32例儿童非亲缘脐血移植临床研究

Clinical Study of Unrelated Umbilical Cord Blood Transplantation in 32 Children Patients

本研究对山东省脐血造血干细胞库2001年8月至2004年6月所提供32例儿童无血缘脐血移植(URCBT)进行回顾性分析,观察非血缘脐血造血干细胞移植(UCBT)的疗效及并发症。常规进行脐血的收集、分离、保存、检测、冻融和输注。用PCR-SSO、PCR-SSP、SBT技术检测HLA-A、B、DR。结果表明32例患血液系统疾病儿童,中位年龄8(2-15)岁;中位体重31.5(14-55)kg,其中急性淋巴细胞白血病(ALL)13例,均为高危型;急性髓系白血病(AML)9例,包括M11例,M24例,M43例,M51例;慢性髓系白血病(CML)2例;重型再生障碍性贫血(SAA)3例;急性混合性白血病(HAL)3例;非何杰金氏淋巴瘤2例。供受者HLA全相合为10例,1个位点不相合为16例,2个位点不相合为6例。供受者ABO血型相合12例,不相合20例。输入有核细胞(TNC)5.57(2.16-12.3)×107/kg,CD34+细胞1.78(0.85-5.92)×105/kg。采用以环磷酰胺(Cy)和全身照射(TBI)为主的Cy/TBI预处理方案4例,以白消安(Bu)和环磷酰胺(Cy)为主的Bu/Cy预处理方案21例,其他方案预处理7例。GVHD预防采用以环孢菌素A为主的方案。结果表明32例患者20例(62.5%)白细胞达到植入标准,ANC≥0.5×109/L的中位时间17(9-38)天;Plt≥20×109/L中位时间42(18-102)天。Ⅱ-Ⅳ度和Ⅲ-Ⅳ度急性GVHD累计发生率分别为35%和15%。1年总生存率59.4%,2年总生存率40.6%。结论HLA相合及1-2个位点不合异基因脐血造血干细胞移植是可行的,尤其在治疗儿童血液病方面具有应用前景。

To evaluate the clinical efficacy of unrelated umbilical cord blood transplantation （UCBT） on the treatment of children with hematologic malignancies and nonmalignancies, between August 2001 and June 2004 , 32 patients were transplanted by using unrelated umbilical cord blood supplied by Shandong Umbilical Cord Blood Bank. Out of them, 13 patients suffered from ALL, 9 from AML, 3 from AA, 3 from HAL, 2 from CML and 2 from NHL. The median age was 8 years （range 2 -15）, the median weight was 31.5 kg （range 14- 55）. All patients received ablative conditioning regiment according to the disease and the disease status. Conditioning regiments Cy/TBI were used for 4 patients and Bu/ Cy for 21 patients , other for 7 patients. All patients received cyclosporin A and/or methotrexate for GVHD prophylaxis. The mean number of infused nuclear cells were 5.57 （2.16 - 12.3 ） × 10^7/kg , CD34^＋ cells 1.78 （0.85 -5.59 ） ×10^5. All of UCB units were tested for HLA-A, -B, and DRB1 using low and high resolution techniques. There were HLA-matched in 10, 5/6 in16 and 4/6 in 6. The results showed that 20 out of 32 patients achieved complete engraftment. Median time of neutrophil ≥0.5 × 10^9/L, and platelet ≥20 × 10^9/L were 17 （ 9 - 38 ） and 42 （ 18 - 102 ） days respectively. The incidence of aGVHD Ⅱ - Ⅳ and aGVHD Ⅲ - Ⅳ were 35% and 15% respectively. After a median follow-up of 18 months （ 1.5 - 28.5 ）, overall survival rate at one year was 59.4%, overall survival rate at two years was 40.6%. It is suggested that UCBT is promising for children patients who is lack of matched bone marrow donors.