Aström E, Söderhäll S
Department of Woman and Child Health, Karolinska Hospital, Stockholm, Sweden.
Acta Paediatr. 1998 Jan;87(1):64-8. doi: 10.1080/08035259850157895.
We report results of 2-5 y treatment with intravenous disodium pamidronate (APD) in three girls with severe osteogenesis imperfecta (OI). Treatment was given as monthly infusions. Additional oral 1,25-dihydroxy-cholecalciferol was given to compensate for a transient decrease in serum calcium levels. During treatment, DEXA measurements showed a gradual increase in bone density in all patients. All parameters analysed in serum (ALP, osteocalcin, PICP, ICTP) and in urine (deoxypyridinoline and pyridinoline) showed a decreased bone turnover. The two younger patients reported a major improvement in well-being, pain and activities of daily life. The effect on the older patient was less pronounced. No negative side effects in clinical or laboratory variables were observed. This study indicates that APD is of value in the symptomatic treatment of children with severe OI.
我们报告了三名患有严重成骨不全症（OI）的女孩接受静脉注射帕米膦酸钠二钠（APD）进行2至5年治疗的结果。治疗方式为每月输注。额外给予口服1,25 - 二羟基胆钙化醇以补偿血清钙水平的短暂下降。治疗期间，双能X线吸收法（DEXA）测量显示所有患者的骨密度逐渐增加。血清（碱性磷酸酶、骨钙素、前胶原I型羧基端前肽、I型胶原交联C末端肽）和尿液（脱氧吡啶啉和吡啶啉）中分析的所有参数均显示骨转换降低。两名较年轻的患者在幸福感、疼痛和日常生活活动方面有显著改善。对年龄较大患者的效果不太明显。未观察到临床或实验室变量方面的负面副作用。本研究表明，APD在严重OI儿童的对症治疗中具有价值。
