Children's Hospital, University of Cologne, Cologne, Germany.
Horm Res Paediatr. 2011;76(5):321-7. doi: 10.1159/000331128. Epub 2011 Sep 27.
BACKGROUND/AIMS: Osteogenesis imperfecta (OI) is a hereditary disease causing increased bone fragility. Pamidronate (PAM), which has to be administered as a 3-day course according to the original protocol by Glorieux, is the most frequently used therapy. Other bisphosphonates like neridronate (NER), which can be infused during an outpatient visit, have also proven to be effective. This is the first analysis comparing the effect of PAM and NER using vertebral morphometry.
28 patients with OI type III and IV were retrospectively analyzed by matched pairs.
No differences were detected between patients treated with PAM or NER at the start of therapy: mean age 4.4 years (p = 0.730), mean height 86.8 cm/85.3 cm (p = 0.854), lumbar vertebral area 208.9 mm(2)/206.0 mm(2) (p = 0.555), and in all vertebral indices. After 1 year of treatment (mean 1.16 years; p = 0.854) both groups showed a significant increase in the vertebral area and improved vertebral indices. Again there were no differences between the groups in the vertebral area (p = 0.590).
In this study there was no difference between patients treated with PAM or NER regarding vertebral morphometry during the first year of therapy. Because of the possibility of an outpatient setting, NER is convenient for these children.
背景/目的：成骨不全症（OI）是一种遗传性疾病，导致骨骼脆弱度增加。帕米膦酸盐（PAM）是最常用的治疗药物，根据 Glorieux 的原始方案，它需要连续 3 天给药。其他双膦酸盐，如可以在门诊输注的奈立膦酸盐（NER），也已被证明有效。这是首次使用椎体形态计量学比较 PAM 和 NER 疗效的分析。
通过配对分析，回顾性分析了 28 例 OI Ⅲ型和Ⅳ型患者。
在开始治疗时，接受 PAM 或 NER 治疗的患者之间未检测到差异：平均年龄 4.4 岁（p = 0.730），平均身高 86.8 cm/85.3 cm（p = 0.854），腰椎面积 208.9 mm(2)/206.0 mm(2)（p = 0.555），以及所有椎体指数。治疗 1 年后（平均 1.16 年；p = 0.854），两组的椎体面积均显著增加，椎体指数得到改善。两组的椎体面积也无差异（p = 0.590）。
在这项研究中，接受 PAM 或 NER 治疗的患者在治疗的第一年中，椎体形态计量学方面没有差异。由于可以在门诊进行，NER 对这些儿童更方便。
