Vallo Alfredo, Rodriguez-Leyva Francisco, Rodríguez Soriano Juan
Division of Paediatric Nephrology, Department of Paediatrics, Hospital de Cruces and Basque University School of Medicine, Bilbao, Spain.
Acta Paediatr. 2006 Mar;95(3):332-9. doi: 10.1080/08035250500434785.
Administration of bisphosphonates represents a beneficial therapy in children and adolescents with severe osteogenesis imperfecta (OI) because it significantly reduces the annual rate of bone fractures.
To evaluate the anthropometric, skeletal and mineral metabolic effects of long-term intravenous pamidronate therapy in OI.
Ten patients, aged 5 mo to 25 y, with OI received cyclical intravenous pamidronate. The yearly dose of pamidronate was approximately 9 mg/kg/d at all ages. Duration of treatment varied from a minimum of 2 y to a maximum of 5 y. Growth, bone mass and mineral metabolic parameters were studied at baseline and repeated every year thereafter. Bone mass was assessed by calculation of bone mineral apparent density (L2-L4 BMAD). This represents the first study on the changes in size-adjusted measures of bone mass observed with such therapy.
While on therapy, all children and adolescents grew normally but did not experience any manifest catch-up growth. A significant decrease in the incidence of bone fractures was observed. In seven patients with severe forms, L2-L4 BMAD increased by 80% after the first 2 y of therapy but tended to stabilize or even decrease over the following years despite maintenance of therapy. A significant inverse correlation could be established between urinary Ca excretion and L2-L4 BMAD (r = -0.30, p < 0.05).
Our results confirm that cyclical pamidronate infusions reduce the incidence of bone fractures and allow normal growth. The improvement in bone mass initially observed after the first 2 y of therapy is not always sustained over the following years despite maintenance of therapy.
双膦酸盐给药对患有严重成骨不全（OI）的儿童和青少年是一种有益的治疗方法，因为它能显著降低每年的骨折发生率。
评估长期静脉注射帕米膦酸治疗OI对人体测量学、骨骼和矿物质代谢的影响。
10例年龄在5个月至25岁的OI患者接受了周期性静脉注射帕米膦酸治疗。所有年龄段的帕米膦酸年剂量约为9mg/kg/d。治疗持续时间从最短2年到最长5年不等。在基线时研究生长、骨量和矿物质代谢参数，此后每年重复研究。通过计算骨矿物质表观密度（L2-L4 BMAD）评估骨量。这是第一项关于这种治疗方法所观察到的骨量大小调整测量变化的研究。
在治疗期间，所有儿童和青少年生长正常，但未出现任何明显的追赶生长。观察到骨折发生率显著降低。在7例严重类型的患者中，治疗的前2年后L2-L4 BMAD增加了80%，但尽管维持治疗，在随后几年中趋于稳定甚至下降。尿钙排泄与L2-L4 BMAD之间可建立显著的负相关（r = -0.30，p < 0.05）。
我们的结果证实，周期性静脉注射帕米膦酸可降低骨折发生率并允许正常生长。尽管维持治疗，但在治疗的前2年后最初观察到的骨量改善在随后几年并不总是持续的。
