脐血移植治疗儿童恶性血液病的临床研究

目的观察脐血移植(CBT)治疗儿童血液系统恶性疾病的植入率、移植相关并发症和生存情况。方法血液系统恶性疾病患儿19例,其中无关供者16例,同胞供者3例。脐血与患儿HLA6个位点全相合6例,1个位点错配10例,2个位点错配3例。移植时疾病状态,高危10例,标危9例。预处理主要采用Bu/Cy或Cy/TBI方案,同时应用抗胸腺球蛋白(ATG)。GVHD预防主要采用环孢菌素A(CsA)、骁悉(MMF)及甲基泼尼松龙(MP)三联方案,3例同胞供者移植则单用CsA预防GVHD。移植脐血的细胞数量是,有核细胞(TNC)5.96×107/kg(2.57～12.20),CD34+细胞2.20×105/kg(0.31～5.80)。结果18例成功重建粒系造血,中位重建时间17(11～35)天;15例血小板植入,中位重建时间38(25～112)天。发生急性GVHD9例,其中Ⅰ度～Ⅱ度7例,Ⅲ度～Ⅳ度aGVHD2例;慢性GVHD3例。移植后随访4～86个月,复发3例,死亡8例,其中移植相关死亡6例,移植相关死亡率(TRM)31.6%。因CMV感染导致间质性肺炎死亡4例,占移植相关死亡的66.7%(4/6)。2年总生存率(OS)和无事件生存率(EFS)分别为62.54%(±11.24%)、44.53%(±12.22%);标危移植组与高危移植组病人2年OS分别为88.89%(±10.48%)、40.00%(±15.49%)。结论本组儿童血液系统恶性疾病脐血移植的植入率、移植相关并发症和生存情况与国外文献报道相似,移植时疾病状态为标危时治疗效果较好,CMV感染所致间质性肺炎是移植相关死亡的主要原因。

Cord blood transplantation for the treatment of childhood hematological malignancies

Objective To investigate the engraftment,transplantation related complications and survival after cord blood transplantation(CBT) for the treatment of hematological malignancies in children.Methods Nineteen children with hematological malignancies were enrolled in this study,16 patients were transplanted with unrelated donors,3 with sibling donors.Six patients received HLA-matched cord blood units,10 patients received HLA-mismatched CB units with a disparity in one antigen,and 3 patients received HLA-mismatched CB units with a disparity in two antigens.Ten patients were transplanted at high risk status,9 at standard risk.Conditioning regimens were mostly Bu/Cy or Cy/TBI;besides,nearly all patients received additional antithymocyte globulin(ATG).GVHD prophylaxis consisted of cyclosporine A(CsA),mycophenolate mofetil(MMF) and methylprednisolone(MP),nevertheless,3 patients with sibling donors received single CsA.The infused cord blood units contained 5.96(2.57~12.20)×107/kg nucleated cells and 2.20(0.31~5.80)×105/kg CD34+ positive cells.Results Of the 19 patients,18 achieved neutrophil engraftment at a median of 17(11~35)days after transplantation.Platelet engraftment was achieved in 15 patients at a median of 38(25~112)days.Acute GVHD occurred in 9 patients,7 patients developed grade-Ⅰ to grade-Ⅱ GVHD,2 patients developed grade-Ⅲ to grade-Ⅳ GVHD.Chronic GVHD occurred in 3 patients.Following-up for 4 to 86 months,3 patients relapsed and 8 patients died.Of the 8 dead patients,6 patients died from transplantation-related complications,the transplantation-related mortality(TRM) was 31.6%.4 patients died of CMV-infectious interstitial pneumonia(CMV-IP),it was about 66.7%(4/6) of cases of TRM.For the studied 19 patients,2 year overall survival(OS) and event-free survival(EFS) are 62.54%(±11.24%) and 44.53%(±12.22%) respectively.For the standard risk and high risk patients,2 year OS are 88.89%(±10.48%) and 40.00%(±15.49%) respectively.Conclusion The engraftment,transplantation related complications and survival of this studied group were about the same as other overseas reports.The outcome of treatmen is better when the transplant was performed in a standard risk status of disease.CMV-IP was the major cause of TRM.