Intravenous pamidronate treatment of infants with severe osteogenesis imperfecta
Intravenous pamidronate treatment of infants with severe osteogenesis imperfecta
复制标题
DOI：
10.1136/adc.2006.096552
复制
发表时间：
2007-04-01
影响因子：
5.2
通讯作者：
Soderhall, Stefan
中科院分区：
文献类型：
--
作者：
Astrom, Eva;Jorulf, Hakan;Soderhall, Stefan
目的：在一项前瞻性的观察研究中，对11例3~13个月(中位数3.6个月)的成骨不全患儿进行了静脉注射帕米磷酸二钠的治疗。结果：在3~6岁(中位数4.5岁)的成骨不全患儿的治疗过程中，3~6岁(中位数4.5岁)的儿童在治疗过程中，腰椎双能X射线吸收测量仪显示骨密度逐渐增加。血清骨代谢参数(碱性磷酸酶、骨钙素、前胶原1羧基末端肽、1胶原硬肽)和尿骨代谢参数(脱氧吡啶)显示骨转换降低。活动能力有了改善，在最新的记录中，在3.3-6.5岁(中位数4.8岁)的儿童都能走路。椎体重塑，椎体高度增加，没有儿童出现脊柱侧弯、后凸或颅底凹陷。所有的儿童都需要股骨髓内棒治疗骨折，5名儿童需要胫骨棒治疗极端的弯曲度，从而阻止功能站立和行走。结论：对于严重成骨不全的婴幼儿，雪崩是一种有效的对症治疗方法，但往往需要额外的骨科手术。早期治疗可预防脊柱侧弯和颅底凹陷。长期的随访很重要。
Objective: Children with the severe forms of osteogenesis imperfecta have in several studies been treated with intravenous pamidronate, but there are only few reports of the effect of early treatment.Aim: To evaluate the effect of treatment started in infancy.Methods: In a prospective observational study, with a historic control group, intravenous disodium pamidronate (APD) was given as monthly infusions to 11 children with osteogenesis imperfecta aged 3 - 13 (median 3.6) months, who had severe osteogenesis imperfecta with congenital bowing of the femora and vertebral compression fractures.Results: During treatment of children aged between 3 and 6 (median 4.5) years, dual-energy x ray absorptiometry measurements of the lumbar spine showed a gradual increase in bone density. Bone metabolism parameters in serum (alkaline phosphatase, osteocalcin, procollagen 1 carboxy-terminal peptide, collagen 1 teleopeptide) and in urine (deoxypyridinoline) indicated a decrease in bone turnover. An improvement of mobility was seen and at the latest recording, at the age of 3.3 - 6.5 (median 4.8) years, the children could all walk. Vertebral remodelling was seen, with increased vertebral height, and no child developed scoliosis, kyphosis or basilar impression. All children required femoral intramedullar rods for fractures, and five needed tibial rodding for extreme curvatures that prevented functional standing and walking. No adverse effects were seen on growth, fracture healing or blood chemistry.Conclusions: APD is an efficient symptomatic treatment for infants with severe osteogenesis imperfecta, but additional orthopaedic surgery is often needed. Early treatment may prevent scoliosis and basilar impression. Long-term follow-up is important.