ANALYSIS OF 462 TRANSPLANTATIONS FROM UNRELATED DONORS FACILITATED BY THE NATIONAL-MARROW-DONOR-PROGRAM
ANALYSIS OF 462 TRANSPLANTATIONS FROM UNRELATED DONORS FACILITATED BY THE NATIONAL-MARROW-DONOR-PROGRAM
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DOI：
10.1056/nejm199303043280901
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发表时间：
1993-03-04
影响因子：
158.5
通讯作者：
BLUME, KG
中科院分区：
文献类型：
--
作者：
KERNAN, NA;BARTSCH, G;BLUME, KG
研究背景与方法。同种异体骨髓移植可以治疗大量血液病、骨髓衰竭、免疫缺陷综合征和某些代谢疾病的患者。不幸的是，只有25%到30%的潜在受体有相同的hla的兄弟姐妹可以作为供体。1986年，美国建立了国家骨髓捐赠计划，以帮助缺乏亲属供体的患者从非亲属供体处寻找和获得合适的骨髓。在项目的头四年里，462名患有后天和先天性淋巴造血障碍或代谢疾病的患者接受了来自非亲属捐赠者的骨髓移植。移植后100天内移植的概率为94%，尽管8%的患者后来出现继发性移植失败。II级、III级或IV级急性移植物抗宿主病的概率为64%，一年后发生慢性移植物抗宿主病的概率为55%。在有良好预后因素的白血病患者中，两年无病生存率为40%，而在高风险患者中，这一比例为19%。29%的再生障碍性贫血患者存活两年，而骨髓增生异常患者两年无病生存率为18%。对于先天性免疫或非免疫疾病的患者，生存率为52%。国家骨髓捐赠计划使大量需要从hla匹配密切的非亲属供体中获得骨髓移植的患者受益，并促进了非亲属供体进入供体库和获得合适骨髓的工作。
Background and Methods. Allogeneic bone marrow transplantation is curative in a substantial number of patients with hematologic cancers, marrow-failure disorders, immunodeficiency syndromes, and certain metabolic diseases. Unfortunately, only 25 to 30 percent of potential recipients have HLA-identical siblings who can act as donors. In 1986 the National Marrow Donor Program was created in the United States to facilitate the finding and procurement of suitable marrow from unrelated donors for patients lacking related donors.Results. During the first four years of the program, 462 patients with acquired and congenital lymphohematopoietic disorders or metabolic diseases received marrow transplants from unrelated donors. The probability of engraftment by 100 days after transplantation was 94 percent, although 8 percent of patients later had secondary graft failure. The probability of grade II, III, or IV acute graft-versus-host disease was 64 percent, and the probability of chronic graft-versus-host disease at one year was 55 percent. The rate of disease-free survival at two years among patients with leukemia and good prognostic factors was 40 percent and among patients at higher risk, 19 percent. Twenty-nine percent of the patients with aplastic anemia were alive at two years, and the rate of two-year disease-free survival among patients with myelodysplasia was 18 percent. For patients with congenital immunologic or nonimmunologic disorders, the probability of survival was 52 percent.Conclusions. The National Marrow Donor Program has benefited a substantial number of patients in need of marrow transplants from closely HLA-matched unrelated donors and has facilitated the recruitment of unrelated donors into the donor pool and the access to suitable marrow.