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低剂量帕米膦酸盐治疗重度脑瘫患儿骨质减少的初步研究。

Low doses of pamidronate to treat osteopenia in children with severe cerebral palsy: a pilot study.

作者信息

Plotkin Horacio, Coughlin Susan, Kreikemeier Rose, Heldt Kathryn, Bruzoni Matias, Lerner Gary

机构信息

Inherited Metabolic Diseases Section, Department of Pediatrics, University of Nebraska Medical Center, NE 68198-5456, USA.

出版信息

Dev Med Child Neurol. 2006 Sep;48(9):709-12. doi: 10.1017/S0012162206001526.

Abstract

The aim of this study was to test the efficacy of low doses of pamidronate in increasing bone mineral density (BMD) in non-ambulatory children and adolescents with cerebral palsy (CP). Twenty-three non-ambulatory children and adolescents (12 females, 11 males; mean age 10y [SD 5y], range 4y 1 mo-17 y 11 mo) with severe spastic quadriplegic CP and low BMD were recruited from a multidisciplinary clinic. Severity of CP was graded at Level IV (n=10) and Level V (n=13) using the Gross Motor Function Classification System. Patients received intravenous pamidronate (4.12 mg/kg/y, maximum 45 mg/d) every 4 months. Lumbar spine and femoral neck BMD were measured at baseline and after 4 and 12 months. Twelve months after the first dose of pamidronate there was a significant increase in lumbar spine and femoral neck BMD (p<0.01 for both sites) and z scores compared with baseline values (p<0.01 for both sites). Mean BMD z scores increased 1.6 points for femoral neck and 1.9 points for lumbar spine after 12 months of pamidronate treatment. Serum intact parathyroid hormone increased significantly and cross-linked N-teleopeptide of type I collagen decreased significantly at 12 months. No significant side effect was noted. Low doses of pamidronate are well tolerated and significantly increase BMD in non-ambulatory children and adolescents with CP.

摘要

本研究的目的是测试低剂量帕米膦酸盐对提高非行走型脑瘫(CP)儿童和青少年骨密度(BMD)的疗效。从一家多学科诊所招募了23名非行走型儿童和青少年(12名女性,11名男性;平均年龄10岁[标准差5岁],范围4岁1个月至17岁11个月),他们患有严重痉挛性四肢瘫脑瘫且骨密度较低。使用粗大运动功能分类系统将脑瘫严重程度分为IV级(n = 10)和V级(n = 13)。患者每4个月接受一次静脉注射帕米膦酸盐(4.12 mg/kg/年,最大45 mg/天)。在基线、4个月和12个月后测量腰椎和股骨颈的骨密度。首次注射帕米膦酸盐12个月后,与基线值相比,腰椎和股骨颈的骨密度显著增加(两个部位p<0.01),z评分也显著增加(两个部位p<0.01)。帕米膦酸盐治疗12个月后,股骨颈的平均骨密度z评分增加1.6分,腰椎增加1.9分。12个月时血清完整甲状旁腺激素显著增加,I型胶原交联N-端肽显著降低。未观察到明显副作用。低剂量帕米膦酸盐耐受性良好,可显著提高非行走型脑瘫儿童和青少年的骨密度。

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