脐血移植治疗37例儿童恶性血液病的临床研究-单中心经验

Clinical effect of umbilical cord blood transplantation in 37 pediatric patients with hematologic malignancies：a single-center experience

摘要:

目的 探讨脐血造血干细胞移植（UCBT）治疗儿童恶性血液病的疗效。方法 回顾性分析接受UCBT 的37 例恶性血液病患儿的临床资料，包括急性淋巴细胞性白血病14 例，急性髓细胞性白血病9 例，幼年粒单细胞白血病5 例，慢性粒细胞白血病和骨髓增生异常综合征各3 例，急性混合型白血病2 例，淋巴肉瘤性白血病1 例。其中34 例非血缘相关，3 例血缘相关。HLA 配型6/6 相合5 例，5/6 相合12 例，4/6 相合11 例，3/6 相合9 例。移植中位年龄5.7 岁，中位体重20 kg。结果 中性粒细胞和血小板植入中位天数分别是12 d 和25 d，植入率分别为95% 和78%。中性粒细胞植入率与CD34⁺ 细胞数呈正相关（P=0.011）。血小板植入率与CD34⁺ 细胞数和有核细胞数均有关（分别P=0.001、0.014）。急性移植物抗宿主病（GVHD）的发生率为49%，慢性GVHD 为11%。随访中位时间54 个月，5 年移植相关病死率、总生存率和无病生存率分别为27%、57%和41%。结论 脐血移植是快速获得的造血干细胞来源之一，为恶性疾病患儿争取了治疗时间。

Abstract:

Objective To evaluate the clinical effect of umbilical cord blood transplantation (UCBT) in children with hematologic malignancies. Methods A retrospective analysis was performed on the clinical data of 37 pediatric patients with hematologic malignancies that consisted of 14 cases of acute lymphocyte leukemia, 9 cases of acute myeloid leukemia, 5 cases of juvenile myelomonocytic leukemia, 3 cases of chronic myeloid leukemia, 2 cases of acute mixed leukemia, 3 cases of myelodysplastic syndrome, and 1 case of lymphosarcomatous leukemia. Thirty-seven children with hematologic malignancies received UCBT from unrelated donors (34 cases) and related donors (3 cases). Grafts were 6/6 HLA-matched in 5 cases, 5/6 HLA-matched in 12 cases, 4/6 HLA-matched in 11 cases, and 3/6 HLAmatched in 9 cases. Before transplantation, these patients received rabbit antithymocyte globulin-containing conditioning regimen. The myeloablative conditioning regimen was given in 36 cases and the reduced-intensity conditioning regimen in one case. The median age of transplantation was 5.7 years, and the median weight was 20 kg. The grafts that contained a median of 6.2×10⁷ total nucleated cells (TNC)/kg and 2.7×105 CD34⁺ cells/kg were infused. Results The median times to neutrophil engraftment and platelet engraftment were 12 days and 25 days, respectively, and the rates of neutrophil engraftment and platelet engraftment were 95% and 78%, respectively. The rate of neutrophil engraftment was positively correlated with the number of CD34⁺ cells (P=0.011), while the rate of platelet engraftment was correlated with the numbers of CD34⁺ cells and TNC (P=0.001; P=0.014). The incidence rates of acute and chronic graft-versushost disease were 49% and 11%, respectively. The median follow-up was 54 months. The 5-year transplant-related mortality, overall survival, and disease-free survival were 27%, 57.4% and 41%, respectively. Conclusions UCBT is an alternative source of hematopoietic stem cells for patients with hematologic malignancies.