Effect of pamidronate treatment in children with polyostotic fibrous dysplasia of bone
Effect of pamidronate treatment in children with polyostotic fibrous dysplasia of bone
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DOI：
10.1210/jc.2003-030050
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发表时间：
2003-10-01
影响因子：
5.8
通讯作者：
Glorieux, FH
中科院分区：
文献类型：
--
作者：
Plotkin, H;Rauch, F;Glorieux, FH
静脉注射双膦酸盐化合物帕米膦酸盐可减少骨痛，据报道可导致成人纤维性骨发育不良（FD）的发育不良病变再填充。在这里，我们描述了这种治疗方法在18名患有多骨不全FD的儿童和青少年（治疗开始时年龄，6.2-17.5岁；8名女孩）中的效果，他们接受帕米膦酸钠治疗1.2-9.1年（中位数，3.8年）。帕米膦酸盐（1-1.5 mg/kg）治疗周期。D（连续3天）每4个月给予一次。血清碱性磷酸酶和尿I型胶原n端肽水平在基线时升高，在治疗的前3年持续下降。在任何患者中，没有溶解性病变充盈或病变周围骨皮质增厚的影像学证据。接受帕米膦酸钠治疗的患者（n = 7，治疗时间1.4-4.8年）骨组织发育不良的组织形态学结果与未接受药物治疗的患者（n = 9）相似。没有发现严重的副作用。综上所述，帕米膦酸盐治疗儿童和青少年多牙性FD是安全的。然而，我们没有发现明确的证据表明帕米膦酸盐对这类患者的发育不良病变有影响。
Intravenous infusions with the bisphosphonate compound pamidronate decrease bone pain and reportedly can lead to refilling of dysplastic lesions in adults with fibrous dysplasia (FD) of bone. Here we describe the effects of this treatment approach in 18 children and adolescents (age at start of therapy, 6.2-17.5 yr; eight girls) with polyostotic FD, who received pamidronate for 1.2-9.1 yr (median, 3.8 yr). Treatment cycles with pamidronate (1-1.5 mg/kg.d on 3 consecutive days) were given every 4 months. Levels of serum alkaline phosphatase and urinary collagen type I N-telopeptide were elevated at baseline and decreased continuously during the first 3 yr of therapy. There was no radiographic evidence of filling of lytic lesions or thickening of the bone cortex surrounding the lesions in any patient. Histomorphometric results in dysplastic bone tissue of patients receiving pamidronate (n = 7; time of therapy, 1.4-4.8 yr) were similar to those of patients without medical therapy (n = 9). No serious side effects were noted. In conclusion, pamidronate therapy appears to be safe in children and adolescents with polyostotic FD. However, we found no clear evidence that pamidronate has an effect on dysplastic lesions in such patients.