Pediatric MDS Including Refractory Cytopenia and Juvenile Myelomonocytic Leukemia
Pediatric MDS Including Refractory Cytopenia and Juvenile Myelomonocytic Leukemia
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DOI：
10.1007/978-3-030-02278-5_74
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发表时间：
2019-01-01
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影响因子：
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通讯作者：
Niemeyer, Charlotte M.
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作者：
Niemeyer, Charlotte M.
对于外周血中有2-19%的原始细胞或骨髓中有5-19%的原始细胞的儿童MDS患者，采用与成人MDS伴过度原始细胞(MDS-EB)相同的诊断标准(WHO)。一些有20%-30%的原始细胞的病例也可能有缓慢进展的疾病，可能缺乏急性白血病的临床特征，因此表现更像MDS而不是AML。这些MDS-EB转化病例(MDS-EB-t)加上MDS-EB病例约占所有儿童MDS的四分之一。在欧洲儿童MDS工作组(EWOG-MDS)的大量MDS-EB/MDS-EB-t儿童队列中，allo-HSCT与完整的MAC包括BU/CY/MEL的组合导致了63%的5岁OS，NRM和复发同样导致治疗失败(Strahm等人。2011年)。最新数据显示，通过使用高分辨率分型，接受9/10或10/10位点匹配的MSD或UD移植的患者的结果是可重叠的(Locatelli和Strahm 2018)。因为12岁的患者患移植物抗宿主病的风险很高，EWOG-MDS建议对从http://ewog-mds.移植的≥老年患者进行强化的移植物抗宿主病预防(CsA+MTX)Org)。结构复杂的核型的存在与预后不良密切相关(Göhring等人)。2010)。
For pediatric patients with MDS and 2–19% blasts in the PB or 5–19% blasts in the BM, the same diagnostic criteria utilized for adults with MDS with excess blasts (MDS-EB) are applied (WHO). Some cases with 20–30% blasts may also have slowly progressive disease, may lack clinical features of acute leukemia, and thus behave more like MDS than AML. These cases of MDS-EB in transformation (MDS-EB-t) together with cases of MDS-EB account for approximately a quarter of all childhood MDS. In the large cohort of children with MDS-EB/MDS-EB-t of the European Working Group of MDS in Childhood (EWOG-MDS), allo-HSCT with a full MAC consisting of the combination of BU/CY/MEL resulted in an OS at 5 years of 63%, with NRM and relapse contributing equally to treatment failure (Strahm et al. 2011). The update shows that the outcome for patients who received a graft from either a MSD or an UD matched for 9/10 or 10/10 HLA-loci by using high-resolution typing is superimposable (Locatelli and Strahm 2018). Because patients≥ 12 years of age had a high risk of NRM, EWOG-MDS recommends an intensified GVHD prophylaxis (CSA+ MTX) for older patients transplanted from a MSD (see http://ewog-mds. org). The presence of a structurally complex karyotype was found to be strongly associated with poor prognosis (Göhring et al. 2010).