Pediatric Endocrinology, Department of Women's and Children's Health, Astrid Lindgren Children's Hospital, Karolinska Institutet, Stockholm, Sweden.
Horm Res Paediatr. 2011;75(5):354-61. doi: 10.1159/000323370. Epub 2011 Feb 7.
BACKGROUND/AIMS: Pamidronate is widely used to treat pediatric patients with osteogenesis imperfecta (OI). We aimed at delineating the effects of monthly pamidronate therapy on the growth of different body segments in prepubertal OI patients.
The study included 14 prepubertal patients (12 boys, 2 girls) with mild forms of OI (type I and IV). The mean age at treatment start was 7:8 years:months (3:7-11:0). Pamidronate was given as monthly intravenous infusions. The patients were measured 1 year before, at treatment start and 1 and 2 years after treatment start.
Height standard deviation score (SDS) and sitting height SDS significantly increased (p < 0.05) during the first year of treatment when compared to the pre-treatment year. No further improvement was detected during the second year of treatment. However, when plotted on disease-specific growth charts (untreated patients with the same OI types), height gain was significant during the first (p < 0.001) and second (p < 0.05) years of treatment. All patients increased their bone mineral density throughout the follow-up.
Monthly pamidronate improves the growth of prepubertal patients with mild OI, where the most prominent growth stimulation is seen in the upper body segment.
背景/目的：帕米膦酸盐广泛用于治疗成骨不全症（OI）的儿科患者。我们旨在描述每月帕米膦酸盐治疗对青春期前 OI 患者不同身体部位生长的影响。
该研究纳入了 14 名轻度 OI（I 型和 IV 型）的青春期前患者（12 名男孩，2 名女孩）。治疗开始时的平均年龄为 7:8 岁：月（3:7-11:0）。帕米膦酸盐作为每月静脉输注给予。在治疗开始前、开始后 1 年和 2 年对患者进行了测量。
与治疗前一年相比，治疗的第一年身高标准差评分（SDS）和坐高 SDS 显著增加（p<0.05）。在第二年的治疗中没有发现进一步的改善。然而，当在疾病特异性生长图表（具有相同 OI 类型的未治疗患者）上绘制时，在第一年（p<0.001）和第二年（p<0.05）的治疗中身高增长显著。所有患者在随访过程中均增加了骨矿物质密度。
每月帕米膦酸盐可改善轻度 OI 青春期前患者的生长，其中上半身部位的生长刺激最为明显。
