65例中国非亲缘性脐血造血干细胞移植的回顾研究

目的1998—2004年,广州脐血库向国内25家移植中心提供非亲缘性脐血治疗恶性及非恶性肿瘤性疾病患者65例,对其进行回顾性分析,探讨与非亲缘性脐血移植密切相关的影响因素。方法脐带血均采自广州市妇婴医院正常分娩的足月顺产产妇,脐血分离、冻存、复温方法参照纽约脐血库的标准及相关文献;选择脐血的依据是HLA相合程度及有核细胞数量。结果65例接受非亲缘性脐血移植的病例中,白血病49例,遗传缺陷性疾病16例;男性42例,女性23例;受者中位年龄10岁(1~33岁);中位体重27kg(10~67kg);HLA-A、B,DRB1匹配全相合9例,1个位点不合43例,2个位点不合13例;总有核细胞(TNC)中位输入量5.7×107/kg[(1.84~20.00)×107/kg]。移植后植入50例有核细胞总数(ANC>500/μl),植入的中位时间为17.5d(7~44d),植入率77%;41例受体发生GVHD,发生率为63%。30例长期存活(无病生存时间>365d),无病存活率为61%。结论非亲缘性脐血移植具有良好的临床应用前景。

Results of unrelated umbilical cord blood stem cell transplantation for 65 patients in China

OBJECTIVE: From December 1998 to April 2004, 3960 umbilical cord blood units were stored in Guangzhou cord blood bank, which provided 100 umbilical cord blood units to 25 transplant center for 83 patients with malignant or non-malignant diseases. To study the related factors affecting unrelated umbilical cord blood stem cell transplantation, the authors analyzed retrospectively the results of transplantation of unrelated umbilical cord blood stem cells for 65 patients. METHODS: ALL (acute lymphocytic leukemia) cord blood units were obtained from full term normal vaginal and cesarean deliveries in Guangzhou Women and Infants Hospital. The fractionation, cryopreservation and thawing of the cord blood were performed according to the regulations of New York umbilical cord blood bank and pertinent literature. The selection of cord blood was based on HLA typing and the number of nucleated cells. The sex and HLA antigens of donors were defined as the evidence of engraftment. Time to engraftment was recorded when the absolute number of neutrophil ANC (absolute neutrophil count) was higher than 5.0 x 10(8) for three days. Event-free survival and graft versus host disease (GVHD) were provided by transplant centers. RESULTS: Out of 65 patients who received unrelated cord blood stem cell transplant, 49 patients were diagnosed as having malignant diseases [including 23 with ALL, 16 with AML (acute myeloid leukemia), 7 with CML (chronic myelogenous leukemia), 3 with lymphoma and one with MDS (myelodysplastic syndrome)], 16 patients had non-malignant disease. The 65 transplanted patients (42 male, 23 female) had a median age of 10 years (range 1 - 33 years) and a median body weight of 27 kg (range 10 - 67 kg). The patients received cord blood stem cells from unrelated 0-locus (n = 9) or 1-locus (n = 43) or 2-locus (n = 13) HLA mismatched donor. The median dose of infused cells was: total neutrophil count (TNC) 5.7 x 10(7), CD(34)(+) 5.1 x 10(5), CFU-GM 3.8 x 10(4). Fifty of 65 (77%) patients had engraftment. GVHD occurred in 41 patients (63%), including acute grade I - II GVHD in 31 patients (76%), acute grade III - IV GVHD in 8 patients (20%) and chronic GVHD in 2 patients (5%). Fifty patients had engraftment (ANC > 5.0 x 10(8)) after a median time of 17 (range 7 to 44) days after transplant, while an autologous hematopoietic reconstitution was observed in 6 patients; 24 patients died of severe pneumonia (n = 8), acute GVHD (n = 4), or sepsis (n = 12) and the disease-free survival probability was 61%. CONCLUSIONS: Unrelated allogeneic umbilical cord blood transplantation may be a good substitution for unrelated allogeneic bone marrow transplantation with a good prospect.