高分辨率CT观察不同来源干细胞移植治疗B型尼曼-皮克病儿童肺间质病变效果

目的 采用高分辨率CT （HRCT）观察不同来源干细胞移植治疗B型尼曼-皮克病（NPD）患儿肺间质病变的效果。方法 回顾性分析6例B型NPD患儿接受造血干细胞移植前后肺部HRCT资料，其中3例接受非亲缘脐带血干细胞移植治疗，3例接受亲缘单倍体骨髓+外周血干细胞移植治疗；于3个层面（主动脉弓层面、气管隆嵴层面及右膈顶层面）观察移植前后肺间质病变（表现为小叶间隔增厚、小叶中心阴影和小叶内线3种CT征象）累及范围并进行评分，计算各征象评分变化速率及征象评分均值变化速率。结果 接受干细胞移植后，6例患儿肺间质病变评分均较移植前呈明显下降趋势；其中3例接受非亲缘脐带血干细胞移植者肺部小叶间隔增厚、小叶中心阴影和小叶内线的征象评分变化速率及征象评分均值变化速率平均值（1.16分/月、1.33分/月、1.11分/月、1.24分/月）均高于3例接受亲缘单倍体骨髓+外周血干细胞移植者（1.14分/月、0.86分/月、0.88分/月、1.03分/月）。结论 干细胞移植可改善B型NPD患儿肺间质病变；非亲缘供体脐带血干细胞移植可能较亲缘单倍体移植治疗效果更佳。

High resolution CT observation on effect of different sources stem cell transplantation for treating pulmonary interstitial lesions of children with Niemann-Pick disease type B

Objective To observe the therapeutic effect of stem cell transplantation from different sources for treatment of pulmonary interstitial lesions in children with Niemann-Pick disease (NPD) type B with high resolution CT (HRCT). Methods Data of lung HRCT of 6 children with type B NPD before and after hematopoietic stem cell transplantation were analyzed. Among 6 cases, 3 underwent unrelated umbilical cord blood stem cell transplantation, and the rest 3 underwent related haploid bone marrow and peripheral blood stem cell transplantation. The involvement range of CT manifestations of pulmonary interstitial lesions, including interlobular septal thickening, intralobular shadow and intralobular lines before and after transplantation were scored from three levels of bilateral lungs, i.e. the aortic arch level, the carina level and the right upper diaphragmatic level, and the change rates of the above manifestations and their mean change rate were calculated. Results After stem cell transplantation, the scores of pulmonary interstitial lesions decreased significantly compared with those before transplantation in all 6 cases of type B NPD. The change rate of lung manifestations of interlobular septal thickening, intralobular shadow and intralobular lines and manifestation mean change rate in 3 cases who underwent unrelated umbilical cord blood stem cell transplantation (1.16 scores/month, 1.33 scores/month, 1.11 scores/month, 1.24 scores/month) was higher than that in the other 3 cases who underwent related haploid bone marrow and peripheral blood stem cell transplantation (1.14 scores/month, 0.86 scores/month, 0.88 scores/month, 1.03 scores/month). Conclusion Stem cell transplantation could improve lung interstitial lesions in children with NPD type B.Unrelated umbilical cord blood stem cell transplantation might be more effective than related haploid transplantation.