中日儿童MDS-RCC治疗策略与预后的临床回顾性分析

本研究回顾性分析中日两国儿童MDS-RCC不同治疗策略对预后的影响。根据2008年WHO髓系肿瘤分型标准,我院儿童血液病诊疗中心2009-2011年间诊治的50例获得性骨髓衰竭综合征及1999至2008年日本小儿血液学会登记录入的222例儿童MDS患儿中,中日两国共107例患儿符合MDS-RCC,对这107例患儿的不同治疗策略与预后进行回顾性比较分析。结果表明:①研究中107例患儿,诊断时中位年龄7.8岁,男女比为1.02∶1;随访中位时间3.5年,3、5年总生存率分别为93.8%和79.7%。②回顾性分成输血依赖和非输血依赖2组,输血依赖组63例,3、5年总生存率分别为89.9%和70.6%,非输血依赖组44例,5年总生存率为100.0%。③输血依赖组治疗分为免疫抑制剂治疗组(IST)〔环孢菌素A(CsA)加或不加抗胸腺细胞球蛋白(ATG)〕和造血干细胞移植(HSCT)两组,IST治疗组38例患儿,5年总生存率为100.0%;HSCT组25例患儿,3、5年总生存率分别为82.9%和30.6%。非输血依赖组分为观察及CsA治疗组,5年总生存率为100.0%。④比较我院33例MDS-RCC患儿与日本74例患儿。我院33例患儿,其中输血依赖15例,均采用IST治疗;非输血依赖18例,其中2例观察,16例给予IST治疗。所有患儿3、5年总生存率均为100.0%。日本74例患儿,3、5年总生存率分别为93.7%和75.0%,其中非输血依赖26例,给予观察,5年总生存率为100.0%;输血依赖48例,IST治疗组5年总生存率为100.0%,HSCT组3、5年总生存率分别为82.9%和30.6%。结论:儿童MDS-RCC疾病进展少见。无输血依赖的患儿仅予以观察,不予特殊治疗,IST可提高生存质量。输血依赖的患儿IST可作为一线治疗方案。HSCT治疗相关死亡率高,影响预后,其在儿童MDS-RCC中的治疗地位有待进一步观察。

Retrospective Analysis of Treatment Strategy and Prognosis in Childhood Myelodysplastic Syndromes from China and Japan

This study was aimed to retrospectively analyse the prognosis of childhood myelodysplastic syndromes（MDS-RCC） from China and Japan.Two hematologists and one pathologist from China and Japan constituted a diagnosis group.According to the criteria of 2008 WHO,33 children with MDS-RCC from 50 chinese children diagnosed as acquired bone marrow failure syndrome from 2009 to 2011,and 74 Japanese children with MDS-RCC in a prospective registration group conducted by the Japanese Society of Pediatric Hematology were enrolled in this study.The outcome of total 107 childhood MDS-RCC treated with different treatment strategies was analyzed retrospectively.The results indicated that：（1） the 3 and 5-year overall survival rates（OS） for all patients were 93.8% and 79.7% respectively.（2） All 107 patients with MDS-RCC were further subclassified into 2 groups： transfusion dependent group and transfusion independent group.The 3 and 5-year overall survival rates（OS） for transfusion dependent group were 89.9% and for transfusion independent group were 70.6% respectively,the 5-year overall survival rate（OS） for transfusion independent group was 100.0%.（3） Treatment strategy： patients from transfusion dependent group were treated w ith immunsuppression the rapy（IST）,in w hich CsA combined w ith or w ithout ATG and patients w ere treated w ith HSCT.The 5-year overall survival rate（OS） w as 100% for IST group.The 3 and 5-year overall survival rates（OS） for HSCT patients were 82.9% and 30.6%,respectively.All the patients from transfusion independent group w ere alive till the last follow up.（4） Compared w ith patients from our hospital and Japan,the 5-year overall survival rate（OS） for patients in our hospital w as 100.0%,the 3-and 5-year overall survival rates（OS） for patients in Japan w ere 93.7% and 75.0%,respectively.It is concluded that children w ith MDS-RCC are seldom progressive.The observation and w ait strategy is applicable for patients w ith MDS-RCC w ho have no transfusion dependent.IST therapy is recommended to those w ho have transfusion-dependent.