儿童血细胞减少伴骨髓增生减低100例临床特征分析

目的：总结中日两国100例儿童血细胞减少伴骨髓增生减低病例临床特征，探讨儿童骨髓增生异常综合征（MDS）有效治疗方案。方法：非随机选取中日两国2006~2011年血细胞减少伴骨髓增生减低病例100例，回顾性分析其临床特点并对我国患者进行预后分析。结果：中日两国病例MDS及获得性再生障碍性贫血（AA）构成比差异无统计学意义。100例患儿中，AA、难治性血细胞减少（RCC）、难治性血细胞减少伴多系发育异常(RCMD) 病例数分别为29、58、13。3组病例网织红细胞绝对值及骨髓增生程度的差异具有统计学意义（P＜0.05）。对我国患儿进行随访，中位随访时间41（16~70）个月。环孢素A(CSA)联合康力龙治疗者，AA、RCC及RCMD 3组病例3个月有效率分别为25%、47%、60%，其6个月的有效率分别为75%、82%、60%。结论：RCC、RCMD、AA 3组外周血网织红细胞绝对值及骨髓增生程度具有显著性差别。CSA联合康力龙治疗儿童获得性AA和低增生性MDS疗效确切，但由于病例数少，随访时间短，仍需扩大病例长期观察。

Clinical features of cytopenia with bone marrow hypoplasia in children: an analysis of 100 cases

Objective To summarize the clinical features of cytopenia with bone marrow hypoplasia in 100 children and to investigate an effective treatment regimen for myelodysplastic syndrome(MDS) in children.Methods A retrospective analysis was performed on the clinical data of 100 children non-randomly selected from Japan and China who were diagnosed with cytopenia with bone marrow hypoplasia between 2006 and 2011.The data of patients from China were subjected to prognostic analysis.Results There was no significant difference in the proportion of MDS cases and acquired aplastic anemia(AA) cases between the Japanese and Chinese children.Of the 100 patients,there were 29 cases of acquired AA,58 cases of refractory cytopenia of childhood(RCC) and 13 cases of refractory cytopenia with multilineage dysplasia(RCMD).There were significant differences in reticulocyte absolute value in peripheral blood and degree of bone marrow proliferation among the three patient groups(P<0.05).The patients from China were followed up for 16-70 months(median,41 months).After being treated with cyclosporine(CsA) combined with stanozolol,the patients with AA had response rates of 25% and 75%,the patients with RCC had response rates of 47.1% and 82.4%,and the patients with RCMD had response rates of 60% and 60% respectively at 3 and 6 months after treatment.Conclusions There are significant differences in reticulocyte absolute value in peripheral blood and degree of bone marrow proliferation among patients with RCC,RCMD and acquired AA.CsA combined with stanozolol has a good therapeutic efficacy in the treatment of acquired AA and hypoplastic MDS in children,but studies of more cases and a longer follow-up duration are needed.