Pamidronate Treatment of Bone Fibrous Dysplasia in Nine Children with McCune-Albright Syndrome, Acta Paediatr, 89:188-93 2000, R. Lala, P. Matarazzo, S. Bertelloi, F. Buzi, F. Rigaon, C de Sanctis
This was a study of nine patients with moderate to severe fibrous dysplasia who were treated with intravenous pamidronate. They were treated over a period of six months to three and a half years. Bone pain and gait abnormality due to pain disappeared after two or three cycles of the drug treatment. Cranial asymmetry and limb length differences remained unchanged. An increase in bone density was also reported. Elevated levels of alkaline phosphatase and urine hydroxyproline were reduced by the treatment. Long term implications are unknown.
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Long Term Effects of Intravenous Pamidronate in Fibrous Dysplasia of Bone, Journal of Bone and Mineral Research, Vol. 12 Number 10, 1997, Roland D. Chapurlat, Pierre D. Delmas, Daniel Liens, Pierre J. Meunier
The aim of this study was to assess the long tern effects of intravenous pamidronate in FD. The severity of bone pain and the number of painful sites appears to be significantly reduced. Al biochemical markers of bone remodeling were substantially lowered. They observed a radiographic response in 9 (of 20) patients with refilling of osteolytic lesions. A mineralization defect was observed in one case and four had stress lines but no fractures.
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Long Term Effects of Intravenous Pamidronate in Fibrous Dysplasia of Bone, The Lancet, Vol. 343, April 16, 1994, Daniel Liens, Pierre D. Delmas, Pierre J. Meunier
Nine patients with FD were treated with intravenous pamidronate and were followed for 18-48 months. The major effect was decreased bone pain. Positive radiological changes were seen in four patients.
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Effect of Pamidnodrate Treatment in Children with Polyostotic Fibrous Dysplasia of Bone, The Journal of Clinical Endocrinology & Metabolism, 88(10):4589-4575, 2003, Horacio Plotkin, Frank Rauch, Leonid Zeitlin, Craig Munns, Rose Travers, And Francis H. Glorieux
Report from a Canadian study of 18 children and adolescents with FD. Patients had bone biopsies before and after treatment with high dose pamidronate. Levels of serum alkaline phosphatase and urinary collagen type I N-telopeptide decreased continuously over the first three years of the study. There was no radiographic evidence of filling of lesions or thickening of the bone cortex surrounding the lesions in any patient. The study concluded that pamidronate therapy appears to be safe for children and adolescents, but there was no clear evidence that pamidronate had any positive effect on dysplastic lesions. This is perhaps the best study to date, in terms of the quality of the design of the study, on the use of bisphosphonates in FD.
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Pamidronate treatment of polyostotic Fibrous Dysplasia: Failure to Prevent Expansion of Dysplastic Lesions During Childhood, Journal of Pediatric Endocrinology and Metabolism, 19: 75-80, 2006, Chan, Brendan and Zacharin, Margaret.
This study followed 3 male children with MAS and polyostotic FD – starting ages 2.5-5 – while they received treatments with biophosphonates (pamidronate). The period of treatment lasted 8-10.5 years. The authors found that the treatment controlled pain effectively, but it did not reduce or limit the size of dysplastic lesions in the children’s bone.
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Pegvisomant for Treatment of Growth Hormone Excess in MAS, Journal of Clinical Endocrinology and Metabolism, 91 (8): 2960-2966, 2006, Sunday Akintoye, et. al.
This article examined 5 patients with fibrous dysplasia with an excess in Growth Hormone (GH). Participants were given pegvisomant, a GH receptor antagonist for a 12 week period. It is an antagonist that was expected to block the binding of certain proteins, decreasing GH levels in the blood. The pegvisomant effectively lowered IGF-1 & IGFBP-3 levels which are binding proteins, essentially lowering the level of GH in the blood. Unfortunately, pegvisomant did not resolve other factors linked to individuals with FD and GH excess, such as fatigue and sweating.
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