双膦酸盐在治疗儿童McCune-Albright综合征的应用

Five Cases of Bisphosphonate Therapy on Children with McCune-Albright Syndrome and Literature Review

目的报告5例McCune-Albright综合征患儿应用唑来膦酸治疗骨纤维异常增殖症,为此类疾病的药物治疗提供参考。方法对北京协和医院内分泌科收治的5例儿童McCune-Albright综合征患者应用唑来膦酸的临床资料进行总结分析,并对应用双膦酸盐治疗的有效性、安全性以及影响双膦酸盐治疗结局的因素进行文献复习。结果双膦酸盐在儿童McCune-Albright综合征的治疗中有一定疗效,安全性较好。结论儿童McCune-Albright综合征可应用双膦酸盐治疗骨纤维异常增殖症,骨骼受累严重程度可能影响双膦酸盐的治疗结局。

OBJECTIVE To provide references for clinical rational drug use through five empirical analysis of zoledronate’s application on children with McCune-Albright syndrome.METHODS The clinical data of five children with McCune-Albright syndrome were analyzed and summarized.Literature review was conducted to analyze long-term out come of bisphosphonate therapy and factors influencing outcome of bisphosphonate therapy in fibrous dysplasia.RESULTS Bisphosphonate therapy achieved certain effects,and there were no serious adverse events occurred.CONCLUSION Bisphosphonate therapy can be used on children with McCune-Albright syndrome,the prognostic factor that influencing outcome of bisphosphonate therapy is the extent of skeletal involvement.