口服二磷酸盐治疗儿童成骨不全症临床疗效的Meta分析

Clinical efficacy of oral bisphosphonates in the treatment of children with osteogenesis imperfecta:a Meta-analysis

目的评价口服二磷酸盐治疗儿童成骨不全症(OI)的临床疗效。方法根据Cochrane系统评价方法,计算机检索PubMed、Embase、CENTRAL、中国生物医学文献数据库等,收集所有关于口服二磷酸盐治疗儿童OI的随机对照试验(RCTs)。按照Cochrane协作网的标准对纳入研究的文章进行质量评估,并提取有效数据进行Meta分析。结果纳入6篇文献,共396例患儿。6篇文献的研究结果显示,二磷酸盐治疗可以降低四肢骨折的发生率(P=0.010),并且可以增加腰椎骨密度的Z分(P=0.000 1)和百分比(P=0.010)。二磷酸盐还可以增加体质量的Z分(P=0.000 1)。在生活质量方面,二磷酸盐治疗后患儿的自我照顾能力得到提高(P=0.000 3)。但是二磷酸盐治疗后,患儿的身高无明显增长(P=0.42),疼痛也未能得到缓解(P=0.13)。2组在上消化道不良事件的发生率方面差异无统计学意义(P=0.82)。结论二磷酸盐治疗可以有效降低四肢骨折的发生率并提高腰椎的骨密度,可以促进生长,部分改善生活质量,并且不会引起上消化道不良事件。

Objective It is to evaluate the clinical efficacy of oral bisphosphonates in the treatment of children with osteogenesis imperfecta（OI）.Methods According to the standards of the Cochrane Collaboration,all of randomized controlled trials（RCTs） in which oral bisphosphonates was compared with placebo treatment for children with OI were searched in PubMed,Embase,CENTAL,CBM et al.The quality of the trials selected was evaluated and effective data was pooled for Meta analysis.Results Six RCTs with a total of 396 patients were screened for the present study.Results of these trials＇ results showed that bisphosphonates treatment was superior to placebo in a lower risk of new peripheral fractures（P = 0.010） and the significant difference favouring bisphosphonates in the increasing of lumbar spine bone mineral density Z-score（P = 0.0001）and percentage（P = 0.010）.For growth,bisphosphonates could increase weight Z-scores（P = 0.0001）.For the quality of life,bisphosphonates treatment was better than placebo in a higher score of the ability of self-care（P = 0.0003） and the mobility（P = 0.07） although the difference was not significant.However,we found no difference in change of the height（P =0.42） and the pain score（P = 0.13）.Finally,the incidence of upper gastrointestinal adverse events were similar between the treatment and placebo groups（P = 0.82）.Conclusion Compared with placebo,oral bisphosphonates treatment could effectively reduce the incidence of new peripheral fractures and increase the lumbar spine BMD.Meanwhile,bisphosphonate therapy also increased the growth and partly improve the quality of life but was not associated with the incidence of upper gastrointestinal adverse events.