MacMillan Margaret L, Davies Stella M, Nelson Gene O, Chitphakdithai Pintip, Confer Dennis L, King Roberta J, Kernan Nancy A
Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, University of Minnesota Blood and Marrow Transplant Program, Minneapolis, Minnesota 55455, USA.
Biol Blood Marrow Transplant. 2008 Sep;14(9 Suppl):16-22. doi: 10.1016/j.bbmt.2008.05.019.
The National Marrow Donor Program (NMDP) has facilitated unrelated donor hematopoietic cell transplants for more than 20 years. In this time period, there have been many changes in clinical practice, including improvements in HLA typing and supportive care, and changes in the source of stem cells. Availability of banked unrelated donor cord blood (incorporated into the NMDP registry in 2000) as a source of stem cells has become an important option for children with leukemia, offering the advantages of immediate availability for children with high-risk disease, the need for a lesser degree of HLA match, and expanding access for those with infrequent HLA haplotypes. Overall survival (OS) in children with acute leukemia transplanted with unrelated donor bone marrow (BM) is markedly better in more recent years, largely attributable to less treatment-related mortality (TRM). Within this cohort, 2-year survival was markedly better for patients with acute lymphoblastic leukemia (ALL) in first complete response (CR1) (74%) versus second complete response (CR2) (62%) or more advanced disease (33%). Similar findings are observed with patients with AML, suggesting earlier referral to bone marrow transplant (BMT) is optimal for survival. Notably, this improvement over time was not observed in unmodified peripheral blood stem cell (PBSC) recipients, suggesting unmodified PBSC may not be the optimal stem cell source for children.
美国国家骨髓捐赠计划（NMDP）推动非亲属捐赠者造血细胞移植已有20多年。在此期间，临床实践发生了许多变化，包括HLA分型和支持治疗的改进，以及干细胞来源的变化。作为干细胞来源的库存非亲属捐赠者脐带血（于2000年纳入NMDP登记处）已成为白血病患儿的重要选择，具有可为高危疾病患儿立即提供、对HLA匹配程度要求较低以及扩大罕见HLA单倍型患者的获取机会等优势。近年来，接受非亲属捐赠者骨髓（BM）移植的急性白血病患儿的总生存率（OS）明显更高，这主要归因于较低的治疗相关死亡率（TRM）。在这一队列中，首次完全缓解（CR1）的急性淋巴细胞白血病（ALL）患者的2年生存率（74%）明显高于第二次完全缓解（CR2）（62%）或疾病进展更严重的患者（33%）。急性髓系白血病（AML）患者也观察到类似结果，这表明早期转诊至骨髓移植（BMT）对生存最为有利。值得注意的是，未接受处理的外周血干细胞（PBSC）受者未观察到随时间推移的这种改善，这表明未接受处理的PBSC可能不是儿童的最佳干细胞来源。
