration of pamidronate in children in our institute is analyzed: 2 McCuneAlbright (MCA), 1 Tripple A syndrome (TA), 1 glycogene storage disease (GSD) type 0. Pamidronate 0.5 mg/kg per day, 3-consecutive-days were given for 3 months interval (6 mg/kg per years).
Results: We have two cases (19 and 5 old ages) of fibrous dysplasia due to MCA who had severe bone pain showing remarkable clinical improvement with pamidronate. We used pamidronate treatment 18-aged-male with TA syndrome that had osteoporosis (BMD-Z score was −3.0) and severe back pain. His BMD-Z score was −2.08 after the treatment. We also treated nine aged-girl with GSD type 0, who had osteopenia (BMD-Z score −1.2 end of the therapy Z score was: −0.5) and chronic bone pain because of restricting diary product in her diet. All patients had normal levels of calcium, phosphorus, and vitamin D, and proper nutrient intake. No advers effects of pamidronate treatment were identified and subjective skeletal pain diminished in all patients. All children had a positive response to the treatment, with rapid and marked clinical improvement in their mobility.
Conclusion: Our experience with the use of bisphosphonates in pediatric patients with diseases other than OI. Intravenous bisphosphonates are well tolerated, and reduce the risk of fracture and ameliorate bone related clinical symptoms.
20 - 22 Sep 2014
European Society for Paediatric Endocrinology