Díaz de Heredia C, González M, Verdeguer A, Elorza I, Rodriguez A, Martinez A, Pérez J M, Badell I, Gonzalez M E, Olivé T, Fernández J M, Maldonado M S, Díaz M A, Sánchez de Toledo J
Servicio de Oncología y Hematología Pediátricas, Hospital Universitario Vall d'Hebron, Universitat Autónoma de Barcelona, Barcelona, Spain.
Unidad de Trasplante, Hospital Niño Jesús, Universidad Autónoma de Madrid, Madrid, Spain.
Bone Marrow Transplant. 2014 Jun;49(6):767-72. doi: 10.1038/bmt.2014.30. Epub 2014 Mar 10.
Outcomes of unrelated cord blood transplants (UCBT) were assessed in 172 consecutive children, median age 5 years (range: 0.5-18), with haematological malignancies treated at nine Spanish hospitals between February 1996 and April 2009. Data were collected from the Spanish Working Party for Blood and Marrow Transplantation in Children (GETMON) database. ALL was diagnosed in 125 patients, AML in 43 and myelodysplastic syndrome in 4. Myeloid engraftment (ANC⩾0.5 × 10(9)/L) occurred in 87.2% at a median of 22 days and was associated with the total nucleated cell (TNC) dose infused and use of a TT-containing conditioning regimen. Cumulative incidence of relapse was 20% at 1 year post transplant and 29% at 3 years, being higher in patients with a diagnosis of ALL, very high risk disease and GVHD grades 0-1. Cumulative incidence of non-relapse mortality (NRM) was 19% at 100 days post transplant and 39% at 1 year. BU-FLU-TT-ATG-conditioned patients had lower NRM. Disease-free survival (DFS) was 40% at 2 years post transplant (for patients transplanted since 2006). On multivariate analysis, TNC dose infused, AML and BU-FLU-TT-ATG-conditioning regimen increased the probability of DFS. It is of paramount importance to select cord blood units with the highest cell dose. As the BU-FLU-TT-ATG-conditioning regimen was associated with better DFS owing to lower NRM, further prospective studies testing this regimen are warranted.
1996年2月至2009年4月期间，在西班牙9家医院对172例连续的血液系统恶性肿瘤儿童进行了非血缘脐血移植（UCBT），这些儿童的中位年龄为5岁（范围：0.5 - 18岁）。数据来自西班牙儿童血液和骨髓移植工作组（GETMON）数据库。125例患者诊断为急性淋巴细胞白血病（ALL），43例为急性髓系白血病（AML），4例为骨髓增生异常综合征。髓系植入（中性粒细胞绝对值（ANC）≥0.5×10⁹/L）发生率为87.2%，中位时间为22天，与输注的总核细胞（TNC）剂量以及含TT的预处理方案的使用有关。移植后1年复发的累积发生率为20%，3年时为29%，在诊断为ALL、极高危疾病以及移植物抗宿主病（GVHD）0 - 1级的患者中更高。移植后100天非复发死亡率（NRM）的累积发生率为19%，1年时为39%。接受白消安 - 氟达拉滨 - TT - 抗胸腺细胞球蛋白（BU - FLU - TT - ATG）预处理的患者NRM较低。移植后2年（自2006年起移植的患者）无病生存率（DFS）为40%。多因素分析显示，输注的TNC剂量、AML以及BU - FLU - TT - ATG预处理方案增加了DFS的概率。选择细胞剂量最高的脐血单位至关重要。由于较低的NRM，BU - FLU - TT - ATG预处理方案与更好的DFS相关，因此有必要进一步开展前瞻性研究来验证该方案。
