Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies

Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies
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DOI:
10.1182/blood-2007-06-098020
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发表时间:
2008-11-15
期刊:
影响因子:
20.3
通讯作者:
Kernan, Nancy A.
Kernan, Nancy A.
中科院分区:
医学1区
文献类型:
--
作者:
Kurtzberg, Joanne;Prasad, Vinod K.;Kernan, Nancy A.

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在一项前瞻性2期多中心试验中,研究了1999年至2003年间入组的191例血液恶性肿瘤儿童(中位年龄7.7岁,中位体重25.9 kg)的非亲缘供者脐带血移植结局(中位随访时间27.4个月)。通过低分辨率HLA-A、-B和高分辨率(HR)DRB 1,入组时人类白细胞抗原(HLA)匹配为6/6(n = 17)、5/6(n = 58)、4/6(n = 111)或3/6(n = 5)。冷冻前总有核细胞(TNC)剂量中位数为5.1 × 107 TNC/kg(范围,1.5-23.7),输注量为3.9 × 107 TNC/kg(范围,0.8-22.8)。植入的中位时间(中性粒细胞绝对计数> 500/mm(3)和血小板50 000/mu L)分别为27天和174天。第42天中性粒细胞植入的累积发生率为79.9%(95%置信区间[CI],75.1%-85.2%);第100天急性III/IV级GVHD为19.5%(95% CI,13.9%-25.5%);第2年慢性GVHD为20.8%(95% CI,14.8%-27.7%)。HR匹配降低了严重急性GVHD的发生率。2年累积复发率为19.9%(95% CI,14.8%-25.7%)。6个月和2年生存率分别为67.4%和49.5%。从HLA部分错配的非亲缘供者脐带血移植可以治愈许多白血病儿童。该研究在www. ClinicalTrials. gov as #NCT00000603.(血。2008; 112:4318-4327)
Outcomes of unrelated donor cord blood transplantation in 191 hematologic malignancy children (median age, 7.7 years; median weight, 25.9 kg) enrolled between 1999 and 2003 were studied (median follow-up, 27.4 months) in a prospective phase 2 multicenter trial. Human leukocyte antigen (HLA) matching at enrollment was 6/6 (n = 17), 5/6 (n = 58), 4/6 (n = 111), or 3/6 (n = 5) by low-resolution HLA-A, -B, and high-resolution (HR) DRB1. Retrospectively, 179 pairs were HLA typed by HR. The median precryopreservation total nucleated cell (TNC) dose was 5.1 x 107 TNC/kg (range, 1.5-23.7) with 3.9 x 107 TNC/kg (range, 0.8-22.8) infused. The median time to engraftment (absolute neutrophil count > 500/mm(3) and platelets 50 000/mu L) was 27 and 174 days. The cumulative incidence of neutrophil engraftment by day 42 was 79.9% (95% confidence interval [CI], 75.1%-85.2%); acute grades III/IV GVHD by day 100 was 19.5% ( 95% CI, 13.9%-25.5%); and chronic GVHD at 2 years was 20.8% (95% CI, 14.8%-27.7%). HR matching decreased the probability of severe acute GVHD. The cumulative incidence of relapse at 2 years was 19.9% (95% CI, 14.8%-25.7%). The probabilities of 6-month and 2-year survivals were 67.4% and 49.5%. Unrelated donor cord blood transplantation from partially HLA-mismatched units can cure many children with leukemias. The study was registered at www. clinicaltrials. gov as #NCT00000603. (Blood. 2008; 112: 4318-4327)