Land Christof, Rauch Frank, Montpetit Kathleen, Ruck-Gibis Joanne, Glorieux Francis H
Genetics Unit, Shriners Hospital for Children and McGill University, Montréal, Québec, Canada.
J Pediatr. 2006 Apr;148(4):456-60. doi: 10.1016/j.jpeds.2005.10.041.
To evaluate the functional abilities and the level of ambulation during pamidronate therapy in children with moderate to severe osteogenesis imperfecta.
Functional abilities, ambulation, and grip force were assessed in 59 patients (mean age, 6.1 years; range, 0.5-15.7 years; 30 girls) during 3 years of pamidronate treatment. Functional skills (mobility and self-care) were both assessed by using the Pediatric Evaluation of Disability Inventory. Ambulation level was assessed by using the modified Bleck score. For 48 patients, results after 3 years of pamidronate treatment could be matched to those of patients with similar age and disease severity who had not received pamidronate.
Mobility and self-care scores increased during the study period (+43% and +30%, respectively). The average ambulation score changed from 0.8 to 1.9. Maximal isometric grip force increased by 63%. Mobility and ambulation scores and grip force measures were significantly higher than in patients who had not received pamidronate. The difference in self-care scores did not reach significance.
This study suggests that cyclical pamidronate treatment improves mobility, ambulation level, and muscle force in children with moderate to severe osteogenesis imperfecta.
评估中重度成骨不全患儿在帕米膦酸治疗期间的功能能力和行走水平。
在59例患者（平均年龄6.1岁；范围0.5 - 15.7岁；30名女孩）接受3年帕米膦酸治疗期间，评估其功能能力、行走能力和握力。功能技能（活动能力和自我护理能力）均采用儿童残疾评定量表进行评估。行走水平采用改良的布勒克评分进行评估。对于48例患者，将3年帕米膦酸治疗后的结果与未接受帕米膦酸治疗的年龄和疾病严重程度相似的患者的结果进行匹配。
在研究期间，活动能力和自我护理能力得分均有所提高（分别提高了43%和30%）。平均行走评分从0.8变为1.9。最大等长握力增加了63%。活动能力和行走评分以及握力测量结果均显著高于未接受帕米膦酸治疗的患者。自我护理能力得分的差异未达到显著水平。
本研究表明，周期性帕米膦酸治疗可改善中重度成骨不全患儿的活动能力、行走水平和肌肉力量。
