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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。
Objective To observe the implantation rate, transplantation-related complications and survival of cord blood transplantation (CBT) in children with hematologic malignancies. Methods A total of 19 children with malignant hematological malignancies were involved, including 16 unrelated donors and 3 sib donors. There were 6 matched HLA-6 loci in umbilical cord blood and children, 10 mismatches in 1 locus and 3 mismatches in 2 loci. Transplantation disease status, high risk in 10 cases, standard risk in 9 cases. Pretreatment mainly used Bu / Cy or Cy / TBI program, while the application of anti-thymoglobulin (ATG). GVHD prophylaxis mainly used cyclosporin A (CsA), MMT and methylprednisolone (MP) triple regimen, and 3 sibling donors only used CsA to prevent GVHD. The number of umbilical cord blood transplanted cells was 5.96 × 107 / kg (2.57 ~ 12.20) for nucleated cells (TNC) and 2.20 × 105 / kg (0.31 ~ 5.80) for CD34 + cells. Results 18 cases successfully reconstructed granulocyte hematopoiesis. The median reconstruction time was 17 (11 ~ 35) days. Fifteen platelets were implanted and the median reconstruction time was 38 (25 ~ 112) days. There were 9 cases of acute GVHD, of which 7 cases were grade Ⅰ ~ Ⅱ and 2 cases were aGVHD with grade Ⅲ ~ Ⅳ; 3 cases were chronic GVHD. After transplantation, the patients were followed up for 4 to 86 months, 3 were relapsed, and 8 were dead. Among them, 6 were graft-related deaths and 31.6% were transplant-related deaths (TRMs). Four patients died of interstitial pneumonia due to CMV infection, accounting for 66.7% (4/6) of transplant-related deaths. The 2-year overall OS and EFS were 62.54% and 44.53% respectively. The 2-year OS of the critically ill and high-risk group was 88.89% (± 10.48%), 40.00% (± 15.49%). Conclusion The implantation rate, transplantation-related complications and survival of malignant hematological malignancies in this group are similar to those reported in foreign countries. The treatment effect is better when the disease state is standard risk when transplanted. Interstitial pneumonia caused by CMV infection Is the main cause of transplant-related deaths.