Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: analysis of engraftment and acute graft-versus-host disease.

Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: analysis of engraftment and acute graft-versus-host disease.
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DOI:
10.1182/blood.v88.3.795.bloodjournal883795
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发表时间:
1996-08
期刊:
影响因子:
20.3
通讯作者:
J. Wagner;J. Rosenthal;R. Sweetman;X. Shu;S. Davies;N. Ramsay;P. Mcglave;L. Sender;M. Cairo
J. Wagner;J. Rosenthal;R. Sweetman;X. Shu;S. Davies;N. Ramsay;P. Mcglave;L. Sender;M. Cairo
中科院分区:
医学1区
文献类型:
--
作者:
J. Wagner;J. Rosenthal;R. Sweetman;X. Shu;S. Davies;N. Ramsay;P. Mcglave;L. Sender;M. Cairo

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为了降低与无关供体骨髓(BM)移植相关的发病率和死亡率,并潜在地扩大合适供体的库,冷冻保存的无关供体脐带血被认为是用于移植的造血干细胞的替代来源。患有白血病、骨髓衰竭综合征或先天性代谢缺陷的患者有资格参加I期临床试验,该试验旨在评估无关供体脐带血移植后移植物衰竭和严重急性移植物抗宿主病的风险。截至1995年12月21日,无关供体脐带血被用于重建18例年龄为0.1至21.3岁、体重为3.3至78.8 kg、患有获得性或先天性淋巴造血系统疾病或代谢性疾病的患者的造血。患者接受由纽约血液中心(纽约,NY)收集和评估的HLA匹配(n = 7)或HLA-1至3抗原不同(n = 11)移植物。无关供体脐带血移植后植入的可能性为100%,迄今为止没有患者发生晚期移植失败。100天时发生III-IV级急性移植物抗宿主病的概率为11%。中位随访时间为6个月(范围:1.6 - 17个月);在该高风险患者人群中,6个月时的生存概率为65%。我们的结论是,从HLA匹配和不匹配的无关供体的冷冻保存的脐带血是一个足够的来源,供者来源的植入和难治性严重急性移植物抗宿主病的风险低的概率高的可移植造血干细胞。关于受者大小和供体HLA差异程度的限制仍有待确定。
To reduce the morbidity and mortality associated with unrelated donor bone marrow (BM) transplantation and potentially extend the pool of suitable donors, cryopreserved unrelated donor umbilical cord blood was considered as an alternate source of hematopoietic stem cells for transplantation. Patients with leukemia, BM failure syndrome, or inborn error of metabolism were eligible for a phase I clinical trial designed to estimate the risk of graft failure and severe acute graft-versus-host disease after transplantation of umbilical cord blood from unrelated donors. As of December 21, 1995, unrelated donor umbilical cord blood was used to reconstitute hematopoiesis in eighteen patients aged 0.1 to 21.3 years weighing 3.3 to 78.8 kg with acquired or congenital lympho-hematopoietic disorders or metabolic disease. Patients received either HLA-matched (n = 7) or HLA-1 to 3 antigen disparate (n = 11) grafts collected and evaluated by the New York Blood Center (New York, NY). The probability of engraftment after unrelated donor umbilical cord blood transplantation was 100% with no patient having late graft failure to date. The probability of grade III-IV acute graft-versus-host disease at 100 days was 11%. With a median follow-up of 6 months (range, 1.6 to 17 months); the probability of survival at 6 months is 65% in this high risk patient population. We conclude that cryopreserved umbilical cord blood from HLA-matched and mismatched unrelated donors is a sufficient source of transplantable hematopoietic stem cells with high probability of donor derived engraftment and low risk of refractory severe acute graft-versus-host disease. Limitations with regard to recipient size and degree of donor HLA disparity remain to be determined.