儿童戈谢病脊柱定量CT骨密度测定随访研究

Vertebral bone mineral density follow-up in children with gaucher disease with QCT

目的比较儿童戈谢病(Gaucher disease,GD)治疗前后脊柱椎体骨密度(bone mineral density,BMD)的变化。方法 30例确诊的GD患儿,其中男18例,女12例,年龄4～17岁,平均年龄11.2±4.1岁,其中5例进行脾脏切除。在规范酶替代治疗和治疗9月后在常规随访CT扫描时,同时采用定量CT(quantitative computed tomography,QCT)测定T12-L4间无明显变形及病理性骨折的椎体骨密度,取各椎体平均骨密度。采用配对t检验比较治疗前后患儿骨密度变化。结果 30例患儿治疗前椎体平均骨密度值176.2±20.0 mg/cm3,治疗9月后平均骨密度值为197.8±18.1 mg/cm3,较治疗前椎体骨密度平均升高12.3%(P<0.01);其中5例脾切除患儿治疗前后平均骨密度值分别为163.7±5.7 mg/cm3和190.3±5.2 mg/cm3,椎体骨密度较治疗前升高13.8%。结论使用QCT骨密度测量技术可以精确敏感的评估患儿骨密度情况及治疗疗效的反应,指导临床进一步治疗方案。

Objective To follow-up the bone mineral density （BMD） of vertebrae using quantitative CT in Gaucher disease （GD） children with enzyme replacement therapy. Methods 30 GD patients （male 18, femall2）with mean age 11.2 ±4.1 years, ranging 4 - 17 years, were treated with enzyme replacement therapy and 5 cases had splenectomy. With the routine CT scan dataset for monitor the progression of the disease, the spine BMD was measured with QCT at baseline and 9 months after treatment. Paired student t test was performed to compare this difference. Results The mean BMD of 30 GD patients was 176.2± 20.0 mg/cm3 at the baseline and 197.8 ±18.1 mg/em3 after 9 months treatment, which represented an average of 12.3% increase in BMD （P 〈 0. 01 ）. In particular the average BMD in the children with splenectomy at baseline was 163.7±5.7 mg/cm3 versus the 190. 3 ± 5.2 mg/cm3 of BMD after treatment, and which was increased about 13.8%. Conclusions Bone mineral density measurement using QCT is very useful to monitoring the treatment effect in children with Gaueher disease.